While rife with unknowns, the biotech Retrophin's series of announcements this morning about advancing its pipeline are difficult to ignore.
Founded and led by hedge fund vet Martin Shkreli, Retrophin has nailed down an agreement to raise $25 million to finance development of a preclinical rare-disease drug and a deal to license a potential autism and schizophrenia compound from an unnamed "major pharmaceutical company." The PIPE financing is expected to close next week.
Retrophin ($RTRX) has forked over an unspecified upfront payment to the mystery drugmaker for "the exclusive right to negotiate a royalty-bearing U.S. license" for the potential autism and schizophrenia product for up to 120 days. There are no details on the compound or the pharma group. The company says that studies have shown results for autistic and schizophrenic patients on the product.
"We are very pleased to have the opportunity to exclusively negotiate a license to a product which may represent an important new therapeutic option for patients with schizophrenia and/or autism spectrum disorders," Shkreli said in a statement. "Numerous existing studies utilizing this product have shown positive results in autistic people and those suffering from schizophrenia. This data, combined with our own due diligence, leads us to believe that this drug may have significant utility in these indications."
In related news, the New York-based biotech outfit revealed upbeat survival results from "interim preclinical tests" of an experimental therapy called RE-024 for the extremely rare disease pantothenate kinase-associated neurodegeneration (PKAN). Yet take what you will from interim tests of mice.
Unknowns aside, Shkreli appears to be staying true to the mission of his company to go after new treatments for rare diseases and those with unmet medical needs. Autism, for example, is an intellectual disability that affects millions of children who lack a single drug that targets the molecular drivers of the disease. And PKAN, which afflicts just 5,000 to 10,000 people, causes a range of nasty neurological problems and often death within 10 years of diagnosis, the company said.
Retrophin plans to host a conference call with investors at 10 a.m. ET this morning. Shkreli will likely face many questions from the listeners.
- see the financing release
- here's the release about the pharma deal
- and the news about the preclinical PKAN study