Being first to market with a new type of drug brings advantages. The first mover sets the bar for what physicians, payers, and patients can expect of that medicine and how much it costs. It grabs market share that followers have to steal away. But the first mover isn’t infallible if someone else has something better. That’s something the The Medicines Co. is counting on.
If MedCo’s cholesterol-lowering drug inclisiran wins FDA approval, it will become the third entry in a new class of medicines that block a cholesterol-boosting protein called PCSK9. In presenting clinical data from a Phase 3 study at a major medical meeting this week, it bolstered its case that being a late-comer may not matter—if it can win over payers in ways that the developers of the other PCSK9 medicines haven’t.
The two approved PCSK9 inhibitors have struggled commercially, in part because insurers balked at their steep prices. MedCo will try to prove that its case is different. Inclisiran is administered twice a year, not once or twice a month like its rivals. Because inclisiran is a lower-cost product to make than other PCSK9 drugs, MedCo can be “flexible” with its pricing and “reduce the price relatively quickly” as more patients get access, CEO Mark Timney told Xconomy this week. MedCo says it’s already talking to payers. In the next year or so, we’ll see whether they embrace inclisiran or MedCo has the same issues getting its drug to patients.
In other news this week we saw a biotech bankruptcy, a couple of IPOs, and some big deals. Let’s get to those stories and more in this week’s news roundup.
THE HEART OF THE MATTER
—The Medicines Co. (NASDAQ: [[ticker:MDCO]]) presented details from the first Phase 3 study of its cholesterol lowering injection inclisiran, supporting expectations that the medicine is on par with other next-generation cholesterol fighting drugs. It’s still unclear if inclisiran will fare better with payers; CEO Mark Timney told Xconomy that MedCo “learned a lot” from watching its PCSK9 rivals struggle and is open to flexible pricing.
—The European Society of Cardiology updated its treatment guidelines to include Amarin’s prescription fish-oil pill Vascepa. But Amarin (NASDAQ: [[ticker:AMRN]]) still faces questions in the US, where regulators will decide later this year whether to expand the drug’s label to include claims that it can reduce risk of heart attacks and strokes.
—Novartis (NYSE: [[ticker:NVS]]) touted positive signs from the Phase 3 PARAGON-HF study of heart failure drug Entresto, which missed its main goal last month. The data were also published in the New England Journal of Medicine.
—Vantage rounded up more headlines from the big cardio meeting here.
DATA DUMPS
—Concert Pharmaceuticals (NASDAQ: [[ticker:CNCE]]) disclosed Phase 2 results for autoimmune disease alopecia areata and plans to move into late-stage testing next year, as it races Pfizer (NYSE: [[ticker:PFE]]) for the first FDA-approved medicine for the condition. Concert and Pfizer’s drugs are JAK inhibitors, which are under regulatory scrutiny due to side effects.
—Aridis Pharamceuticals (NASDAQ: [[ticker:ARDS]]) scrapped an experimental drug for ventilator-assisted pneumonia. In a failed Phase 2 study, more patients on the drug died than those on placebo.
—Nivolumab (Opdivo) from Bristol-Myers Squibb (NYSE: [[ticker:BMY]]) failed another big test to treat the brain cancer glioblastoma. The severity of the cancer and lack of treatments has prompted one woman to spend her fortune on an unusual drug-discovery team, as Xconomy reported earlier this year.
RED LIGHT, GREEN LIGHT, YIELD
—Microbiome test kit maker uBiome declared bankruptcy. It is under federal investigation for its business practices, and there have been media reports of shoddy science. The firm’s temporary CEO said in a court filing that the firm might have misled investors in the process of raising roughly $100 million in private funds.
—Boston’s Zafgen (NASDAQ: [[ticker:ZFGN]]) has begun looking for a buyer or other deals. Its metabolic disease programs have hit many roadblocks over the years, including a hold on an experimental diabetes drug.
—Abeona Therapeutics (NASDAQ: [[ticker:ABEO]]) is also looking for someone to buy the company or its assets. New York-based Abeona is developing cell and gene therapies for rare diseases.
—European regulators approved a combo regimen of the Merck (NYSE: [[ticker:MRK]]) immunotherapy pembrolizumab (Keytruda) and the Pfizer (NYSE: [[ticker:PFE]]) drug axitinib (Inlyta) for newly diagnosed kidney cancer patients.
—Global Blood Therapeutics (NASDAQ: [[ticker:GBT]]) said the FDA has agreed to review its sickle cell treatment voxelotor, a once-a-day pill, and make an approval decision by late February, even though the firm had to drop part of its Phase 3 study that aimed to measure patients’ reports of pain and other outcomes.
—Roche extended the deadline until Oct. 1 for Spark Therapeutics (NASDAQ: [[ticker:ONCE]]) shareholders to agree to Roche’s $4.8 billion buyout bid. The deal has already been delayed by regulatory scrutiny.
—Bay Area startup creator BridgeBio (NASDAQ:BBIO]]) unveiled its latest biotech subsidiary: ML Bio Solutions, a Charlotte, NC, company developing a small molecule drug to treat the rare disease
