The passage of the 21st Century Cures Act by the U.S. Senate earlier this week is a big positive for Revive Therapeutics (RVV.V) and a validation of the company's business model. The Act, which passed through U.S. congress with an overwhelming majority, 94-5 in the senate and 326-24 in the house, is designed to facilitate U.S. FDA approval of new drugs and medical devices and bring innovative medicines to patients faster.
Specifically, the Act enhances current FDA programs like breakthrough designation, accelerated approval, and fast track status, and directs the U.S. FDA to make decisions based on the use of “real world evidence” for approval of new indications for existing drugs. Companies can now include things like observational studies, patient input, anecdotal data, and other retrospective research analysis as part of its petition for U.S. approval. This greatly reduces the cost and time to market compared to conducting large, prohibitively expensive randomized clinical trials and should increase the odds of success reformulations and label expansions of known therapeutic agents.
Revive A Big Winner
Revive Therapeutics' lead drug candidate is bucillamine, a dithiol derivative of tiopronin for the treatment of cystinuria. Bucillamine is a drug that has been approved and used in Japan for over three decades, mostly for the treatment of rheumatoid arthritis (RA). Ayumi Pharma of Japan reported 57 million tablets sold in 2015. Recent data confirm the efficacy of bucillamine in RA, as well as other oxidative conditions such as cisplatin-induced ototoxicity and transplantation-associated reperfusion injury. Revive has previously studied bucillamine in a Phase 2 study in patients with gout, demonstrating efficacy comparable to Takeda's Colcrys®.
See my initiation report on Revive → Revive Targets Cystinuria With Bucillamine (July 2016)
Japanese researchers have tested bucillamine and tiopronin, approved in the U.S. as Thiola® and sold by Retrophin, Inc., in patients with cystinuria. The effectiveness of bucillamine was compared with tiopronin shows that bucillamine was statistically superior (markedly superior in two patients and slightly superior in the third) to Retrophin's $150 million drug. Importantly, bucillamine has an "A" safety rating from the Japanese Ministry of Health.
Bucillamine seems exactly the type of drug that the 21st Century Cures Act is designed to expedite through U.S. approval. By signing the bill into law, President Obama is directing the FDA to look at known safe and proven effective medications like bucillamine and get them to patients in need sooner and with significantly less cost. That's a big positive for tiny Revive Therapeutics and bodes well for the company's business model of focusing on treating neglected diseases, like cystinuria, Wilson's disease, and Rhett syndrome, with known proven molecules like bucillamine and tianeptine.
Revive is expected to begin a Phase 2 trial with bucillamine in cystinuria in the next few weeks. Clinical and preclinical evidence suggests that the drug, which is already approved and safely used outside the U.S. for various indications, not only works for cystinuria but is an improvement over existing medications on the U.S. market today. My valuation work shows Revive to be tremendously undervalued right now, and the 21st Century Cures Act gives the company a shot in the arm with respect to bucillamine development. It also emboldens the business model and I believe will ultimately help to create value for shareholders.
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