Pipeline and regulatory updates were the focus this week with Ultragenyx Pharmaceutical (RARE - Free Report) gaining FDA approval for its first drug while companies like Cytokinetics (CYTK - Free Report) and Acorda Therapeutics (ACOR - Free Report) were hit by pipeline setbacks.
Recap of the Week’s Most Important Stories
Ultragenyx Gets FDA Approval for Rare Genetic Disease Drug: Ultragenyx gained FDA approval for its first drug, Mepsevii (vestronidase alfa), for the treatment of children and adults with mucopolysaccharidosis VII (MPS 7), also known as Sly syndrome. Mepsevii is also the first medicine to be approved for this rare genetic disease, which is estimated to affect about 200 patients in the developed world. Mepsevii will be launched later this month and the FDA has asked for a post-marketing study to evaluate the long-term safety of the product. With MPS 7 being an extraordinarily rare disease, sales should build up gradually as patients are identified and placed on therapy.
Meanwhile, Mepsevii is currently under review in the EU where an opinion from the Committee for Medicinal Products for Human Use (“CHMP”) should be available in the first half of 2018.
In addition to gaining FDA approval for Mepsevii, Ultragenyx also got a Rare Pediatric Disease Priority Review Voucher, which can be used to gain priority review for any subsequent drug application that is not eligible for priority review. The company can also sell the voucher for millions of dollars to any other company that wants to fasten the review process for its candidate. BioMarin, United Therapeutics and Knight Therapeutics are some of the companies that sold their priority review vouchers for millions of dollars (Read more: Ultragenyx's rhGUS Gets FDA Nod for Rare Genetic Disease).
Cytokinetics Down on Phase III Setback: Cytokinetics’s shares plunged 25.7% on negative data from the company’s late-stage study on tirasemtiv in patients with amyotrophic lateral sclerosis (“ALS”). The company said that the study failed to meet the primary endpoint as well as the secondary endpoints. Cytokinetics has decided to suspend the development of tirasemtiv with the focus now shifting to Cytokinetics’s next-generation fast skeletal muscle activator, CK-2127107, which the company believes will be better tolerated and potentially more effective than tirasemtiv. Phase II results on CK-2127107 are expected in 2018. CK-2127107 is also being evaluated for spinal muscular atrophy and chronic obstructive pulmonary disease.
ALS, a progressive neurodegenerative disease, afflicts about 30,000 people in the United States with approximately 6,000 new cases being diagnosed every year in the country. There is high unmet need for new therapies with the average life expectancy of an ALS patient being about three to five years after diagnosis while only 10% of patients survive for more than 10 years. Cytokinetics had previously said that tirasemtiv could generate sales of more than $600 million - $1.1 billion in the United States alone.
Cytokinetics’s shares are down 32.1% year to date, compared to the 0.6% gain recorded by the industry it belongs to.
Acorda Scraps Tozadenant Program: Just a few days after announcing safety issues associated with a late-stage study on its investigational treatment for Parkinson’s disease, Acorda said that it is discontinuing its clinical development program for tozadenant (Read more: Acorda Stock Up Despite Phase III Trial on Tozadenant Stops). The company said that it will immediately discontinue dosing of all participants currently enrolled in its tozadenant studies. The decision to discontinue development was based on new information obtained from the phase III program related to previously disclosed agranulocytosis (the absence of white blood cells, which fight infection) and associated serious adverse events. The company said that it could not be confident that weekly white blood cell count screening would be enough to ensure patient safety.
Last week, Acorda had announced that it had identified cases of agranulocytosis, possibly drug-related, in some cases associated with sepsis and death. At that time, the company had said that there would be a higher frequency of blood cell count monitoring in the phase III program for tozadenant (Read more: Acorda Reports Death in Parkinson's Trials, Stock Down 40%).
Tozadenant, an oral adenosine A2a receptor antagonist, became a part of Acorda’s pipeline following its 2016 acquisition of Biotie (for approximately $376 million). Last year, Acorda had said that three of its pipeline candidates – Inbrija (CVT-301) and tozadenant for Parkinson’s and Plumiaz in epilepsy - represent potential U.S. peak net sales of more than $1 billion. However, in addition to the tozadenant setback, Acorda ran into trouble with Plumiaz and discontinued that program in 2016.
It has also not been smooth sailing for Inbrija. The company had filed a new drug application (“NDA”) for Inbrija in June 2017 but the FDA issued a Refusal to File (“RTF”) letter. Acorda intends to resubmit the NDA by year end.
These pipeline setbacks will weigh on Acorda, which could start facing generic competition for its flagship drug, Ampyra, from August 2018.
Six Months Additional Exclusivity for UTHR’s Adcirca: United Therapeutics (UTHR - Free Report) announced that it has gained pediatric exclusivity in the United States for its pulmonary arterial hypertension (“PAH”) treatment, Adcirca. This means the drug will now enjoy exclusivity through May 21, 2018 before the FDA can grant approval to a generic version (Read more: United Therapeutics PAH Drug's Exclusivity Period Extended).
Alnylam Starts Rolling NDA for Patisiran: Alnylam has started submitting a rolling NDA in the United States for patisiran, an investigational RNAi therapeutic targeting transthyretin (“TTR”) for the treatment of hereditary ATTR (hATTR) amyloidosis. The company expects to finish the submission by year end. Alnylam is seeking priority review for the NDA – if granted, the review process would be cut down to six months. Meanwhile, a regulatory application in the EU will also be submitted around the end of this year. Alnylam has a collaboration agreement with Sanofi for patisiran – once approved, the drug will be marketed by Alnylam in the United States, Canada and Western Europe, while Sanofi will sell the product in the rest of the world (Read more: Alnylam Starts Rolling NDA Submission for RNAi Candidate).
TESARO Ovarian Cancer Drug Gets EU Nod: TESARO’s (TSRO - Free Report) ovarian cancer drug, Zejula, gained approval from the European Commission (“EC”) for use as a monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete response (“CR”) or partial response (“PR”) to platinum-based chemotherapy. This makes Zejula the first PARP inhibitor to be approved in Europe regardless of BRCA mutation or biomarker status. The treatment will initially be launched in Germany and the UK this December. EU approval was largely expected considering the CHMP had issued a positive opinion about Zejula in September. Zejula is already available in the United States where it gained approval earlier this year in March (Read more: TESARO Ovarian Cancer Drug Zejula Gets Approval in EU).
TESARO is a Zacks Rank #3 (Hold) stock. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 1.8% over the last five trading sessions. Among major biotech stocks, Celgene (CELG - Free Report) was up 5.7% while Vertex declined 1.8%. Over the last six months, Biogen (BIIB - Free Report) was up 23.6% while Regeneron lost 14.6% (See the last biotech stock roundup here: Dynavax Vaccine Gets FDA Nod, Loxo Inks Deal with Bayer).
What's Next in the Biotech World?
Watch out for the usual pipeline and regulatory updates.
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