Tag: Biopharmaceutical

Research – ZyVersa Therapeutics Announces a Publication in the Peer-Reviewed Journal, Aging, Linking Inflammasome NLRP3 Activation with Age-Related Structural Changes in the Kidney and Reduced Kidney Function

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Oct 18, 2023

  • Chronic kidney disease is most common in people 65 years and older.
  • NLRP3 inflammasomes signaling in the kidneys’ filtration system (“glomerular podocytes”) occurs as early as middle age, leading to scarring, podocyte loss, and impaired function, which may be a critical contributor to a lower threshold for developing kidney disease in older people.
  • Age-related inflammasome signaling and its effects are compounded in the presence of kidney disease, leading to poorer outcomes.
  • ZyVersa is developing Inflammasome ASC Inhibitor IC 100 which can inhibit up to 12 different inflammasomes (including NLRP3 inflammasomes) and their associated ASC specks which perpetuate damaging inflammation.

WESTON, Fla., Oct. 18, 2023 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, announces publication of an article in the peer-reviewed journal, Aging, demonstrating that inflammasome NLRP3 activation is associated with an age-related decline in kidney health, which is compounded in the presence of kidney disease resulting in worsening outcomes.

In the paper titled, “Inhibiting NLRP3 signaling in aging podocytes improves their life-and health-span,” the authors conducted studies in various mouse models, human kidney tissues, and human kidney organoids. Data demonstrate a critical role for NLRP3 inflammasomes in age-related kidney deterioration, which is exacerbated in the presence of kidney disease. Following are key findings reported in the paper:

  • Glomerular NLRP3 levels in human kidney tissue were associated with pathological changes in the kidneys’ filtration system: higher levels of glomerular scarring (glomerulosclerosis) and enlargement (hypertrophy), and reduced podocyte density.
  • Glomerular NLRP3 inflammasome levels were higher in aged mice with experimental focal segmental glomerulosclerosis (FSGS) compared to age-matched mice without disease, indicating that FSGS injury augments the age-dependent increase of NLRP3 signaling.
  • NLRP3 inflammasome signaling and its effects on podocytes may be a critical contributor to a lower threshold for developing kidney disease in older people.
  • Reduction in NLRP3 signaling, either pharmacologically or by gene deletion, in human kidney organoids and in middle-aged mice decreased age-associated podocyte injury.

The authors stated, “By showing that in aged mice with experimental FSGS, injury augments the age-dependent increase of NLRP3 signaling, it makes this pathway an intriguing therapeutic target for podocyte (kidney) diseases in the elderly.” To read the article, Click Here.

“The research published in the Journal, Aging, demonstrates that age-related NLRP3 inflammasome signaling in the kidneys’ filtration system leads to scarring and podocyte loss, which are exacerbated when superimposed by kidney disease, leading to poorer outcomes,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO and President. “This research provides support for inflammasome inhibition as a promising treatment option for kidney disease, which is most common in the elderly. ZyVersa is developing Inflammasome ASC inhibitor IC 100. Unlike NLRP3 inhibitors, designed to inhibit formation of the NLRP3 inflammasome to block initiation of the inflammatory cascade, IC 100 was designed to inhibit formation of multiple types of inflammasomes, and to uniquely inhibit their associated ASC specks to block perpetuation of damaging inflammation.” To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response.

About ZyVersa Therapeutics, Inc.

ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and other inflammatory diseases. For more information, please visit www.zyversa.com.

Cautionary Statement Regarding Forward-Looking Statements

Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.

New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

Corporate and IR Contact:
Karen Cashmere
Chief Commercial Officer
kcashmere@zyversa.com
786-251-9641

Media Contacts
Tiberend Strategic Advisors, Inc.
Casey McDonald
cmcdonald@tiberend.com
646-577-8520

Dave Schemelia
dschemelia@tiberend.com
609-468-9325

Research – Tonix Pharmaceuticals Announces Publication of Data in the Journal Nature Involving TNX-1500 (Fc-modified dimeric anti-CD40L mAb) for the Prevention of Rejection in Kidney Xenotransplantation in Animal Models

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

October 18, 2023 7:00am EDT

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

TNX-1500 is Enrolling in a Phase 1 Clinical Trial

Tonix is Developing TNX-1500 for Prevention of Kidney Allograft Rejection as the First Indication: Multiple Other Indications, including Autoimmune Disorders, are Planned

CHATHAM, N.J., Oct. 18, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that a study published in the Journal Nature1 by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital (MGH) in collaboration with biotechnology company, eGenesis, utilized TNX-1500 (Fc-modified dimeric anti-CD40L monoclonal antibody [mAb]) as part of the immune modulating regimen to prevent organ transplant rejection. Tonix’s TNX-1500 is in development for the prevention of human kidney organ transplant rejection. The molecular target of TNX-1500 is CD40-ligand (CD40L), which is also known as CD154.

TNX-1500 was invented and developed in-house by Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals, and colleagues. TNX-1500 is a third generation anti-CD40L monoclonal antibody that has been designed by protein engineering to decrease FcγRII binding and to reduce the potential for thrombosis. Preclinical studies in non-human primates demonstrated that TNX-1500 showed activity in preventing allograft and xenograft organ rejection and was well tolerated. The research in the Nature paper was conducted at MGH, led by principal investigator Tatsuo Kawai, M.D., Professor of Surgery, Harvard Medical School and the Center for Transplantation Science. A “News and Views” editorial2 and a News3 story appeared in the same issue of Nature.

The Nature article titled, “Design and testing of a humanized porcine donor for xenotransplantation” includes data that provide additional support for TNX-1500’s activity in preventing pig xenograft organ rejection and for its tolerability in non-human primates. Because anti-CD40L treatment is widely recognized as critical to the success of xeno organ transplant, no animals were transplanted without anti-CD40L treatment. Four of the transplanted animals received prophylactic treatment with TNX-1500. The other animals were treated with a primate-adapted version of mAb 5c8, which is an earlier antibody that was also discovered by Dr. Lederman, when he was an assistant professor at Columbia University.4 The primate-adapted 5c8 anti-CD40L mAb5 has been provided to qualified researchers at a nominal charge for more than 20 years in an National Institute of Health (NIH)-funded program called the “Non-human Primate Reagent Resource Center” (NHPRRC).

“The animal study described in the Nature publication1 supports the growing evidence that the protein engineering behind the invention of TNX-1500 resulted in a dimeric antibody that retains activity to prevent rejection and preserve graft function. These and other data6,7 confirm the rationale for us to pursue development of TNX-1500 to prevent rejection in human transplantation,” said Dr. Lederman. “We are currently enrolling in a Phase 1 trial with TNX-1500 in healthy volunteers to support the development of TNX-1500 for the prevention of allograft rejection. There remains a significant need for new treatments with improved activity and tolerability to prevent organ transplant rejection. We believe TNX-1500 has the potential for treating and preventing organ transplant rejection.”

Dr. Lederman added, “Our primary focus of early development will be allotransplantation in which the donor organ comes from a human volunteer or cadaver. However, long term we hope to develop TNX-1500 for xenograft transplantation in which the donor organ comes from genetically engineered pigs. Several lines of research indicate that anti-CD40L is required for long term xenograft acceptance. I believe it is unlikely for human xenotransplantation to proceed without CD40L blockade. In addition, anti-CD40L monoclonal antibodies have demonstrated efficacy in autoimmune diseases like systemic lupus erythematosus and Sjögren’s Syndrome.”

About TNX-1500

TNX-1500 (Fc-modified anti-CD40L mAb) is a humanized dimeric monoclonal antibody that interacts with the CD40-ligand (CD40L), which is also known as CD154. TNX-1500 is being developed for the prevention of allograft and xenograft rejection, for the treatment of autoimmune diseases including multiple sclerosis and for the prevention of graft-versus-host disease (GvHD) after hematopoietic stem cell transplantation (HCT). A Phase 1 study of TNX-1500 is currently enrolling. TNX-1500 is a third generation anti-CD40L mAb that has been designed by protein engineering to decrease FcγRII binding and to reduce the potential for thrombosis. The disulfide-linked dimeric structure is similar to natural antibodies and in the case of anti-CD40L is believed to confer to TNX-1500 a higher avidity for cell-associated CD40L, relative to soluble CD40L. Two articles were recently published in the American Journal of Transplantation that demonstrate TNX-1500 prolongs nonhuman primate renal and heart allograft survival6,7. Other anti-CD40L mAbs are in development for treating systemic lupus erythematosus, Sjögren’s syndrome and multiple sclerosis.8-10 CD40-L is a member of the TNFα super gene family. Other members have been the targets of successful mAb: TNFα and RANKL for autoimmune diseases and osteoporosis, respectively. Other TNFα super gene family members are targeted by mAbs in development including, TNF-like ligand 1A (TL1A) and CD30L for ulcerative colitis.

  1. Anand R.P., et al. Nature. 2023. 622, 393–401.
  2. Mohiuddin M. Nature. News and Views. 2023. “Pig-to-primate organ transplants require genetic modifications of donor.”
  3. Kozlov M. Nature. New, 2023. “Monkey survives two years after gene-edited pig-kidney transplant.”
  4. Lederman S, et al. J Exp Med. 1992. 175(4):1091-101.
  5. NHPRRC anti-CD154 clone 5C8H1D MassBiologics PR-1547
  6. Lassiter G., et al. Am. J. Transplant. 2023. https://doi.org/10.1016/j.ajt.2023.03.022
  7. Miura S., et al. Am. J. Transplant. 2023. https://doi.org/10.1016/j.ajt.2023.03.025
  8. UCB Pipeline – https://www.ucb.com/our-science/pipeline
  9. BioSpace. September 12, 2022 – https://www.biospace.com/article/releases/horizon-therapeutics-plc-announces-phase-2-trial-evaluating-dazodalibep-for-the-treatment-of-sjoegren-s-syndrome-meets-primary-endpoint/
  10. Business Wire. January 18, 2023 – https://www.businesswire.com/news/home/20230118005359/en/Horizon-Therapeutics-plc-Announces-Phase-2-Trial-Evaluating-Dazodalibep-for-the-Treatment-of-Sj%C3%B6gren%E2%80%99s-Syndrome-Meets-Primary-Endpoint-in-the-Second-Study-Population-Only-Phase-2-Trial-to-Meet-Primary-Endpoint-in-Both-Patient-Populations

Tonix Pharmaceuticals Holding Corp.*

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment of a potentially confirmatory Phase 3 study in the third quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 proof-of-concept study has been completed, and topline results were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 in the third quarter of 2023, with topline results expected in early November of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, a small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the mu-opioid receptor while maintaining activity and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development as a preventive treatment in chronic migraine, and enrollment has completed in a Phase 2 proof-of-concept study with topline data expected in early December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes 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. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.

*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. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. 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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
443-213-0495

Source: Tonix Pharmaceuticals Holding Corp.

Released October 18, 2023

Gilead Partnership Provides Boost to Assembly Bio’s Antiviral Pipeline

Posted on by slightbourne@noblecapitalmarkets.com

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Release – Tonix Pharmaceuticals Completes Clinical Stage of Phase 2 UPLIFT Study of TNX-601 ER for the Treatment of Major Depressive Disorder

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

October 16, 2023 7:00am EDTDownload as PDF

Topline Results Expected Early November 2023

Tianeptine, the Active Ingredient in TNX-601 ER, is Not Associated with Sexual Dysfunction or Weight Gain Which are Treatment-Limiting Side Effects of Many Traditional Antidepressants

Tianeptine Acts by Directly Restoring Neuroplasticity and Neurogenesis and Limiting the Effects of Oxidative Stress in the Brain

CHATHAM, N.J., Oct. 16, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced the completion of the clinical phase of the Phase 2 potential registration-quality, double-blind, randomized, multicenter, placebo-controlled UPLIFT1 study of TNX-601 ER2 (tianeptine hemioxalate extended-release tablets) as a potential treatment for major depressive disorder (MDD). Topline results from the intention-to-treat (ITT) sample (N=132) are expected in early November 2023.

TNX-601 ER is being developed as a monotherapy and first-line treatment for MDD and works by a distinct mechanism of action as compared to traditional monoaminergic antidepressants. Tonix recently reported that tianeptine activates peroxisome proliferator-activated receptors PPAR-β/δ and PPAR-γ. These activities on intracellular molecular targets in neurons and glia in the brain are believed to relate to tianeptine’s ability to restore connectivity between neurons that atrophy under conditions of stress, in animal models.3,4 Tianeptine is the active ingredient of an antidepressant that has been marketed as a three-times-a-day medicine outside the U.S. for more than 30 years. Tonix has developed a novel, patented once-daily oral formulation.

“Because of its unique mechanism, tianeptine avoids some of the treatment-limiting side effects of traditional antidepressants, including sexual dysfunction and weight gain,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Tianeptine restores connectivity between neurons, or neuroplasticity, and limits the effects of oxidative stress in the brain in animal models of depression. In contrast to traditional antidepressants which alter the levels of neurotransmitters in the synapse, tianeptine directly induces mature neurons to sprout new connections and also induces formation of new neurons.”

“With the last patient now treated, we look forward to analysis of the results of this registration-quality study, which will help to inform our plans as we discuss next steps with the U.S. Food and Drug Administration (FDA),” said Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “We would like to thank the participants, their families, and all the investigators and researchers who have been an important part of this journey so far.”

  1. Clinical Trials.gov I.D. NCT05686408
  2. TNX-601 ER is an investigational new drug and is not approved for any indication.
  3. McEwen, B. S., et al. Mol. Psychiatry 2010, 15 (3), 237–249
  4. Sullivan, GM et al. June 1, 2023. Poster presentation at the American Society of Clinical Psychopharmacology, Miami, FL. https://www.tonixpharma.com/wp-content/uploads/2023/06/ASCP-Poster-2023-A-Randomized-Placebo-Controlled-Multicenter-Trial-of-Monotherapy-with-TNX-601-ER.pdf

About the Phase 2 UPLIFT Study

The Phase 2 UPLIFT study, TNX-TI-M201, is a potential registration-quality, double-blind, randomized, multicenter, placebo-controlled study to evaluate the efficacy and safety of TNX-601 ER taken by mouth once-daily for 6 weeks for the treatment of moderate-to-severe MDD. It is a parallel design study with two arms, a TNX-601 ER 39.4 mg arm and a placebo arm. A total of 132 participants were randomized in a 1:1 ratio into the two arms across approximately 27 U.S. sites, enrolling adult patients 18-65 years old with a DSM-5 diagnosis of depression and a duration for the current major depressive episode of at least 12 weeks. The primary efficacy endpoint is mean change from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score at Week 6. Key secondary efficacy endpoints include the Clinical Global Impression of Severity Scale (CGI-S) and the Sheehan Disability Scale (SDS).

For more information, see ClinicalTrials.gov Identifier: NCT05686408.

About Major Depressive Disorder (Depression)

According to the National Institute of Mental Health, an estimated 21 million adults in the U.S. in 2020 experienced at least one major depressive episode1, with highest prevalence among individuals aged 18-25 at a rate of 17.0%. For approximately 2.5 million adults in the U.S., adjunctive therapies are necessary for depression treatment.2,3 Depression is a condition characterized by symptoms such as a depressed mood or loss of interest or pleasure in daily activities most of the time for two weeks or more, accompanied by appetite changes, sleep disturbances, motor restlessness or retardation, loss of energy, feelings of worthlessness or excessive guilt, poor concentration, and suicidal thoughts and behaviors. These symptoms cause clinically significant distress or impairment in social, occupational, or other important areas of functioning. The majority of people who suffer from depression do not respond adequately to initial antidepressant therapy.4

1 Data Courtesy of SAMHSA on Past Year Prevalence of Major Depressive Episode Among U.S. Adults 2020. Retrieved from http://www.nimh.nih.gov/health/statistics/major-depression.shtml

2 IMS NSP, NPA, NDTI MAT-24-month data through Aug 2017.

3 Kubitz N, et al. PLoS One 2013, 8 (10), e76882.

4 Rush AJ, et al. Am J. Psychiatry 2007, 163 (11), 1905-1917.

About TNX-601 ER

TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a novel oral formulation of tianeptine designed for once-daily daytime dosing in development as a candidate for the treatment of MDD, posttraumatic stress disorder, and neurocognitive dysfunction associated with corticosteroid use. Tianeptine sodium (amorphous) immediate release (dosed 3 times daily) was first marketed for depression in France in 1989 and has been available for decades in Europe, Russia, Asia, and Latin America for the treatment of depression. Tianeptine sodium has an established safety profile from decades of use in these jurisdictions. Currently no tianeptine-containing product is approved in the U.S. and no extended-release tianeptine product is approved in any jurisdiction. Tonix discovered a novel oxalate salt of tianeptine that may provide improved stability, consistency, and manufacturability compared to known salt forms of tianeptine. In animal models, tianeptine restores dendritic arborization of pyramidal neurons in the CA3 region of hippocampus and in the dentate gyrus region promotes new neuron formation and integration into hippocampal networks.1 Tianeptine’s enhancement of neuroplasticity in animal models of stress is believed to be mediated by activation of PPAR isoforms PPAR-β/δ and PPAR-γ, which makes TNX-601 ER’s properties distinct from traditional monoaminergic antidepressants in the U.S. and contributes to its potential for clinical indications beyond MDD and stress disorders. Tianeptine and its MC5 metabolite are also weak mu-opioid receptor (MOR) agonists that present a potential abuse liability if illicitly misused in large quantities (typically abused at 8-80 times the therapeutic dose on a daily basis2). In patients who were prescribed tianeptine for depression, the French Transparency Committee found an incidence of misuse of approximately 1 case per 1,000 patients treated3 suggesting low abuse liability when used at the antidepressant dose in patients prescribed tianeptine for depression. Clinical trials have shown that cessation of a therapeutic course of tianeptine does not appear to result in dependence or withdrawal symptoms following 6-weeks4-8, 3-months9, or 12-months10 of treatment. The ER formulation of TNX-601 includes several potentially abuse deterrent ingredients, such as gel forming polymers which impede extraction. In addition, the tablet’s hardness makes it difficult to crush, cut or grind to fine particle size, which potentially hinders efforts to misuse by insufflation or intravenous routes. Tianeptine’s reported pro-cognitive and anxiolytic effects as well as its ability to attenuate the neuropathological effects of excessive stress responses suggest that it may also be used to treat posttraumatic stress disorder (PTSD), and neurocognitive dysfunction associated with corticosteroid use. TNX-601 ER is expected to have patent protection through 2037.

1 McEwen, B. S., et al. Mol. Psychiatry 2010, 15 (3), 237–249.

2 Lauhan, R., et al. Psychosomatics 2018, 59 (6), 547–53.

3 Haute Authorite de Sante; Transparency Committee Opinion. Stablon 12.5 Mg, Coated Tablet, Re- Assessment of Actual Benefit at the Request of the Transparency Committee. December 5, 2012.

4 Emsley, R., et al. J. Clin. Psychiatry 2018, 79 (4)

5 Bonierbale M, et al. Curr Med Res Opin 2003, 19(2):114-124.

6 Guelfi, J. D., et al. Neuropsychobiology 1989, 22 (1), 41–48.

7 Invernizzi, G. et al., Neuropsychobiology 1994, 30 (2–3), 85–93.

8 Lepine, J. P., et al. Hum. Psychopharmacol. 2001, 16 (3), 219–227.

9 Guelfi, J. D. et al., Neuropsychobiology 1992, 25 (3), 140–148.

10 Lôo, H. et al., Br. J. Psychiatry. Suppl. 1992, 15, 61–65. 

Tonix Pharmaceuticals Holding Corp.*

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment of a potentially confirmatory Phase 3 study in the third quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 proof-of-concept study has been completed, and topline results were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 in the third quarter of 2023, with topline results expected in early November of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, a small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the mu-opioid receptor while maintaining activity and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development as a preventive treatment in chronic migraine, and enrollment has completed in a Phase 2 proof-of-concept study with topline data expected in early December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes 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. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.

*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. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. 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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
443-213-0495

Source: Tonix Pharmaceuticals Holding Corp.

Released October 16, 2023

Alzheimer’s Specialist Prothena Surges on Sale Speculation

Posted on by slightbourne@noblecapitalmarkets.com

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.

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

Posted on by slightbourne@noblecapitalmarkets.com

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Posted on by cpereira@noblefcm.com


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

Posted on by slightbourne@noblecapitalmarkets.com

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 aweinsoff@channelchek.com

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
ir@onconova.us
https://www.onconova.com/contact/

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

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

Posted on by slightbourne@noblecapitalmarkets.com

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.

Haemonetics Expands Hospital Portfolio Through $253 Million Acquisition of OpSens

Posted on by slightbourne@noblecapitalmarkets.com

Medical technology firm Haemonetics Corporation recently announced a definitive agreement to acquire OpSens, Inc. in an all-cash deal valued at approximately $253 million. OpSens is a medical device company specializing in innovative fiber optic sensor technology for interventional cardiology applications. This strategic acquisition allows Haemonetics to expand its hospital business into the high-growth interventional cardiology market estimated at $1 billion.

Haemonetics, based in Boston, offers a suite of products for blood and plasma collection, the surgical suite, and hospital transfusion services. With the addition of OpSens’ sensor-guided guidewire and pressure guidewire products for transcatheter aortic valve replacement (TAVR) and percutaneous coronary intervention (PCI), Haemonetics bolsters its portfolio with clinically validated technology to improve patient outcomes.

OpSens’ core offerings include the SavvyWire, the first sensor-guided guidewire for TAVR procedures which enables shorter hospital stays, and the OptoWire, a pressure guidewire used to aid coronary artery disease diagnosis by measuring key parameters like fractional flow reserve (FFR). OpSens leverages proprietary optical technology across its sensor solutions for medical devices and critical industrial applications.

According to Stewart Strong, President of Global Hospital at Haemonetics, this acquisition expands Haemonetics’ leadership in interventional cardiology while providing a foundation for additional growth. By combining OpSens’ innovative technology with Haemonetics’ commercial infrastructure and hospital relationships, there is tremendous potential to increase adoption and improve patient care globally.

Strategically, Haemonetics gains several advantages from the purchase:

  • Access to a $1 billion total addressable market in interventional cardiology, a specialty area witnessing increasing procedure volume. OpSens’ competitive, clinically validated offerings are well-positioned for long-term growth.
  • The ability to accelerate OpSens product adoption leveraging Haemonetics’ existing commercial footprint and depth of penetration in U.S. hospitals for its VASCADE vascular closure portfolio.
  • Expanded product breadth and enhanced diversification into adjacent applications like industrial sensors. OpSens technology can be leveraged across Haemonetics’ hospital business and new markets.
  • Opportunities for continued R&D, clinical study efforts, and other business development activities to augment internal product development. Haemonetics aims to expand its hospital division via organic and inorganic investments.

Financially, Haemonetics expects the deal will be immediately accretive to revenue growth. On an adjusted basis, earnings per share is also expected to be accretive right away. Due to one-time integration costs, GAAP earnings per share may be slightly dilutive in the first full fiscal year before turning accretive.

Haemonetics will finance the transaction through existing cash balances and its revolving credit facility. This will result in a manageable rise in the company’s net debt to EBITDA ratio to around 2.1x. The purchase is anticipated to close by January 2024, subject to customary approvals.

In summary, the acquisition of OpSens for $253 million in cash strengthens Haemonetics’ position in the attractive interventional cardiology space while providing new technologies, commercial synergies, and earnings accretion over the long-term. It signals a bold move to supplement organic growth with value-enhancing strategic M&A, as Haemonetics looks to deliver innovation and drive better patient outcomes through continued expansion.

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

Release – Tonix Pharmaceuticals Provides Overview of TNX-2900 Program for the Treatment of Prader-Willi Syndrome at the Foundation for Prader-Willi Research Family Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

October 09, 2023 7:00am EDTDownload as PDF

CHATHAM, N.J., Oct. 09, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced that Herbert Harris, M.D., Ph.D., Executive Vice President, Translational Medicine of Tonix Pharmaceuticals, provided an overview of Tonix’s TNX-2900 (potentiated intranasal oxytocin) program at the Foundation for Prader-Willi Research (FPWR) Family Conference in Denver, CO. A copy of the presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com. Additional information can be found on the FPWR conference website here.

The presentation highlights preclinical data showing the enhancing effects of magnesium (Mg2+) on the activation of oxytocin receptors. The Mg2+ enhanced formulation of intranasal oxytocin is the basis for TNX-2900, in development to treat hyperphagia, or pathological over-eating, in children and adolescents with Prader-Willi syndrome (PWS). In preclinical studies, Mg2+ increases the potency of oxytocin, which is an anorexigenic hormone that reduces appetite and signals fullness, potentially improving receptor binding and resulting in improved therapeutic action.

TNX-2900 has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of PWS. Tonix plans to submit an investigational new drug (IND) application to the FDA in the fourth quarter of 2023. There is no treatment currently approved for hyperphagia in PWS.

Tonix licensed the technology to treat PWS from Inserm Transfert, the private subsidiary of Inserm (the French National Institute of Health and Medical Research).

About Prader-Willi Syndrome

Prader-Willi syndrome is recognized as the most common genetic cause of life-threatening childhood obesity1 and affects males and females with equal frequency and all races and ethnicities. The hallmarks of Prader-Willi syndrome are lack of suckling in infants and, in children, adolescents, and adults, severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications associated with significant mortality. There is currently no approved treatment for either the suckling deficit in babies or the obesity and hyperphagia in older children associated with Prader-Willi syndrome.

1 Foundation for Prader-Willi Research (fpwr.org).

About TNX-2900 and Tonix’s Potentiated Oxytocin Platform

TNX-2900 is based on Tonix’s patented intranasal potentiated oxytocin formulation which is believed to increase specificity for oxytocin receptors relative to vasopressin receptors as well as to enhance the potency of oxytocin. Tonix is also developing a different intranasal formulation and device, designated TNX-1900, for prophylaxis of chronic migraine and for the treatment of insulin resistance and related conditions. Oxytocin is a naturally occurring human hormone that acts as a neurotransmitter in the brain. It was originally approved by the U.S. Food and Drug Administration as Pitocin®*, an intravenous infusion or intramuscular injection drug, for use in pregnant women to induce labor. An intranasal form of oxytocin was marketed in the U.S. by Novartis to assist in the production of breast milk as Syntocinon®** (oxytocin nasal 40 units/ml), but the product was discontinued, and the New Drug Application was withdrawn.

*Pitocin® is a trademark of Par Pharmaceutical, Inc.

**Syntocinon® is a trademark of BGP Products Operations GmbH

About the Foundation for Prader-Willi Research (FPWR)

FPWR is a global nonprofit organization focused on funding research, advancing scientific understanding, and improving the lives of individuals with Prader-Willi syndrome. By supporting innovative research and forging strategic partnerships, FPWR seeks to find treatments and ultimately a cure for PWS. For more information please visit https://www.fpwr.org/. 

Tonix Pharmaceuticals Holding Corp.*

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment of a potentially confirmatory Phase 3 study in the third quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 proof-of-concept study has been completed, and topline results were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 proof-of-concept study in the third quarter of 2023, with topline results expected in early November of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the µ-opioid receptor while maintaining activity in the rat Novel Object Recognition test in vivo and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development for preventing headaches in chronic migraine, and has completed enrollment in a Phase 2 proof-of-concept study with topline data expected in early December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes 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. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.

*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. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. 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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
(919) 360-3039

Source: Tonix Pharmaceuticals Holding Corp.

Released October 9, 2023

Bristol Myers Squibb to Acquire Mirati Therapeutics for $4.8 Billion

Posted on by slightbourne@noblecapitalmarkets.com

Pharma giant Bristol Myers Squibb (BMY) announced today that it will acquire clinical-stage biotech Mirati Therapeutics (MRTX) for $58 per share in an all-cash deal totaling $4.8 billion. Mirati stockholders will also receive a contingent value right worth up to $12 per share, bringing the total potential deal value to $5.8 billion.

The acquisition will expand Bristol Myers Squibb’s oncology portfolio and pipeline. Mirati’s lead asset is KRAZATI (adagrasib), the first and only FDA-approved drug targeting the KRAS G12C mutation. KRAS mutations occur in about 13% of non-small cell lung cancers (NSCLC) and are linked to poor prognosis.

KRAZATI was granted accelerated approval in October 2022 as a second-line treatment for KRAS G12C-mutated NSCLC. It is also being tested in combination with a PD-1 inhibitor as a potential first-line NSCLC therapy. Beyond lung cancer, KRAZATI has shown promise in colorectal and pancreatic cancers.

“With multiple targeted oncology assets including KRAZATI, Mirati is another important step forward in our efforts to grow our diversified oncology portfolio,” said Bristol Myers CEO Giovanni Caforio. The company aims to leverage its global commercial infrastructure to maximize KRAZATI’s reach.

Mirati’s earlier-stage pipeline includes MRTX1719, an innovative PRMT5 inhibitor, as well as several KRAS-targeted agents. MRTX1719 could be the first targeted therapy for MTAP-deleted tumors, which represent about 10% of cancers.

“Bristol Myers Squibb’s global scale, resources and commitment to innovation will enable Mirati’s therapeutics to benefit more patients, faster,” said Mirati CEO Charles Baum.

Strategic Fit

Lung cancer is the most common cancer and leading cause of cancer death globally. The addition of KRAZATI establishes Bristol Myers as a leader in developing targeted lung cancer therapies. Mirati also expands Bristol Myers’ presence in colorectal and pancreatic cancers.

The acquisition builds on Bristol Myers’ recent deals for Turning Point Therapeutics and Eisai’s oncology business. As patents expire for the pharma giant’s top-selling cancer immunotherapy Opdivo, it aims to refill its oncology pipeline.

“With a strong strategic fit, great science and clear value creation opportunities for our shareholders, the Mirati transaction is aligned with our business development goals,” said Caforio.

Broader Biopharma Implications

The blockbuster Mirati acquisition also has significant implications for the broader biotech and biopharma sector. As large pharmas look to replenish pipelines, M&A activity has intensified. The deal shows that promising clinical-stage biotechs with innovative oncology pipelines continue to be attractive buyout targets.

Analysts note the 52% buyout premium Bristol Myers paid as a sign of their urgency to tap into Mirati’s next-gen oncology science. For startup biotechs pursuing novel approaches in high-value areas like oncology, it underscores the possibility of commanding large premium buyouts from “big pharma” acquirers.

However, smaller players also face the risk of being squeezed out as consolidation accelerates. The Mirati deal exemplifies the scaling up required to compete in cutting-edge areas like targeted cancer therapies. Smaller biotechs could find it increasingly difficult to independently develop and commercialize new drugs in the future.

That said, smaller biotechs may also benefit from big pharma’s growing appetite for M&A. The premiums being offered for innovative science and pipelines create lucrative exit opportunities for startups. And the influx of capital from buyouts can fund the next generation of biotech innovation.

Take a look at some emerging growth biotechnology companies by taking a look at Noble Capital Market’s Senior Research Analyst Robert LeBoyer’s coverage list.

Deal Terms

Under the definitive agreement, Bristol Myers will pay $58 per share for Mirati’s outstanding common stock. This represents a 52% premium over Mirati’s 30-day volume-weighted average price. Including Mirati’s $1.1 billion cash balance, the total equity value comes to $4.8 billion.

Each Mirati shareholder will also receive a CVR worth up to $12 per share. This contingent value right payment is triggered if Mirati’s MRTX1719 is approved within 7 years as a NSCLC therapy after two or fewer systemic treatments. The CVR adds up to $1 billion in potential additional value.

The transaction is expected to close in the first half of 2024, pending approval from regulators and Mirati shareholders. Bristol Myers anticipates the deal will be dilutive to its non-GAAP earnings through 2025 as it integrates Mirati. It plans to finance the acquisition through cash and debt offerings.

Caforio stated: “With a strong strategic fit, great science and clear value creation opportunities for our shareholders, the Mirati transaction is aligned with our business development goals.” The deal furthers Bristol Myers Squibb’s transformation into a leading oncology-focused biopharma.