Only two days after Bridgebio Pharma Inc. impressed investors with data from BBP-418 in limb-girdle muscular dystrophy type 2I/R9, the company was back at it again, this time reporting positive top-line results from its global phase III study of encaleret in autosomal dominant hypocalcemia type 1 (ADH1), a genetic form of hypoparathyroidism. Both datasets share a commonality, however, as CEO Neil Kumar noted that “treated patients aren’t just staving off decline, but are improving.” In the case of ADH1, which results in hypocalcemia, treatment with encaleret resulted in 76% of patients achieving both serum and urine calcium targets at week 24.

Qyuns’ bispecific antibody QX-031N binds $1B deal with Roche

Qyuns Therapeutics Co. Ltd. signed a potential $1.07 billion license deal with Roche Holding AG, granting the latter exclusive rights to QX-031N – a human thymic stromal lymphopoietin and interleukin-33-targeting bispecific antibody. QX-031N is an injectable, long-acting antibody expected to be developed for respiratory diseases like chronic obstructive pulmonary disease and asthma, with potential to become both a first-in-class and best-in-disease therapy, according to Qyuns. Under the terms, Basel, Switzerland-based Roche will gain the exclusive right to develop, manufacture and commercialize the product worldwide. Qyuns will receive a one-time, nonrefundable and noncreditable up-front payment of $75 million and is eligible to receive up to $995 million in milestone payments, along with tiered royalties on product sales.

Kyverna comes through with phase II myasthenia gravis data

Wall Street optimism for KYV-101 in generalized myasthenia gravis was vindicated as Kyverna Therapeutics Inc. rolled out positive interim results from the phase II portion of the registrational KYSA-6 clinical trial testing the drug, a fully human, autologous, CD19 CAR T-cell therapy with CD28 costimulation. The findings were shared in an oral presentation at the Myasthenia Gravis Foundation of America’s scientific session during the American Association of Neuromuscular and Electrodiagnostic Medicine annual meeting in San Francisco. One hundred percent of patients achieved clinically meaningful responses in the Myasthenia Gravis Activities of Daily Living and Quantitative Myasthenia Gravis scores. Shares of Emeryville, Calif.-based Kyverna (NASDAQ:KVTX) were trading at $7.24, down 37 cents.

In a $1B+ deal, Modex joins with Regeneron to develop antibodies

With a modest $7 million up front but an ultimate payout potentially topping $1 billion on the line, Modex Therapeutics Inc. will collaborate with Regeneron Pharmaceuticals Inc. to develop antibodies for treating multiple indications. Modex, which is owned by Opko Health Inc., was one of the companies that saw mRNA-rated contract work de-scoped by the Biomedical Advanced Research and Development Authority in August. Modex CEO Gary Nabel is a former director of vaccine research at the NIH. Modex has collaborated with larger companies before. In March 2023, Modex joined up with Merck & Co. Inc. to develop an Epstein-Barr virus vaccine.

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