Tag: Biotech

How to Determine When a Biotech Stock Could Expect Market-Moving News

Posted on by phoffman@channelchek.com

Does the FDA Provide Information that Helps Pharmaceutical Stock Investors?

Investors with a “Stocks on the Move” or “Market Movers” window open sometimes witness a stock climb double or triple digits during a single trading day. It often turns out that it’s a drug company that just passed an FDA milestone. When this happens, these companies have the potential for large movements. The natural question investors ask is, how does one become more aware that there may be an extreme movement in a biotech, or pharmaceutical stock? For wisdom on this subject, I turned to Robert LeBoyer, the Senior Life Sciences Analyst at Noble Capital Markets. Below, cutting through many complex details and variables, is what I discovered from the veteran equity analyst.

The key is to first understand the framework of the FDA approval process. This will help an investor understand the significance of activity and even where to find key dates and imminent decision periods. Especially toward the end of the process, it is especially then when there are events that could rocket the company stock or cause it to retreat. These are PDUFA calendar deadlines and advisory panel meeting dates. Below is an outline of the process and key dates that may allow investors to position themselves to take advantage of any big jump (or even sudden decline) in a biotech’s stock price.

Understanding The FDA Approval Process

The FDA is responsible for regulating the safety and efficacy of drugs and medical devices in the United States. The review process for new drug applications falls under the legally required format called the Prescription Drug User Fee Act (PDUFA).

PDUFA requires the FDA to collect fees from drug developers to fund the review process, in exchange, the FDA has an obligation to answer the application within ten months. The PDUFA legislation has improved the process for companies seeking FDA approval helping to speed the review process. The fees collected are used to hire additional staff and overall improve the FDA’s review process. This avenue has many benefits. It accelerates the process for the companies that are seeking approval as the FDA can afford greater resources, it benefits the taxpayers as the FDA is then subsidized by those that use its service to review potential products, and it helps those with medical conditions that may benefit from a new drug or class of therapy coming to market sooner as a result of the FDA having greater resources.

The first step is pre-clinical testing in animals for indications of effectiveness and toxicity in a laboratory. If satisfactory, it clears the way for the company to submit an investigational new drug application (IND) to the FDA. The overriding goal of pre-clinical testing is to demonstrate that the product safe to then be tested in humans. The IND application outlines what the sponsor of the new drug proposes for human testing in clinical trials. Once reviewed and granted the company can move to clinical trials.

Clinical Trials

Clinical Trials are done in three phases designed to determine the drug candidate’s safety, characterization, and proof of efficacy.

Phase 1 studies (typically involves 20 to 80 people).

This phase involves testing the drug candidate on a small group of healthy volunteers to assess the drug’s safety and determine the appropriate dosage range. The primary goal is to verify safety and to identify any potential side effects.

Phase 2 studies (typically involve a few dozen to about 300 people).

This phase involves testing the drug on a larger trial group of patients that have the condition the drug is intended to treat. In this phase, the developer determines the drug’s efficacy, optimal dosage, and potential side effects. The primary goal is to assess and characterize the drug’s effectiveness in treating the targeted condition. Stocks will sometimes move on Phase 2 effiacy results.

Phase 3 studies (typically involve several hundred to about 3,000 people).

This final clinical study phase involves testing the drug on an even larger and intentionally diversified group of patients with the very condition the drug is intended to treat. These clinical trials are randomized and controlled to confirm the drug’s safety and efficacy in comparasin to existing treatments, a placebo, or both. The primary goal is to demonstrate statistically significant benefit, as defined by the trial parameters.

The announcement of Phase 3 results is a huge milestone, and by itself ordinarily impacts a stock’s price.

According to the Congressional Budget Office (CBO) only about 12 percent of drugs entering clinical trials are ultimately approved for introduction by the FDA. But it is costly; estimates of the average R&D cost per new drug range from less than $1 billion to more than $2 billion per drug. So in addition to being expensive, it’s an uncertain process – many potential drugs never make it to market. This is why full FDA approval, which isn’t automatic after a successful Phase 3 clinical trial, can create an huge upswing, even when expected.

Several things can go wrong during the three phases; these include unexpected side effects or toxicity, lack of efficacy, or failure to meet the primary endpoints of the clinical trial. The developer may even find that it is less effective than current medications. These issues can lead to delays in the approval process, additional studies, or even the termination of the drug’s development.

However, if the clinical trials are successful, the company is ready to file a New Drug Application with the FDA.

FDA Panels are experts with knowledge specific to what is being reviewed (Source: FDA)

New Drug Application (NDA)

There is a pre-NDA period, just before a new drug application is submitted to the FDA. At this time the company may seek guidance from the FDA on the new drug process.

The Submission of an NDA is the formal step that asks the FDA to consider the drug for approval to market. The FDA then has 60 days to decide whether the application gets filed for review. If the FDA files to review the NDA, an FDA review team is assigned to evaluate the sponsor’s research on the drug’s safety and effectiveness.

The FDA review includes a product label approval which includes how the drug can be used. This is very important because the drug can only be marketed within the label indications. The FDA also will inspect the facilities where the drug will be manufactured as part of the approval process.

FDA reviewers will either approve the application or instead issue a complete response letter.

PDUFA Calendar

The FDA PDUFA calendar is a schedule of dates for upcoming PDUFA decisions. These dates are important to investors in biotech and pharmaceutical companies because they represent the time period when the FDA will make a decision about a new drug application. If a drug is approved, it can eventually generate significant revenue for the company, while rejection can lead to a decline in the stock price as investors are disappointed.

Updates direct from the FDA on their calendar and meeting schedule can be subscribed to here.

Advisory Panel

In addition to PDUFA dates, there are other FDA events that can trigger movement in biotech and pharmaceutical stocks. These events include advisory committee meetings, which are meetings where a panel of experts provides recommendations to the FDA on whether to approve a drug or not. These meetings can provide insight into the FDA’s thinking and can influence the stock price.

A schedule of FDA Advisory Panel meetings can be found here.

Advisory committees make non-binding recommendations to the FDA, which generally follows the recommendations but is not legally bound to do so.

Other events that can impact the stock price include Complete Response Letters (CRLs), which are letters from the FDA that outline deficiencies in a drug application and can delay approval. Additionally, FDA inspections of manufacturing facilities can impact the stock price if there are concerns about quality control or manufacturing processes.

Take Away

Investors looking to grow their watch list to include biotech stocks that are in line to receive positive news that could drive the stock value way up or even disappointing news that would weigh on the price, could pay attention to the FDA approval process.

The process is an important tool for biotech and pharmaceutical companies, investors, and analysts. PDUFA dates represent the time when the FDA will make a decision about a new drug application, and can have a significant impact on the stock price. However, there are other FDA events that can also impact the stock price, such as advisory committee meetings, CRLs, and manufacturing facility inspections. It is helpful to stay informed about these events to make knowledgeable investment decisions in the biotech and pharmaceutical industry.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.fda.gov/advisory-committees/advisory-committee-calendar/april-28-2023-meeting-oncologic-drugs-advisory-committee-meeting-announcement-04282023

https://www.fda.gov/drugs/information-consumers-and-patients-drugs/fdas-drug-review-process-continued#:~:text=Phase%201%20studies%20(typically%20involve,application%20(NDA)%20is%20submitted.

https://www.fda.gov/about-fda/fda-track-agency-wide-program-performance/fda-track-pdufa-meeting-management#subscribe

Biotech Oncology Stocks Have Been Doing Well, Here’s Why

Posted on by phoffman@channelchek.com
Image: Rendering of folate receptors on a cancer cell

Understanding ImmunoGen’s Great Performance, and Related Stocks

Discovering a company developing a novel and more effective mechanism or method of doing something, and then investing in shares, is one reason investors pay attention to small-cap stocks. Innovations that improve results of any kind are valuable and usually rewarded. Nowhere is this more true than in biotech or biopharma stocks. After all, better treatments for frightening diseases will always be in demand. However, the big difference between the biotech industry and say, computer technology, is the approval process. FDA requirements are many and approval is slow and uncertain – overall, it’s a high bar to overcome.

Is it Worth it for Investors?

Over the past two days, ImmunoGen (IMGN:Nasdaq) a U.S. based clinical-stage biotech company, has had the kind of moonshot trajectory that investors dream about. The company reported promising topline phase III data and overall survival benefits in folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer patients. Immunogen plans to submit the drug for full approval in the U.S. and Europe. The company’s therapy is a is a first-in-class ADC comprising folate receptor alpha-binding antibody. The stock during the first four days of this week is up over 145%. The reason for the sudden moonshot is the company announced that it expects full FDA approval of one of its ADC candidates (Elahere). ADC, or antibody-drug conjugates, are a very targeted way to treat some solid tumor cancers, and seem to represent the “more effective mechanisms or method of doing something” mentioned above as sought after by small-cap investors.

Excitement Over ADC

An antibody-drug conjugate consists of an antibody that targets a specific antigen or receptor on cancer cells, it carries with it an impactful anticancer drug. The antibody which is linked to a toxin such as a chemotherapy drug, is found by folate receptors on the cancer cells; they will bind with the receptors on the cancer cells, the toxic payload is then delivered to the cells, which internalize it. Once in the cancer cell, the toxin is released. This therapy is designed to result in the selective killing of cancer cells while minimizing damage to healthy cells. ADCs have continued to show promising results in treating various types of cancer and are an active area of research by a few publicly traded small-cap biotechs developing alternatives in oncology.

Stock Market Behavior

As with other industries, the stocks of the peer group will often respond to news of the other. This was the case this week for the subgroup of stocks that are in various stages of researching ADC therapies against cancer.

As the chart below indicates, since May 1, the S&P 500 sank by more than 1.00%, yet cancer research biotech, which is not highly correlated to the overall market, rewarded investors in companies working with ADC technology for better cancer outcomes.

Source: Koyfin

ADC Companies that Rallied this Week

Among the stocks that seemed to have gotten a boost from Immunogen’s good news are:

Ambrx Biopharma (AMAM:Nasdaq) is a clinical-stage biologics company. The company’s lead product candidate is ARX788, an anti-HER2 antibody-drug conjugate (ADC), which is being investigated in various clinical trials for the treatment of breast cancer, gastric/gastroesophageal junction cancer, and other solid tumors.  

Mersana Therapeutics (MRSN:Nasdaq) is a clinical-stage biopharmaceutical company developing antibody-drug conjugates (ADC) for cancer patients with unmet needs.

Vincerx Pharma (VINC:Nasdaq) is a four-year-old clinical-stage biopharmaceutical company. VIP236, a small molecule drug conjugate that is in Phase 1 clinical trials to treat solid tumors. The company’s preclinical stage product candidates include VIP943 and VIP924 for the treatment of hematologic malignancies.

Sutro Biopharma (STRO:Nasdaq) is a clinical-stage oncology company that develops site-specific and novel-format antibody-drug conjugates (ADC). The company’s candidates include STRO-001, an ADC directed against the cancer target CD74 for patients with multiple myeloma and non-Hodgkin lymphoma, an ADC directed against folate receptor-alpha for patients with ovarian and endometrial cancers, which is in Phase 1 clinical trials.

As these biotech companies that are focused on ADC cancer treatment move their products through clinical trials, each success (or failure) is likely to impact the group. Not yet in the publicly traded group, but also being watched by those involved with ADC cancer stocks is OS Therapies.

OS Therapies (OSTX) is a U.S.-based biotech company that is developing therapies to treat specific cancers. The company completed its filing with the SEC last month to go public through an initial public offering (IPO). The biotech company hopes to list its shares on the NYSE American and trade under the symbol OSTX. It is a clinical-stage phase II biopharmaceutical company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) and other solid tumors. There have not been any new treatments approved by the FDA for Osteosarcoma for more than 40 years.

The lead core product candidates OS Therapies is researching are OST-HER2 and the OST-Tunable Drug Conjugate (OST-TDC) platform. The company says it intends to expand its pipeline beyond osteosarcoma into solid tumors. The OST-Tunable Drug Conjugate (OST-TDC) platform could deliver the next-generation ADC technology with the intent of providing a more potent drug and better efficacy with an improved safety profile, a potential “Best-in-Class”. Importantly, OS Therapies lead ADC drug will target folate receptor alpha-binding utilizing a small molecule ligand the same druggable target to Immunogen’s (IMGN) folate receptor alpha-binding site which is something that could become extremely notable to investors and larger pharmaceutical companies. Immunogen has now proved that folate receptor alpha-binding site can work.

The next generation ADC, according to the company filing, will be targeting ovarian, lung and pancreatic cancers. “Tunable” is a term used in drug development that refers to the properties that can be influenced by chemical modifications, and “antibody-drug conjugate.”

An IPO date for OS Therapies has not yet been confirmed.

Take Away

Stocks tend to trade up or down depending on the mood of the market. The current mood is that the overall market may still be overpriced. As such, 2023 has been marked by the bulls and bears duking it out – without any clear direction.

Biotech stocks tend to be far less correlated to what is going on in other areas of the market. This makes the sector and various peer groups worth a visit in bad markets. For example, when the pandemic began to unfold, many biotech stocks rocketed during the same period the overall market was crashing.

Within biotech, companies those working on the production of related technology typically trade in rough tandem with each other. Biotech stocks developing ADC, presumed to be a breaktrough in treating many types of cancers, have gotten a lift in anticipation of the imminent success of one of their peers.  

To do a deeper dive into small-cap names, scroll up to the search bar found next to the Channelchek logo, then enter a company name, ticker, or other keyword.  

Paul Hoffman

Managing Editor, Channelchek

Source

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10137214/

https://www.sciencedirect.com/topics/medicine-and-dentistry/folate-receptor#:~:text=Folate%20receptors%20(FRs)%20are%20membrane,breast%2C%20bladder%2C%20and%20brain.

https://www.nature.com/articles/s41416-022-02031-x

https://www.elahere.com/

https://www.sec.gov/Archives/edgar/data/1795091/000121390023025493/fs12023_ostherapies.htm#T99001

Are Select Biotech Stocks on the Launchpad?

Posted on by phoffman@channelchek.com
Image Credit: Hagerstown CC (Flickr)

Why Biotech Stocks Experience Significant Price Moves

Has biotech reached the tipping point? Shares of PromethiusBiosciences (RXDX) are up 70% this week after Merck (MRK) announced on Sunday it will pay a 75% premium for the clinical-stage biotech company. This follows last month’s big news when Pfizer announced its intention to acquire Seagen (SGEN), on the same day Sanofi (SNY) announced plans to buy Provention Bio (PRVB). This sent PRVB shares skyrocketing 258%. The question for biotech investors now is, who’s next?

Drug discovery and development is a long, uncertain path that often takes 10–15 years, and costs that could exceed $1–2 billion for any new drug ultimately approved for clinical use. Unlike unregulated products, it’s a significant achievement for a candidate to get as far as clinical trials. Promethius and Provention are both clinical-stage biotech companies. Fortunately for investors, each step along the path to success, whether or not it leads to a product going to market, can cause a significant swing in the stock’s price.

Small-cap biotech stocks can experience these significant price jumps from a variety of events. During the period of research and development, a drug on the path toward success or failure will have other significant events both positive and negative, that will impact the stock price in ways not found in other industry sectors. Here are some examples:

Positive clinical trial results: When a small-cap biotech company releases positive clinical trial results, it can generate significant investor interest and drive up the stock price.

Acquisition rumors or deals: When rumors or announcements of an acquisition by a larger company circulate, it can cause a small-cap biotech stock to rise as investors anticipate a potential buyout premium.

FDA approvals: FDA approvals of drugs or medical devices can significantly boost a small-cap biotech company’s stock price, as it can open up a new revenue stream for the company.

Partnerships and collaborations: Partnerships and collaborations with larger companies can also cause a small-cap biotech stock to rise as it indicates a level of validation for the company’s technology or products.

Analyst upgrades: If an influential analyst upgrades their rating on a small-cap biotech stock, it can increase investor interest and drive up the stock price.

Companies You May Want to Watch

There is information on well-over 200 small-cap biotech companies on Channelchek. Below is a select group that investors may want to follow.  

Cocrystal (COCP): Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses, hepatitis C viruses and noroviruses.

Axcella (AXLA): Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using endogenous metabolic modulator compositions. The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to reset multiple biological pathways, improve cellular energetics, and restore homeostasis.

Tonix Pharmaceutical (TNXP): Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system, rare disease, immunology and infectious disease product candidates.

Onconova Therapeutics (ONTX):   Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

MAIA Biotechnology (MAIA):   MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of cancer patients.

PDS Biotechnology (PDSB):    PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on proprietary T cell-activating technology platforms.

Ocugen (OCGN): Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. The company impacts patient’s lives through innovation that forge new scientific paths.

Take Away

Biotech stocks, especially small-caps have come well off the pandemic era, sky-high price levels they had attained. At the same time, large pharmaceutical companies that are still seeing sales from covid related products have ample cash and are faced with patents that are always inching closer to expiration. Acquisitions tend to cause huge price spikes in all industries, especially biotech.

While pharmaceutical companies will be picky as they may work to own patents on their next generation of product offerings, many other positive (and negative) occurrences could impact the stock price. Channelchek provides current information on small-cap biotech stocks so that investors can determine the likelihood of success and to help them steer from negative investment results.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.channelchek.com/search-new?search=biotech

https://www.fiercebiotech.com/biotech/big-pharma-resisting-temptation-biotech-ma-until-prices-drop-further

https://seekingalpha.com/article/4594550-catalyst-pharmaceuticals-a-one-hit-wonder-with-upside

Investing in the Development of Cancer Drugs May be Considered Defensive

Posted on by phoffman@channelchek.com
Image: Visualization of a cancer cell (nucleus in blue) treated with bi-modular fusion proteins (BMFPs). BMFPs bind to an antigen on the surface of the cancer cell to be destroyed. – Inserm (Flickr)

Understanding the Cancer Treatment and Therapy Segments of Biotech

Transforming research discoveries into new cancer treatments takes investment in time and capital. But no one would argue that the end goals of this healthcare (biotech and pharma) sector are not worth it. Investing in the future of treating tumors, and preventing cancer growth is obviously rewarding from the human standpoint of saving life, but breakthroughs in oncology themselves could provide an investor that understands some of the more promising companies, oversized portfolio rewards. It goes without saying, knowledge and understanding of many companies at different stages of research and development, help the odds of being invested in successful stocks.  

Exploding Growth

According to a report by Grand View Research published in early last year, the global oncology drugs market size was valued at $135.7 billion in 2021 and is expected to grow at a compound annual growth rate (CAGR) of 8.3% from 2022 to 2028. The increasing prevalence of cancer, the rising geriatric population, and advancements in drug development and treatment modalities are some of the key factors driving the growth of the novel oncology drugs market.

Relief managing the side effects of treatments, especially chemotherapy, without opiods is also a part of this market. Changing treatment modalities, and growing demand for personalized medicine is still relatively new, and creating more growth opportunities. In addition, a continuing trend of mergers and acquisitions from pharmaceutical companies to expand their oncology drug pipelines and portfolios plays a part in the growth of this sector.  

Meet the management of Onconova (ONTX) in NY,NY for lunch on March 28. This is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. To request attendance, click the registration link here.

Where to Explore Cancer Treatment/Therapy Companies

Investors use Channelchek as one of their trusted outlets to discover and explore smaller public companies involved in oncology treatments and therapies. Below are five companies with a wealth of information housed on the platform. This includes high quality research and video content. For an expanded list of companies, a simple search on Channelchek under “Oncology” or “Cancer” will provide a wealth of more opportunities to discover.

Worth a Deeper Dive?

Onconova Therapeutics Inc. (ONTX) is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. It has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China. Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer. For more information, please visit www.onconova.com.

Onconova has a roadshow scheduled on March 28 in Manhattan, NY. More information on attending the lunch is available here.

Genprex, Inc. (GNPX) is a clinical-stage gene therapy company focused on developing therapies for patients with cancer and diabetes. Its technologies are designed to administer disease-fighting genes to provide new therapies for populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex’s oncology program utilizes its proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans. ONCOPREX encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is then taken up by tumor cells that express tumor suppressor proteins that are deficient in the body. The Company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC) (with each of these clinical programs receiving a Fast Track Designation from the Food and Drug Administration) and for small cell lung cancer. Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an endoscope and an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes to the pancreas. In models of T1D, the genes express proteins that transform alpha cells in the pancreas into functional beta-like cells, which can produce insulin but are distinct enough from beta cells to evade the body’s immune system. In T2D, where autoimmunity is not at play, it is believed that exhausted beta cells are also rejuvenated and replenished.

In 2022 Genprex was one of the more popular presenters at the NobleCon investor conference. A video replay of its presentation is available here.

Imugene Ltd. (IUGNF) is a clinical stage immuno-oncology company developing a range of new and novel immunotherapies that seek to activate the immune system of cancer patients to treat and eradicate tumours. Our unique platform technologies seek to harness the body’s immune system against tumours, potentially achieving a similar or greater effect than synthetically manufactured monoclonal antibody and other immunotherapies. Our product pipeline includes multiple immunotherapy B-cell vaccine candidates and an oncolytic virotherapy (CF33) aimed at treating a variety of cancers in combination with standard of care drugs and emerging immunotherapies such as CAR T’s for solid tumours. We are supported by a leading team of international cancer experts with extensive experience in developing new cancer therapies with many approved for sale and marketing for global markets.

For more data and information, visit immunogen on Channelchek.

MAIA Biotechnology Inc. (MAIA)  is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. Noble Capital Markets initiated coverage of MAIA on February 21, 2023. A copy of the report can be found here.

PDS Biotechnology Corporation (PDSB) is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune® and Infectimune™ T cell-activating technology platforms. We believe our targeted Versamune® based candidates have the potential to overcome the limitations of current immunotherapy by inducing large quantities of high-quality, potent polyfunctional tumor specific CD4+ helper and CD8+ killer T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the potential to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV-positive cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

As part of the Channelchek TakeAway Series, Senior Life Sciences Analyst, Robert LeBoyer sat down with management and discussed PDS Bio, listen to the discussion, including questions from the audience here.

Take Away

In the investment arena, oncology is a growing part of the healthcare sector, specifically the biotechnology and pharmaceutical segments. Companies that develop and market oncology drugs or provide related services are viewed as uncorrelated to other sectors. The demand for the next generation of improved treatments is expected to be ongoing.  While the approval process and regulatory bottlenecks of biotech are unlike any other product category, there are many reasons to review and consider this largely uncorrelated sector – then  dig deeper to possibly cancer R &D.

For the smaller companies considered to have the most potential, a good starting point is Channelchek where you’ll find articles, research, videos, and data, all in one place.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.marketwatch.com/press-release/oncology-drugs-market-share-and-forecast-till-2028-2023-03-20

https://investingnews.com/daily/life-science-investing/biotech-investing/top-oncology-companies/

Biotech Announcement Sends Stock Up 258%

Posted on by phoffman@channelchek.com
Image Credit: Bradley Johnson (Flickr)

The Power of Small Companies Highlighted in Today’s Biopharma Announcements

Business headlines surrounding Silicon Valley Bank and its customers may take some time to fade from the front page. In the meantime, looking past them, there are some positive news and developments. Two news items involve announcements by biotech/pharmaceutical companies this week. One is a deal you don’t have to dig too deep to find, Pfizer (PFE), the pharmaceutical behemoth, is looking to acquire Seagen (SGEN) for $43 billion. The second is a smaller deal and has been crowded off many newsfeeds. Provention Bio (PRVB) is expected to be purchased by Sanofi (SNY) a large French-based pharmaceutical company.

Seagen shares increased 17% in the first hour of trading after the Pfizer announcement, shares of Provention were up 258% the same morning after the Sanofi announcement. Below is a chart of the month-to-date performance of the two that are to be acquired.

Source: Koyfin

The Power of Flying Below the Radar

Seagen is a borderline household name and has been a known acquisition target for some time. Just last July, Merck offered 40 billion for the company, this known interest in the company has kept the price elevated. Shifting the focus on the power of smaller, less talked about companies, they often have more potential for larger gains because they are less known. And while the numbers ($43 billion vs $2.9 billion) don’t make for compelling headlines, the numbers in the graph above demonstrate the impact can be far more compelling to investors.

The Provention Bio Deal

Sanofi and Provention Bio, a U.S.-based, publicly traded biopharmaceutical company focused on preventing autoimmune diseases, including type 1 diabetes (T1D), entered into an agreement for Sanofi to acquire Provention Bio, Inc., for $25.00 per share in cash.

Under the terms of the agreement, Sanofi will begin a cash tender offer to acquire all outstanding shares of Provention Bio.

The actual completion of the tender is subject to standard conditions, including the tendering of a number of shares of Provention Bio, Inc. common stock that, together with shares already owned by Sanofi or its affiliates, represents at least a majority of the outstanding shares of Provention Bio, Inc. common stock.

If the tender offer is successfully completed, then a wholly owned subsidiary of Sanofi will merge with and into Provention Bio, Inc., and all of the outstanding Provention shares that are not tendered in the offer will be converted into the right to receive the same $25.00 per share in cash offered to Provention Bio, Inc. shareholders as part of the offer. Sanofi plans to fund the transaction with available cash. Subject to the satisfaction or waiver of customary closing conditions, Sanofi expects to complete the acquisition in the second quarter of 2023.

Worth Noting

The largest pharmaceutical companies developed huge cash “war chests” during the pandemic era. While they are prudent and tactical when deciding to grow through acquisition, the earnings on much of their cash stockpiles relative to inflation may be erosive to the pool’s purchasing power. Additionally, many small pharmaceutical and biotech companies that are developing tomorrow’s next wonder drugs are short the cash they need to drive their R&D to the finish line, and then to market. It’s presumed these companies are quietly being reviewed for a possible fit by big pharma. Big pharma’s current patents are also being eroded by time as each day they approach patent expiration. This is added incentive for these large companies to be actively looking for future merger and acquisition targets.

Smaller companies, for their part want their progress and potential more known. It is only through being known, and the more broadly the better, that investors of all types understand the work they do and the potential along with the risk they hold. These companies often hire the service of impartial, highly credible equity analysts to provide details of the pipeline and the successes and challenges of the company. This company-sponsored research provides investors with a third-party window into the company. The window is, at times, as basic as the idea that investors need to know enough about the existence of a small company to want to own shares. Greater investor interest typically increases liquidity which could help the company continue moving forward and developing its products.

Channelchek houses quality company-sponsored research. For Life Sciences company-sponsored research covered by FINRA licensed Sr. Analyst Robert LeBoyer visit this link. For Healthcare Services Sr. Analyst Gregory Aurand visit this link.

Paul Hoffman

Managing Editor, Channelchek

What to Look for in a Biotech Stock

Posted on by phoffman@channelchek.com
Image Credit: Marco Verch (Flickr)

Steps to Discover Which Biotech Stocks May Get Hot

The biotech sector is in and of itself exciting. A company developing an idea that can improve human lives, decrease suffering, or even prevent death, by nature, could be a more rewarding endeavor than investing in a company that, by comparison, does little to make a big difference. If, at the same time, the opportunity to return the investor a multiple over returns available elsewhere in the market, then the motivation to allocate a portion of investment capital increases dramatically. But how does an investor gauge a company in the biotech sector and evaluate its chance of survival and likelihood of success?

As with much of investing, sure things don’t often provide a good return. And adding risk doesn’t necessarily equate to added return. The higher perceived risk of a sector such as biotech needs to be offset by research. Filtering stocks through a selection process is key, so the probability of picking those that survive and thrive is higher than average.

I spoke with Robert LeBoyer, the Senior Life Sciences Analyst at Noble Capital Markets, and asked him to list factors to improve the likelihood of choosing a successful biotech company. His knowledge and enthusiasm for the sector caused me to want to share what I learned.

Differentiation

Companies developing a drug that is different than all that came before for what it proposes to treat or prevent stand a good chance of getting funding to make it through the different phases of study. With a development time of 3-5 years, it is best if there is a clear unmet need for the therapy or no current therapies at all.

Investors should determine if there are treatments presently and ask whether the drug or treatment mechanism is a significant improvement over any current product. Also is the field crowded or will it soon be crowded with alternatives to what a company may bring to market? LeBoyer recommended asking where there is an improvement. He gave the example of many cholesterol-lowering drugs, which he said all target the same enzyme. A company with a drug that similarly targets that enzyme may not be worth exploring. Learning of a company that has a different mechanism of action, one which shows promise of greater efficacy, or, reduced cost, or fewer side effects may be worth exploring further.

As an example of a company that had met these criteria some years back, Gilead received approval for a once-daily tablet to treat hepatitis C. Prior to this, the only treatment options involved a year-long regimen of weekly interferon injections and ribavirin tablets. The side effects were depression, nausea, flu-like symptoms, and a reduction in some blood cells. The cost of the injections and treatments could cost a health insurer $1 million over the life of the patient. The Gilead treatment, which has a price tag of $93,000, is mathematically more cost-effective. The therapy which Gilead got approved in the U.S. in 2013 was a better treatment than what existed, and even better tolerated by patients. The stock went from $20 to $120 in about a years time after approval.

Development

Clinical development was another attribute brought up by Noble’s analyst. With even the best proof of concept or early-stage trial success, assessing the chance that clinical stage trials may fail for pipeline candidates is difficult.  This is why a company with a diverse pipeline with a number of products being developed or in later stages of clinical trials, increases the probability of successful biotech investment. Many companies easily pass stage one trials and even stage two, but don’t get past the final hurdle. LeBoyer shared with me a story of a company he now covers that had a vaccine for Covid-19 early on. The human clinical trials, however, were not done in the U.S., but were instead the result of trials on persons mostly of similar lineage. The FDA required a sampling comparable to the diversification of heritage or gene pool in the U.S.  

Obtaining a basic understanding of the FDA side of development is important for anyone making decisions on biotech stocks.

The drug approval process in the U.S. involves multi-layered (Step One through Step Four) with each representing an important milestone on the path to full approval so the product can be brought to market or meet rejection along the path.

Step One is the development phase, Step Two is research, Step Three is Clinical Trials, and Step Four is FDA Approval.

Knowing where companies stands in the FDA step process can help an investor assess the likelihood of approval. Many products, can get to the last step and not be approved, but those just starting out on Step One are a greater risk both in the time it will take and the chance for something to not be to the FDA’s liking.

Finances

Biotech companies, by and large burn through cash in their research, development and trial periods. Understanding how long the cash on hand and other available sources can last before they need to raise more cash, then comparing this with how close to an expected finish line they are, could help an investor steer away from a company that may have a product in the pipeline that meets other key elements investors should look at, but unfortunately, funds may stand in the way of success.

Robert LeBoyer explained that the current high-interest rate environment, coupled with depressed stock prices, makes this particularly important now. For those companies that can borrow, the cost of money is now far more expensive than it had been in the last decade and a half. And issuing more shares, essentially selling more of the company, dilutes the value of shares currently held. It could become a tricky situation that stockholders or those deciding to become a stockholder should monitor

Take Away

Are there companies with a pipeline that includes drugs that meet a large unmet need (as one example, Alzheimer’s), or can attack a disease like cancer in a unique way that would be embraced by the medical community and patients? “Unicorn” companies do exist, but finding them, assessing them, and doing it before a louder investment buzz occurs takes some digging. A solid place to start digging is under the biotech company section on Channelchek.  Available by clicking here, here investors are exposed to many opportunities and the underlying data, the latest news, and of course, thorough company descriptions.

Biotech companies covered by analyst Robert LeBoyer, along with his current research are available here. Channelchek will be highlighting interesting biotechs in future articles and discussing their work and status against the criteria presented above. Join Channelchek to receive emails and gain free access to these articles, video presentations, updated research reports, and news of company roadshows. Visit Rob LeBoyers coverage list here.

Paul Hoffman

Managing Editor, Channelchek

Was US Pharma Clinical Trial Activity Thwarted During the Pandemic?

Posted on by phoffman@channelchek.com
Image Source: NIH (Flickr)

The Volume Change of Non-Covid Related Medical Studies During the Pandemic

Did Covid19 related efforts by the pharmaceutical and biotech industry pull dollars from or impede the progression of non-Covid medical research? Also, were patient-enrolled studies significantly curtailed or paused during this period? Answers to these questions had been hard to quantify. Public estimates have ranged from the virus as having a minimal impact all the way to the other extreme of tragic decline. Applied Clinical Trials, an industry publication, has found a unique and accurate source from which to remove the guesswork and mine for a conclusion. From this, they were able to come to a definitive conclusion that surprised both extremes in expectations.

Statistical Sources

The US government’s Open Payments program is a national database that discloses payments made by drug and medical device companies to hospitals, physicians, and others in the health service provider field. The purpose of the data is to provide transparency; however, it has ample information to do analysis and provide conclusions on other questions related to clinical research or the broader medical arena.

Applied Clinical Trials discovered from Open Payments that the effect on the total volume of US clinical trial activity was quite limited. The statistics reveal that the overall pharmaceutical industry spending on all US patient enrollment and treatment activities did not decline in the years 2020 and 2021, from the year 2019, which was used as a baseline.

How the Data Was Used

The Open Payments database allowed direct measurement of the impact of COVID-19 on US industry-sponsored clinical trial activity through the end of 2021. More recent data is not yet available. The researchers learned Covid19 may have slowed and hindered the launch and execution of clinical trials in two ways. First, COVID-19 clinical trials may have displaced other clinical trial activity. Second, the Covid19 pandemic caused logistical and operational challenges to most clinical trials. Do you remember the six-foot rule? Recruiting patients, treating these patients, and validating source data are some of the areas where the pandemic created more than the normal amounts of hurdles for clinical trials.

The needed data was isolated by coding Open Payments by individual study indication. Also the U.S. National Library of Medicine (under the NIH) maintains the website clinicaltrials.gov. This website shows little or no decline in the number of US sites opened for Phase II and III clinical trials between 2019, 2020, and 2021. Of course open sites may reveal the amount of patient activity taking place. A Site may be opened, but have less patient activity. Therefore assessing actual activity levels from the ClinicalTrials.gov database is nt perfect. However, Open Payments provides more complete data since the payments are predominately tied to patient enrollment and treatment experienced.

Pharmaceutical company US clinical research spending, reported in Open Payments ($billions)

Overall Conclusion

The research shows there was no decline in non-COVID related study spending during the height of pandemic. Open Payments data show a constant, if undramatic, increase in US clinical trials between 2017 and 2021. Even when Covid19 related spending during the time period was removed, they saw no decline in study spending. Trials continued, through the period, at a rate that is similar to the year just prior to the start of the pandemic.

More About Open Payments

Open Payments is the result of a federal law, the Sunshine Act. Searches and downloads on the website are easy and may only be slowed by the size of many of the files. The data may not reach back beyond ten years because the site has only existed since late 2013. Since, pharmaceutical companies with at least one marketed product have been required to report payments to physicians and other medical professionals. These Open Payments include two types: general and research. General payments relate to pharmaceutical companies’ payments to medical professionals when marketed products are involved. Research payments are more restricted. They are broken down by clinical grant payments. These cover virtually all clinical investigators and their clinical trial experience across all indications.

Investor Take Away

Investors had been concerned that the pipeline at companies manufacturing medical devices and developing drugs, therapies, and other treatments may have a less full pipeline because of the pandemic and the US response to it. While the data doesn’t speak to the speed of the FDA approval process, it alleviates concerns that the related investment sectors were on hiatus and now behind on phase II and phase III trials.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.appliedclinicaltrialsonline.com/view/the-limited-impact-of-covid-19-on-us-clinical-trial-activity

https://openpaymentsdata.cms.gov/

“Self-Boosting” Vaccines for a Myriad of Applications

Posted on by phoffman@channelchek.com
Image: Second Bay Studios

Microparticles Could be Used to Deliver “Self-Boosting” Vaccines

Anne Trafton | MIT News Office  

Most vaccines, from measles to Covid-19, require a series of multiple shots before the recipient is considered fully vaccinated. To make that easier to achieve, MIT researchers have developed microparticles that can be tuned to deliver their payload at different time points, which could be used to create “self-boosting” vaccines.

In a new study, the researchers describe how these particles degrade over time, and how they can be tuned to release their contents at different time points. The study also offers insights into how the contents can be protected from losing their stability as they wait to be released.

Using these particles, which resemble tiny coffee cups sealed with a lid, researchers could design vaccines that would need to be given just once, and would then “self-boost” at a specified point in the future. The particles can remain under the skin until the vaccine is released and then break down, just like resorbable sutures.

This type of vaccine delivery could be particularly useful for administering childhood vaccinations in regions where people don’t have frequent access to medical care, the researchers say.

“This is a platform that can be broadly applicable to all types of vaccines, including recombinant protein-based vaccines, DNA-based vaccines, even RNA-based vaccines,” says Ana Jaklenec, a research scientist at MIT’s Koch Institute for Integrative Cancer Research. “Understanding the process of how the vaccines are released, which is what we described in this paper, has allowed us to work on formulations that address some of the instability that could be induced over time.”

This approach could also be used to deliver a range of other therapeutics, including cancer drugs, hormone therapy, and biologic drugs, the researchers say.

Jaklenec and Robert Langer, the David H. Koch Institute Professor at MIT and a member of the Koch Institute, are the senior authors of the new study, which appears today in Science Advances. Morteza Sarmadi, a research specialist at the Koch Institute and recent MIT PhD recipient, is the lead author of the paper.

Staggered Drug Release

The researchers first described their new microfabrication technique for making these hollow microparticles in a 2017 Science paper. The particles are made from PLGA, a biocompatible polymer that has already been approved for use in medical devices such as implants, sutures, and prosthetic devices.

To create cup-shaped particles, the researchers create arrays of silicon molds that are used to shape the PLGA cups and lids. Once the array of polymer cups has been formed, the researchers employed a custom-built, automated dispensing system to fill each cup with a drug or vaccine. After the cups are filled, the lids are aligned and lowered onto each cup, and the system is heated slightly until the cup and lid fuse together, sealing the drug inside.

This technique, called SEAL (StampEd Assembly of polymer Layers), can be used to produce particles of any shape or size. In a paper recently published in the journal Small Methods, lead author Ilin Sadeghi, an MIT postdoc, and others created a new version of the technique that allows for simplified and larger-scale manufacturing of the particles.

In the new Science Advances study, the researchers wanted to learn more about how the particles degrade over time, what causes the particles to release their contents, and whether it might be possible to enhance the stability of the drugs or vaccines carried within the particles.

“We wanted to understand mechanistically what’s happening, and how that information can be used to help stabilize drugs and vaccines and optimize their kinetics,” Jaklenec says.

Their studies of the release mechanism revealed that the PLGA polymers that make up the particles are gradually cleaved by water, and when enough of these polymers have broken down, the lid becomes very porous. Very soon after these pores appear, the lid breaks apart, spilling out the contents.

“We realized that sudden pore formation prior to the release time point is the key that leads to this pulsatile release,” Sarmadi says. “We see no pores for a long period of time, and then all of a sudden we see a significant increase in the porosity of the system.”

The researchers then set out to analyze how a variety of design parameters, include the size and shape of the particles and the composition of the polymers used to make them, affect the timing of drug release.

To their surprise, the researchers found that particle size and shape had little effect on drug release kinetics. This sets the particles apart from most other types of drug delivery particles, whose size plays a significant role in the timing of drug release. Instead, the PLGA particles release their payload at different times based on differences in the composition of the polymer and the chemical groups attached the ends of the polymers.

“If you want the particle to release after six months for a certain application, we use the corresponding polymer, or if we want it to release after two days, we use another polymer,” Sarmadi says. “A broad range of applications can benefit from this observation.”

Stabilizing the Payload

The researchers also investigated how changes in environmental pH affect the particles. When water breaks down the PLGA polymers, the byproducts include lactic acid and glycolic acid, which make the overall environment more acidic. This can damage the drugs carried within the particles, which are usually proteins or nucleic acids that are sensitive to pH.

In an ongoing study, the researchers are now working on ways to counteract this increase in acidity, which they hope will improve the stability of the payload carried within the particles.

To help with future particle design, the researchers also developed a computational model that can take many different design parameters into account and predict how a particular particle will degrade in the body. This type of model could be used to guide the development of the type of PLGA particles that the researchers focused on in this study, or other types of microfabricated or 3D-printed particles or medical devices.

The research team has already used this strategy to design a self-boosting polio vaccine, which is now being tested in animals. Usually, the polio vaccine has to be given as a series of two to four separate injections.

“We believe these core shell particles have the potential to create a safe, single-injection, self-boosting vaccine in which a cocktail of particles with different release times can be created by changing the composition. Such a single injection approach has the potential to not only improve patient compliance but also increase cellular and humoral immune responses to the vaccine,” Langer says.

This type of drug delivery could also be useful for treating diseases such as cancer. In a 2020 Science Translational Medicine study, the researchers published a paper in which they showed that they could deliver drugs that stimulate the STING pathway, which promotes immune responses in the environment surrounding a tumor, in several mouse models of cancer. After being injected into tumors, the particles delivered several doses of the drug over several months, which inhibited tumor growth and reduced metastasis in the treated animals.

Reprinted with permission from MIT News ( http://news.mit.edu/ )

3D-Printed Human Hearts (Patient Specific)

Posted on by phoffman@channelchek.com
Image Credit: Melanie Gonick, MIT

Custom, 3D-Printed Heart Replicas Look and Pump Just Like the Real Thing

Jennifer Chu | MIT News Office

No two hearts beat alike. The size and shape of the heart can vary from one person to the next. These differences can be particularly pronounced for people living with heart disease, as their hearts and major vessels work harder to overcome any compromised function.

MIT engineers are hoping to help doctors tailor treatments to patients’ specific heart form and function, with a custom robotic heart. The team has developed a procedure to 3D print a soft and flexible replica of a patient’s heart. They can then control the replica’s action to mimic that patient’s blood-pumping ability.

The procedure involves first converting medical images of a patient’s heart into a three-dimensional computer model, which the researchers can then 3D print using a polymer-based ink. The result is a soft, flexible shell in the exact shape of the patient’s own heart. The team can also use this approach to print a patient’s aorta — the major artery that carries blood out of the heart to the rest of the body.

To mimic the heart’s pumping action, the team has fabricated sleeves similar to blood pressure cuffs that wrap around a printed heart and aorta. The underside of each sleeve resembles precisely patterned bubble wrap. When the sleeve is connected to a pneumatic system, researchers can tune the outflowing air to rhythmically inflate the sleeve’s bubbles and contract the heart, mimicking its pumping action.

The researchers can also inflate a separate sleeve surrounding a printed aorta to constrict the vessel. This constriction, they say, can be tuned to mimic aortic stenosis — a condition in which the aortic valve narrows, causing the heart to work harder to force blood through the body.

Doctors commonly treat aortic stenosis by surgically implanting a synthetic valve designed to widen the aorta’s natural valve. In the future, the team says that doctors could potentially use their new procedure to first print a patient’s heart and aorta, then implant a variety of valves into the printed model to see which design results in the best function and fit for that particular patient. The heart replicas could also be used by research labs and the medical device industry as realistic platforms for testing therapies for various types of heart disease.

“All hearts are different,” says Luca Rosalia, a graduate student in the MIT-Harvard Program in Health Sciences and Technology. “There are massive variations, especially when patients are sick. The advantage of our system is that we can recreate not just the form of a patient’s heart, but also its function in both physiology and disease.”

Rosalia and his colleagues report their results in a study appearing today in Science Robotics. MIT co-authors include Caglar Ozturk, Debkalpa Goswami, Jean Bonnemain, Sophie Wang, and Ellen Roche, along with Benjamin Bonner of Massachusetts General Hospital, James Weaver of Harvard University, and Christopher Nguyen, Rishi Puri, and Samir Kapadia at the Cleveland Clinic in Ohio.

Print and Pump

In January 2020, team members, led by mechanical engineering professor Ellen Roche, developed a “biorobotic hybrid heart” — a general replica of a heart, made from synthetic muscle containing small, inflatable cylinders, which they could control to mimic the contractions of a real beating heart.

Shortly after those efforts, the Covid-19 pandemic forced Roche’s lab, along with most others on campus, to temporarily close. Undeterred, Rosalia continued tweaking the heart-pumping design at home.

“I recreated the whole system in my dorm room that March,” Rosalia recalls.

Months later, the lab reopened, and the team continued where it left off, working to improve the control of the heart-pumping sleeve, which they tested in animal and computational models. They then expanded their approach to develop sleeves and heart replicas that are specific to individual patients. For this, they turned to 3D printing.

“There is a lot of interest in the medical field in using 3D printing technology to accurately recreate patient anatomy for use in preprocedural planning and training,” notes Wang, who is a vascular surgery resident at Beth Israel Deaconess Medical Center in Boston.

An Inclusive Design

In the new study, the team took advantage of 3D printing to produce custom replicas of actual patients’ hearts. They used a polymer-based ink that, once printed and cured, can squeeze and stretch, similarly to a real beating heart.

As their source material, the researchers used medical scans of 15 patients diagnosed with aortic stenosis. The team converted each patient’s images into a three-dimensional computer model of the patient’s left ventricle (the main pumping chamber of the heart) and aorta. They fed this model into a 3D printer to generate a soft, anatomically accurate shell of both the ventricle and vessel.

The team also fabricated sleeves to wrap around the printed forms. They tailored each sleeve’s pockets such that, when wrapped around their respective forms and connected to a small air pumping system, the sleeves could be tuned separately to realistically contract and constrict the printed models.

The researchers showed that for each model heart, they could accurately recreate the same heart-pumping pressures and flows that were previously measured in each respective patient.

“Being able to match the patients’ flows and pressures was very encouraging,” Roche says. “We’re not only printing the heart’s anatomy, but also replicating its mechanics and physiology. That’s the part that we get excited about.”

Going a step further, the team aimed to replicate some of the interventions that a handful of the patients underwent, to see whether the printed heart and vessel responded in the same way. Some patients had received valve implants designed to widen the aorta. Roche and her colleagues implanted similar valves in the printed aortas modeled after each patient. When they activated the printed heart to pump, they observed that the implanted valve produced similarly improved flows as in actual patients following their surgical implants.

Finally, the team used an actuated printed heart to compare implants of different sizes, to see which would result in the best fit and flow — something they envision clinicians could potentially do for their patients in the future.

“Patients would get their imaging done, which they do anyway, and we would use that to make this system, ideally within the day,” says co-author Nguyen. “Once it’s up and running, clinicians could test different valve types and sizes and see which works best, then use that to implant.”

Ultimately, Roche says the patient-specific replicas could help develop and identify ideal treatments for individuals with unique and challenging cardiac geometries.

“Designing inclusively for a large range of anatomies, and testing interventions across this range, may increase the addressable target population for minimally invasive procedures,” Roche says.

This research was supported, in part, by the National Science Foundation, the National Institutes of Health, and the National Heart Lung Blood Institute.

Reprinted with permission from MIT News ( http://news.mit.edu/ )

Cultivating a Microbiome that Reduces the Incidence of Cancer

Posted on by phoffman@channelchek.com
Image Credit: NIH (Flickr)

Microbes in Your Food Can Help or Hinder Your Body’s Defenses Against Cancer – How Diet Influences the Conflict Between Cell ‘Cooperators’ and ‘Cheaters’

The microbes living in your food can affect your risk of cancer. While some help your body fight cancer, others help tumors evolve and grow.

Gut microbes can influence your cancer risk by changing how your cells behave. Many cancer-protective microbes support normal, cooperative behavior of cells. Meanwhile, cancer-inducing microbes undermine cellular cooperation and increase your risk of cancer in the process.

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of Gissel Marquez Alcaraz, Ph.D. Student in Evolutionary Biology, Arizona State University and Athena Aktipis, Associate Professor of Psychology, Center for Evolution and Medicine, Arizona State University.

We are evolutionary biologists who study how cooperation and conflict occur inside the human body, including the ways cancer can evolve to exploit the body. Our systematic review examines how diet and the microbiome affect the ways the cells in your body interact with each other and either increase or decrease your risk of cancer.

Cancer is a Breakdown of Cell Cooperation

Every human body is a symphony of multicellular cooperation. Thirty trillion cells cooperate and coordinate with each other to make us viable multicellular organisms.

For multicellular cooperation to work, cells must engage in behaviors that serve the collective. These include controlled cell division, proper cell death, resource sharing, division of labor and protection of the extracellular environment. Multicellular cooperation is what allows the body to function effectively. If genetic mutations interfere with these proper behaviors, they can lead to the breakdown of cellular cooperation and the emergence of cancer.

Cancer cells can be thought of as cellular cheaters because they do not follow the rules of cooperative behavior. They mutate uncontrollably, evade cell death and take up excessive resources at the expense of the other cells. As these cheater cells replicate, cancer in the body begins to grow.

Cancer is fundamentally a problem of having multiple cells living together in one organism. As such, it has been around since the origins of multicellular life. This means that cancer suppression mechanisms have been evolving for hundreds of millions of years to help keep would-be cancer cells in check. Cells monitor themselves for mutations and induce cell death, also known as apoptosis, when necessary. Cells also monitor their neighbors for evidence of abnormal behavior, sending signals to aberrant cells to induce apoptosis. In addition, the body’s immune system monitors tissues for cancer cells to destroy them.

Cells that are able to evade detection, avoid apoptosis and replicate quickly have an evolutionary advantage within the body over cells that behave normally. This process within the body, called somatic evolution, is what leads cancer cells to grow and make people sick.

Microbes Can Help or Hinder Cell Cooperation

Microbes can affect cancer risk through changing the ways that the cells of the body interact with one another.

Some microbes can protect against cancer by helping maintain a healthy environment in the gut, reducing inflammation and DNA damage, and even by directly limiting tumor growth. Cancer-protective microbes like Lactobacillus pentosus, Lactobacillus gasseri and Bifidobacterium bifidum are found in the environment and different foods, and can live in the gut. These microbes promote cooperation among cells and limit the function of cheating cells by strengthening the body’s cancer defenses. Lactobacillus acidophilus, for example, increases the production of a protein called IL-12 that stimulates immune cells to act against tumors and suppress their growth.

Other microbes can promote cancer by inducing mutations in healthy cells that make it more likely for cellular cheaters to emerge and outcompete cooperative cells. Cancer-inducing microbes such as Enterococcus faecalis, Helicobacter pylori and Papillomavirus are associated with increased tumor burden and cancer progression. They can release toxins that damage DNA, change gene expression and increase the proliferation of tumor cells. Helicobacter pylori, for example, can induce cancer by secreting a protein called Tipα that can penetrate cells, alter their gene expression and drive gastric cancer.

Healthy Diet with Cancer-Protective Microbes

Because what you eat determines the amount of cancer-inducing and cancer-preventing microbes inside your body, we believe that the microbes we consume and cultivate are an important component of a healthy diet.

Beneficial microbes are typically found in fermented and plant-based diets, which include foods like vegetables, fruits, yogurt and whole grains. These foods have high nutritional value and contain microbes that increase the immune system’s ability to fight cancer and lower overall inflammation. High-fiber foods are prebiotic in the sense that they provide resources that help beneficial microbes thrive and subsequently provide benefits for their hosts. Many cancer-fighting microbes are abundantly present in fermented and high-fiber foods.

In contrast, harmful microbes can be found in highly-processed and meat-based diets. The Western diet, for example, contains an abundance of red and processed meats, fried food and high-sugar foods. It has been long known that meat-based diets are linked to higher cancer prevalence, and that red meat is a carcinogen. Studies have shown that meat-based diets are associated with cancer-inducing microbes including Fusobacteria and Peptostreptococcus in both humans and other species.

Microbes can enhance or interfere with how the body’s cells cooperate to prevent cancer. We believe that purposefully cultivating a microbiome that promotes cooperation among our cells can help reduce cancer risk.

A Good Place to Start to Evaluate Specific Biotech Companies?  

Posted on by phoffman@channelchek.com
Image Credit: Andrea Piacquadio (Pexels)

Exploring and Discovering Biotech Stocks

When it comes to hand-selecting companies for investment, a critical ingredient for success is information. This ingredient becomes even more critical with biotech companies. Each year, many companies have been involved in medicine, medical equipment, genetics, and wellness that take off and provide investors with double or triple-digit gains. During the same years, there are stocks in the sector that, on the surface seem to have just as much going for them, yet a diligent peak below the surface demonstrates their success is less probable.

The ability to get below the surface is one reason the JPMorgan Health Symposium draws between 8,000 and 10,000 attendees each year. Attending is an expensive commitment, but firsthand information, insights from others that are in-the-know, and exposure to scientific paths, trends and research that barely existed a few years earlier, can pay off.

If you were not among the 8,000 counted as attending at the 2023 JP Morgan Health Symposium, you’ll want to know, Noble Capital Markets, teamed with Channelchek to provide a video recap with insights and key takeaways on some of the biotech trends that may be worth exploring. This takeaway, coupled with select company presentations and questions from two top equity analysts in the field is sure to build on your current health sector knowledge.  Go Here For More Information (and free access).  

Trends Worth Exploring

Molecular diagnostics, involves taking DNA or RNA which is our unique and easily obtainable genetic code, and analyzing the sequences for red flags. These markers can pinpoint the chance for emergence of specific diseases. This field has expanded rapidly in recent years, with some products now being used regularly. But the potential is for far more to be developed and approved for use. This provides for tremendous profit potential.

Alternative pain relief, non-opioid and non-NAISD pain medications for chronic sufferers, could benefit millions who suffer eah day. The potential runs the gamut from chronic headaches or back pain to situations where one is recovering from surgery, sports injuries, or accidents. Millions of prescriptions are written each year for pain medications. This has, in part fed into the opioid crisis in the U.S. It has prompted an almost emergency-level need for replacing older addictive medications with effective alternatives.  There are a number of companies making gains in this area of great need.

Gene therapy is a technique to treat or cure disease by modifying one’s genes. In many cases, the hope is that it leads to a permanent cure. New gene therapies are being developed for a wide swath of ailments including life-threatening disease. It is expected to be in many cases the next generation of cure. The methods for gene therapy include replacing a disease-causing gene with a healthy copy, or inactivating the disease-causing gene. In other cases a modified gene may be introduced to help treat the disease. The research and development include cancers, infectious disease, organ failures, and autoimmune problems. Many of these companies will be opening the door to welcome life improvements for the some people, and curing what are now incurable diseases for others.

Drilling Down at the Company Level

It may feel uncomfortable to suggest that investing in and backing the right companies that resolve health issues can be profitable to you. But, the truth is, without investments and interest in stock ownership, tomorrow’s miracle drugs would never come to exist.

Watch the Takeaway from the JP Morgan conference with an eye toward what the company presenters deem important, and then listen to the analysts that also drill deeper beyond concept and stage of development, they discuss finances, which for many less experienced biotech investors, isn’t focused on enough. The companies selected for the Noble Capital Markets Takeaway all fall within one the fields mentioned above.  Register Here.

Possible Side-Effects

The J.P. Morgan Healthcare Symposium was held in mid-January. It is one of life science’s largest and most frenzied sharing of information related to the industry. Not everyone gets to go. We’re enthusiastic to be bringing you a slice of the excitement in hopes that you deepen your understanding of not just these companies, but what to look for in others as well.

Paul Hoffman

Managing Editor, Channelchek

Source

https://www.jpmorgan.com/solutions/cib/insights/health-care-conference

https://www.sfchronicle.com/sf/article/JPMorgan-health-care-conference-brings-8-000-to-17706261.php#:~:text=The%20JPMorgan%20health%20care%20conference%2C%20the%20largest%20industry,the%20first%20time%20since%202020%2C%20drawing%208%2C000%20attendees.

What’s Driving New Investor Interest in Biotech Stocks?

Posted on by phoffman@channelchek.com
Image: JP Morgan 41st Health Symposium

More Singles and Doubles for Investors in Biotech Expected (Few Home Runs)

Biotech has been highlighted by us a few times in recent weeks because of the potential the current financial dynamics could have for companies and investors. This past weekend, fresh out of the JP Morgan Health conference, a number of major publications have echoed a similar sentiment. A weekend piece in Barron’s in particular, caught my attention — its overall conclusion is the same as our readers have seen on Channelchek, but the path taken to get to the conclusion is somewhat different.

Health Landscape

From March 2020 until February 2021, biotech stocks were on a tear. The increase of 155%, as measured by the XBI, can be attributed to the intense focus on healthcare during the period.  Higher demand for anything healthcare-related drove share prices among the companies in this sector. This went a long way to provide capital to companies whose very nature are high costs and low revenue. The strength of the sector brought up the deserving, along with others that benefitted from biotech’s overall momentum.

The peak was nearly two years ago. Just as interest in biotech strengthened less than deserving companies, the weakness that followed has brought down many companies that would likely be valued much higher if not for the “throw the baby out with the bathwater” effect, especially with so many sector index fund investors.

This weakness must have been a painful transition for management of companies that are enthusiastic about the prospects of their research and development but now find they may be in survival mode and now spend more time pitching their story and plans while hoping for an overall rise in interest in the sector.

Biotech Mood 2023

The challenge for smaller biotech and medical device companies, which ordinarily spend many years developing products, while benefiting from few or none on the market, is that current valuations have made it a steeper uphill battle to raise new funds for their work and if they do, they may over dilute current shares.

There is a change in the mood of life sciences companies. the Barron’s article, titled, Tanking Biotech Stocks Will Mean a Big Year for Deals. Who Could Benefit? wrote, “With reality setting in, it’s a buyer’s market for companies looking for acquisitions and partnerships, according to the pharmaceutical and medical technology execs who gathered at the J.P. Morgan healthcare investor conference.” JP Morgan describes this annual event as the largest and most informative healthcare investment symposium in the industry. It connects global industry leaders, emerging fast-growth companies, innovative technology creators and members of the investment community.

The conference had been on hiatus for a couple of years in response to pandemic concerns. Certainly there was a lot of new and interesting information to be absorbed and understood.

An overall impression coming from this 41st health symposium is that management of the cash-starved small firms are in a situation where they either have to make a deal with a partner or acquirer or perish. The realization has set in that terms or prices they may have once been able to command are not today’s reality.

Geoff Martha , CEO of Medtronic (MDT), a medical device manufacturer, is quoted as telling Barron’s “We’re getting lots of calls from companies that literally we talked to six months ago.” He explained, back we’d offer, “we’ll buy you for X amount,” and the response would be, “No way, we’re worth [two times that].” The Medtronic CEO said they are now calling back trying to restart the conversations.

Source: Koyfin

Tide Turning

Speaking about the terms now expected, the chief financial officer of Gilead Sciences (GILD) is quoted as saying, “It’s changed completely in terms of both the deal structures they’ll contemplate, the valuations that they’re thinking about,”

Large pharmaceutical companies such as Gilead have the means to provide a non-dilutive source of funds; they are coming off a number of very profitable years and are looking for more rewarding uses of their cash. This doesn’t mean they are willing to cut large acquisition checks; the current trend seems to be more partnering deals – collaborations that keep the best ideas moving forward.

The risk-reward analysis by the large pharmaceutical companies is versus low-return financial assets on the balance sheet. “We can make a lot of investments because it’s not high cost,” says Anat Ashkenazi, CFO of Eli Lilly (LLY). “And we know some of these will fail, some will succeed. That’s how we operate.”

This has ushered in a health industry where large companies with cash to spend are capable of placing many well analyzed bets on future devices and drugs from small companies that now must make a deal or risk perishing.

Take Away

When a small biotech company gets an infusion of cash from collaborating with a big pharmaceutical company, its stock typically reacts very positively. This is not the same level of reaction as an outright purchase, but worthwhile just the same. There is an atmosphere where these partnerships and collaborations are likely to occur with more frequency. This could add to the number of small biotech stock potential winners early in 2023.

Discover Inner Details from the Health Symposium

Investors eager to discover more about the companies at the JPM conference, what was said, where the industry is going, and actionable investor possibilities, can immerse themselves in this info deeper next week.

Here’s how.

Noble Capital Markets’ equity analysts and investment bankers attended the meetings, lunches, cocktail events, and interviewed company management. Next week they will share their collective takeaways. It is perhaps better than having endured the unusually bad weather yourself in San Francisco, get more information here!

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.jpmorgan.com/solutions/cib/insights/health-care-conference

https://www.barrons.com/articles/biotech-partnerships-mergers-acquistions-51673643442

https://www.channelchek.com/news-channel/takeaway-series-on-channelchek-j-p-morgan-healthcare-conference

Organs-On-A-Chip Minimize Late-Stage Drug Development Failures

Posted on by phoffman@channelchek.com
Image: Lung-on-a-Chip,  National Center for Advancing Translational Sciences (Flickr)

Organ-On-A-Chip Models Allow Researchers to Conduct Studies Closer to Real-Life Conditions – and Possibly Grease the Drug Development Pipeline

Bringing a new drug to market costs billions of dollars and can take over a decade. These high monetary and time investments are both strong contributors to today’s skyrocketing health care costs and significant obstacles to delivering new therapies to patients. One big reason behind these barriers is the lab models researchers use to develop drugs in the first place.

Preclinical trials, or studies that test a drug’s efficacy and toxicity before it enters clinical trials in people, are mainly conducted on cell cultures and animals. Both are limited by their poor ability to mimic the conditions of the human body. Cell cultures in a petri dish are unable to replicate every aspect of tissue function, such as how cells interact in the body or the dynamics of living organs. And animals are not humans – even small genetic differences between species can be amplified to major physiological differences.

Fewer than 8% of successful animal studies for cancer therapies make it to human clinical trials. Because animal models often fail to predict drug effects in human clinical trials, these late-stage failures can significantly drive up both costs and patient health risks.

To address this translation problem, researchers have been developing a promising model that can more closely mimic the human body – organ-on-a-chip.

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of Chengpeng Chen, Assistant Professor of Chemistry and Biochemistry, University of Maryland, Baltimore County

As an analytical chemist, I have been working to develop organ and tissue models that avoid the simplicity of common cell cultures and the discrepancies of animal models. I believe that, with further development, organs-on-chips can help researchers study diseases and test drugs in conditions that are closer to real life.

What are Organs-On-Chips?

In the late 1990s, researchers figured out a way to layer elastic polymers to control and examine fluids at a microscopic level. This launched the field of microfluidics, which for the biomedical sciences involves the use of devices that can mimic the dynamic flow of fluids in the body, such as blood.

Advances in microfluidics have provided researchers a platform to culture cells that function more closely to how they would in the human body, specifically with organs-on-chips. The “chip” refers to the microfluidic device that encases the cells. They’re commonly made using the same technology as computer chips.

Not only do organs-on-chips mimic blood flow in the body, these platforms have microchambers that allow researchers to integrate multiple types of cells to mimic the diverse range of cell types normally present in an organ. The fluid flow connects these multiple cell types, allowing researchers to study how they interact with each other.

This technology can overcome the limitations of both static cell cultures and animal studies in several ways. First, the presence of fluid flowing in the model allows it to mimic both what a cell experiences in the body, such as how it receives nutrients and removes wastes, and how a drug will move in the blood and interact with multiple types of cells. The ability to control fluid flow also enables researchers to fine-tune the optimal dosing for a particular drug.

The lung-on-a-chip model, for instance, is able to integrate both the mechanical and physical qualities of a living human lung. It’s able to mimic the dilation and contraction, or inhalation and exhalation, of the lung and simulate the interface between the lung and air. The ability to replicate these qualities allows researchers to better study lung impairment across different factors.

Bringing Organs-On-Chips to Scale

While organ-on-a-chip pushes the boundaries of early-stage pharmaceutical research, the technology has not been widely integrated into drug development pipelines. I believe that a core obstacle for wide adoption of such chips is its high complexity and low practicality.

Current organ-on-a-chip models are difficult for the average scientist to use. Also, because most models are single-use and allow only one input, which limits what researchers can study at a given time, they are both expensive and time- and labor-intensive to implement. The high investments required to use these models might dampen enthusiasm to adopt them. After all, researchers often use the least complex models available for preclinical studies to reduce time and cost.

This chip mimics the blood-brain barrier. The blue dye marks where brain cells would go, and the red dye marks the route of blood flow. Vanderbilt University/Flickr

Lowering the technical bar to make and use organs-on-chips is critical to allowing the entire research community to take full advantage of their benefits. But this does not necessarily require simplifying the models. My lab, for example, has designed various “plug-and-play” tissue chips that are standardized and modular, allowing researchers to readily assemble premade parts to run their experiments.

The advent of 3D printing has also significantly facilitated the development of organ-on-a-chip, allowing researchers to directly manufacture entire tissue and organ models on chips. 3D printing is ideal for fast prototyping and design-sharing between users and also makes it easy for mass production of standardized materials.

I believe that organs-on-chips hold the potential to enable breakthroughs in drug discovery and allow researchers to better understand how organs function in health and disease. Increasing this technology’s accessibility could help take the model out of development in the lab and let it make its mark on the biomedical industry.