Breakthrough therapy

Tonix Pharmaceuticals Announces Pricing of $4.5 Million Public Offering

Retrieved on: 
Friday, September 29, 2023

The closing of the public offering is expected to take place on or about October 3, 2023, subject to the satisfaction of customary closing conditions.

Key Points: 
  • The closing of the public offering is expected to take place on or about October 3, 2023, subject to the satisfaction of customary closing conditions.
  • The gross proceeds of the offering will be approximately $4.5 million before deducting placement agent fees and other estimated offering expenses payable by the Company.
  • Brookline Capital Markets, a division of Arcadia Securities, LLC is acting as co-placement agent for the offering.
  • The offering will be made only by means of a prospectus supplement and accompanying base prospectus, as may be further supplemented by any free writing prospectus and/or pricing supplement that Tonix may file with the SEC.

Tonix Pharmaceuticals Announces Proposed Public Offering

Retrieved on: 
Thursday, September 28, 2023

CHATHAM, N.J., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (“Tonix” or the “Company”), a biopharmaceutical company, today announced that it intends to offer and sell shares of its common stock (or common stock equivalents in lieu thereof) and warrants to purchase shares of common stock in a public offering.

Key Points: 
  • CHATHAM, N.J., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (“Tonix” or the “Company”), a biopharmaceutical company, today announced that it intends to offer and sell shares of its common stock (or common stock equivalents in lieu thereof) and warrants to purchase shares of common stock in a public offering.
  • All of the securities to be sold in the offering are to be offered by Tonix.
  • The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.
  • Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995 including those relating to the completion of the public offering, the timing and size of the public offering, the intended use of proceeds from the public offering and other statement that are predictive in nature.

Verastem Presents Avutometinib and Defactinib Combination Program Updates at the 5th Annual RAS-Targeted Drug Development Summit

Retrieved on: 
Thursday, September 28, 2023

Verastem Oncology, (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced the presentation of scientific background and clinical trial updates on the avutometinib and defactinib programs at the 5th Annual RAS-Targeted Drug Development Summit in Boston, Massachusetts.

Key Points: 
  • Verastem Oncology, (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced the presentation of scientific background and clinical trial updates on the avutometinib and defactinib programs at the 5th Annual RAS-Targeted Drug Development Summit in Boston, Massachusetts.
  • The updates are part of two oral presentations by Jonathan Pachter, PhD, Chief Scientific Officer and Louis Denis, MD, Chief Medical Officer at Verastem Oncology.
  • The first presentation titled “Vertical Inhibition of RAS, RAF & MEK: Enhancing Antitumor Efficacy of KRAS G12C & G12D Inhibitors with RAF/MEK Clamp Avutometinib”, includes scientific rationale for clinical combinations with avutometinib and defactinib in various RAS pathway-driven cancers.
  • The second presentation titled, “Introducing Rational Combinations of RAF/MEK Clamp Avutometinib: Breakthrough Therapy Designation & Beyond,” discusses novel combination treatment approaches and provides an overview of the avutometinib and defactinib clinical development program.

Pancreatic Cancer Research Gains Momentum Amidst Soaring Young-Onset Cases

Retrieved on: 
Tuesday, September 26, 2023

VANCOUVER, British Columbia, Sept. 26, 2023 /PRNewswire/ -- USA News Group - The seriousness of pancreatic cancer continues to grow, with increasing alarm since The Lancet published an analysis back in April 2023, showing a rising incidence of pancreatic cancer among individuals younger than 55 years. Another study published in JAMA Network Open showed that early-onset cancer in people younger than 50 rose over the last decade, with gastrointestinal cancers seeing the fastest increase. This has led to increased calls from pancreatic cancer organizations such as Pancreatic Cancer Action Network (PanCAN) for more action in the space, including advocating that the US Congress invest more in cancer research funding now. Among the ongoing efforts that are drawing more attention in the pancreatic cancer space have been the developments coming from Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Merus N.V. (NASDAQ: MRUS), Guardant Health, Inc. (NASDAQ: GH), Medtronic plc (NYSE: MDT), and even NVIDIA Corporation (NASDAQ: NVDA).

Key Points: 
  • This has led to increased calls from pancreatic cancer organizations such as Pancreatic Cancer Action Network (PanCAN) for more action in the space, including advocating that the US Congress invest more in cancer research funding now.
  • "By teaming up with PanCAN, we believe we may be able to expedite development and provide pancreatic cancer patients with a bespoke immunotherapeutic treatment option."
  • Pelareorep delivered the company a big win near the end of 2022, when the FDA rewarded it with a Fast Track Designation (FTD) for treating advanced or metastatic pancreatic cancer.
  • "With a five-year survival rate of 12%, pancreatic cancer patients cannot afford to wait for new treatment options," said Julie Fleshman, JD, MBA, President and CEO of PanCAN.

Pancreatic Cancer Research Gains Momentum Amidst Soaring Young-Onset Cases

Retrieved on: 
Tuesday, September 26, 2023

VANCOUVER, British Columbia, Sept. 26, 2023 /PRNewswire/ -- USA News Group - The seriousness of pancreatic cancer continues to grow, with increasing alarm since The Lancet published an analysis back in April 2023, showing a rising incidence of pancreatic cancer among individuals younger than 55 years. Another study published in JAMA Network Open showed that early-onset cancer in people younger than 50 rose over the last decade, with gastrointestinal cancers seeing the fastest increase. This has led to increased calls from pancreatic cancer organizations such as Pancreatic Cancer Action Network (PanCAN) for more action in the space, including advocating that the US Congress invest more in cancer research funding now. Among the ongoing efforts that are drawing more attention in the pancreatic cancer space have been the developments coming from Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Merus N.V. (NASDAQ: MRUS), Guardant Health, Inc. (NASDAQ: GH), Medtronic plc (NYSE: MDT), and even NVIDIA Corporation (NASDAQ: NVDA).

Key Points: 
  • This has led to increased calls from pancreatic cancer organizations such as Pancreatic Cancer Action Network (PanCAN) for more action in the space, including advocating that the US Congress invest more in cancer research funding now.
  • "By teaming up with PanCAN, we believe we may be able to expedite development and provide pancreatic cancer patients with a bespoke immunotherapeutic treatment option."
  • Pelareorep delivered the company a big win near the end of 2022, when the FDA rewarded it with a Fast Track Designation (FTD) for treating advanced or metastatic pancreatic cancer.
  • "With a five-year survival rate of 12%, pancreatic cancer patients cannot afford to wait for new treatment options," said Julie Fleshman, JD, MBA, President and CEO of PanCAN.

Press Release: Once-weekly ALTUVIIIO® approved in Japan as a new class of factor VIII therapy for hemophilia A

Retrieved on: 
Monday, September 25, 2023

The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO®[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy.

Key Points: 
  • The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO®[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy.
  • ALTUVIIIO is indicated for control of bleeding tendency in patients with hemophilia A (factor VIII deficiency).
  • ALTUVIIIO was also recently approved by the Taiwan Food and Drug Administration for treatment of adults and children with hemophilia A on August 31, 2023.
  • The high-sustained factor activity levels will enable patients and physicians to reimagine living with hemophilia.

89bio Announces U.S. FDA has Granted Breakthrough Therapy Designation for Pegozafermin in Nonalcoholic Steatohepatitis (NASH)

Retrieved on: 
Thursday, September 21, 2023

SAN FRANCISCO, Sept. 21, 2023 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to pegozafermin in patients with nonalcoholic steatohepatitis (NASH).

Key Points: 
  • SAN FRANCISCO, Sept. 21, 2023 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to pegozafermin in patients with nonalcoholic steatohepatitis (NASH).
  • “We believe pegozafermin is well positioned as a leading FGF21 analog treatment option with demonstrated strong histology data, best-in-class tolerability, and dosing convenience to date.
  • It has the potential to meet the treatment needs for F2/3 patients as well as compensated cirrhotic (F4) patients.
  • BTD is an FDA program designed to expedite the development and review of product candidates intended for serious or life-threatening conditions.

Zai Lab Obtains Breakthrough Therapy Designation for Efgartigimod Alfa Injection (Subcutaneous Injection) in Patients with Chronic Inflammatory Demyelinating Polyneuropathy in China

Retrieved on: 
Monday, September 18, 2023

SHANGHAI, China and CAMBRIDGE, Mass., Sept. 18, 2023 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for efgartigimod alfa injection (subcutaneous injection) (efgartigimod SC) for the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP).

Key Points: 
  • SHANGHAI, China and CAMBRIDGE, Mass., Sept. 18, 2023 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for efgartigimod alfa injection (subcutaneous injection) (efgartigimod SC) for the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP).
  • The Breakthrough Therapy Designation for efgartigimod SC was supported by data from both global and Chinese patients enrolled in the ADHERE study.
  • “We have seen how efgartigimod SC can meaningfully improve and stabilize disease symptoms in these patients.
  • Existing treatment options are quite limited, and problematic given the general reliance on long-term steroid or chronic immunoglobulin therapy.

Eloxx Pharmaceuticals Reports Independent Confirmation of Positive Biopsy Results in All Patients Treated with ELX-02 in Phase 2 Clinical Study for Alport Syndrome

Retrieved on: 
Monday, September 18, 2023

WATERTOWN, Mass., Sept. 18, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported additional independent confirmation on the positive TEM assessment results from its proof-of-concept Phase 2 open-label clinical trial (NCT05448755) of ELX-02 for the treatment of Alport syndrome after eight weeks of treatment. Visual assessment of TEM images from kidney biopsies showed an improvement in foot process effacement in all three treated patients consistent with disease regression. Alport syndrome is a rare genetic kidney disorder caused by mutations in COL4A3/4/5 genes, characterized by podocyte injury and impaired kidney filter function leading to proteinuria.

Key Points: 
  • Visual assessment of TEM images from kidney biopsies showed an improvement in foot process effacement in all three treated patients consistent with disease regression.
  • Alport syndrome is a rare genetic kidney disorder caused by mutations in COL4A3/4/5 genes, characterized by podocyte injury and impaired kidney filter function leading to proteinuria.
  • Eloxx recently submitted an Investigational New Drug application (IND) to the FDA for ELX-02 for the treatment of Alport syndrome with nonsense mutations.
  • The IND, if allowed, would enable the inclusion of U.S.-based sites in the planned pivotal trial.

Cybin Completes Enrollment in Phase 2 Study of CYB003 in Major Depressive Disorder

Retrieved on: 
Thursday, September 21, 2023

Cybin Inc. (NYSE American:CYBN) (NEO:CYBN) (“Cybin” or the “Company”), a clinical-stage biopharmaceutical company committed to revolutionizing mental healthcare by developing new and innovative psychedelic-based treatment options, is pleased to announce that it has completed enrollment in its Phase 2 study of CYB003, a proprietary deuterated psilocybin analog program being developed for the potential treatment of major depressive disorder (“MDD”).

Key Points: 
  • Cybin Inc. (NYSE American:CYBN) (NEO:CYBN) (“Cybin” or the “Company”), a clinical-stage biopharmaceutical company committed to revolutionizing mental healthcare by developing new and innovative psychedelic-based treatment options, is pleased to announce that it has completed enrollment in its Phase 2 study of CYB003, a proprietary deuterated psilocybin analog program being developed for the potential treatment of major depressive disorder (“MDD”).
  • To date, CYB003 has demonstrated a favorable safety and tolerability profile at all doses evaluated in the five completed cohorts (1mg, 3mg, 8mg, 10mg, and 12mg).
  • The CYB003 program is supported by a recently granted U.S. patent covering composition of matter claims until 2041.
  • “The completion of enrollment in our Phase 2 CYB003 study is a significant milestone for Cybin that brings us closer than ever to understanding the potential antidepressant effects and clinical advantages of CYB003.