- Shire enters the home stretch with its newer formulation of asparaginase.
- Acceleron publishes early-stage results in muscular dystrophies.
- Antisense Therapeutics gets the go-ahead on trials in Duchenne muscular dystrophy.
Note: Subscribers to Avisol Capital Partners Total Pharma Tracker got an early look at this publication.
Welcome to another edition of "3 Things In Biotech" a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.
Shire enters the home stretch with its newer formulation of asparaginase
Company: Shire Group (SHPG)
Therapy: Calaspargase pegol
Disease: Acute lymphoblastic leukemia
News: SHPG announced that the FDA has accepted the application for approval for its newer formulation of asparaginase called calaspargase pegol. Pegylated asaparginase forms an important piece of the puzzle for the treatment of acute leukemia, particularly in children. The FDA has set an action date for review of this application on December 22.
Edit: This article originally stated that the calaspargase pegol was approved in Europe. This was an oversight and has been corrected.
Looking forward: In the realm of therapy for this form of leukemia, asparaginase isn't exactly the sexiest ticket today. That would be the CAR-T cells or other immunotherapeutic strategies. However, asparaginases form an important cornerstone for the treatment of acute leukemia, and its use is being better recognized in the United States for non-pediatric patients. So a novel formulation of the drug that helps to circumvent issues relating to dosing and resistance. Oncaspar nets some $100 million in sales in Europe, so it is likely to give a big boost to SHPG if approved in the US, as well.
Overall, this is a favorable sign for SHPG, but it is just one piece of a big puzzle for it.
Acceleron publishes early-stage results in muscular dystrophies
Company: Acceleron Pharmaceuticals (XLRN)
Disease: Muscular dystrophies
News: XLRN announced the publication of the final results from a phase 1 study in the journal Muscle and Nerve. These results describe the use of its myostatin-boosting agent ACE-083 in healthy volunteers, demonstrating muscle increases ranging from 8.9% to 14.5%, with no serious adverse events or discontinuations due to toxicity. Meanwhile, patients in the placebo arm experienced very little muscular growth in the treatment period.
Looking forward: While very early days for ACE-083, these findings demonstrate encouraging tolerability and a signal of muscle-boosting activity. Later-stage clinical trials are now underway to investigate ACE-083 in patients with either facioscapulohumeral dystrophy or Charcot-Marie-Tooth disease, two forms of muscle wasting with no approved therapies.
This alone wouldn't make me buy into the XLRN thesis, but it does signal a favorable path for ACE-083, as far as we can tell from such early trial data.
Antisense Therapeutics gets the go-ahead on trials in Duchenne muscular dystrophy
Company: Antisense Therapeutics (OTCPK:ATHJF)
Disease: Duchenne muscular dystrophy
News: ATHJF announced that its immunomodulatory therapy for Duchenne muscular dystrophy has been granted approval to move forward with a phase 2 clinical study in Australia. This trial will test a single dose of ATL1102 in wheelchair-bound boys between the ages of 10 and 18, with a primary goal of establishing safety and tolerability. The company hopes that the six-month treatment period will also give it sufficient time to assess potential efficacy changes in the patients.
Looking forward: Although effective treatment options are beginning to emerge for Duchenne muscular dystrophy (most notably Sarepta's (SRPT) eteplirsen), it should be noted that many patients will not benefit at all from new therapies, and the unmet need remains strong here. Therefore, it is encouraging to see new treatment options enter the clinic. An approved product here would also benefit Ionis Pharmaceuticals (IONS), which outlicensed the technology to ATHJF.
Overall, it is too early to buy on this one, but we may have primary results of the study by the end of the year, at which time it will be important to revisit this company.
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