Mavacamten

Today, a brief rundown of news involving Bluebird bio and Affimed, as well as updates from Cytokinetics, BridgeBio Pharma and Acelyrin that you may have missed.Carlyle and SK Capital Partners, two investment firms attempting to buy Bluebird bio, have amended their offer to give Bluebird shareholders a choice of payouts. Holders of Bluebird stock can either receive $3 per share plus rights to $6.84 more if a certain sales milestone is achieved Carlyle and SK Capitals original bid or $5 per share. Shareholders previously had a deadline of May 12 to tender their stock to the original deal, but now have until May 29 to do so. Bluebirds board is in favor of accepting Carlyle and SK Capitals offer, warning investors in a statement Wednesday that, absent a deal, the company is at significant risk of default. Ned PagliaruloCancer drug developer Affimed has filed for insolvency, warning investors Tuesday that there is substantial doubt the company can sustain itself. As a result of the filing, Affimed shares will be suspended from trading on the Nasdaq stock exchange beginning May 20. The company has been talking with potential investors and partners about deals or other ways in which it might raise capital, but has so far been unsuccessful. Affimed, which has three drugs in clinical testing, last reported cash holdings of 24 million euros as of Sept. 30, 2024. Ned PagliaruloCytokinetics on Tuesday said its experimental drug aficamten outperformed beta blockers in a Phase 3 study of people with obstructive hypertrophic cardiomyopathy. U.S. regulators are already reviewing aficamtens use in the inherited heart condition, with an approval decision now expected by late December after a recent delay. The latest results, though generally expected by investors, could help boost its case with physicians and insurers, wrote RBC Capital Markets analyst Brian Abrahams. If approved, aficamten would compete for market share with Bristol Myers Squibbs Camzyos, which booked $602 million in sales last year. Ben FidlerBridgeBio Pharma has begun dosing patients in a first-of-its-kind Phase 3 trial testing whether its drug, Attruby, can prevent or delay the onset of transthyretin amyloidosis in people more likely to inherit it. The trial, ACT-EARLY, will evaluate Attruby as a prophylactic treatment in carriers of a disease-causing variant of the TTR gene thats implicated in the hereditary form of the condition. Attruby is one of three marketed drugs that can slow progression of a type of transthyretin amyloidosis that affects the heart. No medicines have been proven to prevent the disease, however. Ben FidlerShareholders in Acelyrin and Alumis voted Tuesday to combine the two biotechnology companies, ending a twisting merger saga that featured an unsolicited bid from an entity controlled by Tang Capital and a push from Trium Capital to force Acelyrin to liquidate its assets and return cash to its stockholders. Since announcing the planned combination, Alumis has tweaked the terms of the deal, saying its shareholders would own 52% of the new company, while Acelyrins would hold 48%. The merger is now set to close in the second quarter of 2025. Gwendolyn Wu '

Cytokinetics said its cardiac myosin inhibitor aficamten — already under review at the FDA as a potential treatment for obstructive hypertrophic cardiomyopathy (HCM) — delivered positive topline results in another Phase III trial, this time comparing it to a commonly used beta-blocker.The new data emerged about a week after the FDA extended its timeline for a decision by three months due to the company's failure to include a Risk Evaluation and Mitigation Strategy (REMS) in its original application.The 175-patient MAPLE-HCM trial met its primary endpoint, demonstrating significant improvement in peak oxygen uptake (pVO2) from baseline to week 24 compared to metoprolol. The head-to-head comparison could help aficamten stand out against Bristol Myers Squibb's first-to-market cardiac myosin inhibitor Camzyos (mavacamten) in the obstructive HCM space.Camzyos, approved by the FDA in 2022, recently received updated labelling that reduced some of its monitoring requirements and eased certain restrictions.In a release Tuesday, Cytokinetics' R&D chief Fady Malik said the MAPLE-HCM results are "the first evidence that aficamten may be used as monotherapy," and also "provide important context to the benefit of this potential new medicine compared to the current standard of care."Cytokinetics faced investor disappointment earlier this month when the FDA pushed its decision date for aficamten from September 26 to December 26 after the agency decided that the company needs to submit a REMS, which the FDA then classified as a major amendment requiring more review time.The filing is largely based on the Phase III SEQUOIA-HCM study. FDA hasn't asked for any additional data, and Cytokinetics is "still moving forward full steam ahead" with its commercial readiness plans, a company representative told FirstWord earlier this month. See – ViewPoints: Cytokinetics' R&D head on why aficamten is worth the wait.Morgan Stanley analysts have said aficamten is likely to be approved for obstructive HCM in 2025. "We see greater upside potential from the MAPLE-HCM study, which could position aficamten in first-line therapy and provide differentiation from Camzyos," they added.