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ONCE > A Cure for Hemophilia within Reach

http://www.nejm.org/doi/full/10.1056/NEJMe1713888

In a dose-escalation study involving nine patients, six of seven participants who received the highest dose (6×1013 vg per kilogram) had normal levels of factor VIII over a period of 20 to 24 weeks. After 52 weeks, the median level was still 77 IU per deciliter (range, 19 to 164). The seventh patient had levels of 12 to 32 IU per deciliter, which is similar to levels that are associated with mild hemophilia. All seven patients temporarily received glucocorticoids. Although all the patients were positive for antibodies to AAV5 capsid peptides at week 8, no AAV5 cellular immune responses were detected at any time point. Despite the high dose of vector, there was no major liver toxicity. Inhibitors developed in none of the participants. Vector DNA levels in biologic fluids decreased over time. No vector DNA was found in purified sperm cells obtained from two participants.

Lighting a Spark for Gene Therapy
http://www.cetusnews.com/business/Lighting-a-Spark-for-Gene-Therapy.Bkm8nsL_WG.html

Its shares have come under pressure lately, falling over 20% from an October high of $91 as a result of what Yee termed “investor nervousness on Spark” related to its gene-therapy treatment for a retinal condition that leads to blindness. The treatment is expected to get approval from the Food and Drug Administration in about a month. Yee wrote that investor worries dealing with the manufacturing of the Spark retinal treatment and other issues “seem overblown.” The Spark gene therapy for the retinal disease is expected to be the first replacement gene therapy program approved by the FDA. Many more FDA approvals could come in the next few years, including a treatment for spinal muscular atrophy, a fatal condition, being developed by Avexis

Spark will get limited financial benefit from the hemophilia B treatment if it gets regulatory approval because its partnership gives Pfizer the bulk of the profits. (Pfizer is down about 0.5%, at $35.37) And the hemophilia B market is a fraction of the size of the much larger hemophilia A market. Hemophilia A is the much more common form of the disease, which afflicts an estimated 20,000 Americans.

The potential market for a hemophilia treatment is large, with an estimated $7 billion in global drug and other spending estimated for this year, mostly for the replacement factor.

Probably the key gene-therapy treatment being developed by Spark is one for hemophilia A. The company has reported very encouraging preliminary data on three patients and is expected to say more about the program at the coming ASH meeting. That treatment is expected to go into a Phase 3 trial in 2019.

Spark has full rights to its hemophilia A treatment which means that potential financial benefit to success in that program could greatly exceed the profits from its hemophilia B treatment. The hemophilia A market is competitive with BioMarin Pharmaceutical also developing a treatment.

One analyst estimates that Pfizer owns an estimate 85% of the hemophilia B economics, but adds that the program validates Spark’s approaches to adenoid associate virus gene therapy. And the hemophilia A business is wholly owned and represents a market that is four times larger than the one for hemophilia B.