Tag: Biotech

Three Biotech Industry Challenges Investors Should Monitor

Posted on by phoffman@channelchek.com

Regulatory, Competitive, and Pricing Challenges That Analysts Believe Biopharma Will Face

Trends in biotech are changing, and if certain events play out, the field could become more challenging on three fronts, according to a new report from analysts from the biotech team at RBC Capital Markets. The report discusses advancements in science that could trump others currently in use or some nearing the end of pipelines, along with regulations that could alter scarcity and prices that stem from recent actions.

In its industry report released on August 4, RBC’s biotech team discussed how deep discounts may be dictated by government programs. It also details how new competition could arise for drugs now offered by Biogen (BIIB) and Incyte (INCY) companies. And also how improvements in public-health from new weight-loss drugs and from gene-editing treatments may reduce demand for other products.

Pricing Challenges

The Inflation Reduction Act in its current form will reduce the prices paid for drugs by federal programs like Medicare, the analysts warn. Mandatory discounts, which will rise from 25% to 60%, will phase in for covered medicines beginning at the ninth to 16th years on the market. Any increases in the prices charged to the government program Medicare would be capped at the inflation rate.

The mechanisms by which these pricing measures will be rolled out by the Centers for Medicare Services will become more clear later this year. On the positive side for the industry, RBC noted a number of biotech companies have asked federal courts to block the law enacted in 2022. And if Republicans win controlling seats in the House in the fall, or even the Executive Branch next year, this would increase the chances of a rollback of the federal pricing provisions.

Exclusivity Challenges

A separate issue is that a lapse of exclusivity protection could affect companies such as Biogen, says RBC. That company’s big-selling product Tysabri, a multiple sclerosis treatment brings in about $2 billion a year. But lower protections may cause it to face competition from a generic “biosimilar” version from the Sandoz unit of Novartis (NVS). Earlier this summer, a court denied Biogen’s request for a preliminary injunction that would have kept the Sandoz biosimilar off the market while Biogen would press a patent infringement claim. The RBC analysts see signs that the Food and Drug Administration (FDA) is gearing up to approve the Sandoz biosimilar. The analysts mentioned that any sales losses at Biogen could be offset by the successful growth of its new anti-Alzheimer’s treatments.

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Another exclusivity threat concerns the product Jakafi, which is a successful Incyte cancer and immune disorder drug, its annual sales could exceed $3.5 billion in the next few years. But the expiration of Jakafi’s patent protection in 2029 could bring a stream of generics it would compete with. This places the maker Incyte under pressure to develop products to extend its Jakafi franchise, says RBC.

Medical Success Challenges

The team at RBC writes that some welcome medical advances might prove so successful that they would reduce the need for other products. They gave examples that include the class of diabetes and obesity treatments known as GLP-1 drugs, which they expect are on their way to becoming enormous sellers for Novo Nordisk (NOVO) and Eli Lilly (ELI).

In addition to the dramatic weight loss achieved by the drugs, the GLP-1s may also become useful to reduce cardiovascular and liver disease. While the public health aspect would be cause to celebrate, investors should be cognizant that the prospects for other drugs sold to treat illnesses such as the liver disease called nonalcoholic steatohepatitis, or NASH, may get new competition.

Another promising breakthrough are gene-editing therapies, the one-time treatments that use the Nobel Prize-winning technology known as CRISPR to permanently blunt genetic illnesses.

Intellia Therapeutics (NTLA) and Verve Therapeutics (VERV) are testing such approaches as treatments for nerve and heart disorders that otherwise require continuing doses of medicines sold by other biotech companies. The RBC team says that the success of the one-shot editing therapies could trim the need for drugs that at present must be continually taken.

Take Away

Analysts don’t have a crystal ball, but investors do rely on their expertise to gain insight as to industry trends and individual company insights. There are many changes occurring in the biotech and biopharma space that could change the competitive landscape, some better for investors, some better for patients, and some even better for taxpayers and social security and Medicaid recipients.

Investors that wish to increase their knowledge of the industry and smaller innovative companies within the segment should explore the data and information in this section of Channelchek and dig even deeper into companies specifically covered by the industry analyst at Noble Capital Markets by using this link.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.rbccm.com/en/insights/industries-in-motion/podcast.page?dcr=templatedata/article/podcast/data/2021/09/how_biotech_breakthroughs_are_changing_healthcare

https://www.barrons.com/articles/biotech-stocks-changing-regulation-disruption-1b4b3ee

https://www.fiercebiotech.com/biotech/peter-marks-says-base-editing-could-be-incredible-game-changer

Biotech Companies to Benefit from AI Efficiencies and Analysis

Posted on by phoffman@channelchek.com

Enabling Better Drug Discovery Outcomes with Machine Learning

Can the long road to bring new medical treatments or therapies to market be shortened by introducing artificial intelligence? AI applied to the early stage of the discovery process, which often involves new insight into a disease or treatment mechanism, may soon provide researchers many more potential candidates or designs to evaluate. AI can also help in the sorting and evaluation of these candidates to improve the success rates of those that make it into the lab for further study.

Benefits AI Brings to Biotech Research

The cost of bringing a single drug to market in terms of time and money is substantial. Estimates are in the $2.8 billion range, and the average timeline for drug development exceeds a decade. On top of this, there is a low level of certainty of taking a promising molecule all the way to market. The success rate of translating preclinical research findings into effective clinical treatments is low; failure rates are estimated to be around 90%.

The refinement of digital sorting and calculating with advanced computational technologies, such as artificial intelligence (AI) and machine learning (ML), have the potential to revolutionize pharmaceutical research and development (R&D). Despite it still being a young technology, AI-enabled applications and algorithms are already making an impact in drug discovery and development processes.

One of the significant benefits of ML in drug development is its ability to recognize patterns and unveil insights that might be missed by conventional data analysis or take substantially less time to recognize. AI, and ML technologies can help a biotech company do precursory evaluation, accelerate the design and testing of molecules, streamline the testing processes, and provide a faster understanding along the way if the molecule will perform as expected. With improved clinical success and reduced costs throughout the development pipeline, AI may be shot in the arm the industry needs.

Adoption of AI in Biotechnology

While any full-scale adoption of AI in the pharmaceutical industry is still evolving and finding its place, implementation and investment are growing. Top global pharmaceutical companies have increased their R&D investment in AI by nearly 25% over the past three years – this indicates a recognition of the perceived benefits.

The interest and investment in AI drug discovery is fueled by several factors. As touched on earlier, a more efficient and cost-effective drug development process would be of great benefit. AI can significantly reduce both time and cost. And the sooner more effective treatments are available, the better. Chronic diseases, such as cancer, autoimmune problems, neurological disorders, and cardiovascular diseases, creates an ongoing demand for improved drugs and therapies. AI’s ability to analyze vast amounts of data, identify patterns, and then learn from the information at an accelerated rate can allow researchers to shorten timelines to final conclusions.  

Even more exciting is the growing availability of large datasets thanks to the rise of big data. With an increase in the volume, variety and velocity of data, and the AI-assisted ability to make sense of it, outcomes are expected to be improved. These datasets, obtained from various sources like electronic medical records and genomic databases, allow successful AI applications in drug discovery. Technological advancements, especially in ML algorithms, have been contributing to the growth of AI in medicine. And they are growing more sophisticated, allowing for accurate pattern identification in complex biological systems. Collaborations between academia, industry, and government agencies have further accelerated growth sharing knowledge and resources.

Trends in AI and ML Biotechnology

While considered a young technological field, AI-enabled drug discovery is being shaped by a number of new trends and technologies. Modern AI algorithms are now capable of analyzing intricate biological systems and foretelling the effects of medications on human cells and tissues. By detecting probable adverse effects early on in the development phase, the predictive ability helps prevent failures in the later stages.

By generating candidates that fit certain requirements, generative models can accelerate the design of completely new medications. But other technology is also now available to assist. By offering scalable processing resources, cloud computing dramatically cuts down on both time and expense. By simulating the interaction of hundreds of chemicals with disease targets, virtual drug screening enables the fast screening of drugs.

A higher understanding of disease biology and the discovery of new therapeutic targets is being made possible by integrative techniques that incorporate many data sources not available a short while ago.

Constraints on AI-Assisted Biotech Research

While AI can speed up certain aspects of drug discovery, it cannot replace most traditional lab testing. Hands-on experimentation and data collection on living organisms are expected to always be necessary, many of these processes during the clinical trial stages cannot be sped up.

Regulatory bodies, like the FDA, are also cautious about embracing AI fully, raising concerns about transparency and accountability in decision-making processes.

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Take Away

The near future of artificial intelligence and machine learning assuming a larger role in enabling drug discovery and more efficient R&D looks bright. The technology offers real promise for more efficient and cost-effective drug development processes – this would address the need for new therapies for chronic diseases.

The time-consuming process of testing on real subjects is not expected to be replaced or overly streamlined by technology, but finding subjects and evaluating results can also benefit from the new technology.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://5058440.fs1.hubspotusercontent-na1.net/hubfs/5058440/cold%20outreach%20use%20case%20images/Pathways%20for%20Successful%20AI%20Adoption%20in%20Drug%20Development%20-%20VeriSIM%20Life.pdf

https://www.mckinsey.com/industries/life-sciences/our-insights/ai-in-biopharma-research-a-time-to-focus-and-scale

https://www.drugdiscoveryonline.com/doc/the-global-market-for-ai-in-drug-discovery-to-sextuple-by-0001

https://www.mckinsey.com/industries/life-sciences/our-insights/we-can-invent-new-biology-molly-gibson-on-the-power-of-ai

https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process

More Proof There is Significant Value in Biotech Stocks

Posted on by phoffman@channelchek.com

Another Sign Conditions are Improving for Biotech Stocks

Biotech stocks seem to be exhibiting unusual value for any stock segment. A subset of the healthcare sector, biotechnology includes companies researching, and developing what may be the next-generation medical preventatives and treatments. There had been a huge decline in interest in the segment that coincided with FDA approval of the first Covid19 vaccine in late Summer 2021. Since then, the average price has been more than cut in half (see XBI/IBB chart) for biotechs.

The chart below is the S&P SPDR Biotech ETF and iShares Biotech ETF. They represent 280 stocks. According to an article in Barron’s dated June 4, 2023, of these, 23 (almost 10%) are trading below enterprise value (EV). Many more are on the cusp of trading right at the value of their net assets. For example, the article mentioned Atea Pharmaceuticals (AVIR), a biotech developing antiviral therapeutics for Hep-C and Covid. “If you bought all Atea’s shares and paid off all of its debt, the cash and other liquid assets remaining on its balance sheet would be worth more than what you spent,” wrote Barron’s.

Source: Koyfin

Of the 280 stocks, nearly 60 have enterprise values below $100 million – the current conditions are not sustainable. At some point, the “invisible hand” of the market is will work to correct it. Last week, Atea, which had been trading near $3.70 recently, was offered $5.75 per share. Concentra Biosciences, which is controlled by the investment fund Tang Capital Partners, made the offer with some contingencies tied to licenses or sales of Atea’s products.

The peculiar condition of the market valuing biotech companies below EV or even cash came to my attention at a RoadShow that was arranged by Noble Capital Markets that featured Cocrystal Pharma (COCP). Cocrystal has several products advancing toward clinical milestones. It was presented by a member of Cocrystals executive management team in South Florida. While the myself and the other investors became familiar with COCP’s development pipeline, and data like the rate of cash usage, the amount of cash on hand, and the market value, it became quite apparent the company had far more cash than the amount the stock market had priced the entire company. And at its cash burn rate (amount of cash used to cover expenses each month), that there might be a significant valuation disconnect.

Many believe disconnects like this will be resolved as the markets always are seeking value and seemingly mispriced companies. There are already many examples this in 2023 as big pharma either has partnered with, or outright acquired companies. This, of course, can cause the stock prices to skyrocket. In fact, while the news was focused on Silicon Valley Bank last March, Provention Bio (PRVB) shot up 258% after a deal was announced.

Smaller biotechs need money to spend on developing drugs, and can’t rely on product sales. Even with what might seem like a huge war-chest of cash, low market values have stifled the ability to raise new money. The road to the next wonder drug is long and requires management to take comfort that they can secure funds when needed.

The extent of this challenge is unique to each company. For many, since the biotech segment valuations came down from the pandemic-inspired dizzying heights,  they might have cash, but not enough to go an extended period until funding conditions improve. The offer last week by Concentra is a sign that conditions are changing. It isn’t just pharmaceutical companies shopping now for biotech bargains to own, it seems investment partnerships are also recognizing the extreme value in some companies.

For data and current information on almost 250 biotech companies, visit the biotech industry section here, on Channelchek.  

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.barrons.com/articles/biotechs-negative-enterprise-values-5e289e8e?mod=Searchresults

News From the Biotech Sector has become Increasingly Rewarding

Posted on by phoffman@channelchek.com

2023 May Be the Year the Biotech Sector Cures Itself of Malaise

A pivotal point for biotech stocks seems to have been reached. It’s almost mid-2023, and barely a week goes by without news driving a biotech company’s stock price upward to gain returns that one would expect to take years to achieve in a broad basket index position. The most recent news causing this price action is yet another clinical-stage therapeutic company. Shares of PDS Biotech are up 27% since yesterday and 41% month-to date. This week’s move is attributable the company reporting positive trial results.

PDS Biotechnology (PDSB) is a clinical-stage immunotherapy company with a developing pipeline of targeted immunotherapies for cancer and infectious disease.

The impetus for the price move was the announcement of interim data from a Phase 2 trial investigating its PDS0101 in combination with Merck’s  KEYTRUDA®. The trials were in patients with unresectable, recurrent or metastatic human papillomavirus head and neck cancers.

The results were quite positive and will be featured in a poster presentation and in a head and neck cancer expert panel discussion at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting being held June 2-6 in Chicago.

These are highlights of the results showing the interim data of the efficacy of PDS Bio’s PDS0101 in combination with KEYTRUDA®:

The estimated 12-month overall survival rate was 87.1%. Published results are 36-50% with approved ICIs used alone*.

  • Median progression-free survival was 10.4 months (95% CI 4.2, 15.3). Published results are median PFS of 2-3 months for approved ICIs when used as monotherapy in patients with similar PD-L1 levels*.
  • A disease control rate (disease stabilization or tumor shrinkage) of 70.6% (24/34)
  • Confirmed and unconfirmed objective response rate was 41.2% (14/34 patients), which is identical to the preliminary response rate data PDS Biotech previously reported at ASCO 2022 (7/17 patients). To date these responses have been confirmed in nine of the 34 patients (26.5%), including one complete response.
  • 15/34 patients (44.1%) had stable disease.
  • 9/34 patients (26.5%) had progressive disease.
  • 4/48 (8.3%) of patients had a Grade 3 treatment-related adverse event (TRAE). No Grade 4 or higher TRAEs were observed.

A main driver of the stock market enthusiasm can be found in the safety and efficacy results in the interim data. “This data showed an estimated 12-month survival rate of 87% and a progression-free survival of 10.4 months, which is very encouraging given the poor prognosis these patients face,” stated Lauren V. Wood, M.D., PDS Biotech’s Chief Medical Officer and a co-author of the study. “Furthermore, we remain encouraged by the safety profile of PDS0101 in combination with KEYTRUDA®, with only 8% of patients experiencing a Grade 3 treatment-related adverse event without more serious Grade 4 or 5 events. We believe these data are encouraging for HNSCC patients and indicate that the addition of the HPV16-targeted immunotherapy PDS0101 to KEYTRUDA® should be further evaluated for its potential to enhance survival in HPV16-positive head and neck cancer patients.”

To understand the company PDS Biotechnology better, visit the research page on Channelchek, and also review this Channelchek video that shares key information about PDSB.

The Nature of Biotech Investing

Drug discovery and development is a long, uncertain path that often takes 10–15 years, with 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. Attaining interim results showing high efficacy and tolerance is a very positive sign and one that will most often cause a large price jump. Negative results can have the opposite effect.

Events that cause small-cap biotech stocks to experience significant price jumps could include:

  • Positive clinical trial results: As with PDS Biotechnology, when a small-cap biotech company releases positive clinical trial results, it can generate significant investor interest and drive up the stock price.
  • FDA approvals: Taking is a step further, 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 cause a small-cap biotech stock to rise as it indicates a level of validation for the company’s technology or products, and provide needed funding to bring research and development along the lengthy timeline.
  • 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.
  • 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 data and 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.

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.

PDS Biotechnology (PDSB):  This was positive news for PDS Biotech, but there work isn’t finished and they have other immunotherapy products in their pipeline based on proprietary T cell-activating technology.

Take Away

As we approach the halfway point of 2023, biotech stocks that had traded sky-high during the pandemic era had been paid far less attention to since. But the tide appears to be turning as news such as that reported by PDSB, and partnerships and even acquisitions have been on the rise.

It’s an interesting sector that, for better or worse, is barely correlated with the rest of the stock market.

Paul Hoffman

Managing Editor, Channelchek

Sources

PDS Biotechnology Press Release (May 25, 2023)

PDS Biotechnology Video

Biotech M&A is Finally Rewarding Patient Investors

Posted on by phoffman@channelchek.com

The Acceleration of Biotech Acquisitions, Why it Should Continue

The pace of mergers and acquisitions (M&A) in the biotech sector has accelerated in 2023 compared to 2022 and 2021. The factors driving this increase are numerous, and there is increasing urgency on both sides, the acquirers and acquirees to find a fit. This is playing out with deal value up significantly in 2023, with noticeable acceleration as the year has progressed. During the first quarter, investors in at least eight biotech companies found themselves in enviable positions. Below is a recap of what has already happened and the perceived trend. If it continues, it could lead to 2023 seeing far more biotech deals than the previous two.

According to data from William Blair’s quarterly biopharma review, the total deal volume in the sector was elevated, although far below record highs. Total M&A value for the first quarter was $52 billion versus $88 billion for all of 2022, and $77 billion in 2021. The report shows the average deal was $630 million (versus $367 million in 2022). The upfront cash and equity has doubled from the prior year at $508 million (versus $249 million in 2022).

Later stage companies seem to be what pharmaceutical firms have the most appetite for. The phase of development of the companies most sought was Phase II or later with 100% or all of the public acquisitions in this stage. Five of the eight were in the commercial stage.  

Source: The Quarterly Rx: Q1 2023 U.S. Biopharma Recap (William Blair)

The details above are of the eight public companies that merged or were acquired during the first quarter. However, just this week alone, there have been three more biotech acquisitions announced:

  • On May 16, 2023, Merck & Co. announced that it would acquire Acceleron Pharma for $11.5 billion. Acceleron is a clinical-stage biopharmaceutical company, the acquisition will give Merck access to Acceleron’s lead drug candidate, luspatercept, which is currently in Phase 3 clinical trials for the treatment of anemia associated with chronic kidney disease.
  • On May 17, 2023, Gilead Sciences announced that it would acquire Immunomedics for $21 billion. Immunomedics is a clinical-stage biopharmaceutical company the acquisition will give Gilead access to Immunomedics’ lead drug candidate, Trodelvy, which is currently in Phase 3 clinical trials for the treatment of triple-negative breast cancer.
  • On May 18, 2023, AstraZeneca announced that it would acquire Daiichi Sankyo’s oncology business for $6.9 billion. Daiichi Sankyo’s oncology business includes a portfolio of marketed and late-stage cancer drugs. The acquisition will give AstraZeneca a broader portfolio of cancer drugs and will help the company to expand its presence in the oncology market.

What is Driving the Acceleration?

There are a number of “not-so-secret” factors that are helping the acceleration of M&A activity in the biotech sector. One factor is the increasing cost of product development. The average cost of developing a new drug has increased from $1 billion to $2.6 billion in the past decade. This has made it increasingly difficult for small and mid-sized biotech companies to develop viable candidates independent of big-pharma’s help. As a result, small companies are increasingly looking to merge, partner or be acquired by larger companies with deeper pockets.

Another factor driving the acceleration of M&A activity in the biotech sector is the increasing focus on innovation. Large pharmaceutical companies don’t have the talent that exists in the universe of small biotech companies. So they are increasingly looking to acquire companies with innovative technologies. These innovations can help them provide new drugs that can compete with the blockbuster drugs coming off patent in the next few years.

Finally, the acceleration of M&A activity in the biotech sector is also being driven by the increasing consolidation of the industry. In recent years, a number of large pharmaceutical companies have merged with or acquired each other. This has led to a smaller number of bigger companies that are now dominant in the industry. These companies are increasingly looking to acquire smaller companies in order to expand their product portfolios and overall reach.

Take Away

While deals in many industries, both public and private, have decelerated to a crawl, the cash-rich pharmaceutical industry giants are tactically looking to build their portfolios of next-generation treatments. And many biotech companies are in need of a lifeline to get their pipeline products the research dollars they deserve. This dynamic has accelerated public and private deals in the industry in 2023.  

The acceleration of M&A activity in the biotech sector is a trend that is not expected to end soon. This is because the factors that are driving deals are likely to remain in place. Investors looking to explore smaller biotech companies may want to keep in mind the nuances of the average company attributes that found deals in the first quarter. Top-tier research on a number of smaller companies, provided by the sectors equity analysts at Noble Capital Markets can be found here.    Company information and data on many other biotech and life sciences companies can be discovered by going to this link.

Paul Hoffman

Managing Editor, Channelchek

Sources

file:///C:/Users/prese/Downloads/WilliamBlair-Biopharma-Quarterly-Review-Q1-2023.pdf

https://community.ionanalytics.com/ma-highlights-1q23?account_created=1

https://dkf1ato8y5dsg.cloudfront.net/uploads/79/598/mahighlights1q23-final.pdf

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/ )