→ In its second rare disease startup launch in a matter of days, SF’s BridgeBio is unveiling a new venture Wednesday that’s set to develop up a trunk of assets it licensed from St. Jude Children’s Research Hospital. The company, called CoA Therapeutics, is developing small molecules to increase Coenzyme-A levels in rare genetic disorders that involve a deficiency of the enzyme. The company’s lead compound will focus on pantothenate kinase-associated neurodegeneration (PKAN), an autosomal recessive genetic disorder resulting from mutations in the pantothenate kinase 2 (PANK2) gene estimated to affect three in every million people.
BridgeBio says it’s financially backing the company toward clinical trials, with plans to enter the clinic in 2019.
“PKAN is a progressive, debilitating disease with no current approved therapy, and patients and their families currently rely on supportive treatments, which partially address the symptoms but not the root cause,” said Shafique Virani, CEO of CoA, in a statement. “We believe that our novel approach, using a highly brain-penetrant compound to directly target enzyme activity in neurons, can safely increase CoA levels, ease patients’ symptoms and make a meaningful difference in their quality of life. Our novel approach also holds promise for other diseases with defects in CoA metabolism, including the organic acidemias.”
CoA also brought in Adam Shaywitz, the former executive director in clinical sciences at BioMarin, to serve as the startup’s new CMO. He’ll also serve as the CMO-in-residence at BridgeBio, the company said.
Just days ago, BridgeBio announced it had snatched an Alexion asset to fund and launch Origin Biosciences, BridgeBio’s first foray into ultra-rare diseases.
→ Not surprisingly, scPharmaceuticals $SCPH has received a complete response letter from the FDA for their drug candidate for edema, with regulators looking for more study data and device modifications before they give it another look. The biotech reported a couple of weeks ago that the FDA had found deficiencies in their application, signaling the red flag that dropped today. The stock, which had plunged earlier, slid another 21% this morning.
→ In the latest move to bulk its gene therapy capabilities, Sarepta announced Wednesday that it’s inked a clinical and commercial manufacturing deal with Brammer Bio. The two will partner to design and build dedicated commercial manufacturing capacity within Brammer’s facility to take on the “unusually high demands typical for systemic administration of the micro-dystrophin therapy for DMD,” the companies said in a statement. The partnership gives Sarepta the manufacturing capacity it needs for its micro-dystrophin DMD gene therapy program, along with future gene therapy programs like its Limb girdle muscular dystrophy effort.
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