FDA & Clinical Trials
OS Therapies Reports FDA Alignment on OST-HER2 Phase 3 Design
OS Therapies Inc. (NYSE American: OSTX) announced on September 21, 2026, that it completed a Type C Statistical Methods Meeting with the U.S. Food and Drug Administration regarding OST-HER2, its investigational immunotherapy for recurrent osteosarcoma. According to the company, the FDA aligned with the updated adaptive design of a planned confirmatory Phase 3 study. OS…
TML-6 Enters a Field Where Rival Drugs Are Already Approved
Merry Life Biomedical, a privately held Taiwan biotechnology company, announced Sept. 21 that its investigational Alzheimer’s pill TML-6 is advancing in a global Phase 2 study now spanning 20 clinical sites in Taiwan, Sweden, and the United States. The trial will enroll about 210 people with early Alzheimer’s disease, testing whether a drug built around…
Longeveron Phase 2b Heart Trial Misses Primary Endpoint
Longeveron Inc. (Nasdaq: LGVN) has reported that its Phase 2b ELPIS II trial of laromestrocel in infants with hypoplastic left heart syndrome did not meet its primary efficacy endpoint. The randomized, double-blind trial enrolled 40 infants and evaluated laromestrocel as an addition to Stage 2 palliative heart surgery. Participants received either a single dose of…
Ultragenyx Wins FDA Approval for Fayuvi in Sanfilippo Syndrome
Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) has received standard full approval from the U.S. Food and Drug Administration for Fayuvi, making it the first approved treatment for children with Sanfilippo syndrome type A. The approval covers Fayuvi, formerly known as UX111, for treating the neurological manifestations of mucopolysaccharidosis type IIIA in pediatric patients with preserved neurodevelopmental…
Skyhawk’s SKY-0515 Holds Up at 15 Months in HD Trial
Skyhawk Therapeutics, a privately held biotech based in Waltham, Massachusetts, announced final 15-month results Monday from its Phase 1/2 trial of SKY-0515, an experimental once-daily pill for Huntington’s disease. Patients who took the drug held steady or slightly improved on a standard measure of disease severity through 15 months. A comparison group of untreated patients,…
Optimi Health Plans Phase 3 MDMA Trial for PTSD
Optimi Health Corp. (Nasdaq: OPTH; CSE: OPTI) plans to begin a Health Canada-authorized Phase 3 clinical trial evaluating MDMA-assisted therapy for post-traumatic stress disorder in 2027. According to the company’s September 16 announcement, the multicenter Canadian study is expected to enroll up to 100 veterans and current or former frontline workers. Eligible groups will include…
Zipalertinib Combo Halves Lung Cancer Progression Risk
A Phase 3 trial presented Sunday at the International Association for the Study of Lung Cancer’s (IASLC) 2026 World Conference on Lung Cancer (WCLC) in Seoul found that adding the experimental oral drug zipalertinib to standard chemotherapy roughly doubled how long previously untreated patients with a specific, biomarker-defined form of advanced non-small cell lung cancer…
Tam-Peli Cuts Death Risk 54% in Relapsed Lung Cancer
Results from the Phase III TAISHAN-302 trial, presented at the International Association for the Study of Lung Cancer’s 2026 World Conference on Lung Cancer, showed that tambotatug pelitecan (Tam-Peli, YL201), an antibody-drug conjugate (ADC) targeting a protein called B7-H3, significantly improved survival compared with topotecan, the long-standing second-line chemotherapy, in patients with relapsed small-cell lung…
Medicus Pharma Selects 200 µg SkinJect Dose After Phase 2 Review
Medicus Pharma Ltd. (Nasdaq: MDCX) has selected the 200 µg dose of SkinJect as its proposed pivotal dose after completing a Phase 2 study in patients with nodular basal cell carcinoma. According to the company’s September 10 announcement, an abridged clinical study report for SKNJCT-003 has been posted on ClinicalTrials.gov. The randomized, double-blind study enrolled…
Gan & Lee Doses First Patient in Oral GLP-1 Pill Trial
Gan & Lee Pharmaceuticals Co., Ltd. (SSE: 603087.SH) announced on September 9 that the first participant has been dosed in a first-in-human Phase 1 study of GZC8072, an experimental once-weekly oral peptide GLP-1 receptor agonist, marking the compound’s formal entry into human testing. This is an early, safety-focused milestone, not an efficacy result, and no…
