Category: Health

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

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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.


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*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

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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).


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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

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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.


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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. 

Alzheimer’s Specialist Prothena Surges on Sale Speculation

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Shares of clinical-stage biotech Prothena Corp skyrocketed over 20% on Monday amid reports the company is exploring strategic options including a potential sale. Prothena specializes in developing therapies for neurodegenerative diseases like Alzheimer’s, making it a hot target for larger pharmaceutical firms hungry for new assets in this space.

Based in Ireland and spun off from Elan Corp in 2012, Prothena focuses on protein misfolding disorders. Its pipeline features several promising programs targeting proteins believed to play a key role in Alzheimer’s and other neurological conditions with high unmet need.

Prothena’s lead candidate is birtamimab, an antibody-based therapy for AL amyloidosis in late-stage studies. Results expected later this year or in early 2024 could support regulatory filings. Beyond amyloidosis, the company is going all-in on Alzheimer’s research.

The most advanced Alzheimer’s asset is PRX012, an antibody targeting amyloid beta proteins thought to drive disease progression. Prothena is also developing a dual vaccine dubbed PRX012/PRX123 against amyloid beta and tau proteins. Reducing both proteins simultaneously may provide better clinical benefit.

These programs have already attracted partnership interest. Prothena has neuroscience collaborations with pharmaceutical giants Bristol Myers Squibb, Roche, and Novo Nordisk. Just this month, Bristol Myers exercised an option to license PRX005, Prothena’s anti-tau antibody, for nearly $60 million upfront.

But Prothena may seek an acquirer willing to buy the entire company outright. The potential payoff from Alzheimer’s success is massive given the huge unmet need. With late-stage data upcoming, now may be the optimal time for a sale.

Any acquirer would gain full access to Prothena’s Alzheimer’s pipeline, along with its programs for Parkinson’s disease and other neurodegenerative disorders. Adding these diverse assets to an existing neuroscience portfolio could create exciting synergistic opportunities.

Prothena’s small size also makes it financially digestible for big pharma buyers. The company’s market cap sits around $2.5 billion, presenting a worthwhile gamble for majors like Roche or Bristol Myers with scores of billions in annual revenue.

News of the company exploring strategic options sent Prothena shares surging 25% on nearly 6x normal volume. Investors are betting a buyout could be announced in coming months. Prothena’s pipeline progress makes it an alluring target.

Upcoming clinical results will prove whether Prothena’s Alzheimer’s bet pays off scientifically. But from a business perspective, the stars may be aligning for a near-term acquisition. Cash-rich pharmas need new prospects to bolster aging portfolios, and Prothena boasts some of the most exciting neurological assets out there.

If Prothena’s Alzheimer’s programs demonstrate strong efficacy, bidding wars could drive the buyout price sky-high. With biotech valuations rebounding from lows, management may see now as the perfect time to capitalize on these assets. Whether via sale, partnership or remaining independent, Prothena’s future direction should become clearer soon.

Pfizer’s Earnings Forecast Drops Due to COVID Products Decline

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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?  

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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.

Release – Eledon Pharmaceuticals to Present Data from Ongoing Phase 1b Trial of Tegoprubart in Patients Undergoing Kidney Transplantation at the American Society of Nephrology Kidney Week 2023 Annual Meeting

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Research News and Market Data on ELDN

IRVINE, Calif., Oct. 13, 2023 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that the Company will present a poster at the American Society of Nephrology’s upcoming Kidney Week 2023 Annual Meeting taking place in Philadelphia, PA from November 2-5, 2023. The poster will highlight new data from Eledon’s ongoing open-label Phase 1b trial evaluating tegoprubart for the prevention of rejection in kidney transplantation.

Details on the poster presentations are below:

Title: Tegoprubart for the prevention of rejection in kidney transplant: update of emerging data from an ongoing trial
Presenter: Steve Perrin, Ph.D., President and Chief Scientific Officer, Eledon Pharmaceuticals
Poster Number: TH-PO835
Session Title: Transplantation: Clinical – I [PO2102-1] 
Session Date and Time: November 2, 2023 from 10:00 AM to 12:00 PM EDT

Following the presentation, a copy of the poster can be found on the Investor section of the Company’s website at https://ir.eledon.com/events-and-presentations/presentations.

About Eledon Pharmaceuticals and Tegoprubart (formerly AT-1501)

Eledon Pharmaceuticals is a clinical stage biotechnology company with immunology expertise that is developing therapies to protect and prevent rejection of transplanted organs, as well as to treat amyotrophic lateral sclerosis (ALS). The Company’s lead compound in development is tegoprubart, an anti-CD40L antibody with high affinity for CD40 Ligand (also called “CD154”), a well-validated biological target with broad therapeutic potential. Eledon is headquartered in Irvine, California. For more information, please visit the company’s website at www.eledon.com.

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Investor Contact:

Stephen Jasper
Gilmartin Group
(858) 525 2047
[email protected]

Media Contact:

Jenna Urban
Berry & Company Public Relations
(212) 253 8881
[email protected]

Source: Eledon Pharmaceuticals

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

Posted on by [email protected]


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.


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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

Posted on by [email protected]

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.

Release – Encouraging Rigosertib Data Presented at EADV As Late Breaker

Posted on by [email protected]

Research News and Market Data on ONTX

Oct 12, 2023

PDF Version

Reporting responses in patients with RDEB-associated SCC, a devastating disease with a significant unmet need

NEWTOWN, Pa., Oct. 12, 2023 (GLOBE NEWSWIRE) — (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”, the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that a late-breaking abstract studying rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB), associated with advanced/metastatic squamous cell carcinoma (SCC), will be presented today in the Late Breaking News session at the European Academy of Dermatology and Venereology (EADV) in Berlin, Germany, by Professor Dr. Johann Bauer, from the University Hospital Salzburg, Dermatology and Allergology of the PMU, Salzburg, Austria, an Investigator on the investigator-led study (IST).

“RDEB-associated SCC is a devastating disease with few if any treatment options for advanced patients. We are very pleased for additional data on the potential use of rigosertib in patients with RDEB-associated SCC to be presented as a ‘late breaker’ at EADV 2023,” said Steven Fruchtman, M.D., President and CEO of Onconova. “We believe that rigosertib’s profile and impact on key cell signaling pathway targets, including PLK-1 kinase, could make rigosertib a very attractive approach for this indication and other cancers. We are employing an “investigator-sponsored trial” strategy to evaluate rigosertib and believe the data that are being presented at EADV 2023 highlight the potential clinical utility of the compound.”

Professor Bauer commented, “I have been working with patients and their families affected by RDEB and the devastating sequalae of squamous cell carcinoma. I am pleased to share the initial encouraging patient experience with rigosertib at this conference. We are keen to further enroll other potential patients on this study.”

Presentation Details

Date:Thursday, October 12, 2023
Time:4:00 to 4:15 p.m. in Germany/10:00 to 10:15 a.m. ET
Abstract Title:Efficacy and Safety of Rigosertib in Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) Associated Advanced/Metastatic Squamous Cell Carcinoma (SCC)
Presenter:Prof. Dr. Johann Bauer, M.D., MBA
Session:D2T01.3: Late breaking news
  

Abstract Details

The abstract outlines promising results from an open-label, single arm safety and efficacy study of four patients with recessive dystrophic epidermolysis bullosa (RDEB) associated advanced/metastatic squamous cell carcinoma (SCC) treated with intravenous/IV or oral rigosertib. Two patients achieved complete cutaneous responses of 12 months or more, one patient experienced metastatic disease progression, and one patient remains on study. Responding patients included one patient who achieved both cutaneous and histological remission of 16 months. The patient’s rigosertib dose was reduced following the report of a grade 2 adverse event (irritative cystitis), which led to symptomatic relief. The other responding patient achieved a complete cutaneous remission and completed the protocol-specified 12-months of therapy. The authors suggest that the preliminary study results indicate rigosertib as a potential treatment for RDEB-associated SCC. Additional patients are being sought for the trial.

A copy of the Abstract will be available on the Scientific Presentations section of the Onconova website following the Presentation.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company’s product candidates, narazaciclib and rigosertib, are proprietary, targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Narazaciclib, Onconova’s novel, multi-kinase inhibitor (formerly ON 123300), is being evaluated in a Phase 1/2 combination trial with the estrogen blocker letrozole, in advanced endometrial cancer (NCT05705505). Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova believes narazaciclib has broad potential and is also evaluating opportunities for combination studies with narazaciclib and letrozole in additional indications, including breast cancer.

Rigosertib is being studied in an investigator-sponsored trial strategy to evaluate the product candidate in multiple indications, including 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 (NCT04263090), a Phase 2 program evaluating oral or IV rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC) (NCT03786237, NCT04177498), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma (NCT05764395).

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

Forward Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding its clinical development and trials, its product candidates, its business and financial position. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Mark Guerin
Onconova Therapeutics, Inc.
267-759-3680
[email protected]
https://www.onconova.com/contact/

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
[email protected]

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

Posted on by [email protected]


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.


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

Posted on by [email protected]

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.

Release – PDS Biotech Announces Interim Safety and Immune Response Data from Phase 1/2 Clinical Trial Evaluating Novel Antibody Drug Conjugate PDS0301 Combined with Docetaxel to Treat Metastatic Prostate Cancer

Posted on by [email protected]

Research News and Market Data on PDSB

  • First study of patients with metastatic prostate cancer to evaluate standard-of-care chemotherapy (docetaxel) combined with an antibody drug conjugate (PDS0301).
  • Decrease in prostate specific antigen (PSA) levels was seen in all patients at all three tested doses of PDS0301.
  • The combination was well-tolerated at all tested dose levels.
  • Data to be presented by National Cancer Institute as an oral presentation at Cytokines 2023.

PRINCETON, N.J., Oct. 11, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or the Company), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell activating platforms, today announced interim safety and immune response data for the first-in-human Phase 1/2 clinical trial evaluating PDS0301, a novel investigational tumor-targeting, antibody-conjugated Interleukin 12, in combination with current standard-of-care (SOC) chemotherapy, docetaxel, to treat metastatic castration sensitive (mCSPC) and castration resistant (mCRPC) prostate cancer. The data will be featured in an oral presentation by Ravi A. Madan, MD, Head, Prostate Cancer Clinical Research Section, Genitourinary Malignancies Branch, Center for Cancer Research of the National Cancer Institute, an Institute of the National Institutes of Health, at the 11th Annual Meeting of the International Cytokine & Interferon Society (Cytokines 2023) in Athens, Greece.

“We are encouraged by the preliminary data from the Phase 1/2 clinical trial evaluating PDS0301 in combination with docetaxel for patients with metastatic prostate cancer which has the potential to improve treatment outcomes for patients with advanced and refractory prostate cancers that have spread to other parts of the body,” said Lauren V. Wood, MD, Chief Medical Officer of PDS Biotech.

Eighteen patients (11 with mCSPC and 7 with mCRPC) with a median age of 69 years (range 39-82) were evaluated for clinical activity and toxicity. Three dose levels of PDS0301 (8.0 mcg/kg, 12.0 mcg/kg, and 16.8 mcg/kg) in combination with docetaxel (75 mg/m2) were administered every three weeks beginning with the second cycle of treatment. The dose-limiting toxicity (DLT) window spanned the 6 weeks after initiating docetaxel. While all doses of PDS0301 were well-tolerated, the 12.0 mcg/kg dose of PDS0301 with chemotherapy provided the best combination of immune response and tolerability.

Interim data highlights to be presented at Cytokines 2023 include:

  • Decrease in PSA levels was seen in all patients at all three tested doses of PDS0301 and ranged from -4% to -100%.
  • All doses of the combination were well-tolerated with one patient experiencing Grade 4 neutropenia.
  • Administration of the combination was associated with decreases in T reg cells and increases in activated natural killer (NK) cells, memory CD8 T cells, proliferating CD4 and CD8 T cells and cytokines INF-γ and Interleukin 10 (IL-10).
  • The changes in immune responses with the combination were independent of the PDS0301 dose.

“The interim data show that adding PDS0301 to docetaxel was associated with increases in peripheral activated natural killer cells, central memory CD8, proliferating CD4 and CD8 cells in addition to cytokines interferon-gamma and Interleukin 10 as well as decreases in T regulatory cells,” said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. “As the first clinical study to evaluate docetaxel and an immunocytokine, we were pleased to see that the combination can be administered every 3 weeks and look forward to its continued evaluation and impact on clinical outcomes for the treatment of metastatic prostate cancer.”

For patients interested in enrolling in this clinical trial, please contact NCI’s toll-free number: 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615), email [email protected], or visit https://trials.cancer.gov using the identifier NCT04633252.

About PDS0301
PDS0301 is a novel investigational tumor-targeting antibody drug conjugate of Interleukin 12 (IL-12) that enhances the proliferation, potency and longevity of T cells and natural killer (NK) cells in the tumor microenvironment. PDS0301 is given by subcutaneous injection and is designed to improve the safety profile of IL-12 and to enhance the anti-tumor response.

About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune®, Versamune® plus PDS0301, and Infectimune® T cell-activating platforms. We believe our targeted immunotherapies have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the ability to reduce and shrink tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers in multiple Phase 2 clinical trials and will be advancing into a Phase 3 clinical trial in combination with KEYTRUDA® for the treatment of recurrent/metastatic HPV16-positive head and neck cancer in 2023. 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.

Forward Looking Statements
This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune® based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune® based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to the Company’s currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. 

Versamune® and Infectimune® are registered trademarks of PDS Biotechnology Corporation. KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

Investor Contact:
Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: [email protected]

Media Contact:
Gina Cestari
6 Degrees
Phone: +1 (917) 797-7904
Email: [email protected]