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Key regulatory updates biotech specialist trust investees

Key regulatory updates biotech specialist trust investees

Key regulatory updates biotech specialist trust investees

Two stocks that core holdings for biotech specialist trusts have reported important regulatory updates in the past week, with several other companies approaching key events, as detailed below.

Array BioPharma (Nasdaq: ARRY), a top ten holding for International Biotechnology Trust (IBT), received FDA approval for Braftovi in combination with Mektovi for the treatment of unresectable or metastatic melanoma with a BRAF mutation. The doublet will be the third such combination to reach the market after drug pairings from Novartis (Tafinlar + Mekinist)  and Roche’s (Zelboraf + Cotellic).  Array intends to market its drug pairing in the US against these giants, but has a potential advantage in terms of efficacy and side effect profile.

Puma Biotechnology (Nasdaq: PBYI), an investee for Worldwide Healthcare Trust (WWH), Biotech Growth Trust (BIOG) and IBT, received a positive opinion for its breast cancer drug Nerlynx (neratinib) from the European Medicines Agency, after an appeal against an earlier decision to reject the application. The opinion covers Nerlynx for extended adjuvant  treatment of early stage HER2-positive, hormone receptor positive breast cancer. This is a slight difference from the approved patient population in the US, where HR status is not specified. Extended adjuvant treatment is one that follows after one year of adjuvant (post surgical) therapy with a Herceptin-based regime.

Alnylam (Nasdaq: ALNY), a top 10 holding for Biotech Growth (3.6% of NAV) that is also held by BB Biotech and is the largest single holding for Edinburgh Worldwide Investment Trust (EWI, 5.8% of NAV), should see the outcome of the FDA’s review of patisiran for hereditary transthyretin-mediated amyloidosis (or hATTR) by the target date of 11 August.

Ionis Pharmaceuticals (Nasdaq: IONS), the largest shareholding of BB Biotech (9.8% of NAV), should see the outcome of the FDA’s review of Waylivra (volanesorsen) for familial chylomicronemia syndrome (FCS) by 30 August. The application has already been reviewed by an FDA’s advisory committee, which voted narrowly (10-8) in favour of approval in May. Meanwhile, Ionis’s 75%-owned affiliate, Akcea Therapeutics (Nasdaq: AKCA), should see the outcome of the FDA’s review of inotersen for  hereditary TTR amyloidosis (hATTR) by 6 October.

Sage Therapeutics (Nasdaq: SAGE),  an important holding for BB Biotech (4.6% of NAV) and IBT (3.7% of NAV), should see an FDA advisory committee meeting convened to consider its application for brexanolone  for treatment of postpartum (post natal) depression in October/November. This would normally be expected to take place a few months ahead of the target review completion date of 19 December.  Investors will be able to review briefing documents published a few days ahead of the advisory committee meeting.

Alexion Pharmaceuticals (Nasdaq: ALXN), a core holding for Biotech Growth (6.7% of NAV) and Worldwide Healthcare Trust (5% of NAV), but is also included in Swiss-listed BB Biotech (BBB, 4%) among others, has filed ALXN1210, the long-acting C5 complement inhibitor, for the treatment paroxysmal nocturnal hemoglobinuria (PNH) in the EU. This follows the US filing earlier this month. ALXN1210 is critical to Alexion’s strategy to maintain its dominance in the C5 space, against the threat from potential biosimilars or other copy-type products to its blockbuster Soliris in the 2020s.

Stemline Therapeutics (Nasdaq: STML), an investee for IBT and Polar Capital Global Healthcare Trust (PCGH), completed its US submission for Elzonris (tagraxofusp), a potential treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN). The company has been awarded Breakthrough Therapy Designation (BTD), which confers priority review – which should mean an approval decision before the end of the year.  BPDCN is a very rare condition and efficacy has been established in a trial in just 42 patients. Elzonris is also being evaluated in clinical trials for chronic myelomonocytic leukemis, myelofibrosis, acute myeloid leukemia and myeloma.

Incyte (Nasdaq: INCY), an important holding for BB Biotech (8% of NAV) and PCGH, plans to file regulatory applications for Jakafi for steroid-refractory acute graft-versus-host disease (GvHD), a problematic side effect associated with stem cell transplants. This follows positive results from the “Reach1” Phase II trial. Jakafi is currently approved for myelofibrosis.

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