Attention has turned to the regulatory path worldwide after Belite Bio Inc. unveiled positive top-line data from the phase III Dragon study with oral, daily tinlarebant in Stargardt disease type 1, the most common form of the rare affliction.
The U.S. Centers for Medicare & Medicaid Services (CMS) rolled out negotiated costs of the second batch of drugs subject to such bargaining under the Inflation Reduction Act. Wall Street was not surprised to learn that the numbers amount to much greater cuts than the Biden administration managed for 2026. CMS said the adjusted maximum fair prices would have achieved 44% lower net spending had they been implemented in 2024 – 36% if forgiven discounts from the part D redesign of the Medicare prescription drug benefit are figured in. Fifteen drugs are listed.
Success in Japan may further bolster Protara Therapeutics Inc. in its push with TARA-002 in pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs), for which the New York-based firm disclosed interim results from an ongoing phase II trial.
Nervgen Pharma Corp. rolled out expanded findings from the Connect phase Ib/IIa study with NVG-291 in spinal cord injury patients showing that the 35-amino acid peptide derived from the intracellular wedge domain of phosphatase sigma provided durable functional gains that continued at week 16 and after.
Success in Japan may further bolster Protara Therapeutics Inc. in its push with TARA-002 in pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs), for which the New York-based firm disclosed interim results from an ongoing phase II trial.
Roche AG’s Genentech unit landed another clinical victory with giredestrant that provided a major stock boost for competitor Olema Pharmaceuticals Inc. Made public were positive phase III data from the Lidera study testing selective estrogen receptor degrader and complete estrogen receptor antagonist (CERAN) as an adjuvant endocrine treatment for people with ER-positive, HER2-negative, early stage breast cancer (BC).
About a month after rolling out positive phase I/II data with its candidate for metastatic, castrate-resistant prostate cancer (mCRPC), Halda Therapeutics Inc. disclosed the plan by Johnson & Johnson (J&J), which is paying to take over the company for $3.05 billion in cash.
The pricier offer by Lundbeck A/S for Avadel Pharmaceuticals plc – which follows last month’s agreement for a takeover by Alkermes plc – had Wall Street buzzing as pundits weighed the odds for each suitor.
Korro Bio Inc.’s latest update on RNA editing prospect KRRO-110 may mean one less competitor in alpha-1 antitrypsin deficiency (AATD), and shares of the firm (NASDAQ:KRRO) closed Nov. 13 at $6.50, down $24.92, or 79%. As part of the third-quarter earnings report, Korro said KRRO-110 produced functional protein in AATD patients but fell short of projected levels of functional protein after a single administration.
At the busy annual congress of the American College of Surgeons in Chicago, attendees heard about a study that found rectal bleeding in younger adults – those under 50 years of age – increased their odds of a colorectal cancer (CRC) diagnosis by a shocking 850%. Already, the demographic has turned up sharply higher cases of the disease in recent years. The ACS meeting was held Oct. 4-7.
