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Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.
Celgene shows some OPTIMISMM in myeloma
Company: Celgene (CELG)
Disease: Relapsed/refractory multiple myeloma
News: CELG announced top-line data from its phase 3 OPTIMISMM study, which assessed the addition of its immunomodulatory drug pomalidomide to bortezomib and dexamethasone therapy for patients with relapsed/refractory multiple myeloma. The company advised that the primary endpoint, progression-free survival, was met for the pomalidomide arm, and safety data were consistent with previous reports.
Looking forward: This was the first study to assess the potential benefit of a "triplet" regimen in patients who had previously progressed on CELG's blockbuster titan lenalidomide (branded Revlimid). At this point, it is not known how strong the PFS data are, but this announcement definitely suggests that CELG will be able to make a move toward extending the reach of pomalidomide, which would ensure continued strong market share in the treatment of myeloma.
Overall, this is great news that bodes well for CELG; expect a supplemental filing sometime in 2018.
Glaxo lands a breakthrough in meningitis vaccination
Company: GlaxoSmithKline (GSK)
Therapy: Bexsero, a recombinant DNA vaccine
Disease: Meningitis B
News: GSK announced that the FDA has granted Breakthrough Therapy designation for Bexsero in the prevention of meningococcal group B infection in patients aged 2-10 years. The designation suggests an imminent extension of Bexsero's use out of the patient age of 10-25 years range, assuming it files for and receives accelerated approval as it did in 2015.
Looking forward: The fact that this drug is already approved in adolescents and young adults, in addition to the signal from the FDA, suggests that GSK is not going to have much trouble getting this vaccine approved in the younger patients, as well. This should help to ensure the growth of a franchise that is already projected to reach over $1 billion in sales per year.
In all, this bit of news probably won't impact GSK much in the near- to mid-term, but it does bear a lot of the weight for continued growth in the coming years, if approved in this setting.
Abeona demonstrates promise in rare pediatric disorder
Company: Abeona Therapeutics (ABEO)
Disease: Sanfilippo syndrome B
News: ABEO announced preliminary findings from the phase 1/2 study involving its gene therapy ABO-101 for the treatment of Sanfilippo syndrome B. The first patient dosed with the therapy appears to have favorable distribution of the therapy, and glycosaminoglycan levels were reduced in several compartments, including the CSF, blood plasma, and urine. These findings provide an early signal that adeno-associated, virus-based therapy can provide therapeutic benefit in Sanfilippo syndrome B.
Looking forward: We covered this drug in a previous edition of 3 Things, and it led to a bit of interesting conversation. Shareholders care about Sanfilippo syndromes more than their incidence might suggest. With this news, ABEO is striking out early, although it is indeed very preliminary. I will eagerly await more news on this before making a strong recommendation one way or the other.
However, if you're the risk-taking type, this announcement could be the first sign that ABEO is on to something big for treating this serious, albeit rare, condition.
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