Biopharmaceutical Archives - Page 5 of 25

Rare Disease Pharma Play: Cycle Bids $466M for Vanda

Posted on June 6, 2024June 6, 2024 by slightbourne@noblecapitalmarkets.com

Cycle Pharmaceuticals Ltd., a rapidly growing pharmaceutical company laser-focused on rare diseases, has set its sights on acquiring Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) for $8.00 per share in an all-cash transaction valuing Vanda at $466 million.

The unsolicited proposal, disclosed publicly on June 6th, represents an attractive 98% premium to Vanda’s share price prior to an earlier $4.05 per share acquisition offer from Future Pak LLC announced in April. Cycle’s $8.00 bid also represents a 58% premium to Vanda’s closing price on June 5th.

Vanda, which has been publicly traded since 2006, currently markets therapies for sleep disorders, jet lag, and schizophrenia, with additional pipeline candidates in development. The company’s shares have struggled over the past year, trading as low as $3.30 before the Future Pak offer surfaced.

Cycle was founded in 2012 with the sole mission of developing and commercializing treatments for underserved rare disease patients. The company has quickly built an arsenal of six approved drugs, including recent U.S. launches of TASCENSO ODT for multiple sclerosis in 2023 and TIOPRONIN for a rare metabolic disorder in 2024.

In disclosing its proposal publicly, Cycle cited its “extensive U.S. operational footprint and distribution,” stating this makes it “a strong strategic fit” to maximize the value of Vanda’s commercial products and pipeline. Cycle reported $109 million in 2023 net sales and $40 million in operating profits.

The proposal represents “immediate, compelling and certain cash value” for Vanda shareholders according to Cycle. Its $8.00 per share cash bid exceeds the cash component of Future Pak’s most recent $23 per share revised offer on May 7th, which included stock and contingent value rights.

Cycle stated it has substantial cash reserves on hand and is highly confident it can secure committed debt financing for the transaction after limited due diligence. The firm is aiming to complete diligence within 2-3 weeks and finalize a definitive merger agreement shortly thereafter.

While Cycle stated a preference to reach an agreement privately with Vanda’s board, it has gone public with its proposal “for the benefit of Vanda shareholders” to encourage them to voice support for the premium cash bid.

The rare disease focus of both companies could make this an intriguing strategic fit, while Cycle’s bold premium cash offer puts the onus on Vanda’s board to either embrace this higher-valued bid or make a compelling case that greater long-term value could be unlocked by rejecting it. Regardless, this acquisition play instantly ratchets up the stakes in Vanda’s strategic review process.

Release – Tonix Pharmaceuticals Announces Two Oral Presentations and One Poster Presentation Involving TNX-1500 (Fc-modified humanized anti-CD40L mAb) at the American Transplant Congress 2024

Posted on June 6, 2024June 6, 2024 by aweinsoff@channelchek.com

Research News and Market Data on TNXP

June 06, 2024 8:00am EDTDownload as PDF

TNX-1500 displays potential as a monotherapy or combination therapy to prevent rejection in organ transplantation in allograft and xenograft animal models

Research Directed by Faculty of the Center for Transplantation Sciences, Massachusetts General Hospital

Transplantation is also believed to be a model for treating autoimmunity

CHATHAM, N.J., June 06, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, announces two oral presentations and a poster presentation at the American Transplant Congress 2024, held June 1-5, 2024 at the Pennsylvania Convention Center, Philadelphia, Pa. A copy of the oral and poster presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com.

“We remain encouraged by the potential of our TNX-1500, Fc-modified humanized anti-CD40L monoclonal antibody therapy for the prevention of rejection in solid organ transplantation,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Despite advancements in the field of solid organ transplantation, there remains a significant need for new treatments with improved activity and tolerability. Several lines of research indicate that anti-CD40L therapy may improve outcomes for allograft acceptance, and that anti-CD40L therapy may be required for long term xenograft acceptance. We are excited about the broad potential of TNX-1500, on the prevention of allograft and xenograft rejection and also for the treatment of autoimmune diseases like systemic lupus erythematosus and Sjögren’s Syndrome.”

In the oral presentation titled, “Combined Blockade of the CD154 and CD28 Co-Stimulation Pathways Attenuates Pathogenic Alloimmunity and Prolongs Survival in Cynomolgus Cardiac Allografts”, by Kinoshita, K. et al., data demonstrated that the combined use of TNX-1500 and anti-CD28 monoclonal antibody, VEL-101 is associated with durable protection and graft survival and function in a nonhuman primate model.

In the oral presentation titled, “Extended Survival of 9- and 10-Gene-Edited Pig Heart Xenografts with Ischemia Minimization and CD154 Costimulation Blockade-Based Immunosuppression”, Sanatkar, A. et al., data demonstrated that TNX-1500 has promise to prevent rejection of 9-, or 10-gene-edited (GE) pig hearts.^1,2

The poster presentation titled, “Experience with a Novel Delayed Immune Tolerance Protocol in Nonhuman Primates Based on Anti-CD154, Anti-CD2, and Anti-CD28”, by Ileka, I. evaluated whether a modified protocol based on targeting CD28 and CD2 promotes expansion of peripheral regulatory T-cells and is sufficient to promote heart allograft acceptance.

In February 2024, Tonix announced completion of the clinical stage of its Phase 1 single ascending dose study of TNX-1500 in healthy volunteers. The primary objectives of the study are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of intravenous TNX-1500. This first-in-human study is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients.

About TNX-1500

TNX-1500 (Fc-modified humanized anti-CD40L mAb) is a humanized monoclonal antibody that binds and blocks the CD40-ligand (CD40L), also known as CD154. TNX-1500 is being developed for the prevention of allograft and xenograft rejection, for the prevention of graft-versus-host disease (GvHD) after hematopoietic stem cell transplantation (HCT) and for the treatment of autoimmune diseases. A first-in-human Phase 1 trial of TNX-1500 has completed the clinical phase. The primary objective of the Phase 1 trial is to assess the safety, tolerability, PK, and pharmacodynamics of intravenous (i.v.) TNX-1500. Eligible participants enrolled in the Phase 1 trial were distributed across three dosing cohorts (3 mg/kg, 10 mg/kg, and 30 mg/kg, respectively) and evaluated regularly over a 120-day period after dosing. The Phase 1 trial is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients. Two published articles in the American Journal of Transplantation demonstrate TNX-1500 prevents rejection, prolongs survival and preserves graft function as a single agent or in combination with other drugs in non-human primate renal and heart allografts. ^3,4

Revivicor 9-GE pigs: GalKO.β4GalNT2KO.GHRKO.hCD46.hCD55.hTBM.hEPCR.hCD47.hHO-1
Revivicor 10-GE pigs: GalKO.β4GalNT2KO.CMAHKO.GHRKO.hCD46.CD55.hTBM.hEPCR.hCD47.hHO-1.
Lassiter G., et al. Am J Transplantation. 2023. https://doi.org/10.1016/j.ajt.2023.03.022
Miura S., et al. Am J Transplantation. 2023. https://doi.org/10.1016/j.ajt.2023.03.025

Tonix Pharmaceuticals Holding Corp.^*

Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya¹, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace^® SymTouch^® (sumatriptan injection) 3 mg and Tosymra^® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

¹Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Katie Dodge
LaVoieHealthScience
kdodge@lavoiehealthscience.com
(978) 360-3151

Source: Tonix Pharmaceuticals Holding Corp.

Released June 6, 2024

Eledon Pharmaceuticals (ELDN) – Data Update From Phase 1b Trial Shows Continued Tegoprubart Benefits

Posted on June 4, 2024June 4, 2024 by slightbourne@noblecapitalmarkets.com

Tuesday, June 04, 2024

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 From Phase 1b Trial Updated. Eledon presented data from the Phase 1b trial testing tegoprubart, its drug for prevention of kidney transplant rejection, at the American Transplant Congress. We found the data to continue to show improved kidney function during the first year after transplantation, a strong indicator of organ survival, with continued safety and tolerability.

Kidney Function Measures Continue To Show Improvements Over Tacrolimus. The Phase 1b data included 13 patients who had reached 30-day post-transplant evaluation. Their mean eGFR was above 60 mL/min/1.73m² at each reported time point. The overall mean eGFR after day 30 reached 70.5 mL/min/1.73m². This is an improvement over standard-of-care immunosuppressive regimens that have eGFR rates around the 50ml/min/1.73m² level during the first year after the transplant.

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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.

Release – Cadrenal Therapeutics Highlights Presentation Of New Trial Data At ISHLT Conference Demonstrating The Importance Of Anticoagulation Quality In LVAD Patients

Posted on June 3, 2024June 3, 2024 by aweinsoff@channelchek.com

Research News and Market Data on CVKD

Tecarfarin, which recently received Orphan Drug Designation from the FDA for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device, has the potential to improve the time in therapeutic range a factor correlated in the ARIES-HM3 trial with better patient outcomes

PONTE VEDRA, Fla., June 3, 2024 — Cadrenal Therapeutics, Inc., (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage novel oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions, today highlighted a groundbreaking presentation at the International Society for Heart & Lung Transplantation (ISHLT) 44th Annual Meeting & Scientific Sessions.

These new findings from secondary analyses of the ARIES-HM3 trial were released in a presentation titled, “Impact of Vitamin K Antagonist (VKA) Therapy On Outcomes In a Randomized Controlled Trial of Aspirin Removal In Left Ventricular Assist Device (LVAD) Patients – A Pre-Specified Analysis From the Aspirin and Hemocompatibility Events With a Left Ventricular Assist Device in Advanced Heart Failure, or the ARIES-HM3, Randomized Clinical Trial.”

The ARIES-HM3 trial data demonstrated that lower time in therapeutic range, or TTR, translated directly to excessive bleeding events. The “average” patient in the ARIES-HM3 study had a 30% rate of serious bleeding events even after aspirin was eliminated as part of the antithrombotic regimen, and persistent bleeding was inversely correlated with TTR.

The ARIES-HM3 clinical study was sponsored by Abbott (NYSE: ABT), which evaluated a new clinical approach to patient management that included removal of aspirin as part of the antithrombotic regimen warfarin. The data is currently under review by the FDA. Labeling changes related to the antithrombotic regimen have not been approved by the FDA at this time.

Dr. Mandeep Mehra, who chaired the ARIES-HM3 study, holds the William Harvey Distinguished Chair in Advanced Cardiovascular Medicine and is Executive Director of the Center for Advanced Heart Disease at Brigham and Women’s Hospital, commented, “This comprehensive analysis identifies adequacy of VKA use (as measured by TTR) as a significant risk marker for bleeding events and provides new clinical direction for further mitigation of bleeding to enhance hemocompatibility with the HeartMate 3 LVAD.” Mehra continued, “Each incremental improvement of 10% above the median of 56% TTR trends in a significant further reduction in bleeding rate. Tecarfarin could potentially be an important therapy for patients with LVADs who all require chronic anti-coagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin and deserves further study.”

“The ARIES-HM3 trial data underscores the deficiencies of warfarin and the need for a new VKA therapy for patients with LVADs. We believe our drug candidate, the next-generation VKA tecarfarin, with its unique retrometabolic design that provides for more stable anticoagulation than warfarin, is the much-needed replacement therapy. We intend to pursue a pivotal trial evaluating tecarfarin effectiveness for LVAD patients,” said Quang Pham, Founder, Chairman and Chief Executive Officer of Cadrenal Therapeutics. “Cadrenal commends Abbott’s commitment to LVAD patients in sponsoring this important trial and analyses.”

On April 9, 2024, Cardenal Therapeutics announced that the United States Food and Drug Administration (FDA) had granted tecarfarin Orphan Drug Designation for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device, which includes the left ventricular assist device (LVAD).

The current market-leading direct oral anticoagulants (DOACs), such as Eliquis, are not indicated for patients with LVADs due to a lack of evidence of benefit, while the level of anticoagulation achieved with warfarin was achieved in the target range only 56% of the time in the ARIES-HM3 trial. Patients whose level of anticoagulation was in the therapeutic range > 56% had better outcomes than those with lower levels. This highlights the potential role for investigating new VKA agents in improving clinical outcomes in LVAD patients.

VKA anticoagulation is prescribed for the prevention of LVAD-related clotting. However, the only available VKA is warfarin, which was approved for human use in 1954. Tecarfarin has been shown to improve TTR, particularly in patients taking multiple medications, and be more stable in patients with renal dysfunction which is common in LVAD patients.

ABOUT CADRENAL THERAPEUTICS, INC.
Cadrenal Therapeutics is developing tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions. Tecarfarin has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease (ESKD) and atrial fibrillation (AFib) and just received orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices (VADs). Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome (APS). Tecarfarin is a next-generation Vitamin K Antagonist (VKA) specifically designed to use a different metabolism pathway than the oldest and most commonly prescribed VKA warfarin. Tecarfarin has been evaluated in eleven (11) human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit: www.cadrenal.com.

Safe Harbor Statement
Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding tecarfarin having the potential to improve the time in therapeutic range which analysis of the ARIES-HM3 trial reveals is associated with better patient outcomes. TTRs as predicting increased risk for bleeding events, the VKA tecarfarin, filling the market void for a next-generation VKA, the Company pursuing a pivotal trial evaluating tecarfarin effectiveness for LVAD patients. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve the time in therapeutic range, the ability of the Company to advance tecarfarin with patients with left ventricular assist devices (LVADs), thrombotic APS, and those with AFib and ESKD and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the SEC, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.

For more information, please contact:

Cadrenal Therapeutics:
Matthew Szot, CFO
858-337-0766
press@cadrenal.com

Investors:
Lytham Partners, LLC
Robert Blum, Managing Partner
602-889-9700
CVKD@lythampartners.com

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SOURCE Cadrenal Therapeutics, Inc.

Release – Tonix Pharmaceuticals Presented New Data on Tonmya™ Suggesting Activity for Improvement in Fibromyalgia-Associated Depression Severity in an Oral Presentation at ASCP Annual Meeting

Posted on June 3, 2024June 3, 2024 by aweinsoff@channelchek.com

Research News and Market Data on TNXP

June 03, 2024 8:00am EDT

Tonmya treatment was associated with improvement in depressive symptoms as measured by the Beck Depression Inventory-II

In addition, post-hoc analyses showed improvement in depression, anxiety, memory and energy items on the Fibromyalgia Impact Questionnaire-Revised

New Drug Application (NDA) submission to the FDA on track for the second half of 2024

CHATHAM, N.J., June 03, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, presented new data from the Phase 3 RESILIENT trial of Tonmya (TNX-102 SL, cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia in an oral presentation at the American Society of Clinical Psychopharmacology (ASCP) Annual Meeting on May 29, 2024 in Miami Beach, Fla. A copy of the presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com.

In the oral presentation titled, “Effects of Bedtime TNX-102 SL (Sublingual Cyclobenzaprine HCl) on Mood and Anxiety Symptoms in Fibromyalgia: Results of the Phase 3 RESILIENT Trial,” Seth Lederman, MD, Chief Executive Officer, presented new data suggesting activity for improvement in depressive symptoms with Tonmya.

Depression was frequent among patients enrolled in the RESILIENT trial: ~47% reported experiencing depression within the past 6 months upon fibromyalgia diagnosis and ~25% of the intent-to-treat (ITT) population had experienced a lifetime major depressive episode (MDE). The effect of Tonmya on depressive symptoms was studied using the Beck Depression Inventory-II (BDI-II). Patients started with a baseline mean (standard deviation) for placebo of 10.0 (6.72) and Tonmya of 9.6 (6.32). The BDI-II score separated at Week 2 with a nominal p-value of <0.01. By Week 14, the total BDI-II score in the TNX-102 SL group improved over placebo with a nominal p-value of 0.005 and an effect size of 0.27.

Dr. Lederman said, “Although pain is the prototypic symptom in fibromyalgia and the validated FDA endpoint for the approval of a new drug, depression severity is also a prominent factor in the quality of life for fibromyalgia sufferers. In one study, depressive symptoms had a higher correlation with impaired quality of life than any other symptom, including pain frequency and intensity.² The improvement in depression observed in the Phase 3 RESILIENT was particularly striking since the mean entry score of 10 reflects mild depression. Others have struggled to show benefits of traditional antidepressants in mild depression and consequently many antidepressants have been studied in moderate or severely depressed patients and the benefits of such drugs for patients with mild depression have been inferred.”

Dr. Lederman continued, “In addition to the BDI-II score, in post hoc analyses several individual items on the Fibromyalgia Impact Questionnaire-Revised (FIQR) also improved in the Tonmya-treated group, including : depression (p < 0.001), anxiety (p = 0.001), sensitivity (p = 0.020), memory problems (p = 0.001) and energy (p < 0.001), for which these p-values were not corrected for multiplicity. Together these findings indicate that Tonmya has broad-spectrum activity against fibromyalgia symptoms and may improve fibromyalgia at the syndromal level.”

In the RESILIENT trial, as previously reported, Tonmya improved overall daily pain (p=0.00005), the pre-specified primary endpoint, making it the second Phase 3 study of Tonmya in the management of fibromyalgia to reach statistical significance on the pre-specified primary endpoint. Tonmya also demonstrated statistically significant and clinically meaningful results in all six pre-specified key secondary endpoints including those related to improving sleep quality, reducing fatigue, and improving patient global ratings and overall fibromyalgia symptoms and function.”

In the RESILIENT trial, there were no new safety signals, low rates of systemic adverse events, and a favorable tolerability profile.

Tonix remains on track to submit an NDA to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for Tonmya for the management of fibromyalgia and has scheduled a Type B pre-NDA meeting with FDA for the second quarter of 2024.

Tonix Pharmaceuticals Holding Corp.^*

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

²Offenbaecher M, et al. Pain is not the major determinant of quality of life in fibromyalgia. Rheumatology International 2021; 41:1995–2006

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Katie Dodge
LaVoieHealthScience
kdodge@lavoiehealthscience.com
(978) 360-3151

Source:

Released June 3, 2024

Release – Ocugen Set to Join Russell 3000® Index Effective June 28, 2024

Posted on May 28, 2024May 28, 2024 by slightbourne@noblecapitalmarkets.com

Research News and Market Data on OCGN

MALVERN, Pa., May 28, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced its expected upcoming inclusion in the Russell 3000® Index, according to preliminary Russell reconstruction information posted on the FTSE Russell website. The newly reconstructed index will take effect after the market closes on June 28, 2024.

“Inclusion of Ocugen to the Russell 3000® Index is our latest milestone, adding to what has already been a transformational year for the Company with three of our game-changing modifier gene therapies targeting blindness diseases—both rare and those affecting millions—in clinical trials,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This ranking signifies the value of our pipeline, including the recently initiated Phase 3 liMeliGhT clinical trial of OCU400 for broad retinitis pigmentosa, and robust growth strategy, supporting our efforts to enable long-term shareholder value, garner significant visibility of Ocugen within the investment community, and broaden our shareholder base.”

The annual Russell 3000® Index reconstitution measures the performance of the largest 3,000 U.S. companies representing approximately 96% of the investable U.S. equity market as of Tuesday, April 30.

Membership in the U.S. Russell 3000® remains in place for one year and means automatic inclusion in the appropriate growth and value style indexes. FTSE Russell determines membership for its Russell indexes primarily by objective, market-capitalization rankings, and style attributes.

Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Russell’s U.S. indexes serve as the benchmark for about $10.5 trillion in assets as of the close of December 2023. Russell indexes are part of FTSE Russell, a leading global index provider.

About FTSE Russell:
FTSE Russell is a leading global provider of benchmarking, analytics, and data solutions for investors, giving them a precise view of the market relevant to their investment process. A comprehensive range of reliable and accurate indexes provides investors worldwide with the tools they require to measure and benchmark markets across asset classes, styles, or strategies.

FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create ETFs, structured products, and index-based derivatives.

FTSE Russell is focused on applying the highest industry standards in index design and governance, employing transparent rules-based methodology informed by independent committees of leading market participants. FTSE Russell fully embraces the IOSCO Principles, and its Statement of Compliance has received independent assurance. Index innovation is driven by client needs and customer partnerships, allowing FTSE Russell to continually enhance the breadth, depth and reach of its offering.

FTSE Russell is wholly owned by London Stock Exchange Group. For more information, visit: https://www.lseg.com/en/ftse-russell.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the Company’s expected inclusion in the Russell 3000 ® Index, qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that changes may be made to the preliminary Russell reconstruction lists prior to finalization, that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com

Release – YS Biopharma Announces Name Change to LakeShore Biopharma

Posted on May 24, 2024May 24, 2024 by aweinsoff@channelchek.com

GAITHERSBURG, Md., May 24, 2024 /PRNewswire/ — YS Biopharma Co., Ltd. (Nasdaq: YS) (“YS Biopharma” or the “Company”), a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer, today announced it is changing its legal name from “YS Biopharma Co., Ltd.” to “LakeShore Biopharma Co., Ltd”.

Dr. David Shao, Director, President, Co-Chief Executive Officer, and Chief Business Officer of the Company, commented, “We have decided to change our company name to LakeShore Biopharma to align with our global market positioning and international brand image. This change marks one of the first steps for us to expand our market reach, and we are eager to move forward and drive success under our new company name.”

The name change and trading symbol change will not affect any rights of shareholders or the Company’s operations and financial position. The Company’s ordinary shares and warrants will continue to be listed on the Nasdaq. Effective with the opening of the trading day on May 28, 2024, the ticker symbol of the Company’s ordinary shares and warrants will change from “YS” to “LSB” and from “YSBPW” to “LSBPW”, respectively. There is no action required by the Company’s current shareholders with respect to the company name or ticker symbol changes. The Company’s CUSIP will not change in connection with the name change.

About LakeShore Biopharma (formerly known as YS Biopharma)

LakeShore Biopharma, previously known as YS Biopharma, is a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer. It has developed a proprietary PIKA® immunomodulating technology platform and a new generation of preventive and therapeutic biologics targeting Rabies, Coronavirus, Hepatitis B, Influenza, Shingles, and other virus infections. The Company operates in China, the United States, Singapore, and the Philippines, and is led by a management team that combines rich local expertise and global experience in the biopharmaceutical industry. For more information, please visit investor.ysbiopharma.com.

Investor Relations Contact

Alyssa Li
Director of Investor Relations
Email: ir@yishengbio.com

Robin Yang
Partner, ICR, LLC
Tel: +1 (212) 537-4035
Email: YSBiopharma.IR@icrinc.com

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Behind the Scenes of Biotech’s Battle for the Billion-Dollar Diet Pill

Posted on May 24, 2024May 24, 2024 by slightbourne@noblecapitalmarkets.com

For biotech investors scouring for the next big opportunity, the massive manufacturing expansions underway at pharma giant Eli Lilly (LLY) are worth a close look. The company just announced another staggering $5.3 billion investment into a key production facility in Indiana to crank up supply of its blockbuster obesity and diabetes drugs.

This commitment brings Lilly’s total investment at the Lebanon, Indiana site to an incredible $9 billion – representing the firm’s largest-ever manufacturing bet in its nearly 150-year history. It highlights the huge demand that Lilly’s game-changing medications like Mounjaro and Zepbound are facing from physicians and patients alike.

For small biotech firms developing the next generation of weight loss, diabetes and metabolism therapies, Lilly’s supply chain moves send an important signal – this market is headed for explosive growth in the coming years. Companies sitting on promising pipeline candidates could emerge as attractive buyout targets.

At the heart of Lilly’s expansion plans are its incretin drugs, which mimic gut hormones to suppress appetite and regulate blood sugar. Mounjaro, approved for diabetes, and Zepbound, greenlit for chronic weight management, both contain the active ingredient tirzepatide.

Since their launches, demand for these effective and convenient once-weekly injectable treatments has far outstripped supply. Shortages have been widespread in the U.S. as Lilly raced to build out its production infrastructure.

The new $9 billion Indiana campus will be instrumental in increasing Lilly’s capacity to manufacture tirzepatide at scale. When fully operational in 2028, it will employ around 900 skilled workers including scientists, engineers and technicians.

But this plant is just one piece of Lilly’s supply chain mobilization for incretin drugs. Since 2020, the company has plowed over $18 billion into building, acquiring and expanding manufacturing sites in the U.S. and Europe. New facilities are also coming online in North Carolina, Ireland and Germany through 2026.

These investments are already paying dividends. On its latest earnings call, Lilly hiked its 2023 revenue guidance by $2 billion, citing greater visibility into ramping up production of Mounjaro, Zepbound and its incretin pipeline over the remainder of the year.

For small biotechs, the supply chain frenzy at Lilly underscores the commercial opportunity in obesity, diabetes and metabolism. With over 40% of U.S. adults classified as obese, safe and effective chronic weight management regimens like Lilly’s incretin franchise could disrupt a massive global market worth billions annually.

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

The manufacturing expansions suggest appetite for these therapies will continue to surge, fueling demand for the next generation of medications offering better efficacy, tolerability and dosing schedules. Smaller drug developers operating in this space could become prime M&A candidates as deep-pocketed pharmas look to build out their obesity and diabetes portfolios.

Case in point: Lilly itself acquired Zepbound through its $8 billion buyout of Protunor Biopharma in 2022. Several major deals have already reshaped the incretin drug landscape in recent years, including Pfizer’s $6.7 billion purchase of Akero Therapeutics for its NASH/diabetes pipeline.

With its bold investments, Lilly is putting its money where its mouth is when it comes to obesity and metabolic disease. For lean biotechs advancing the next wave of therapies in this booming treatment category, that could spell opportunity knocking in the form of lucrative buyout offers or partnerships down the line.

Keep an eye on this space as Lilly’s supply chain moves underscore that the fight against fat is only just beginning for the pharmaceutical industry.

Release – Traws Pharma’s ICAR Poster Highlights Potency Of COVID-19 Candidate

Posted on May 23, 2024May 23, 2024 by aweinsoff@channelchek.com

Research News and Market Data on TRAW

May 23, 2024

Differentiated resistance profile positions Traws’ program as a potential class leader

NEWTOWN, Pa., May 23, 2024 (GLOBE NEWSWIRE) — Traws Pharma, Inc. (“Traws” or “Traws Pharma”), a clinical stage biopharmaceutical company developing oral small molecules for respiratory viral diseases and cancer, today announced presentation of a poster at the annual International Conference on Antiviral Research (ICAR2024) which is being held from May 20^th to May 24^th in Gold Coast, Australia. The poster highlights positive results of preclinical experiments using patient isolates of COVID19 virus to define the resistance profile of ratutrelvir, formerly known as travatrelvir or TRX01, Traws’ ritonavir-free Mpro protease inhibitor for COVID19, currently in Phase 1 single and multiple ascending dose (SAD/MAD) escalation studies.

“We are very pleased to present the results of resistance studies for ratutrelvir at ICAR2024. Resistance studies are an important part of establishing a differentiation profile for an antiviral agent. Our initial data indicate that ratutrelvir has superior activity compared to nirmatrelvir against a range of omicron variants, based on a comparison of EC₅₀. We believe the results presented at ICAR provide additional positive data to indicate that ratutrelvir has a differentiated resistance profile compared to nirmatrelvir,” said Werner Cautreels, Ph.D., Chief Executive Officer of Traws Pharma.

Dr. Cautreels continued, “Some resistance mutations are common to both drugs but a large part of resistance to nirmatrelvir was not seen with ratutrelvir. Together, ratutrelvir’s differentiated resistance profile, promising pharmacokinetic effects, potential to be used without ritonavir and the accelerated pace of Phase 1 enrollment put the compound on track to advance to Phase 2 studies in H2 2024 and position ratutrelvir as a potential class-leading therapy for COVID- 19.”

C. David Pauza, Ph.D., Chief Scientific Officer, Virology at Traws Pharma said, “Understanding the patterns of resistance mutations gives us the ability to identify circulating viruses that may already be resistant to individual antiviral agents; selecting the right drug can be guided by this information. The resistance studies presented at ICAR compared ratutrelvir to nirmatrelvir, the only Mpro inhibitor approved in the U.S. Our data suggest that ratutrelvir exhibited greater activity compared to nirmatrelvir against wild type Mpro, and remained active against Mpro variants with specific mutations that are associated with nirmatrelvir resistance.”

Dr. Pauza added, “An important finding was that critical mutations accounting for much of the resistance to nirmatrelvir were not found using ratutrelvir, indicating important differences between the two compounds. These differences were most apparent when the nirmatrelvir-resistant mutants P252L or T304I were involved in drug resistance, as these mutations were not observed after ratutrelvir selection.

The data were reported in a poster entitled, “Travatrelvir, an inhibitor of SARS-CoV-2 Main Protease now in Phase 1 Clinical Trials: in vitro Drug Resistance Compared to Nirmatrelvir” (Abstract 356V) presented at ICAR by Bhargava Teja Sallapalli, Master of Veterinary Science, Department of Veterinary Medicine, Virginia-Maryland School of Veterinary Medicine, College Park Maryland et al. The poster can be found on the Scientific Presentations section of the Traws Investor Relations web page.

About Ratutrelvir, the Phase 1 Program and Planned Next Steps

Ratutrelvir (also previously known as 83-0060) was designed as an inhibitor of the SARS-CoV-2 Mpro (3CL protease). It has demonstrated in vitro activity against the original strain of the virus as well as the delta and omicron variants and is more active than nirmatrelvir (PAXLOVID®, Pfizer’s Mpro inhibitor) in preclinical studies. Also in preclinical studies, ratutrelvir did not require co-administration with a human cytochrome P450 (CYP) inhibitor, such as ritonavir, and so it is expected to avoid associated drug:drug interactions, potentially permitting wider patient use. The drug candidate’s pharmacokinetic (PK) profile may enable a once daily, treatment regimen and reduce the likelihood of viral rebound.

The Phase 1 study will evaluate single and multiple ascending doses of ratutrelvir in a double-blinded, placebo-controlled clinical trial to assess safety, tolerability, and pharmacokinetics. Subjects will be randomized 3:1 in five fasted and one fed single ascending dosing (SAD) cohorts and two multiple ascending dosing (MAD) cohorts. Topline data from the study, which is being conducted in Australia (NCT06402136), and the initiation of an international Phase 2 study in subjects with moderate to severe COVID19, are expected to take place in H2 2024.

About Traws Pharma, Inc.

Traws Pharma is a clinical stage biopharmaceutical company developing oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes an oral inhibitor of the SARS-CoV-2 Mpro (3CL protease), ratutrelvir, and tivoxavir marboxil, a new oral antiviral drug candidate for influenza which targets the influenza cap-dependent endonuclease and has shown activity in cell-based assays against drug resistant viruses as well as against avian flu.

In the cancer program, Traws is developing the novel, proprietary multi-kinase CDK4-plus inhibitor narazaciclib for refractory endometrial cancer and potentially other cancers. Narazaciclib targets pathways involved in the development of resistance to CDK inhibitors.

Traws Pharma is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we build solutions for important medical challenges, aiming to alleviate the burden of viral infections and cancer.

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 including statements regarding preclinical studies to evaluate the resistance profile of ratutrelvir, the Phase 1 study of ratutrelvir in Australia and its design, timing and potential results and the timing of a planned Phase 2 study. Traws 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 Traws 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 Traws’ clinical trials, collaborations, market conditions and those discussed under the heading “Risk Factors” in Traws’ filings with the Securities and Exchange Commission. Any forward-looking statements contained in this release speak only as of its date. Traws 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.

Traws Pharma, Inc. Contact:
Mark Guerin
Traws Pharma, Inc.
267-759-3680
www.trawspharma.com

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
bmackle@lifesciadvisors.com

Release – Tonix Pharmaceuticals to Deliver an Oral Presentation and Present Two Posters at the American Society of Clinical Psychopharmacology (ASCP) Annual Meeting

Posted on May 22, 2024May 22, 2024 by aweinsoff@channelchek.com

Research News and Market Data on TNXP

May 22, 2024 8:00am EDT

Oral Presentation of Tonmya™ (TNX-102 SL) for Fibromyalgia; NDA preparation in progress

Posters Highlighting Other TNX-102 SL Programs In Clinical Development; Long COVID and Acute Stress Disorder

CHATHAM, N.J., May 22, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the Company will deliver an oral presentation and present two posters at the American Society of Clinical Psychopharmacology (ASCP) Annual Meeting being held May 28-31, 2024 at the Loews Miami Beach Hotel in Miami Beach, Fla.

The oral presentation will detail findings of studies of Tonmya (TNX-102 SL, sublingual cyclobenzaprine HCl) in fibromyalgia. One poster will describe the Phase 2 proof of concept study of TNX-102 SL in fibromyalgia-type Long COVID. The second poster will describe the upcoming investigator-initiated Phase 2 trial of TNX-102 SL in treating acute stress disorder and preventing posttraumatic stress disorder after motor vehicle collision, which will be conducted by the University of North Carolina, the sponsor of the study.

TNX-102 SL is a centrally acting, non-opioid medication, which is trade named Tonmya™ for the management of fibromyalgia. As previously announced, the second statistically significant Phase 3 study of Tonmya, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo in participants with fibromyalgia (p=0.00005). Statistically significant and clinically meaningful results (p=0.001 or better) were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function.

Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for Tonmya for the management of fibromyalgia and has scheduled a Type B pre-NDA meeting with FDA for the second quarter of 2024.

Copies of the Company’s presentation and posters will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference. Additional meeting information can be found on the ASCP website here.

Oral Presentation Details

Presenter:	Seth Lederman, M.D., Chief Executive Officer
Title:	Effects of Bedtime TNX-102 SL (Sublingual Cyclobenzaprine (CBP) HCl) on Mood and Anxiety Symptoms in Fibromyalgia: Results of the Phase 3 RESILIENT Trial
Date/Time:	May 29, 2024, 3:00 p.m. ET

Poster Presentation Details

Presenter:	Herbert Harris, M.D., Ph.D., Executive Vice President, Translational Medicine
Title:	Effect of Bedtime Sublingual Cyclobenzaprine (TNX-102 SL) on Pain, Sleep, Fatigue and Cognition in Fibromyalgia-Type Long COVID: Results of a Double-Blind Randomized Proof-of-Concept Phase 2 Study
Date/Time:	May 30, 2024, 12:30 p.m. ET
Presenter:	David Hsu, Ph.D., Senior Scientist
Title:	Optimizing Acute Stress Reaction (ASR) Interventions with TNX-102 SL (Sublingual Cyclobenzaprine HCl) – The OASIS Trial: Sustaining Civilian Performance Post-Trauma by Reduction of ASR and Prevention of ASD/PTSD
Date/Time:	May 30, 2024, 12:30 p.m. ET

Tonix Pharmaceuticals Holding Corp.^*

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Katie Dodge
LaVoieHealthScience
kdodge@lavoiehealthscience.com
(978) 360-3151

Source: Tonix Pharmaceuticals Holding Corp.

Released May 22, 2024

Biogen’s Bold $1.8B Kidney Disease Treatment Acquisition

Posted on May 22, 2024May 22, 2024 by slightbourne@noblecapitalmarkets.com

Biogen Inc. is doubling down on novel therapies for rare diseases, announcing an acquisition of Human Immunology Biosciences that could be valued at up to $1.8 billion. The deal gives Biogen full rights to HI-Bio’s lead drug candidate felzartamab, which is being studied for several chronic kidney conditions with large unmet medical needs.

The transaction reflects Biogen’s strategic shift under CEO Christopher Viehbacher to diversify beyond its core neuroscience franchise. Just months after the $6.5 billion buyout of kidney disease specialist Reata Pharmaceuticals, Biogen is opening its checkbook again to beef up its pipeline of potential rare disease medicines.

HI-Bio’s felzartamab has completed mid-stage trials for two types of kidney disorders – primary membranous nephropathy and transplant glomerulopathy where the immune system attacks a transplanted organ. Importantly, it is also being evaluated for IgA nephropathy, a leading cause of kidney failure with no approved treatments available.

For Biogen, the deal provides another shot on goal as it navigates an uncertain period. While its newly-launched Alzheimer’s drug Leqembi has shown promise, the company was forced to abandon its previous Alzheimer’s treatment Aduhelm after years of controversy. Biogen’s older multiple sclerosis franchises are facing rising competitive threats as well.

The HI-Bio acquisition gives Biogen added pipeline diversification into nephrology and autoimmune diseases. Felzartamab has a unique approach, as it is an anti-FcRn antibody that targets pathogenic IgG antibodies which can damage kidneys and other organs.

If felzartamab can demonstrate positive efficacy and safety in broader Phase 3 testing, it could eventually have multi-billion dollar peak sales potential across its targeted kidney indications according to analyst forecasts. However, there is no guarantee of clinical or regulatory success.

From a financial perspective, Biogen is paying $1.15 billion upfront for private HI-Bio, along with contingent value rights worth up to $650 million if certain development and commercial milestones are achieved. This is relatively modest compared to Biogen’s $6.5 billion acquisition of Reata announced in February.

The HI-Bio deal continues Biogen’s aim to revamp its R&D pipeline through a series of bold acquisitions and partnerships under Viehbacher. The company is betting that assembling a portfolio of high-risk, high-reward clinical candidates for diseases like Alzheimer’s and kidney disorders will ultimately pay off.

For the healthcare sector and public markets, Biogen’s aggressive business development approach is emblematic of the ongoing consolidation wave. With rising costs of drug development and payer pricing pressures, large biopharma companies are increasingly looking to acquisitions of smaller, more focused biotechs to source external innovation.

While Biogen’s M&A strategy carries substantial financial risk, the HI-Bio deal gives it a promising asset that could reshape treatment for serious kidney diseases if it can overcome the high hurdle of clinical success. For healthcare investors, absorbing Biogen’s evolving pipeline story will be crucial in evaluating the company’s future growth prospects.

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This $850M Biotech Deal Could Disrupt the Atopic Dermatitis Market

Posted on May 17, 2024May 17, 2024 by slightbourne@noblecapitalmarkets.com

In a strategic move to strengthen its dermatology portfolio, pharmaceutical giant Johnson & Johnson has agreed to acquire Proteologix, a privately-held biotech developing bispecific antibody therapies for inflammatory skin diseases like atopic dermatitis. The $850 million all-cash deal gives J&J access to promising clinical and preclinical stage assets.

The crown jewel of the acquisition is Proteologix’s lead candidate PX128, a Phase 1-ready bispecific antibody targeting two key drivers of atopic dermatitis and asthma – interleukin-13 (IL-13) and thymic stromal lymphopoietin (TSLP). By simultaneously blocking these complementary inflammatory pathways, PX128 could provide a substantial efficacy boost over current monospecific antibody treatments.

Proteologix’s second asset, the preclinical bispecific PX130, goes after IL-13 and IL-22 for the treatment of moderate to severe atopic dermatitis. J&J cited the differentiated design of these dual-acting antibodies, highlighting their potential for infrequent, convenient dosing that could improve adherence.

The acquisition aligns with J&J’s strategic focus on building an immunology pipeline centered around bispecific antibodies for improved disease control across a range of inflammatory conditions.

Atopic dermatitis, a chronic inflammatory skin disease, impacts over 100 million adults worldwide, representing a massive market opportunity. However, up to 70% of patients fail to achieve remission on standard systemic treatments, underscoring a significant unmet need.

“We see an opportunity for best-in-disease efficacy for both PX128 and PX130,” said David Lee, global immunology therapeutic area head at J&J. The company believes the bispecifics could be game-changers for underserved patient subgroups by more comprehensively targeting the heterogenous drivers of atopic dermatitis.

The deal comes on the heels of positive Phase 3 data from Eli Lilly’s IL-13 antibody lebrikizumab in atopic dermatitis. After manufacturing setbacks, Lilly resubmitted its lebrikizumab filing in April and anticipates a decision later this year, setting up a potential commercial clash with J&J’s dual-acting antibodies down the road.

Proteologix, based in California, will be eligible for additional milestone payments on top of the $850 million upfront cash paid by J&J. The transaction, expected to close in mid-2024 pending regulatory approval, will fold in Proteologix’s other preclinical bispecific antibody programs focused on autoimmune diseases and cancer.

For J&J, the deal provides a promising path toward next-generation, differentiated therapies for the significant population of atopic dermatitis patients struggling with existing treatment options. Proteologix’s dual-acting bispecific antibodies represent potentially transformative medicines for a disease area that has proven stubbornly difficult to treat.

The acquisition reinforces J&J’s commitment to immunology and dermatology while bolstering its pipeline with innovative, clinically advanced assets that could drive future growth. As the atopic dermatitis market heats up, J&J has made a preemptive strike to secure a competitive edge through its newest biotech addition.

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Release – Traws Pharma Reports First Quarter 2024 Financial Results And Provides Business Update

Posted on May 16, 2024May 17, 2024 by aweinsoff@channelchek.com

May 16, 2024

PDF Version

Completed acquisition of Trawsfynydd and concurrent $14 million Capital Raise

Excellent pipeline progress, led by candidates for COVID 19, influenza, and oncology programs

Poised to initiate Phase 2 studies in H2 2024 for our influenza candidate and
ritonavir-free COVID 19 protease inhibitor

NEWTOWN, Pa., May 16, 2024 (GLOBE NEWSWIRE) — Traws Pharma, Inc. (“Traws” or “Traws Pharma”), a clinical stage biopharmaceutical company developing oral small molecules for respiratory viral diseases and cancer, today announced financial results for the first quarter of 2024, and provided a business update.

“2024 has already been a transformative year for Traws Pharma to advance our portfolio of novel treatments for serious respiratory infections and oncology programs. We completed the acquisition of Trawsfynydd and concluded a concurrent $14 million private placement financing. In addition, we initiated first-in-human dosing for our COVID 19 product candidate, including completion of the first cohort dosing group. Furthermore, we completed the last dose escalation cohort for our CDK4+ inhibitor, narazaciclib,” stated Werner Cautreels, Ph.D., Chief Executive Officer of Traws Pharma. “We believe that we are poised to make even more meaningful progress in the second half of 2024, as we advance our influenza treatment and ritonavir-free protease inhibitor for COVID 19 into expanded Phase 1 dose escalation studies and begin Phase 2 development.”

“Based on the preclinical profile and early clinical data from our infectious disease candidates and narazaciclib, I am optimistic about the outlook for Traws’ portfolio and look forward to updating our investors with our progress through the year,” concluded Dr. Cautreels.

Traws Proprietary Portfolio Highlights:

TRX100 (tivoxavir marboxil): a cap-dependent endonuclease inhibitor for influenza: Phase 1

Targets the cap-dependent endonuclease of influenza and is a potent inhibitor of influenza virus replication including A and B strains
First Phase 1 study demonstrated safety and tolerability in healthy volunteers with pharmacokinetics and pharmacodynamics (PK/PD) data to support the potential use of a single oral dose for treatment or prophylaxis
We plan to initiate Phase 1 dose extension to evaluate one additional, higher dose prior to the initiation of Phase 2 studies in H2 2024. Topline data from the Phase 2 study are expected in H1 2025

TRX01 (ratutrelvir): a ritonavir-free Mpro protease inhibitor for COVID19: Phase 1

Potent oral inhibitor of SARS-CoV-2 Mpro (3CL protease), effective against the original, delta, and omicron variants of SARS-CoV-2, that does not require co-administration with ritonavir, reducing the risk of drug-drug interactions. Preclinical data support once-daily dosing for 10days which could overcome viral rebound seen with other agents.
We are in the process of conducting a Phase 1 first-in-human single ascending dose/multiple ascending dose (SAD/MAD) study in normal volunteers. The second dosing cohort is underway and topline data are expected H2 2024. A Phase 2 study is also planned to begin in H2 2024 in patients with moderate to severe COVID19. Topline data are expected H1 2025

Narazaciclib: CDK 4+ to treat solid tumors: Phase 1/2

Available preclinical and clinical data suggest that narazaciclib is active in numerous tumor types, inhibiting CDK 2/4/6, CSF1R and ARK 5/NUAK1. Preclinical studies also showed reduced neutropenia, as compared to palbociclib, and inhibition of palbociclib resistant cancer cells.
A dose escalation study to define the recommended Phase 2 dose (RP2D) recently enrolled the last cohort. A review of the clinical and PK/PD data is underway. We intend to utilize these data to define the clinical strategy, including selection of a lead indication and next steps in its development.

First Quarter 2024 Financial Results

Cash and cash equivalents as of March 31, 2024, were $16.4 million, compared with $20.8 million as of December 31, 2023.

In April 2024, the Company raised gross proceeds of $14 million from the sale of common and preferred stock to TPAV, LLC, an affiliate of Torrey Pines, and OrbiMed Private Investments VIII, LP, an affiliate of OrbiMed Advisors.

The Company believes that its cash and cash equivalents will be sufficient to fund ongoing clinical trials and business operations into the fourth quarter of 2024.

Revenue was fifty-six thousand dollars for the first quarter of 2024, consistent with the same period in 2023.

General and administrative (G&A) expenses were $3.4 million for the first quarter of 2024, compared with $2.1 million for the same period in 2023. The increase in G&A expenses was caused by higher legal and professional fees related to the Trawsfynydd acquisition on April 1, 2024, partially offset by lower bonus accrual as well as lower insurance, meeting, and public company expenses.

Research and development (R&D) expenses were $1.9 million for the first quarter of 2024, compared with $4.1 million for the same period in 2023. The decrease was primarily caused by lower costs related to narazaciclib drug substance and drug product manufacturing, a reduction in clinical development and consulting costs and lower personnel expenses due to lower bonus accrual.

Net loss for the first quarter of 2024 was $5.0 million, or $0.24 per share on 20.8 million weighted average shares outstanding, compared with a net loss of $5.8 million, or $0.28 per share for the same period in 2023, based on 20.8 million weighted average shares outstanding.

About Traws Pharma, Inc.

In the cancer program, Traws is developing the novel, proprietary multi-kinase CDK4-plus inhibitor narazaciclib potentially for refractory endometrial cancer and other solid tumor cancers. Narazaciclib targets pathways involved in the development of resistance to CDK inhibitors.

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 including statements regarding the Phase 1 study of ratutrelvir in Australia and its design, timing and potential results and the timing of a planned Phase 2 study. Traws 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 Traws 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 Traws’ clinical trials, collaborations, market conditions and those discussed under the heading “Risk Factors” in Traws’ filings with the Securities and Exchange Commission. Any forward-looking statements contained in this release speak only as of its date. Traws 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.

Traws Pharma, Inc. Contact:
Mark Guerin
Traws Pharma, Inc.
267-759-3680
www.trawspharma.com

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
bmackle@lifesciadvisors.com