It was a relatively slow week for the biotech sector though key pipeline updates were provided by companies like Cellectis CLLS and Insmed INSM . While Insmed skyrocketed on positive data on its lead pipeline candidate, Cellectis was hit by a clinical hold on a couple of phase I studies.
Recap of the Week's Most Important Stories
Cellectis Hit by FDA Clinical Hold: Cellectis' shares were hit by a clinical hold placed by the FDA on a couple of early-stage studies being conducted on UCART123. While one study is for acute myeloid leukemia ("AML"), the other is for blastic plasmacytoid dendritic cell neoplasm (BPDCN). The hold came into place after the company reported a death in the BPDCN study.
Cellectis, which is focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), said that it is working with the FDA and investigators for the resumption of the studies with an amended protocol including a lower dosage. Although shares were down 20.3% on the clinical hold, Cellectis has gained 51.4% year to date, significantly outperforming the industry's 15.9% rally.
Insmed Shoots Up on ALIS Data: Insmed's shares shot up 119.6% on positive top-line data from a late-stage study (CONVERT) for treatment-refractory nontuberculous mycobacterial ("NTM") lung disease caused by mycobacterium avium complex ("MAC"). Results showed that the addition of ALIS (amikacin liposome inhalation suspension) to guideline-based therapy ("GBT") eliminated evidence of NTM lung disease caused by MAC in sputum by the sixth month in 29% of patients, compared to 9% of patients on GBT alone.
Based on this data, Insmed intends to seek accelerated approval and priority review of ALIS under subpart H. ALIS has breakthrough therapy designation and fast track status in the United States and is also a qualified infectious disease product ("QIDP") under the Generating Antibiotic Incentives Now ("GAIN") Act, which will provide it with an additional five years of exclusivity.
ALIS is the lead candidate in Insmed's pipeline. According to the company, about 75,000 - 105,000 patients will be diagnosed with NTM lung disease in 2018, of which 40,000 - 50,000 will be treated for NTM lung disease caused by MAC.
Akcea/Ionis Submit FCS Drug for FDA Approval: Akcea Therapeutics AKCA and Ionis Pharmaceuticals IONS announced the submission of a New Drug Application ("NDA") for volanesorsen for the treatment of familial chylomicronemia syndrome ("FCS"), a severe, rare disorder characterized by extremely high levels of triglycerides. The company is looking to launch volanesorsen globally in 2018 provided it gains timely approval. While the efficacy data on volanesorsen is promising, concerns remain about the safety profile of the candidate given injection site reactions and the fall in platelet count.
Volanesorsen is currently being evaluated in a late-stage study for familial partial lipodystrophy ("FPL") with topline data expected in 2019.
Ligand Diabetes Drug Fares Well in Mid-Stage Study: Ligand Pharmaceuticals LGND announced positive top-line results from a mid-stage study on its investigational type II diabetes treatment, LGD-6972, an oral, small molecule, glucagon receptor antagonist ("GRA"). In addition to showing robust, statistically significant, dose-dependent reductions from baseline in hemoglobin A1c after 12 weeks of treatment, LGD-6972 was found to be safe and well tolerated. Although there are several diabetes treatments available in the market, there remains significant need for additional treatments considering diabetes is a growing global epidemic affecting millions across the world. Ligand is a Zacks Rank #1 (Strong Buy) stock. You can see the complete list of today's Zacks #1 Rank stocks here .
Amgen's Mimpara EU Label Expanded: Amgen AMGN gained approval in the EU for a pediatric formulation of Mimpara for the treatment of secondary hyperparathyroidism ("HPT") in children aged three years and above with end stage renal disease ("ESRD") on maintenance dialysis therapy. Approval was largely expected as the Committee for Medicinal Products for Human Use ("CHMP") of the European Medicines Agency ("EMA") had adopted a positive opinion for the pediatric formulation earlier this year in June (Also read: Amgen Gets EU Nod to Expand Hyperparathyroidism Drug Label ).
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 2.8% over the last three trading sessions. Among major biotech stocks, Alexion was up 4.5%. Over the last six months, Vertex VRTX was up 76.4% while Amgen slipped 0.6% (See the last biotech stock roundup here: CAR-T in Focus on Gilead-Kite Deal, Incyte Up on Baricitinib Update ).
What's Next in the Biotech World?
Watch out for the usual regulatory and pipeline updates.
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