Key highlights in the biotech sector over the past week include earnings results from companies like Regeneron Pharmaceuticals, Inc. REGN and the lifting of the clinical hold imposed by the FDA on a couple of studies being conducted on Cellectis’s CLLS UCART123.
Recap of the Week’s Most Important Stories
Solid Q3 Results from Regeneron: Regeneron’s third quarter results were strong with the company surpassing both earnings and revenue expectations. Flagship product, Eylea, continues to perform well. On the third quarter call, Regeneron said that the diabetic macular edema (“DME”) indication represents significant growth opportunity considering only 10% of patients with DME in the United States are currently treated with an anti-VEGF agent. Regeneron is looking to expand Eylea’s label into diabetic retinopathy and expects to file for regulatory approval in the second half of 2018 if results from a late-stage study are positive. A supplementary biologics license application (“BLA”) for every 12-week dosing of Eylea for wet age-related macular degeneration (“AMD”) is set to be filed by year end.
The Dupixent launch for atopic dermatitis is ongoing while Regeneron and its partner Sanofi expect to file for approval for the asthma indication by year end. Meanwhile, data from the ODYSSEY OUTCOMES study on PCSK9 inhibitor, Praluent, should be out in the first quarter of 2018. A regulatory application for PD-1 inhibitor, cemiplimab, is also slated for the first quarter of 2018 (Read more: Regeneron Beats on Q3 Earnings & Sales, Shares Up).
Regeneron has gained 13.1% year to date, outperforming the industry’s 3% rally.
Dicerna-Boehringer Ingelheim to Work Together for NASH Treatment: Dicerna Pharmaceuticals DRNA, which is focused on the development of investigational RNA interference (RNAi) therapeutics, has signed up with Boehringer Ingelheim for the discovery and development of novel RNAi therapeutics based on Dicerna’s proprietary technology platform, GalXC, for the treatment of chronic liver diseases. The initial focus will be on nonalcoholic steatohepatitis (“NASH”), a chronic liver disease with no treatment options currently approved though several companies are conducting research in this area.
The agreement could see Dicerna receive more than $200 million from Boehringer, including an upfront payment, development and commercial milestone payments, and R&D reimbursement for a GalXC candidate addressing an undisclosed NASH target. Dicerna could also receive staggered royalties (up to double-digits) on worldwide net sales. Dicerna’s shares jumped 28.2% following the announcement of the deal.
Keryx Down Despite Auryxia Label Expansion: Keryx Biopharmaceuticals, Inc. KERX, which is focused on developing medicines for renal disease, gained FDA approval for the label expansion of Auryxia. Auryxia can now be used for the treatment of patients with iron deficiency anemia and non-dialysis dependent chronic kidney disease (“CKD”). However, while approval for this indication, which significantly expands the patient population for Auryxia, is a positive, the company’s shares were down on disappointing third quarter results (Read more: Keryx Q3 Loss Wider Than Expected, Stock Declines).
FDA Lifts Clinical Hold on Cellectis Studies: The FDA lifted the clinical hold, previously announced on Sep 4, 2017, on a couple of early-stage studies being conducted on Cellectis’s UCART123 for acute myeloid leukemia (“AML”) and blastic plasmacytoid dendritic cell neoplasm (“BPDCN”). The hold was placed after the company had reported a death in the BPDCN study.
Cellectis has agreed to several changes in the study protocols for the hold to be lifted. These include lowering the cohort dose level as well as the cyclophosphamide dose of the lympho-depleting regimen, including specific criteria on the day of UCART123 infusion, ensuring that the next three patients who will be treated in each protocol are under the age of 65 and ensuring that enrollment will be staggered across the UCART123 protocols AML123 and ABC123 with at least 28 days elapsing between the enrollments of two patients across the two studies.
Although the lifting of the clinical hold comes with several restrictions, it is a relief for the company, which is focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART).
Cellectis is a Zacks Rank #2 (Buy) stock. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 0.2% over the last five trading sessions. Among major biotech stocks, Regeneron was up 4.6% reflecting strong third quarter results while Gilead GILD was down 2.3%. Over the last six months, Vertex VRTX was up 28.1% while Celgene lost 14.1% (See the last biotech stock roundup here: Celgene, Amgen, Gilead Hit by Q3 Results, Vertex Beats on All Fronts).
What's Next in the Biotech World?
Several small- and mid-cap companies will be reporting results in the next few days. Moreover, companies like Dynavax Technologies Corporation DVAX and Heron Therapeutics, Inc. should get a response from the FDA for regulatory applications filed by them. It could be third time lucky for Dynavax which is awaiting approval for its hepatitis B vaccine, Heplisav-B, with the FDA expected to respond today. Heron is awaiting a decision from the FDA for Cinvanti, which is under review for the prevention of chemotherapy-induced nausea and vomiting (“CINV”). The FDA is expected to give its decision on November 12.
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