Rapport Therapeutics

Christine Marie Utter, PTC Therapeutics' SVP and Chief Accounting Officer, sold shares totaling $132,492 on Jan 7-8, 2024, to cover tax obligations from vested RSUs, retaining over 64,000 shares. PTC, valued at $3.4B, shows strong financial health and a 56% return over the past year. Recent developments include a lease amendment, FDA application for FA treatment, Morgan Stanley upgrade, Novartis collaboration, and strong Q3 earnings.

Massachusetts biopharma companies secured $7.89 billion in VC funding in 2024, marking a post-pandemic increase. Boston surpassed Cambridge in venture dollars, signaling a shift for mid- to late-stage firms. The state led in drug development, with significant FDA approvals and a strong global pipeline presence.

Dr. Horan, Dr. Sauder, Dr. Ramsay, and Dr. Yohn are Bristol Myers Squibb employees. Dr. Harvey has affiliations with multiple companies, including Bristol Myers Squibb. Dr. Ramsay holds Bristol Myers Squibb stock. Dr. Keefe consults for various companies and has received royalties. Dr. Davis consults for several companies. Dr. Paul has roles at Karuna Therapeutics, Sage Therapeutics, and Voyager Therapeutics. Dr. Brannan was with Karuna Therapeutics and now consults for Bristol Myers Squibb.

A 30% reduction in long episodes correlates with a ≥50% reduction in clinical seizures, validating the biomarker for RAP-219's Phase 2a proof-of-concept trial in focal epilepsy, with topline data expected in mid-2025.

FDA placed a clinical hold on Rapport Therapeutics' RAP-219 for diabetic peripheral neuropathic pain, granted accelerated approval to PTC Therapeutics' gene therapy for AADC deficiency, cleared icobrain aria AI tool for ARIA detection in Alzheimer treatment, expanded age indication for Theranica's Nerivio Migraine device in pediatric patients, and issued a CRL for Applied Therapeutics' govorestat for classic galactosemia.

Timmerman Report celebrates its 10th anniversary with events in Boston (Mar. 6) and Seattle (Mar. 13), featuring biotech leaders discussing future trends in biopharma.

FDA places clinical hold on Rapport Therapeutics' phase 2a trial for RAP-219 in diabetic peripheral neuropathic pain; Alnylam Pharmaceuticals to assess mivelsiran in cAPPricorn-1 for cerebral amyloid angiopathy; FDA accepts PTC Therapeutics' resubmitted NDA for ataluren in nonsense mutation Duchenne muscular dystrophy.

Rapport Therapeutics announces Q3 2024 financial results, appoints new Board members, and updates on RAP-219 trials in focal epilepsy, peripheral neuropathic pain, and bipolar disorder. The company ended Q3 with $320.7 million in cash, expected to fund operations through 2026.

The FDA has placed a clinical hold on Rapport Therapeutics' phase 2a trial for RAP-219, an epilepsy treatment for diabetic peripheral neuropathic pain, requesting additional protocol information. Rapport is conducting ongoing phase 2a trials for focal epilepsy and bipolar disorder unaffected by this hold. The company is also progressing with MAD-2 and PET trials, expecting results in 2025, and developing a long-acting injectable formulation of RAP-219.