The good news for Sarepta Therapeutics Inc. is bad news for Pfizer Inc. as the phase III study of its mini-dystrophin gene therapy in Duchenne muscular dystrophy has missed its primary endpoint. Now Sarepta’ s Elevidys (delandistrogene moxeparvovec-rokl), a single-dose, adeno-associated virus-based gene transfer therapy, is barrelling toward a June 21 PDUFA date with the U.S. FDA as the near competition shrinks in the rearview mirror. Pfizer’s fordadistrogene movaparvovec failed to hit the mark on motor-function movement in ambulatory boys ages 4 to 7 when compared to placebo. It's another blow to Pfizer as about six weeks ago a boy in its phase II Daylight study of Duchenne died after receiving the same gene therapy.

Urogen soars on duration of response data in bladder cancer

Urogen Pharma Ltd. shares (NASDAQ:URGN) were trading at $17.90, up $5.18, or 40%, on word of an 82.3% (95% CI, 75.9%, 87.1%) 12-month duration of response data by Kaplan-Meier estimate (n=108) from its phase III Envision study. The finding was made in in low-grade, intermediate-risk, non-muscle invasive bladder cancer patients who achieved complete response (CR) at three months after the first instillation of UGN-102 for intravesical solution. Princeton, N.J.-based Urogen previously disclosed that Envision met its primary endpoint by showing that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of the drug, a sustained release, hydrogel-based version of the antineoplastic antibiotic mitomycin.

Itch takes a powder as Cara scratches difelikefalin

Following a phase II/III failure, Stamford, Conn.-based Cara Therapeutics Inc. discontinued its only pipeline program, that of oral difelikefalin for moderate to severe pruritis in adults with notalgia paresthetica, placing the company’s future in question. While the intravenous version of the first-in-class kappa opioid receptor agonist gained U.S. FDA approval under the brand name Korsuva in August 2021 for pruritis in patients undergoing hemodialysis, sales coming to Cara are meager compared to the company’s expenses. For 2023, Cara recorded a net loss of $118.5 million, which accounted for $108.5 million in R&D expenses and $21 million in total revenue – including $12.9 million in Korsuva profit-share revenue from partner CSL Vifor. “Given no obvious value drivers from here … we downgrade Cara to a neutral rating with no price target,” said H.C. Wainwright analyst Oren Livnat. Cara’s shares (NASDAQ:CARA) plunged 36% to 43 cents in early trading June 13.

$168M series A, B rounds propel Santa Ana Bio

Santa Ana Bio Inc. made its debut with combined series A and B rounds that pulled down $168 million for precision immunology push in inflammatory diseases. Candidates are expected to enter the clinic next year. Founding investor Versant Ventures led the series A, joined by TPG and Google Ventures (GV); the $125 million series B was spearheaded by GV.

China’s AI-powered Quantumpharm raises $126M in Hong Kong IPO

Chinese artificial intelligence (AI)-driven drug discovery firm Quantumpharm Inc., also known as Xtalpi, began trading on the Hong Kong stock exchange June 13, listing under a new special technology listing track that lured it away from an IPO in the U.S. Shenzhen, China-headquartered Xtalpi initially priced its HKEX offering of 187.37 million shares at HK$5.28 per share for net proceeds of HK$989.33 million (US$126 million). The company’s shares began trading under stock code 2228, peaking at HK$6.58 in intraday trading and closing at HK$5.78, recording a gain of 9.46% and market capitalization of HK$19.69 billion.

Nektar Therapeutics drives upward momentum in drug developers index

The BioWorld Drug Developers Index (BDDI) mirrored the movements of the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA), up in May after a decline at the end of April. BDDI ended May up 1.82% after closing in April down 4.8% from the beginning of the year. The NBI took a similar path, closing in May up 0.7% after falling 4.76% at the end of April. The DJIA had fallen to 0.34% at the end of April, then closed May up 2.64%. This represents a shift from the end of February, when the BDDI had surged by 4.41%, outperforming both the NBI and DJIA.

Biosecure Act misses first congressional ride

While the Biosecure Act missed its first chance at a congressional ride, that doesn’t mean it will be stranded by the wayside. Although it enjoyed strong bipartisan support, the bill wasn’t included as a rider to the U.S. House version of the National Defense Authorization Act, a must-pass defense spending bill for fiscal 2025. However, there’s still significant momentum to pass the bill, possibly as an add-on to the Senate version of the defense spending bill, as stand-alone legislation or as part of an end-of-year health legislative package, RBC Capital Markets LLC analyst Leonid Timashev noted.

Supremes shoot down abortion pill challenge

The U.S. Supreme Court today unanimously upheld the FDA’s relaxed regulation of the abortion drug mifepristone when it ruled that the organizations challenging those changes lacked the standing to do so. “A plaintiff’s desire to make a drug less available for others does not establish standing to sue,” Justice Brett Kavanaugh wrote for the court. While Justice Clarence Thomas agreed with the rest of the court, he issued a concurring opinion to express his concerns about the expansion of associational standing over the years. He encouraged the court to “explain just how the Constitution permits associational standing” when an appropriate case arises.

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