Another phase III failure of Theravance Biopharma Inc.’s norepinephrine reuptake inhibitor ampreloxetine to treat symptomatic neurogenic orthostatic hypotension, this time in patients with the rare disease multiple system atrophy, means the end of the company’s R&D efforts. Cutting its workforce in half and terminating all development of ampreloxetine, its only pipeline product, the Dublin-based company’s shares (NASDAQ:TBPH) sank by 26%, or $4.99, to close March 3 at $13.96.

BioWorld tracked 144 clinical trial readouts across phases I through III in January 2026, down from 215 in December. January included 15 phase III trials reporting positive results and three that failed to meet key endpoints. By phase, the month’s updates consisted of 60 from phase I, 44 from phase II and 40 from phase III.

The busy Prader-Willi syndrome (PWS) space took another hit as did shares of Aardvark Therapeutics Inc. (NASDAQ:AARD), which closed March 2 at $5.47, down $7.02, or 56%, after the company disclosed a voluntary pause of the phase III Hunger Elimination or Reduction Objective (HERO) trial testing ARD-101 as a treatment for hyperphagia, or intense hunger, in patients with the disease.

Uniqure NV is the latest firm to get caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies. The company disclosed in its latest earnings report that U.S. regulators are calling for a sham-controlled study before they will consider approval of gene therapy AMT-130 in Huntington’s disease, a requirement that could set the program back by two to three years and raises potential ethical issues.

In a boon for licensee Bristol Myers Squibb Co., Systimmune Inc.’s next-generation cancer drug, izalontamab brengitecan (iza-bren), hit both progression-free survival and overall survival endpoints in a phase III trial of Chinese patients with advanced triple-negative breast cancer.

The psychedelic and psychedelic-like drug space made another stride as Ataibeckley Inc. disclosed positive top-line data from a double‑blind, placebo‑controlled, first-in-patient phase IIa study testing oral R-MDMA therapy EMP‑01 in adults with social anxiety disorder (SAD).

Almost three months to the day after Protara Therapeutics Inc. made known positive interim data from the phase II trial testing cell-based therapy TARA-002 in pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs), Palvella Therapeutics Inc. scored positive top-line results from the phase III Selva study evaluating Qtorin 3.9% rapamycin anhydrous gel for microcystic LMs.

Ascletis Pharma Inc. raised HK$843.53 million (US107.93 million) in a placement on the Hong Kong Stock Exchange to advance its lead program, ASC-30, a small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist that can be dosed once monthly subcutaneously and once daily orally for treating obesity.

A world-first pediatric mRNA cancer vaccine trial is launching in Australia that could open new possibilities for children with aggressive brain tumors.

An “outsized placebo response” is not stopping Gossamer Bio Inc. from seeking a path to potential approval of inhaled tyrosine kinase inhibitor seralutinib in pulmonary arterial hypertension (PAH), but the missed primary endpoint in the phase III Prosera study sent shares of the company (NASDAQ:GOSS) tumbling 80% and left investors skeptical going forward.