Month: December 2021

BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases

12/27/2021

Excerpt from the Press Release: SAN RAFAEL, Calif. and SHANGHAI, Dec. 16, 2021 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, today announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated…

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Glycomine Announces First Dosing in Phase 1 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG

12/24/2021

Excerpt from the Press Release: SAN CARLOS, Calif.–(BUSINESS WIRE)–Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that the company has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for GLM101 for the treatment of PMM2-CDG and has initiated dosing healthy volunteers…

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Vincerx Pharma Announces First Patient Dosed in Phase 1 Dose-Escalation Study of VIP152 in Relapsed or Refractory Chronic Lymphocytic Leukemia or Richter Syndrome

12/24/2021

Excerpt from the Press Release: PALO ALTO, Calif., Dec. 17, 2021 (GLOBE NEWSWIRE) — Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today announced that the first patient has been dosed in the Company’s Phase 1 dose-escalation study of VIP152 in…

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Apnimed Announces Dosing of First Patient in Phase 2 MARIPOSA Confirmatory Trial of AD109, a Potential Nighttime Oral Treatment for Obstructive Sleep Apnea (OSA)

12/24/2021

– Study to Inform Phase 3 Program Design of AD109 and Advance Program for AD504 – Topline Results Expected Mid-2022 Excerpt from the Press Release: CAMBRIDGE, Mass.–(BUSINESS WIRE)–Apnimed, a clinical-stage pharmaceutical company focused on developing oral pharmacologic treatments to address obstructive sleep apnea (OSA) and related disorders, today announced the dosing of the first patient in the…

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Passage Bio Announces Positive Interim Safety and Biomarker Data and Advances Phase 1/2 Trial of PBGM01 in GM1 Gangliosidosis

12/23/2021

– Independent Data Monitoring Committee recommends proceeding with two additional planned patient cohorts, which are now being recruited in parallel – Positive safety profile, including no serious adverse events, no complications related to intra-cisterna magna injection and no evidence of dorsal root ganglion toxicity – Intra-cisterna magna delivery resulted in substantial increases in beta-galactosidase enzyme…

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EVUSHELD long-acting antibody combination retains neutralizing activity against Omicron variant in independent FDA study

12/23/2021

Only antibody authorized in the US for pre-exposure prophylaxis of COVID-19 Excerpt from the Press Release: WILMINGTON, Del.–(BUSINESS WIRE)–AstraZeneca’s EVUSHELD (tixagevimab co-packaged with cilgavimab), a long-acting antibody combination for the prevention of COVID-19, retained neutralizing activity against the Omicron SARS-CoV-2 variant (B.1.1.529), according to new preclinical data. In this study,EVUSHELD’s Inhibitory Concentration 50 (IC50), a measure…

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Notable Presents Clinical Validation Data from Two Phase 2 Trials in Adult and Pediatric Acute Myelogenous Leukemia at the 63rd American Society of Hematology (ASH) Annual Meeting

12/23/2021

-Studies demonstrate high correlation between predicted response by Notable’s predictive precision medicines platform and actual clinical response, short-term and at one year- -Interim analyses of studies with MD Anderson Cancer Center and Texas Children’s Hospital investigators corroborate platform prediction data from Standford University collaboration- Excerpt from the Press Release: FOSTER CITY, Calif.–(BUSINESS WIRE)–Notable Labs, Inc.…

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Vaxart to Test Cross-Reactivity of its COVID-19 Oral Tablet Vaccine Against Omicron

12/22/2021

Cross-reactivity will be assessed using samples from Phase II clinical participants and in a parallel animal challenge study Oral tablet’s immunogenicity profile suggests it may provide cross-protection against SARS-CoV-2 variants, including Omicron Excerpt from the Press Release: SOUTH SAN FRANCISCO, Calif., Dec. 16, 2021 (GLOBE NEWSWIRE) — Vaxart, Inc. (NASDAQ: VXRT) said today it plans to…

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Kura Oncology Doses First Patient in Phase 1/2 Clinical Trial of Tipifarnib in Combination with Tipifarnib in Head and Neck Squamous Cell Carcinoma

12/22/2021

– Preclinical data suggest HRAS and PI3Kα are co-dependent oncogenes in HNSCC – – Combination has potential to address up to 50% of patients with HNSCC – – Initial cohort comprised of patients with PIK3CA-dependent HNSCC – SAN DIEGO, Dec. 16, 2021 (GLOBE NEWSWIRE) —  Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed…

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Artax Biopharma Announces First-in-Human Dosing in Phase 1 Clinical Trial for AX-158, Company’s First-in-Class Oral Immunomodulator to Treat T Cell-Mediated Diseases

12/22/2021

The Phase 1 Trial, Expected to Conclude in 2022, Will Evaluate Safety, Exposure, and Pharmacokinetics of AX-158 in Healthy Volunteers AX-158, a Novel, Oral Small Molecule Immunomodulating Agent for the Treatment of T Cell- Mediated Diseases, Selectively Modulates T Cell Responses without Suppression of Immune System Function Excerpt from the Press Release: CAMBRIDGE, Mass., Dec. 15,…

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