Investigational New Drug

Tonix Pharmaceuticals Announces Positive Results from Clinical Pharmacokinetic Bridging Study of Tonmya™ to Support Development and Partnering in Japan and China

Retrieved on: 
Tuesday, February 27, 2024
Fibromyalgia, Tonix Pharmaceuticals, Investigational New Drug, New Drug Application, Marketing, Female, Hydrogen chloride, Data, PMDA, Patient, Male, Tablet, IND, NDA, NMPA, CTN, NCE, Pharmaceutical industry

CHATHAM, N.J., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, announced positive results from its clinical pharmacokinetic (PK) bridging study of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in healthy adult male and female ethnic Japanese and Chinese volunteers. Results indicate that key pharmacokinetic parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study. Tonmya was generally well tolerated in the ethnic Japanese and Chinese healthy volunteers. The company expects these data to fulfill the requirement for a bridging study, and to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE). Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.

Key Points: 
  • Results indicate that key pharmacokinetic parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study.
  • The company expects these data to fulfill the requirement for a bridging study, and to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE).
  • Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.
  • “This bridging study is an important first step as we begin evaluating the potential for approval and marketing Tonmya in Japan and China.

Derm-Biome Pharmaceuticals prepares to begin IND-enabling studies for DB-007-4, a potential best-in-class treatment for inflammatory skin diseases

Retrieved on: 
Tuesday, February 27, 2024
Investigational New Drug, Toxicology, Acne, Toxicity, Atopic dermatitis, Drug development, Skin, Risk, GLP, Safety, Human, Rosacea, IND, Pharmaceutical industry

Toxicology studies are an essential part of drug development as they help to evaluate the potential safety and toxicity of a drug candidate before it is tested on humans.

Key Points: 
  • Toxicology studies are an essential part of drug development as they help to evaluate the potential safety and toxicity of a drug candidate before it is tested on humans.
  • There is a real need for skin disease drugs that offer high efficacy and a low risk of adverse effects with long-term use.
  • For conditions like AD, an inflammatory skin disease that affects up to 25% of babies and young children, there is a need for safer treatment options.
  • DB-007-4 is a topical multi-disease treatment that targets important pathways that play a vital role in the development of inflammatory skin diseases while being well-tolerated by skin.

Immix Biopharma 12 Month Review Progress Update

Retrieved on: 
Wednesday, February 21, 2024
Boston University, Research, Autoimmune disease, Toxicity, Doctor of Philosophy, Boston Medical Center, PMID, Health, Multiple myeloma, Patient, Poster, Blood, Investigational New Drug, Presentation, Bank, Johnson & Johnson, GMP, BCMA, Amyloidosis, BioMarin Pharmaceutical, Autoimmunity, MBBS, Pfizer, ASH, European, IMS, Conference, ODD, Haematologica, IND, Public, Gene, MD, FDA, Food, U.S. FDA, Cell, ALA, Letter, Society, Publication, BMC, Pharmaceutical industry

LOS ANGELES, Feb. 21, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, today announced its 12-month progress update including shareholder letter.

Key Points: 
  • LOS ANGELES, Feb. 21, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, today announced its 12-month progress update including shareholder letter.
  • “The past 12 months have seen landmark achievements for Immix Biopharma, cementing our position as a leading cell therapy company in autoimmune disease.
  • NXC-201 is the first and only ‘Single-Day CRS‘ CAR-T, a critical advancement for autoimmune diseases like AL Amyloidosis,” said Ilya Rachman, MD PhD CEO Immix Biopharma.
  • As we reflect on the last 12 months of progress, we wish to connect with you offering an update on Immix Biopharma.

CERo Therapeutics, Inc. Announces Publication of Preclinical Research Supporting the Use of Its Clinical Candidate CER-1236 to Treat AML Patients

Retrieved on: 
Thursday, March 7, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Clinical Trials, Therapy, NSCLC, Investigational New Drug, Bone marrow, AML, RUNX1, Cancer, B-cell lymphoma, ASXL1, Clinical Cancer Research, Classification, Patient, CERO, IND, Cero, Vaccine

The results in the paper found that the target for CER-1236 is found in the large majority (83%) of leukemic cells extracted from the bone marrow from patients, and that more importantly CER-1236 effectively eliminated leukemic cells in the company’s experiments.

Key Points: 
  • The results in the paper found that the target for CER-1236 is found in the large majority (83%) of leukemic cells extracted from the bone marrow from patients, and that more importantly CER-1236 effectively eliminated leukemic cells in the company’s experiments.
  • Finally, the target for CER-1236 was found by CERo to be highly expressed and detectable across common AML genetic classification subtypes, including patient samples with adverse risk mutations in TP53, ASXL1 and RUNX1.
  • “We’re very pleased with this publication in Clinical Cancer Research supporting our near term plans to advance CER-1236 into the clinic.
  • As we have previously reported, CERo plans to file an Investigational New Drug (IND) application in the first half of 2024, and is targeting initial treatment of AML patients as well as B Cell lymphoma patients before the end of the year,” said Brian G Atwood, CERo’s Chairman and Chief Executive Officer.

Coya Therapeutics Presents Biomarker Data on Neuroinflammatory Pathways in Frontotemporal Dementia (FTD) at the AD/PD 2024 Conference

Retrieved on: 
Wednesday, March 6, 2024
Research, Mental Health, Neurology, Clinical Trials, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Central nervous system, Investigational New Drug, Houston Methodist Hospital, Chemokine, Frontotemporal dementia, Messenger, FDA, COYA, Conference, FTD, Therapy, Pathology, Patient, Poster, Role, IND, Pharmaceutical industry

The poster presentation was shared for the first time today at the AD/PD 2024 Conference in Lisbon, Portugal and can be found here .

Key Points: 
  • The poster presentation was shared for the first time today at the AD/PD 2024 Conference in Lisbon, Portugal and can be found here .
  • We intend to file an Investigational New Drug (IND) application with the FDA for COYA 302 in FTD later this year and initiate a Ph.
  • 2 trial in FTD patients shortly thereafter,” stated Fred Grossman, Chief Medical Officer at Coya Therapeutics.
  • In addition, several inflammation transcripts in monocyte genes known to be involved in neuroinflammatory signaling pathways were dysregulated in FTD, compared to controls.

MAIA Biotechnology CEO Details Immuno-Oncology Cancer Treatment Candidates and Development Pipeline in Letter to Shareholders

Retrieved on: 
Tuesday, March 5, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, Clinical Trials, NSCLC, Survival, Lung cancer, Letter, Investigational New Drug, IND, Cancer, Pharmaceutical industry

MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA” or the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today published a 2024 Letter to Shareholders by Chairman and Chief Executive Officer Vlad Vitoc, M.D., detailing the Company’s immuno-oncology cancer treatment candidates and development pipeline.

Key Points: 
  • MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA” or the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today published a 2024 Letter to Shareholders by Chairman and Chief Executive Officer Vlad Vitoc, M.D., detailing the Company’s immuno-oncology cancer treatment candidates and development pipeline.
  • “At MAIA Biotechnology, our tenacious pursuit of innovative medicines to improve and extend people’s lives has led us to the forefront of cancer research.
  • Along with NSCLC, MAIA’s pipeline of immuno-oncology therapies includes multiple hard-to-treat cancers.
  • Company’s pipeline includes THIO-102 Phase 2 and THIO-103 Phase 2/3 clinical trials (planning stage), and Investigational New Drug (IND)-enabling studies for second-generation telomere targeting agents.

Astria Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides a Corporate Update

Retrieved on: 
Monday, March 4, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Allergy, Investigational New Drug, PD, Research, YTE, Hereditary angioedema, Investment, Atopic dermatitis, Richard Driehaus, Safety, OX40, HAE, Therapy, Pharmacokinetics, Patient, Pharmaceutical industry

Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update.

Key Points: 
  • Astria Therapeutics, Inc. (NASDAQ:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and provided a corporate update.
  • The trial is evaluating safety and tolerability, changes in HAE attack rate, pharmacokinetics (PK), pharmacodynamics (PD), and quality-of-life assessments.
  • Pending proof-of-concept results from the ALPHA-STAR trial, Astria expects to progress directly to a pivotal Phase 3 program which is anticipated to initiate in the first quarter of 2025.
  • Cash Position: As of December 31, 2023, Astria had cash, cash equivalents and short-term investments of $246.5 million, compared to $188.8 million as of September 30, 2023.

Children’s Hospital Los Angeles Awarded $6 Million from CIRM to Advance CAR T-Cell Therapies in Recurring Solid Tumors in Children

Retrieved on: 
Friday, March 1, 2024
Biotechnology, Health Technology, Health, Oncology, Neoplasm, Division, Senior, Investigational New Drug, CHLA, Regenerative medicine, Doctor of Philosophy, Multimedia, CIRM, Transplant, FDA, CBDI, TGFB, Sarcoma, Cell, Radiation, Interim, Survival rate, Bone marrow, Hematology, Virus, Tumor microenvironment, USC, Patient, Clinical trial, Survival, Research, Medicine, Vaccine

Children’s Hospital Los Angeles has received a multi-year $6 million award from the California Institute for Regenerative Medicine (CIRM) to develop innovative stem cell approaches to treat children and adolescents with recurrent solid tumors.

Key Points: 
  • Children’s Hospital Los Angeles has received a multi-year $6 million award from the California Institute for Regenerative Medicine (CIRM) to develop innovative stem cell approaches to treat children and adolescents with recurrent solid tumors.
  • (Photo: Business Wire)
    When cancer reoccurs in children and young adults with solid tumors such as sarcomas and neuroblastomas, there are few good treatment options.
  • This is why Children’s Hospital Los Angeles researchers focus on finding novel therapies using the patient’s own immune cells to treat childhood cancers.
  • In 2022, CIRM awarded $8 million to the Keck School of Medicine of USC and Children’s Hospital Los Angeles to establish a CIRM Alpha Clinic.

Atara Biotherapeutics Receives FDA Clearance of IND Application in Lupus Nephritis for ATA3219, an Allogeneic CAR T Therapy

Retrieved on: 
Thursday, February 29, 2024
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Investigational New Drug, Woman, Autoimmune disease, Physician, FDA, Food, CD19, Epstein–Barr virus, Risk, Leukapheresis, Prevalence, Safety, Lupus, Kidney, ATRA, EBV, Disease, Patient, Lupus nephritis, SLE, IND, Tissue, Mortality, Cancer

We are eager to address the significant unmet need in lupus nephritis as we initiate our Phase 1 trial,” said Pascal Touchon, President and Chief Executive Officer of Atara.

Key Points: 
  • We are eager to address the significant unmet need in lupus nephritis as we initiate our Phase 1 trial,” said Pascal Touchon, President and Chief Executive Officer of Atara.
  • Subjects will receive lymphodepletion treatment followed by ATA3219 at a dose of 40, 80, or 160 x 106 CAR+ T cells.
  • “CAR T cells can naturally infiltrate deep into target tissues to mediate B-cell depletion and produce durable responses.
  • The ATA3219 IND submission included in vitro data reflecting the CD19 antigen-specific functional activity of ATA3219 and CAR-mediated activity against B cells from SLE patients.

Neurenati Therapeutics Concludes a 1.2 Million $ Seed Financing Round to Develop NEU-001 as a Promising Therapy for Hirschsprung Disease

Retrieved on: 
Thursday, February 29, 2024
Infectious Diseases, Biotechnology, Pharmaceutical, Health, Investigational New Drug, Enterocolitis, Inflammatory Bowel Diseases, Drug development, UQAM, Pediatrics, Life, Ipsen, Therapy, Patient, Intellectual property, IND, Gastroenterology, Partnership, Pharmaceutical industry

Neurenati Therapeutics is delighted to announce the successful closing of its seed funding round, securing 1.2 million $ from Genson Capital, on the 2024 Rare Disease Day.

Key Points: 
  • Neurenati Therapeutics is delighted to announce the successful closing of its seed funding round, securing 1.2 million $ from Genson Capital, on the 2024 Rare Disease Day.
  • Neurenati, a Québec-based biotech company, focuses on the development of various rare diseases therapies, including pediatric conditions.
  • Maxime Ranger, President and CEO of Neurenati Therapeutics commented, “Currently, surgery remains the only option to newborns with HSCR.
  • As fourth member of the Board, he joins Rodolphe Soret (cofounder), Benoit Doré (Axelys), and Maxime Ranger (Neurenati).