Noticias y eventos de Beam Therapeutics Inc. (BEAM)BEAMBeam Therapeutics Inc.

Beam Therapeutics Reports Initial Data from BEAM-301 Clinical Trial

Beam Therapeutics reported initial data from the ongoing Phase 1/2 clinical trial evaluating BEAM-301 for the treatment of glycogen storage disease type Ia, or GSDIa. The data will be presented during a poster session at the 33rd Annual Congress of the European Society of Gene and Cell Therapy, or ESGCT, in Hamburg, Germany. BEAM-301 is an investigational liver-targeting lipid-nanoparticle formulation of base editing reagents designed to correct the R83C mutation, one of the most prevalent disease-causing mutations in GSDIa and one that is associated with the most severe form of the disease. BEAM-301 is being evaluated in an open-label Phase 1/2 dose-exploration trial to investigate its safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy in patients with GSDIa who are homozygous or compound heterozygous for the R83C variant. As of the August 17, data cutoff, six patients had received a single intravenous infusion of BEAM-301, including three patients treated with 20 mg and three patients treated with 40 mg. Median follow-up across both cohorts was 7.6 months. BEAM-301 demonstrated an acceptable safety profile consistent with LNP-based therapies, with no dose-limiting toxicities, Grade 4 or 5 treatment-emergent adverse events, or TEAEs, or treatment discontinuations due to TEAEs reported. All patients experienced at least one TEAE considered related to BEAM-301. Four of six patients experienced serious TEAEs considered related to BEAM-301, including three Grade 3 infusion-related reactions, or IRRs. Premedication with shorter-acting steroids, which are typically used in this patient population, may have contributed to the Grade 3 IRRs observed. The steroid premedication and infusion rate regimens have since been modified. Two other serious TEAEs of diarrhea and rhabdomyolysis occurred in two patients; both have resolved. With the exception of a single Grade 2 elevation in aspartate aminotransferase, or AST, all transaminase elevations were Grade 1. All liver enzyme elevations resolved without treatment, and total bilirubin remained within normal limits. BEAM-301 demonstrated dose-dependent improvements in time to fasting hypoglycemia, or TTH, a key measure of glucose regulation in patients with GSDIa. Median TTH increased from 2.9 hours at baseline to 4.3 hours at Day 36 in the 20 mg cohort, and from 2.5 hours at baseline to 15.0 hours at Day 36 in the 40 mg cohort. Two of three patients in the 40 mg cohort achieved the protocol-defined maximum controlled fasting duration of 15 hours, consistent with restored endogenous glucose production. During the 15-hour fasting challenges, blood glucose levels were generally maintained within the euglycemic range. One patient in the 40 mg cohort did not show an increase in TTH at the first post-dose assessment; several factors may have contributed, including elevated body mass index, or BMI, and heterozygosity for the R83C mutation. Daily starch intake and daily number of starch doses generally decreased following treatment with BEAM-301, with two of three patients in the 40 mg cohort achieving a 100% reduction in daily starch requirements and no longer requiring starch supplementation at their latest reported assessments. Continuous glucose monitoring demonstrated increased time spent in the euglycemic range and decreased time in the hyperglycemic range following treatment with BEAM-301, despite reductions in daily starch intake. Patients generally did not experience an increase in time spent in hypoglycemia. Triglyceride levels were generally comparable to baseline at the latest evaluable time point. Based on the safety and clinical findings observed to date, Beam is advancing BEAM-301 into the 60 mg dose-escalation cohort, with enrollment currently underway. Beam also plans to engage with the FDA regarding a potential path to approval.

Beam Therapeutics Presents Initial Data on BEAM-301 at ESGCT

Beam Therapeutics announced that the company will present initial data from the ongoing Phase 1/2 clinical trial evaluating BEAM-301 for the treatment of glycogen storage disease type Ia, or GSDIa, at the 33rd Annual Congress of the European Society of Gene and Cell Therapy, or ESGCT, taking place October 27-30, in Hamburg, Germany. BEAM-301 is a liver-targeting lipid-nanoparticle, or LNP, formulation of base editing reagents designed to correct the R83C mutation, one of the most prevalent disease-causing mutations in GSDIa and one that is associated with the most severe form of the disease. As of the June 26, data cutoff, six patients received a single intravenous infusion of BEAM-301, including three patients treated with 20 mg and three patients treated with 40 mg. Median follow-up was 313 days in the 20 mg cohort and 53 days in the 40 mg cohort. One patient in the 40 mg cohort was not evaluable for efficacy as of the data cutoff. BEAM-301 demonstrated an acceptable safety profile that was consistent with LNP-based therapies. All patients experienced at least one treatment-emergent adverse event considered related to BEAM-301. Five serious adverse events were reported and resolved within two to 10 days, including three Grade 3 infusion-related reactions. The use of shorter-acting steroids as premedication, which is typical in this patient population, may have contributed to the Grade 3 IRRs observed. The steroid premedication regimen has since been optimized. All transaminase elevations were transient and resolved without intervention. With the exception of a single Grade 2 elevation in aspartate aminotransferase, all transaminase elevations were Grade 1. No dose-limiting toxicities, treatment discontinuations due to treatment-emergent adverse events, or deaths were reported. Dose-dependent improvements in time to fasting hypoglycemia were observed. At baseline, median TTH was 2.9 hours in the 20 mg cohort and 2.5 hours in the 40 mg cohort. By Day 36, median TTH increased to 4.3 hours in the 20 mg cohort, while both evaluable patients in the 40 mg cohort achieved the protocol-defined maximum controlled fasting duration of greater than or equal to15 hours, consistent with restored endogenous glucose production. Reductions in prescribed daily starch intake were observed following treatment with BEAM-301. Decreases from baseline in prescribed daily starch intake guided by continuous glucose monitoring were 74%, 71%, and 11% in the 20 mg cohort, and 71% and 92% in the 40 mg cohort. Corresponding daily starch doses decreased by 0-4 and 2-4 doses/day in the 20 mg cohort and the 40 mg cohort, respectively. Triglyceride levels were comparable to baseline at last follow-up. Updated data from the ongoing Phase 1/2 trial will be presented during the ESGCT poster session.

Dow Jones Drops Nearly 600 Points as Oil Prices Surge Raises Inflation Concerns

The major averages closed broadly lower, with the Dow Jones losing nearly 600 points as a renewed jump in oil prices and escalating Middle East tensions stoked inflation concerns.The rise in oil prices is particularly problematic coming after Friday's stronger-than-expected August jobs report. The U.S. added 162,000 jobs, nearly three times economists' expectations, pushing markets to increase bets that the Federal Reserve could raise rates at its September 16 meeting. The probability of a hike is now around 60%, while the 10-year Treasury yield has climbed to roughly 4.8%.Get caught up quickly on the top news and calls moving stocks with these five Top Five lists.1. STOCK NEWS:An Amazoncargo plane overshot the runway at Miami International Airport this weekend,Qualcommannounced awith AmazonGE AerospaceConsolidated Precision Products for $11.75BBoston Scientificsaid it isits Q3 and FY26 net sales and adjusted earnings per share guidanceNovartisshares fell after its Phase 3 HARBOR study for DM12. WALL STREET CALLS:Intelto Outperform at Northland after "material" turnaround progressPelotonto Underweight at Morgan StanleyAirbnbto Outperform at Raymond JamesBest Buyto Neutral at DA DavidsonSpaceXwith a Buy at Pivotal Research3. AROUND THE WEB:Netflixplans to report box office grosses for six upcoming films, Variety reportsNvidia-backedFirmus signed a multi-year deal with OpenAI to supply computing capacity to two Malaysian data centers, Reuters reportsAhead of Oura's upcoming IPO filing, Robinhoodhas been listed as an underwriter, WSJ saysNovo Nordiskhas halted two trials testing of ziltivekimab, its experimental heart disease medicine, would help patients with heart failure, Bloomberg reportsThe Independent Communications Authority of South Africa, South Africa's telecommunications regulator, plans to investigate prices charged by providers such as Netflixand Meta'sWhatsApp, Bloomberg reports4. MOVERS:Roivant Sciencesgained in New York after announcing its Phase 3 PHocus clinical trialPharvarisincreased in New York after announcing its CHAPTER-3 Phase 3 studyD-Wave Quantumwas higher afterwith the U.S. Department of CommerceSigma Lithiumfell after a preliminary judicial rulingBeam Therapeuticswas lower after announcing updated clinical data from its5. EARNINGS/GUIDANCE:United Natural Foodsand provided guidance for FY27ABMand raised its outlook for FY26, with CEO Scott Salmirs highlighting "record quarterly revenue, robust EPS growth and substantial cash generation"Solaris Energyraised itsGeneral Millsreaffirmed itsGameStop, with EPS higher year-over-yearINDEXES:The Dow fell 628.18, or 1.18%, to 52,786.07, the Nasdaq lost 85.58, or 0.32%, to 26,421.41, and the S&P 500 declined 45.08, or 0.58%, to 7,673.52.