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Biotech Stock Roundup: Exelixis Soars on Priority Review, FDA Panel Supports Spark Drug

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With biotech sector earnings season round the corner, there were not too many updates this week. Important news this week include priority review for Exelixis’s (EXEL - Free Report) label expansion plans for Cabometyx and a positive FDA panel outcome for Spark Therapeutics’s Luxturna.

Recap of the Week’s Most Important Stories

Exelixis Up on Priority Review from FDA: Exelixis’s shares shot up 17.2% on news that the FDA has granted priority review to the company’s regulatory application for the label expansion of Cabometyx. The company is looking to get Cabometyx approved for use in treatment-naïve advanced renal cell cancer patients. With the FDA granting priority review, a response should be out on Feb 15, 2018.

Cabometyx is currently approved for the treatment of patients with advanced renal cell carcinoma who have received prior anti-angiogenic therapy. According to the American Cancer Society, kidney cancer is among the top ten most commonly diagnosed forms of cancer among both men and women in the United States.

The five-year survival rate for patients with advanced or late-stage metastatic renal cell carcinoma is only 12%. According to information provided by the company in its press release, about 14,000 patients in the United States need first-line treatment for advanced kidney cancer every year (Read more: FDA Grants Priority Review for Exelixis' RCC Drug Cabometyx).

Cabometyx is being evaluated for other types of cancer as well. In addition to announcing the priority review status, Exelixis presented positive late-stage data on Cabometyx for advanced hepatocellular carcinoma (“HCC”), an aggressive form of advanced liver cancer. The study achieved its primary endpoint of overall survival and the company intends to submit a supplemental New Drug Application (sNDA) in the first quarter of 2018. Cabometyx has orphan drug status in the United States for this indication.

Spark’s Luxturna Gets Full FDA Panel Support: Spark Therapeutics got a boost with the FDA’s advisory panel unanimously supporting the company’s investigational treatment for use in patients with biallelic RPE65-mediated inherited retinal disease.

Timely approval would make Luxturna the first pharmacologic treatment for an inherited retinal disease (“IRD”) as well as the first gene therapy for a genetic disease in the United States.

The gene therapy company should get to know about the approval status of the treatment by Jan 12, 2018. Luxturna, which is under priority review, has orphan drug, breakthrough therapy and rare pediatric disease designations in the United States. The gene therapy is under review in the EU as well.

Regeneron’s Dupixent Tops in Allergic Inflammatory Disease Study: Regeneron Pharmaceuticals (REGN - Free Report) and partner Sanofi announced positive phase II results on Dupixent (dupilumab) in adults with active moderate-to-severe eosinophilic esophagitis, a chronic, allergic inflammatory disease. Patients receiving Dupixent weekly reported a significant improvement in the ability to swallow versus placebo (Read more: Sanofi/Regeneron's Dupixent Succeeds in Phase II Study). The market could be huge for Dupixent if it gains timely approval given the lack of any FDA-approved treatments. Dupixent has orphan drug status in the United States for this indication.

Dupixent, which has blockbuster potential, is currently approved for eczema and is being evaluated for additional indications apart from eosinophilic esophagitis. In fact, last month, the companies had announced positive top-line data from a late-stage study on Dupixent for uncontrolled persistent asthma.  

Regeneron and Sanofi are also collaborating with Aimmune Therapeutics, a clinical-stage company focused on the development of treatments for potentially life-threatening food allergies. Under the clinical collaboration, Aimmune’s AR101 will be evaluated with adjunctive Dupixent in peanut-allergic patients in a mid-stage study that is scheduled to commence next year. The study will be sponsored by Regeneron while Aimmune will provide clinical supply of AR101 and food challenge materials.

Regeneron has gained 20.6% year to date, outperforming the industry’s 12.5% rally.

Ionis to get $10M Milestone Payment from Biogen on Study Initiation: Ionis Pharmaceuticals (IONS - Free Report) is set to receive a $10 million milestone payment from partner Biogen (BIIB - Free Report) related to the initiation of a phase I/IIa study on IONIS-MAPTRx in patients with mild Alzheimer's disease (“AD”). Biogen has an option for the development and commercialization of the antisense drug. The companies already have a partnered drug, Spinraza (spinal muscular atrophy) on the market and are collaborating for other drugs for diseases like amyotrophic lateral sclerosis and other neurodegenerative diseases.

Ionis is a Zacks Rank #1 (Strong Buy) stock. You can see the complete list of today’s Zacks #1 Rank stocks here.

Immune Design Pivotal Study Design gets Positive FDA Feedback: The FDA has given its nod to Immune Design’s phase III clinical study design for CMB305 in patients with synovial sarcoma. The pivotal study, which will evaluate the cancer vaccine as a monotherapy compared to placebo, is scheduled to commence in mid-2018. While progression free survival ("PFS") followed by overall survival ("OS") are the co-primary endpoints, the FDA said that it could support full approval if the PFS endpoint is met (Read more: Immune Design's Sarcoma Candidate to Enter Phase III in 2018).

Performance

Medical - Biomedical and Genetics Industry 5YR % Return

The NASDAQ Biotechnology Index slipped 0.5% over the last five trading sessions. Stocks were hit earlier this week by reports of President Trump criticizing high drug prices again. Among major biotech stocks, Biogen was up 3.5% with a couple of analyst upgrades boosting the stock. Regeneron was down 4% during this period. Over the last six months, Vertex was up 35.1% (See the last biotech stock roundup here: Amgen's Imlygic Data Promising, Regeneron Scores Win in PCSK9 Lawsuit).

What's Next in the Biotech World?

Watch out for third quarter earnings results from companies like Amgen, Biogen and Vertex. Alexion should also get a response from the FDA regarding the label expansion of its flagship product, Soliris (eculizumab), for refractory generalized myasthenia gravis. The agency is expected to give a response by October 23.

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