Bio Roundup: Amarin’s Stunner, Lung Cancer News, Data Dumps & More

Xconomy National — 

Six years ago, a biotech called Amarin won FDA approval of a prescription fish-oil pill, Vascepa, because it could lower triglycerides, a type of fat in the blood. But Amarin didn’t have the evidence that lowering triglycerides with fish oil would really help people. Sales lagged. Amarin’s shares sank. While other groups tested other fish oils and found no evidence of concrete cardiovascular benefits, Amarin pressed on with a massive study, bleeding cash for years.

The strategy seems to have paid off. Amarin said this week that the REDUCE-IT study showed Vascepa lowered the risk of heart disease, marking a huge surprise victory for the company and a new turn in a long-running scientific debate.

Elsewhere, there was plenty of news out of a big lung cancer meeting in Toronto, a bushel of biotechs went public, and the second-ever RNA interference drug could soon be headed for an FDA review. Let’s get the roundup rolling.


—For the first time, a pill containing fish oil showed in a clinical trial that it might help reduce the chance of heart disease in people already taking statins to control high cholesterol. The pill is Vascepa, from Amarin (NASDAQ: AMRN), and the 8,379-patient trial called REDUCE-IT concluded that patients were 25 percent less likely, relative to those on statins and a placebo, to suffer a cardiovascular event. Crucial full details will be released in November, but investors aren’t waiting. Amarin’s shares have skyrocketed more than 360 percent this week.

—Gilead Sciences (NASDAQ: GILD) announced it would begin making generic versions of its own hepatitis C drugs that cost up to 75 percent less. The branded versions have years of patent life remaining but are facing legal and financial challenges.


—AstraZeneca (NYSE: AZN) released positive Phase 3 lung cancer data for its drug durvalumab (Infimzi), which when combined with chemotherapy bested chemo alone in patients with advanced cancer.

—Roche detailed the results of a study, IMpower132, that tested a combination of its immunotherapy atezolizumab (Tecentriq) and chemotherapy in newly diagnosed advanced non-small cell lung cancer patients. Though cross-trial comparisons come with caveats, the overall results didn’t appear to match an immunotherapy-chemo combo from Merck (NYSE: MRK). Still, Roche’s regimen notably showed promise in peoples whose tumors don’t express the protein PDL1, EP Vantage reports.

—Roche is trailing rivals in non-small cell lung cancer, but in the less common, more aggressive form—small-cell lung cancer—its drug combination is ahead. Roche detailed the results of IMpower133, through which an atezolizumab-chemo regimen extended the lives of people with SCLC by two months, compared to chemo alone. Roche’s rivals aren’t far behind with their own combos, however, as Reuters reports here. (And Bristol-Myers Squibb’s nivolumab (Opdivo) was cleared for SCLC patients last month.)

—Roche also said it will file for approval of entrectinib for patients whose non-small cell lung cancer has a specific genetic alteration, a ROS1 fusion. Roche is also testing entrectinib in a variety of tumors with so-called NTRK fusions and is aiming for a tissue-agnostic drug approval—that is, clearance to treat tumors with NTRK fusions regardless of where in the body they form.

—A combination of the Blueprint Medicines (NASDAQ: BPMC) targeted cancer drug BLU-667 and osimertinib (Tagrisso), from AstraZeneca, showed promise in two patients with lung cancer and an EGFR mutation whose tumors resisted prior treatment.


—Six life science companies closed out the final week of the third quarter with IPOs, raising a total of $586 million in new capital. Among the newcomers to Wall Street: Gritstone Oncology (NASDAQ: GRTS), a developer of personalized cancer vaccines; Arvinas (NASDAQ: ARVN), which is advancing protein degradation drugs; and Sutro Biopharma (NASDAQ: STRO, a developer of protein-based drugs with a long-running alliance with Celgene (NASDAQ: CELG).

—Equillium of La Jolla, CA, filed for an IPO to finance clinical testing of a drug to treat graft-versus-host disease.

—LogicBio Therapeutics, a gene therapy developer, filed for an IPO to fund clinical tests of its experimental treatment for a rare inherited liver disease.


—KSQ Therapeutics, a Cambridge, MA, startup using the CRISPR gene editing system as a drug discovery tool, raised another $80 million and revealed a pipeline of 13 experimental cancer drugs.

—Morphic Therapeutic, a startup from Boston-area scientific entrepreneur Tim Springer, raised an $80 million Series B to bring its first oral, integrin-blocking drugs to human tests.

—Novartis (NYSE: NVS) is laying off 2,550 people in Switzerland and the U.K., part of a shift among big drug makers from high-volume pills to more expensive “specialty” drugs, like biologics. Swiss unions are pushing back. Reuters has more.

—The FDA approved the latest in a new crop of drugs meant to reduce the frequency of migraines, galcanezumab (Emgality) from Eli Lilly (NYSE: LLY). The drug has a list price of $575 per month, or $6,900 per patient, per year.

—The FDA cleared Sarepta Therapeutics (NASDAQ: SRPT) to resume testing its experimental gene therapy for Duchenne muscular dystrophy, which has shown promise in an early study. The FDA temporarily halted the study in July due to a manufacturing issue.

—The FDA approved duvelisib (Copiktra), from Verastem (NASDAQ: VSTM), for people with chronic lymphocytic leukemia, small lymphocytic leukemia, or follicular lymphoma who have failed at least two treatments. Duvelisib, which Verastem bought from Infinity Pharmaceuticals in 2016, is now the company’s first marketed product. It has a monthly list price of $11,800, which comes to $141,600 per patient, per year.

—The agency also cleared dacomitinib (Vizimpro), from Pfizer (NASDAQ: PFE), for advanced lung cancer patients whose tumors have one of two specific genetic alterations.

—Merck ended a policy mandating that its CEO retire at age 65, and announced current top executive Ken Frazier has agreed to stay aboard until at least December 2019, when he turns 65.

—Epizyme (NASDAQ: EPZM) said that the FDA has allowed the resumption of trials involving its top experimental drug tazemetostat. The green light comes five months after a new case of cancer in a patient prompted a partial hold on all tazemetostat trials. Epizyme enjoyed a brief stock bump, but shares remain well below their 52-week high.

—Lyra Therapeutics, a maker of ear, nose, and throat medicines, has raised $29.5 million to fund Phase 2 testing of its lead drug.

—Janssen Biotech terminated a four-year alliance with Geron (NASDAQ: GERN) and returned to the biotech full rights to the experimental blood disorder drug imetelstat. Geron’s stock price plummeted 68 percent on the news.


 —Alnylam Pharmaceuticals (NASDAQ: ALNY) could file for accelerated approval of its second RNA interference medicine, givosiran, by the end of the year. Interim results from a Phase 3 study showed the drug had an impact on people with acute hepatic porphyria, a potentially deadly genetic disease.

—Alexion Pharmaceuticals (NASDAQ: ALXN) is preparing to file for approval of its drug eculizumab (Soliris) after positive Phase 3 data in neuromyelitis optica spectrum disorder, a rare disease affecting the central nervous system. The blockbuster drug is already approved to treat two rare blood disorders.

—TG Therapeutics (NASDAQ: TGTX) delayed the release of Phase 3 data for a combination of drugs for the chronic lymphocytic leukemia and changed the goals of the study. TG did not say that the study failed. Instead, it blamed the delay on not having enough long-term information about the patients who responded to the drug. Investors weren’t buying it, sending shares down more than 40 percent this week. Here’s more from STAT.

—Shares of Lexington, MA-based Aldeyra Therapeutics (NASDAQ: ALDX) surged 35 percent after the company reported encouraging results from a mid-stage study of an anti-inflammatory drug, reproxalap, in dry eye disease. A Phase 3 study should begin next year.

 —The New England Journal of Medicine published interim results from a 3,573-patient Phase 2 study of M72/AS01e, an experimental vaccine from GlaxoSmithKline (NYSE: GSK) for tuberculosis. The vaccine may be able to reduce the incidence of tuberculosis in adults who don’t have HIV infections, but the results will have to be confirmed with further testing.


—Alexion paid $400 million in cash up front to acquire Syntimmune, a New York and Waltham, MA, startup developing a drug for rare autoimmune diseases.

—Women’s health company Hologic (NASDAQ: HOLX) bought Focal Therapeutics for $125 million cash. Privately held Focal has commercialized a marker used in breast-conserving surgery.

—Roche bought Tusk Therapeutics, a U.K.-based developer of cancer immunotherapy drugs focused on immune cells known as regulatory T cells, for about $81 million up front.

—Eli Lilly (NYSE: LLY) paid Chugai $50 million up front for an oral diabetes drug on the verge of its first human tests. The drug gives the Indianapolis pharmaceutical giant a new asset to challenge Novo Nordisk (NYSE: NVO) in the race to bring a pill version of the injectable GLP-1 diabetes drugs that help control patients’ blood sugar.

—Cerecor (NASDAQ: CERC) is paying approximately $27 million in stock to buy Ichorion Therapeutics, a developer of drugs for rare metabolic diseases.


—Don’t miss “Boston’s Life Science Disruptors” on Oct. 17 at the Koch Institute for Integrative Cancer Research. This year we’ll be featuring behind-the-scenes looks at startups aiming to target RNA with drugs, take CRISPR gene editing to the next level, and study the genetic activity of single cells. You can register here.

Frank Vinluan and Alex Lash contributed to this report.