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companies to watch- 2013 Roundup Snapshot analyses of selected companies developing new life sciences products and technologies DEVELOPMENTS TO DATE • September: Oral and IV solithromycin became the only antibiotic to receive the FDA's qualified infections product designation (QIPD) for the treatment of CABP (communityacquired bacterial pneumonia). "Solithromycin demonstrated safety and tolerability in patients with chronic liver disease without a change in pharmacokinetics so that there will be no change in dosing of patients with hepatic insufficiency." • October: Taksta gained orphan drug designation for the treatment of prosthetic joint infections. • November: Preclinical results suggested solithromycin may provide effective prevention and treatment of intrauterine infections during pregnancy. "No new antibiotic has been developed for infections in pregnancy in over 20 years." COMING IN 2014 • Mid-year: release of Solitaire-Oral Phase 3 trial top-line data in CABP July — Soligenix Biodefense drives this company. With U.S. government support, Soligenix continues to develop an anti-ricin vaccine, a treatment for radiation poisoning, and other products based on its ThermoVax platform. In September, it nearly doubled its previous funding with a new $23.6 million BARDA contract and a $6.4 million NIAID (National Institute of Allergy and Infectious Diseases) contract for its radiation drug OrbeShield. Not bad for a company with a tiny market cap of only about $40 million (twice what it was in June). "Now with the proper funding in hand, our primary focus moving into 2014 is on the quality execution of all our development programs," says Christopher Schaber, chairman, president, and CEO. DEVELOPMENTS TO DATE • December: initiated two Phase 2 clinical studies: SGX942, the company's first-in-class innate defense regulator technology in oral mucositis; and orBec in chronic GI graft-versushost disease (GVHD), supported by a $300 million NIH grant COMING IN 2014 • initiation of a Phase 2/3 clinical study with SGX203 in pediatric Crohn's disease and a Phase 2 clinical study with SGX201 in acute radiation enteritis • completion of oral mucositis Phase 2 clinical study and chronic GI GVHD Phase 2 clinical study • preclinical data supporting vaccine/biodefense programs, most notably OrbeShield, RiVax (ricin toxin vaccine), and ThermoVax (vaccine heat stabilization technology August — CogRx (Cognition Therapeutics) Still a tough cookie in a tough area — this developer of 34 LifeScienceLeader.com February 2014 neuroscience drugs for Alzheimer's disease (AD) and possibly other conditions, such as Parkinson's and ALS, has been chalking up a series of firsts since its appearance in Companies to Watch. "CogRx's small molecule drug candidates represent the first ever reported to directly target toxic Abeta oligomer proteins and their receptors and stop their bad effects on memory," CEO Hank Safferstein says. "CogRx is rapidly advancing these exciting drug candidates toward clinical trials." DEVELOPMENTS TO DATE According to the company, CogRx has: • become "the first group to demonstrate siRNA (small interfering RNA) knockdown of a specific receptor on neurons and glia; lowers oligomer binding more than 90 percent • become "the first group to demonstrate dose-dependent reduction in Abeta oligomer binding in post-mortem human AD brain sections using our proprietary small molecule drug candidates and antibodies raised against specific receptor epitopes" • "developed first-in-class, highly brain-penetrant, orally bioavailable small molecule receptor antagonist (IND candidates) that stop memory deficits in Alzheimer's disease models" COMING IN 2014 • conducting a pre-IND meeting with the FDA with the goal of filing an IND by the end of the year • closing on Series B to fund the AD program into the clinic and into patients diagnosed with Alzheimer's disease • "Use of funds will also support further mechanistic work on the soluble Abeta oligomer protein receptor/ receptor complex we have identified and its potential role in other CNS diseases characterized by abnormal protein aggregation" September — Esperion Therapeutics On track and on target. Another company with blockbuster potential, and one of the fresh crop of IPOs in 2013, this drug developer aims at the still needy area of cholesterol reduction. Esperion's strategy is carefully planning studies and trials to prove its concept and define its target patient groups. "We are on track to deliver, in 2014, top-line results from two large robust Phase 2b clinical trials that will transform Esperion into the leading developer of an oral, once-daily, small molecule LDL-C (LDL cholesterol) lowering therapy for the treatment of patients with hypercholesterolemia," says CEO Tim Mayleben. He says the company set clear goals for its LDL-C lowering drug, ETC-1002, in 2013 and achieved all of them on time. DEVELOPMENTS TO DATE • October: commenced a large Phase 2b study of ETC-