After Study 1’s independent data monitoring committee said the experiment will likely fall short of its primary endpoint with ulixacaltamide in essential tremor, Praxis Precision Medicines Inc. plans to wait for full data from Study 1 as well as Study 2 in the Essential 3 phase III program before deciding on regulatory moves.
Astrazeneca plc’s good news with its oral selective estrogen receptor degrader (SERD) and estrogen receptor antagonist, camizestrant, when used as part of a combo in breast cancer raised optimism for the approach, which has caught on in various biopharma quarters.
As Wall Street looks ahead to phase III data due soon with Mineralys Therapeutics Inc.’s lorundrostat for uncontrolled or resistant hypertension, analysts are also weighing the odds of the firm in phase II with the same compound for obstructive sleep apnea.
Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.
Once high-flying Bluebird Bio Inc. has found a way out of its financial squeeze, as funds managed by global investment firms Carlyle and SK Capital Partners LP, along with a team of biotech executives, will be taking over the company.
Research seems to be gaining ground in Wilson disease, a rare inherited condition that causes copper levels to accumulate in the liver, brain and eyes. Most people are diagnosed as children or around middle age, but the age spectrum can be wide. Monopar Therapeutics Inc. has drawn Wall Street’s attention in the space.
Solid Biosciences Inc. is preparing for a sit-down with the U.S. FDA this year to discuss the firm’s results with the next-generation gene therapy SGT-003 for Duchenne muscular dystrophy (DMD).
Corbus Pharmaceuticals Inc. has placed its chips on “bets where somebody else is ahead of us,” CEO Yuval Cohen said. “The idea is to have a de-risked asset” that proves better in the clinic. At the 2025 American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium in San Francisco, Corbus offered data from the U.S. and U.K. first-in-human dose-escalation study – the Western trial – with Nectin-4-targeting antibody-drug conjugate (ADC) CRB-701 (SYS-6002).
Xilio Therapeutics Inc. is banking $52 million up front from Abbvie Inc., including a $10 million equity investment, plus about $2.1 billion in contingent payments for option-related fees and milestones, plus tiered royalties, as the pair embarks on developing tumor-activated, antibody-based immunotherapies, including masked T-cell engagers.
The closely watched chronic spontaneous urticaria (CSU, or hives) space chalked more early stage KIT inhibitor data as Third Harmonic Bio Inc. rolled out results from the phase I single and multiple ascending-dose trial with THB-335 in healthy volunteers, along with plans to move the oral candidate into phase II development by the middle of 2025.
