Proteostasis shares whacked hard as CF drug falls short; Advaxis axes trial plans in wake of a clinical hold

Proteostasis tried mighty hard to persuade investors that its positive biomarker data from a study of its cystic fibrosis drug PTI-801 underscored the drug’s potential. But with a fail on improving lung function, added to some caustic criticism from a prominent analyst, the stock got whacked. 

“While PTI’s corrector binds to a different site on the CFTR protein than Vertex’s medicines, the failure to match the high efficacy bar established by Vertex $VRTX is a testament to the difficult development path for competitors with small molecule therapies for cystic fibrosis,” noted Leerink’s Geoffrey Porges. 

Next up, notes Leerink, is Galapagos’ GLPG- 2737 $GLPG, which will also be measured against Vertex’s dominant drug. Galapagos will have to get around Vertex’s loyal legion of analysts, who are quick to attack rivals seeking to approach the throne. But data are data.

Shares of the company $PTI plunged 27% in pre-market trading Thursday.

→ With its lead drug on clinical hold following the death of a patient in a study, Advaxis $ADXS is reworking its pipeline plans to slash its burn rate and guard cash. The biotech reported Thursday morning that it will cut its investment in axalimogene filolisbac and amp up work on other therapies while carving $30 million out of its annual budget. Execs are also looking for a partner, saying that if they can’t strike a pact fairly soon they will stop their “AIM2CERV trial in high-risk locally advanced cervical cancer, and will not conduct the ADVANCE PD-1 combination trial in metastatic cervical cancer, which has not yet been initiated. Advaxis has determined to focus future development efforts for AXAL on HPV-positive head-and-neck cancer through cost-effective clinical studies that are currently being explored.”

→ Microcap biotech Ophthotech $OPHT shot up 30% — and counting — Thursday morning after announcing a gene therapy collaboration with University of Pennsylvania and University of Florida. The New York-based company now has a license to develop and commercialize an adeno-associated virus (AAV) gene therapy that treats an orphan monogenic disease called rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP). Affecting around 11,000 patients in the US and five major European markets, the disease triggers severe vision loss leading to blindness. The preclinical product coming out of Penn and Florida, Ophthotech says, is designed to simultaneously knock down mutant rhodopsin protein and deliver a functional replacement. This marks the second gene therapy deal for Ophthotech, which is also collaborating with the University of Massachusetts Medical School. In addition to working toward an IND, the biotech also made a separate sponsored research pact with Penn to conduct further preclinical studies.

→ Engulfed in a curious series of regulatory and financial woes, MiMedx $MDXG is letting its CFO Michael Senken go after wrapping up an internal investigation into its sales and distribution practices. Also shown the door: John Cranston, the corporate controller and treasurer. An interim CFO, Edward Borkowski, has been brought in to restate financial statements from the past six years, as the company claims that information can no longer be relied upon. That’s a conclusion of the still ongoing investigation by its own audit committee, which began this February and delayed the release of 2017’s financial results (the company still doesn’t have a timeline for filing its 10-K). The internal review was quickly followed by a reported preliminary probe by the US Justice Department in the same matter as well as whether the tissue graft maker overcharged the government for its products. Marietta, Georgia-based MiMedx, which has a troubled history with short sellers, saw its stock fall by close to 13%.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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