A journey of a thousand miles begins with a single step, and a big one in this case. BioLineRx (BLRX) announced this morning that the FDA has granted Orphan Drug Designation to its lead oncology candidate, BL-8040, for the treatment of pancreatic cancer. This designation by the FDA entitles BL-8040 to seven years of market exclusivity, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.
BioLineRx shares reacted to the news, jumping nearly 23% to $0.79.
Since 1983, the FDA has granted orphan-drug designation to over 3,500 drugs. Biopharmaceutical companies are attracted to the orphan-drug market, because it can be very lucrative. Around 30 million Americans suffer from 7,000 or so rare diseases. Market research firm EvaluatePharma projects that worldwide orphan-drug sales will top $200 billion by 2022.
“Orphan Drug Designation in pancreatic cancer is a very important milestone in the development plan of BL-8040, and joins previously approved orphan designations by the FDA for BL-8040 in AML and stem-cell mobilization,” stated Philip Serlin, Chief Executive Officer of BioLineRx. “Despite advances in the treatment of various cancers with immune checkpoint inhibitors, pancreatic cancer is refractory to these treatment options, and remains an area of significant unmet medical need. We have previously reported encouraging clinical data supporting the potential of BL-8040 as part of an immunotherapy combination treatment in pancreatic cancer, and we look forward to top-line results from our ongoing pancreatic clinical studies later this year.”
BL-8040 is currently being investigated in clinical studies for the treatment of pancreatic cancer under two separate immuno-oncology collaborations – one with Merck & Co., Inc., Kenilworth, N.J., USA (known as MSD outside the United States and Canada), and a second collaboration with Genentech, a member of the Roche Group.
BLRX stock has one of the best ratings by the Street. TipRanks reveals that the stock has a Strong Buy analyst consensus rating with 4 back-to-back ‘buy’ ratings in the last three months. Meanwhile the average analyst price target of $3.00 suggests the stock still has upside potential of nearly 280% from the current share price for the next 12 months. (See BLRX’s price targets and analyst ratings on TipRanks)