Breaking Boundaries: Lilly’s Investment in Base Editing for Heart Disease

Pharmaceutical giant Eli Lilly is expanding its efforts in cardiovascular disease research through a new deal with Beam Therapeutics and Verve Therapeutics worth up to $600 million. The deal centers around base editing, an emerging gene editing technology that Beam and Verve are pioneering for new precision genetic medicines.

Under the agreement announced today, Lilly will acquire Beam’s opt-in rights to co-develop and co-commercialize several of Verve’s base editing programs targeting cardiovascular disease. This includes lead programs focused on PCSK9 and ANGPTL3 – two high profile genes involved in cholesterol regulation and metabolism. A third undisclosed target related to liver-mediated cardiovascular disease is also included.

In exchange, Beam will receive a hefty $200 million upfront payment along with a $50 million equity investment from Lilly. Beam is further eligible for up to $350 million in future milestone payments as the programs advance through clinical trials and regulatory approvals.

For Lilly, this deal provides access to a promising new approach to treating cardiovascular disease, an area where the company already has a major presence. Lilly has been a leader in cholesterol drugs like statins for decades, and more recently entered the PCSK9 market through its ownership of Repatha. But despite effective medications, cardiovascular disease remains a top killer globally.

Base editing offers a way to precisely and permanently modify disease-causing genes in order to lower cholesterol and potentially deliver stronger treatment effects than current options. Early human trials have already shown base editing of PCSK9 can lower LDL cholesterol. Verve recently initiated a clinical trial using base editors to target both PCSK9 and ANGPTL3 simultaneously.

Take a moment to take a look at Ocugen (OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines.

As a technology pioneer, Beam is widely considered the leader in base editing. The company has uncovered a series of natural enzymes that can be programmed to make single letter DNA changes at targeted sites without cutting the double strand like traditional CRISPR gene editing. This opens possibilities for more precise control while minimizing unintended effects.

Beam CEO John Evans highlighted base editing as a core strategic priority, and views creative partnerships as a key path to accelerate development. “Our initial collaboration with Verve and this new transaction with Lilly are exemplary of our execution of that strategy,” Evans said. “This deal provides meaningful upfront capital to advance our portfolio of clinical- and research-stage programs, with significant additional value achievable as the Verve programs advance.”

For Beam, the capital influx provides fuel to advance its broader base editing pipeline including programs in sickle cell disease, alpha-1 antitrypsin deficiency, and glycine encephalopathy. The company now expects its cash runway to extend into the second half of 2026.

Lilly’s history with Verve also preceded this acquisition. In 2021, Lilly led Verve’s $105 million Series B financing and took a stake in the company. That marked another early mover deal to tap into base editing. Verve CEO Sekar Kathiresan said “Lilly’s extensive capabilities in drug development and commercialization make them an ideal partner for Verve as we work together to advance base editing programs aimed at reducing CVD risk through genome editing.”

Beam and Verve join a short list of biotechs focused on realizing the promise of base editing. But Lilly’s involvement marks a huge vote of confidence from the pharma world. As base editing advances toward the clinic, deals like this suggest major players view the technology as more than just hype.

Lilly has been aggressively scouting the latest biotech innovations through both in-house R&D and external deals. The past year saw Lilly acquire hot companies like POINT Biopharma and Repare Therapeutics for large sums. Base editing adds a new tool in Lilly’s toolkit for next generation therapeutic approaches.

Cardiovascular disease also represents an attractive area for investment despite already having effective medications. Heart attacks, strokes and other complications remain a top cause of mortality globally. In the US alone, 1 in 4 deaths are attributable to heart disease each year. Even modest improvements in treatment can translate into major public health benefits.

The risk for Lilly and other major pharmaceutical companies is that base editing and gene editing fail to live up to their early promise in the clinic. However, most experts are optimistic the technology will usher in a new wave of therapies over the next decade. For a pharma giant like Lilly with over $28 billion in yearly revenue, the potential reward is well worth the investment risk to stay on the cutting edge.

Eledon Pharmaceuticals (ELDN) – C-Suite Interview With Eledon CEO Available On ChannelChek


Thursday, October 26, 2023

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Our Discussion Included Eledon’s Science, Markets, and Company Strategy.  We recently spoke with the Eledon CEO, Dr. DA Gros, as part of our C-Suite Interview Series. Our interview included the scientific principles on which tegoprubart is based, the unmet need in kidney transplantation, and the current clinical trials. Dr. Gros also discussed additional tegoprubart indications, company finances, and future milestones.

Eledon Is Pursuing An Unmet Need In Kidney Transplantation. Dr. Gros described how tegoprubart blocks a step in the immune response that has applications in many immune disorders. The first application in development is for prevention of kidney transplant rejection, where the current standard of care drugs are effective but have toxic side effects that lead to organ failure after 10 to 12 years. Tegoprubart is currently in two clinical trials for this indication.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

MAIA Biotechnology (MAIA) – THIO-101 Data Update Shows Improved Disease Control Rates


Wednesday, October 25, 2023

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. For more information, please visit www.maiabiotech.com.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

ESMO Data Shows Unusually High Survival Rates. MAIA Biotechnology presented preliminary data from the THIO-101 trial in non-small cell lung cancer (NSCLC) at the European Society for Medical Oncology conference. The disease control rate (DCR) reached 100% in second line patients compared with about 53% to 64%  for standard of care therapies. DCR for third line patients reached 88%, compared with about 30% expected. We see these results as extremely strong, and believe DCR could correlate with overall survival (OS), a primary endpoint of the trial.

Preliminary Data Shows Strong Response. The results showed a disease control rate (DCR, consisting of complete responses, partial responses, or stable disease) of 100% or 19 out of 19 patients in second line therapy and 88% or 14 out of 16 patients in third line. Published data from 74 clinical trials with standard of care therapies show 53% to 64% DCR in second line and about 30% in third line. We see this as a substantial improvement over results from standard of care therapies.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Gilead Partnership Provides Boost to Assembly Bio’s Antiviral Pipeline

Clinical-stage biotech Assembly Biosciences gained a powerful ally in developing next-generation antivirals through a new collaboration with pharma giant Gilead Sciences. The deal provides Gilead access to Assembly Bio’s portfolio of early-stage assets while giving Assembly funding and expertise to advance its programs.

Under the 12-year partnership announced Monday, Gilead will provide $100 million upfront, including an equity investment at a premium. Assembly Bio gains potential milestone payments, royalties, and profit-sharing as programs progress.

For investors, the collaboration validates Assembly Bio’s antiviral pipeline and represents significant long-term revenue potential. The deal also propels Assembly closer to becoming a fully integrated biotech firm.

Assembly’s current clinical assets target major viral diseases like hepatitis B virus (HBV) and herpes simplex virus (HSV). Its preclinical pipeline holds promise against hepatitis D virus (HDV), human cytomegalovirus, and more.

Take a look at other emerging biotechnology companies by taking a moment to look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage list.

Gilead brings extensive experience developing and commercializing leading antiviral drugs in HIV and hepatitis C. The pharma giant now doubles down on virology via Assembly’s early-stage assets.

Opting into a program after proof-of-concept could cost Gilead at least $45 million, signaling confidence in Assembly’s science. The biotech leads R&D initially, with Gilead taking over once assets transition to late-stage trials and commercialization.

For Assembly investors, this deal structure provides valuable de-risking of the pipeline. The company secures funding to advance programs while sharing in the upside if Gilead opts in to lucrative late-stage development and sales.

Assembly is eligible for up to $330 million per program in milestones post opt-in. Royalties range from high single digits to high teens. The biotech can also opt into U.S. profit-sharing.

These economics help offset the risk of clinical failure for Assembly’s shareholders. Meanwhile, Gilead pays for the privilege of accessing Assembly’s innovating antiviral pipelines.

The partnership enhances Assembly’s financial position and extends its operating runway. This enables advancing other preclinical programs beyond the leads Gilead can opt into.

For example, Assembly recently unveiled a promising pan-herpesvirus asset that could treat multiple herpes infections from one oral pill. Such follow-on compounds ensure future revenue potential.

Meanwhile, major progress by Gilead with Assembly’s lead assets could generate substantial royalties and profit-sharing income. Upside from the deal should become clearer as Gilead opts to license drugs entering late-stage testing over the 12-year term.

With a strengthened balance sheet and veteran partner at its side, Assembly Bio seems poised for a breakthrough as a developmental biotech focused on next-gen antivirals.

The Gilead deal provides third-party validation of Assembly’s science and a critical launchpad toward integrated status combining R&D, late-stage trials, and commercialization.

For Assembly investors, these benefits significantly de-risk the journey to having approved antiviral products on the market. If clinical programs pan out as hoped, the payoff from this partnership could be huge.

MAIA Biotechnology (MAIA) – Journal Publishes THIO Data In Pediatric Brain Cancer Models


Monday, October 16, 2023

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. For more information, please visit www.maiabiotech.com.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Data Shows Efficacy In An Aggressive Pediatric Brain Cancer. MAIA announced that preclinical data on the use of THIO, its lead product for multiple cancers, was published in the journal Neuro-Oncology. The presentation tested THIO in diffuse intrinsic pontine glioma, an aggressive form of brain cancer in which radiotherapy is the only treatment but has only marginal efficacy. The data shows that THIO was able to activate immune pathways in the tumors, sensitizing the tumors to radiotherapy. In addition to a potential new indication, we see this as consistent with the theorized mechanism of action and provides further proof of concept for THIO.

THIO’s Mechanism Of Action Improved Responses To Radiotherapy. THIO targets the telomers of dividing cancer cells, causing damage that leads to cell death. As discussed in our report on February 21, the DNA released from the dead cancer cells is detected by a protective pathway known as cGAS-STING that activates the immune system pathways and produces an anti-tumor response. The study found that the THIO treatment also sensitized the tumors to radiotherapy, leading to improved efficacy.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Eledon Pharmaceuticals (ELDN) – Journal Publishes Study On Tissue Engineering For Xenotransplantation Surgery


Monday, October 16, 2023

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Potential Breakthroughs In Xenotransplantation. The journal Nature has published a preclinical study by Eledon’s research collaborator detailing genetic changes to porcine (pig) cells and the results of their transplantation to monkeys. In September, the second successful transplant of an engineered pig heart to a human patient used Eledon’s tegoprubart for immune suppression. Both developments have overcome many technological barriers and could make trans-species organ transplantation a clinical reality.

Publication Details Some Of The Changes To Make Xenotransplants Compatible.  Transplanting organs from a non-human species has many additional challenges than transplantation from one human to another (allografts). The Nature article, from Eledon’s collaborator eGenesis, describes the design, creation, and function of kidney grafts from a genetically engineered porcine transplant into a cynomolgus monkey model. The article describes 69 genomic edits to the donor tissue, addition of 7 human genes, and inactivation of endogenous retroviruses. The transplanted monkeys survived up to 758 days (108 weeks).


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Baudax Bio (BXRX) – Shareholders Approve Merger-Related Preferred Stock Conversion


Monday, October 16, 2023

Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.

Gregory Aurand, Senior Vice President, Equity Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Meeting Held October 12, 2023.  In an October 13th filing, Baudax Bio reported that proposals submitted to shareholders for a Special Meeting of Shareholders passed.  In particular, the Series X convertible preferred stock conversion to common shares was approved. The preferred shares were issued in conjunction with the TeraImmune merger announcement in June. In the merger agreement, Baudax Bio issued 1.212 million shares of common stock, and 27,089.719 shares of Series X convertible preferred to TeraImmune shareholders, with each preferred share convertible into 1,000 shares of common stock.  

Amended and Restated Articles of Incorporation Approved Shareholders also approved an amendment to effect a reverse stock split to be determined by the Board of Directors at any time in the next year, at the Board’s discretion.  The Board can set the reverse split within a range of 1:10 to 1:40.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Pfizer’s Earnings Forecast Drops Due to COVID Products Decline

Pharmaceutical giant Pfizer stunned investors on Friday by making drastic reductions to its 2023 earnings and revenue guidance, driven entirely by rapidly declining demand for its COVID-19 vaccine and treatment.

The company now expects full-year sales of $58-61 billion, far below its previous projection of $67-70 billion. Adjusted earnings per share were cut even more drastically, from an expected $3.25-3.45 down to just $1.45-1.65.

The huge forecast reduction was prompted by a projected $7 billion drop in sales of Pfizer’s COVID treatment Paxlovid and a $2 billion decline for its Comirnaty vaccine. This comes amidst clear signs of “COVID fatigue” as vaccination rates slow and cases become milder.

Pfizer’s update led to a sell-off of its stock on Friday. Rival vaccine maker Moderna’s shares also dropped on the news, reflecting similar downbeat trends for COVID products industrywide.

However, Pfizer regained some losses after executives held a call on Monday to outline plans for weathering the abrupt COVID revenue downturn. This includes a new cost-cutting program aiming to deliver $1 billion in savings this year and $2.5 billion by 2024.

The planned cuts will touch all business segments and regions, though details remain scarce. Pfizer says one-time costs to implement the reductions will be approximately $3 billion, including severance charges and other expenses.

This belt-tightening comes after Pfizer hinted in August it was prepared to trim expenses if COVID product sales continued to deteriorate. “We are in the middle of the COVID fatigue. Nobody wants to speak about COVID,” acknowledged CEO Albert Bourla.

Indeed, uptake for Pfizer’s updated Omicron booster has been lackluster since launching last month. Logistical hurdles and lack of awareness around eligibility have hampered rollout. Waning concern over infections has also lowered demand.

Paxlovid prescriptions have similarly collapsed as immunity from vaccines and prior disease leaves most cases mild. Bourla said this means the remaining demand is coming from the minority focused on prevention and protection.

Looking beyond COVID products, Pfizer still expects to complete its pending $12 billion acquisition of cancer detection leader Seagen on schedule. Executives said the belt-tightening and forecast revisions will not impact those plans.

Pfizer is not alone in adapting to new COVID realities. Moderna has delayed advancing new boosters and vaccines meant to target emerging variants. Merck has paused production of its Molnupiravir antiviral.

But Pfizer’s aggressive pandemic investments leave it most exposed to lasting changes in demand. The company marshaled its resources early on to supply over 3.5 billion vaccine doses worldwide, along with millions of Paxlovid courses.

Now, the record revenues these products delivered are evaporating almost overnight. And as the market leader, Pfizer’s woes signal a new chapter for the entire vaccine and antiviral space.

Of course, the pandemic is not over, and COVID will remain a threat demanding vaccines and treatments. But with most people vaccinated, reinfected, or both, demand and profits are inevitable casualties absent a dangerous new variant.

For pharmaceutical firms like Pfizer and Moderna, the cash windfall from COVID spending is clearly drawing to a close. With customers, cash flows and share prices dropping, a reckoning has arrived. Massive cost cuts offer one path forward, with layoffs and restructuring the vaccines’ unintended side effect.

Trick or Treat: Is Recent Uptick in Small Cap Biotech M&A Activity the Catalyst for an Industry Turnaround?  

With the recent rise in mergers and acquisitions in the biotech sector, some analysts believe that now is an opportune time for investors to start positioning themselves in small and microcap biotech stocks. Though these smaller companies have been out of favor with investors in the post-pandemic environment, the current conditions suggest their fortunes may soon change.

The biotech sector saw a surge of interest during the pandemic, as companies raced to develop vaccines, treatments and diagnostics for COVID-19. Many smaller biotechs saw their values skyrocket on the hope that they would come up with a breakthrough. However, once the initial rush of COVID-related products came to market, investors began rotating out of pandemic darlings and into recovery plays. This led to a dramatic decline in the valuations of micro and small cap biotechs.

Despite this negative sentiment, mergers and acquisitions in biotech have been increasing over the past year. Pharma giants have been scooping up smaller firms to replenish their drug pipelines. While the big deals have gotten all the headlines, analysts say more deal-making is starting to occur further down the market cap scale. This suggests that the fundamentals of select smaller biotechs remain strong, though valuations do not yet reflect it.

Take Eledon Pharmaceuticals (ELDN) for example. This clinical stage biotech has a market cap of only $35 million, which is about half its cash on their 6/30/23 balance sheet . Though its lead drug candidate is in Phase 2 trials for several indications, the company’s stock price has languished. However, with ample cash to support ongoing trials and an approved IND for another preclinical asset, Eledon seems significantly undervalued based on peers.

In fact, many small biotechs appear mispriced today based on the potential of their underlying technology and pipelines. These companies are developing innovative new platforms and drug candidates across therapeutic areas like oncology, rare diseases, neurology, and ophthalmology. While risks are high during the R&D process, achieving clinical milestones and a path to successful commercialization could drive exponential growth.

Consider the market opportunity for breakthrough platforms like CRISPR gene editing or cell therapy. Many smaller firms are advancing novel applications of these cutting-edge technologies. If proven successful in clinical studies, even niche indications could generate billions in peak annual sales.

Additionally, smaller firms tend to be more targeted in their R&D approach. Rather than spreading efforts across numerous indications, microcaps often concentrate on 1-2 assets with large treatment populations. This focused strategy allows them to achieve key milestones more economically. Partnerships with larger biopharma companies can also help offset expenses in later stage development.

The current biotech environment shares some similarities with pandemic euphoria. Today’s misery and fear echoes the extreme euphoria felt by investors three years ago. Just as sentiment eventually turned negative on pandemic darlings, the same could occur for today’s recovery favorites. Already, tech giants like Meta, Nvidia and Tesla have fallen substantially from their highs, suggesting a potential peak.

While rotating out of pandemic favorites made sense as reopening plays gained momentum, the economic backdrop is cloudier now. High inflation, rising rates, geopolitical tensions and recession fears have driven significant equity declines and increased market volatility this year. This has led some investors to question whether stocks still offer favorable risk-reward profiles.

With bonds and equity markets declining in tandem, some investors have turned to cash equivalents like money market funds. While these instruments can provide principal protection, their yields could still lag inflation, given its uncertain outlook. Accounting for taxes owed on interest earned further reduces the chances of a real return. Therefore, holding too much cash during periods of high inflation could erode purchasing power over time.

Rather than accept guaranteed, but potential negative real returns in cash, investors may want to revisit beaten-down assets with asymmetric upside, like small cap biotech stocks. Some of these companies offer innovative technologies that could drive explosive growth if their development and commercialization efforts prove successful, and investor sentiment in this sector turns around.

To identify promising opportunities in the space, investors need to educate themselves on individual companies, study various ideas and leverage resources like Channelchek. While risks for small biotech stocks are always high, those able to discern winners from losers can potentially generate substantial outsized gains.

After years of hype around pandemic favorites, optimism needs to be rekindled for forgotten pockets of the market like micro and small cap biotech stocks. For investors with the appropriate time horizon and risk tolerance, now could be the ideal time to start building positions. If M&A activity continues apace, it likely will not be long before positive fundamentals translate into rising valuations.

Onconova Therapeutics (ONTX) – Rigosertib Data Presented In Ultra-Rare Disease


Friday, October 13, 2023

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. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications. Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies. These studies include a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Rigosertib Data In RDEB-SCC Presented. Onconova presented data from an Investigator-initiated trial (IIT) testing rigosertib in RDEB-associated SCC (recessive dystrophic epidermolysis bullosa associated with squamous cell carcinoma), an ultra-rare but fatal disease with no effective treatment. The data was presented at the European Academy of Dermatology and Venereology (EADV) in Berlin. Results showed complete remission for 2 of the 4 patients tested for over 12 months with one patient still in treatment.

RDEB-SCC Is A Rare But Fatal Disease. RDEB-SCC is an ultra-orphan disease in which mutations in the genes for collagen VII result in structural abnormalities in the skin. This results in fragile skin with failure of the epidermal layer to anchor properly, causing severe blisters and a cycle of chronic injury. The development of squamous cell carcinoma (SCC) often causes death by age 45. Tumors in RDEB-SCC were found to be sensitive to inhibition of an enzyme involved in cell cycle regulation known as PKL-1 (polo-like kinase-1). Rigosertib is an inhibitor of  PLK-1 and was selected for the ITT trial.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

OptimizeRx to Acquire Medicx in $95 Million Deal, Expanding Omnichannel Platform

OptimizeRx Corp. announced Thursday it will acquire Scottsdale-based Medicx Health for $95 million, expanding its platform reach to healthcare consumers.

The deal combines OptimizeRx’s solutions focused on healthcare providers (HCPs) with Medicx’s consumer-centric technologies. Together, the companies can engage over 2 million HCPs and a majority of U.S. healthcare consumers.

“Our acquisition of Medicx is expected to be a major business accelerator for us,” said OptimizeRx CEO Will Febbo.

For OptimizeRx, the acquisition enhances its digital health platform that helps life sciences companies educate and engage HCPs and patients. Medicx brings new omnichannel marketing and analytics capabilities aimed at consumers.

Reaching Healthcare’s Key Stakeholders

OptimizeRx’s lead solution is a digital point-of-care network enabling pharma marketing and engagement integrated within EHR and e-prescribing workflows. This allows drug makers to reach HCPs through digital touchpoints at the point of care.

Medicx has developed a Micro-Neighborhood® Targeting Platform using advanced identity resolution to reach healthcare consumers. Combining both solutions offers an end-to-end way for pharma companies to connect with HCPs and patients—healthcare’s two most important stakeholders.

“Coupling consumer and HCP marketing strategies is a natural next step for many of our customers,” said OptimizeRx Chief Commercial Officer Steve Silvestro.

Profitable Addition to Fuel Growth

The acquisition is expected to significantly benefit OptimizeRx’s growth and profitability. Medicx is a highly profitable company that will immediately add to revenue, EBITDA, and earnings per share.

On a combined basis, the deal will bring OptimizeRx’s revenue run-rate close to $100 million. Medicx also opens substantial new opportunities for customer penetration and cross-selling.

“I’m extremely proud of the leading patient-focused omnichannel platform the Medicx team has built,” said Medicx CEO Michael Weintraub. “Integrating with a leading HCP-focused enterprise provides numerous efficiencies.”

Weintraub added the combined platforms can now inform and educate patients and HCPs in a cohesive way no single company has done before.

Funded for Growth

OptimizeRx will pay $95 million in total consideration to acquire Medicx. The deal will be funded through OptimizeRx’s cash on hand, short-term investments, and a new $40 million credit facility from Blue Torch Capital.

Certain members of Medicx’s management will invest approximately $10.5 million of their proceeds into OptimizeRx common stock.

The acquisition is expected to close in Q4 2023. Medicx will operate as a subsidiary under its current management team.

Strong Quarterly Performance

In tandem with the acquisition announcement, OptimizeRx preannounced strong third quarter 2023 results.

The company expects Q3 revenue between $15.2-$15.5 million, ahead of consensus estimates. Non-GAAP net income is projected at $0.6-$1 million.

OptimizeRx saw accelerated organic growth in messaging driven by its recently enhanced Dynamic Audience Activation Platform.

The deal marks OptimizeRx’s largest acquisition to date as it leverages M&A to expand its platform. Medicx’s addition is expected to be immediately accretive while funding future growth.

PDS Biotechnology Corp. (PDSB) – Data For PDS0301 Phase 1/2 To Show Immune Response and Safety Data


Thursday, October 12, 2023

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technology platforms. Our Versamune®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

First PDS 0301 Prostate Cancer Data To Be Presented. PDS Biotech announced that data from a Phase 1/2 study testing PDS0301 in combination with docetaxel to treat prostate cancer will be presented by National Cancer Institute (NCI) scientists at the Cytokines 2023 meeting on October 15-18. This combination uses PDS0301 with docetaxel to stimulate an immune response and kill the cancer cells. Preliminary data from the trial shows increases in populations of immune cells, pro-inflammatory cytokines, and decreases in PSA levels.

Preliminary Results Show Immune Stimulation With Tolerability. The presentation includes 18 patients with metastatic castrate resistant prostate cancer (mCRPC, n=11) and sensitive prostate cancer (mCRPC, n=7). Patients received one of three dose levels tested in combination with a standard dose of docetaxel every three weeks. Results showed decreases in PSA ranging from -4% to -100%, with increases in CD4 and CD8 immune cells. Cytokines INF-gamma and IL-10 were increased, with decreases in the Treg (suppressive regulatory) cells. These responses are consistent with our expectations for an effective immune response.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Harmony Biosciences Bets on Cannabinoid Therapy for Rare Disorders with $200 Million Zynerba Buyout

Harmony Biosciences aims to expand its pipeline into rare neuropsychiatric disorders through the acquisition of Zynerba Pharmaceuticals and its innovative cannabinoid gel technology.

Harmony, known for its narcolepsy drug Wakix, announced Monday that it will acquire Zynerba in a deal worth up to $200 million. The buyout provides Harmony with Zynerba’s lead asset, Zygel, a synthetic cannabidiol gel in mid-stage trials for Fragile X syndrome and 22q11.2 deletion syndrome.

Zygel could become the first FDA-approved drug for managing Fragile X if it succeeds in pivotal trials. Harmony sees the drug as a way to expand beyond sleep disorders while tackling high unmet needs in orphan neuropsychiatric conditions.

Fragile X affects 80,000 in the U.S., causing intellectual disability and behavioral challenges. Zynerba’s focus aligns with Harmony’s mission in rare neurological diseases, said Harmony CEO Jeffrey Dayno, M.D. in Monday’s announcement.

“With Harmony’s scale, resources and proven commercial excellence, they are well positioned to potentially bring to market the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome and to maximize the value of Zygel,” added Zynerba Chairman and CEO Armando Anido.

No FDA-Approved Options Today

Fragile X syndrome stems from mutations in the FMR1 gene which codes for FMRP, a protein vital for synaptic function and neural connections. The lack of FMRP causes cognitive impairments. Around 60% of Fragile X patients don’t produce any FMRP due to methylation.

While Fragile X affects tens of thousands in the U.S. alone, there are no FDA-approved treatments. Patients rely on behavioral interventions and off-label drug use.

Zynerba’s Zygel aims to modulate the endocannabinoid system impacted by the loss of FMRP. The gel contains synthetic cannabidiol, absorbing through the skin to avoid first-pass metabolism.

Zygel already secured FDA orphan drug status for both Fragile X and 22q deletion syndrome. It also won Fast Track designation for Fragile X.

Now in pivotal Phase 3 trials, Zygel showed positive Phase 2 data in both indications. Harmony believes the drug can help patients manage behavioral symptoms if approved.

Betting Up to $200M on Approval

Under the acquisition terms, Harmony will pay $60 million upfront for Zynerba’s shares, or $1.1059 per share in cash. Zynerba shareholders will also receive one contingent value right (CVR) worth up to $2.5444 per share more.

The CVR payments depend on Zygel hitting clinical, regulatory and sales milestones:

  • $15M for completing Phase 3 Fragile X trial
  • Up to $30M for Phase 3 data (timing-based)
  • $35M for FDA approval in Fragile X
  • $15M for approval in a second indication
  • Up to $45M for reaching sales milestones

Altogether, the deal is valued at up to $200 million if Zygel secures FDA approval and reaches peak sales targets. Harmony expects the buyout to close in Q4 2023.

“Innovative potential new therapeutic option for rare/orphan neuropsychiatric disorders with high unmet medical needs,” said Harmony CEO Dayno.

Doubling Down on Rare Diseases

The acquisition aligns with Harmony’s focus on innovative treatments for overlooked neurological diseases. Zynerba’s work in underserved neuropsychiatric disorders complements Harmony’s leading drug in narcolepsy.

Harmony markets Wakix, a first-in-class H3 receptor antagonist that hit $230 million in net sales over the twelve months ending June 30, 2022. But the company sees untapped growth opportunities in adjacent rare diseases.

The Zynerba deal provides pipeline diversification into high-value orphan drug development. Harmony now has new opportunities in gene mutation disorders like Fragile X and 22q deletion syndrome.

With a profitable commercial engine already built, Harmony is betting its expertise and $430 million cash position can maximize Zygel’s impact if approved. The company sees Zynerba’s cannabinoid therapy as both a strategic fit and a long-term growth driver if milestones are met.

For shareholders, the buyout provides Harmony an approved orphan drug asset with potential peak sales upside. And for patients, it brings hope for the first therapy to address Fragile X symptoms.