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COMPANIES TO WATCH Column LIFESCIENCELEADER.COM NOVEMBER 2014 14 Finances Research partnership funding Vital Statistics BEAT BIOTHERAPEUTICS B y W. Koberstein $4M Seed Financing 10 Employees Headquarters Seattle, WA SNAPSHOT BEAT BioTherapeutics, aka BEATBio, believes it has a gene-therapy candidate, BB-R12, that could greatly improve the quality of life for heart-fail- ure (HF) patients. The therapy uses a "human- ized construct" or antibody as a vector to induce cardiomyocytes (heart muscle) cells to express an "optimized" form of ribonucleotide reduc- tase, producing dATP (deoxyadenosine triphos- phate), a "superior" form of the cellular fuel ATP (adenosine 5'-triphosphate). dATP appears to give an extra boost to the cells and thus improve the heart's performance. BB-R12 is still in pre- clinical studies, planned for a Phase 1 trial in early 2016. Most of its seed money will fund that trial as well as development of the construct and manufacturing scale-up. KEY MILESTONES Demonstrated that BB-R12 restores ejection fraction and overall cardiac performance in ani- mal models of heart failure and improves con- traction and relaxation in healthy and depressed heart muscle cells following injury. No safety issues identified to date. Developed and manufactured a humanized gene construct and scaled up manufacturing using a system licensed from the NIH. Confirmed earlier rodent experiments using the human construct. Completed a successful proof-of-concept study with the humanized construct in a large-animal (swine) myocardial infarction/heart failure model. Held a pre-IND meeting with the FDA and con- firmed development plans and timeline to enter the clinic in early 2016. WHAT'S AT STAKE When a tiny gene-therapy company wants to conquer the heart-failure market, with its patient population of almost 6 million in the U.S. alone, it is only logical to ask some questions. BEATBio is worth watching because it could be an early sign that this long-moribund space, littered with recent failures, is heating up. But the company faces a long haul ahead in proving its treatment can simultaneously deliver strong benefits to millions of patients and meet the challenges of a cost-driven healthcare environment. At this early stage, the company leaves most of the hard questions unanswered. How practi- cal is gene therapy in such a large population, or will the company target a smaller segment, say, advanced or acute cases only? What will be the likely procedure for the gene therapy — how cumbersome or complicated will it be to treat the cardiac cells? What clinical efficacy endpoints must the ther- apy meet for regulatory approval, simply quality of life or survival? Are there practical concerns in the medical care of heart-failure patients, espe- cially older ones, that would limit or affect use of the therapy? The last concern has plagued older HF therapies. For example, infirmity can limit a patient's ability to answer the call of diuretics in the middle of the night. Any medical procedure can challenge such patients. When I attended BEATBio's presentation at the BIO Investor Forum in October, I asked whether pushing heart cells might exhaust already dam- aged heart muscles. CEO Michael Kranda's answer was that BB-R12 is "not driving a damaged heart," but using a self-regulating mechanism that boosts healthy-cell performance as needed. Only healthy cardiomyocytes produce dATP as a result. Of course, in such a large potential space, potential competition is strong. Big Pharma companies have a number of candidates in line for HF. Other small companies, such as Juventas with its stem-cell therapy for advanced HF in Phase 2, are also vying for a place in the space. May the best MOA (mechanism of action) win, but other factors also count. To its credit, BEATBio has a seasoned team in place. Kranda has both VC and company experience going all the way back to Immunex. The newly hired CMO has been tested by fire; Sam Teichman, M.D., was formerly at Cothera, developer of blood-vessel relaxer serelaxin, which had an application for treating acute heart failure rejected by the FDA last May. l BEATBio's founders, from the University of Washington, are recognized experts in cardiovascular biology, muscle physiology, and bioengineering and have received nearly $50M of NIH funding. W A Y N E K O B E R S T E I N Executive Editor With what it believes is a breakthrough gene-therapy approach, this early-stage company hopes to shake up the huge heart-failure space. BEAT BIOTHERAPEUTICS $2.5M April 2013: Seed-stage Investment led by CET Capital Partners, with the W Fund, others. Now raising Series A f nancing to complete IND (Investigational New Drug)-enabling studies and commence Phase 1 human trials. @WayneKoberstein