Ann Arbor-area startup Gemphire Therapeutics just raised $2.7 million, according to a regulatory filing, for its drug that treats homozygous familial hypercholesterolemia.
The drug candidate – called gemcabene – is being licensed from Pfizer, according to a December profile from Crain’s Detroit Business.
The startup’s led by Mina Sooch, formerly CEO of Michigan’s ProNAi Therapeutics, which raised an impressive $59.5 million financing round last year – the largest VC deal in Michigan. Sooch left the cancer drug startup last September, in advance of ProNAi’s (still forthcoming) plans to go public this year.
“I looked at opportunities locally and at interesting opportunities nationally,” Sooch told Crain’s. “I looked at Harvard spinoffs and I looked at San Diego. I spent a lot of time kicking tires. I wanted to lower the risk of whatever I was going to do.”
She settled again in Michigan, developing a drug that has already had its risk profile evaluated by numerous Pfizer trials. Crain’s writes:
The risk was reduced by the tens of millions of dollars Pfizer spent between 1999 and 2004 on trials for gemcabene, a drug taken orally once a day. A total of 952 patients with varying levels of LDL — the so-called bad cholesterol — were tested in a total of 17 human trials. Ten were phase-one trials, which found little toxicity, and seven phase-two trials, which showed clinical effectiveness in lowering LDL and triglycerides.
Unfortunately, the clinical train wreck that was Torcetrapib led Pfizer to jettison its R&D on cardiovascular drugs – it spent $1 billion on the drug, only to have it fail in phase 3 when patients began dying. So, as many midstage drugs are wont to do, gemcabene gathered dust for years on Pfizer’s shelves, until Gemphire cofounders Charles Bisgaier and David Lowenschuss brought it back to life.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
It’s being used to treat homozygous familial hypercholesterolemia because of the scarcity of effective, safe drugs on the market – the two existing therapies can cause severe liver damage. The company received orphan status last February from the FDA. Sooch told Crain’s that she projects it should take just $25 million to get the drug to market as opposed to the standard hundreds of millions, thanks to the heavy R&D already conducted by Pfizer.
