The vision of Life Science Leader is to help facilitate connections and foster collaborations in pharma and med device development to get more life-saving and life-improving therapies to market in an efficient manner. Connect, Collaborate, Contribute
Issue link: https://lifescienceleadermag.epubxp.com/i/216720
Biopharm Development & Manufacturing inhibitors that "take the brakes" off T-cell production (also the rationale for combination therapies). But it remains to be seen if such a complex "regenerative medicine" model can translate to a practical business proposition. Mirna Therapeutics represents the RNA wing of new cancer-Tx approaches. Its technology employs tumor-suppressor microRNA technology to "kill cancer cells by regaining control over multiple oncogenic pathways." This involves restoring and regaining control of the cell's messaging system — a network that stubbornly resists manipulation. As with all RNA-based therapeutic modalities, Mirna faces an uphill battle proving safety and efficacy in human cancers. But of course the RNA crowd is not alone. I offered this tweet in the midst of the cancerrelated presentations: "All new cancer drugs invoke the same sad memory of this: 'We've cured cancer thousands of times — in mice!'" uses the MOA of reducing weight, inflammation, and cardiovascular disease by changing how the body processes fat. Weight loss and cardiometabolic data served as biomarkers to make a strong, if early, case for the drug. Zafgen plans to launch beloranid with narrow labeling for severe obesity, then expand to cardiometabolic disease — a classic case of an orphan- or niche-drug's potential to open up a blockbuster space. LET YOUR VALUES GUIDE YOU DISEASE-AREA DIVERSITY Another notable trend at BIF this year was the greater representation of non-cancer-niche orphan drugs. Cancer and cancer niches have dominated past events because of the dominant interest of Big Pharma in oncology partnerships. In fact, orphan drugs or not, it was refreshing to see a more diverse lineup of companies in general this year. Oxthera is developing its lead product Oxabact for hyperoxaluria, which creates stones and damages liver cells, possibly to prevent dialysis or transplant. It is seeking FDA accelerated-review status for the drug, now in Phase 2, because there is no other treatment option for patients. ContraFect, which like many VC-founded start-ups that sport a board and management full of ex-Big Pharma execs, is going for the (blockbuster) gold with a lead drug to fight blood-borne infections. Labrys Biologics is going up against Amgen, Alder, and Arteus in chronic migraine with a mAb to block binding of human CGRP (calcitonin gene-related peptide) to the CGRP receptor, which causes vasodilation — saying the more than four million chronic patients represent the major unmet need in migraine. Toxicology is a big issue in chronic care, and Labrys' LBR-101 looks good in preclinical tox studies; now, on to Phase 1. Zafgen's beloranid, having yielded supporting data from a Phase 2 study in obesity, YOUR VALUES: THE AMRI SMARTSOURCING™ CORE At AMRI, keeping your values at the center of everything we do enhances our shared mission to improve outcomes and the quality of life. The tenets of AMRI SMARTSOURCING™ align with what you, our customer, values most: Quality, Customer Service, Commitment to Purpose, High Performance, Satisfaction, Ethics, Good Citizenship, and Safety. From mastering creativity in discovery and nurturing your drug development programs to engineering your fnal product, choosing AMRI SMARTSOURCING™ means partnering with a pharmaceutical services organization performing at the highest scientifc levels. You can rely on our global facilities, capabilities and culture to bring your programs from discovery biology through API manufacturing and aseptic fll and fnish. Let your values defne your outsourcing goals. Discover, Develop, Manufacture and Deliver drugs with AMRI SMARTSOURCING™. Global Headquarters: 26 Corporate Circle, Albany, New York, USA 12203 1.518.512.2345 james.grabowski@amriglobal.com www.amriglobal.com amrismartsourcing.com linkedin.com/company/amri December 2013 @amriglobal Drug Discovery Drug Development Manufacturing Aseptic Fill & Finish LifeScienceLeader.com 31