Friday Five – The pharma week in review (19 December 2019)

Roche finally gets green light for Spark Therapeutics deal

Having initially announced its intention to acquire Spark Therapeutics for $4.3 billion in February, Roche finally received clearance to proceed with the deal from both the US Federal Trade Commission (FTC) and the UK Competition and Markets Authority (CMA) this week.

Both regulators had conducted lengthy reviews to assess whether the acquisition would lessen future competition in the haemophilia A market, where Roche commercialises the antibody product Hemlibra. Spark is developing a 'one time' gene therapy for the same indication.

The FTC in particular appears to have become more stringent in its assessment of how pipeline drugs may potentially impact future market competition as a result of industry consolidation. Earlier this year, Bristol-Myers Squibb announced divesture of the psoriasis treatment Otezla (to Amgen) to meet FTC demands pertaining to its proposed acquisition of Celgene.

Investors will likely be buoyed by confirmation that Roche's deal for Spark  can precede in the hope that other gene therapy developers could be Big Pharma acquisition targets.

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Alnylam hits a hat-trick

Alnylam Pharmaceuticals announced on Tuesday that a Phase III study of the investigational RNAi therapeutic lumasiran met its main goal, significantly reducing urinary oxalate levels relative to placebo in patients with primary hyperoxaluria type 1 (PH1). The company said that based on results from the ILLUMINATE-A trial, it plans to submit marketing applications for the drug in the US and Europe in early 2020.

CEO John Maraganore noted that these results "mark our third positive Phase III study readout in 2019, positioning Alnylam with the potential for four marketed products by the end of 2020, assuming positive regulatory reviews." Maraganore added that Alnylam is confident of beating its financial guidance for next year and building, by the end of 2020, a "global, multi-product, commercial-stage company with a robust portfolio of clinical stage programmes.”"

Alnylam's RNAi platform has certainly been validated by a succession of positive clinical readouts, though whether it can translate this success into profits remains to be seen

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GlaxoSmithKline shows its hand in BCMA contest

GlaxoSmithKline had been hoping to present data for its BCMA-targeting antibody drug conjugate (ADC) belantamab mafodotin – for the treatment of multiple myeloma - at the recent annual meeting of the American Society of Hematology (ASH).

Alas, it failed to secure a spot among the late breaking abstracts, prompting data to be released this week – simultaneously via press release and publication in The Lancet Oncology.

Belantamab mafodotin still looks approvable, but data for other BCMA-targeting drugs presented at ASH served to reiterate what a crowded field this has become; what's more, some competitors look to be more efficacious when used after anti-CD38 antibodies, a new standard of care in early-line therapy.

GlaxoSmithKline has put a lot of effort into accelerating the development programme for belantamab mafodotin and is keen to make a renewed push into the oncology market. Success, however, will not come easily.

See also – KOL Views: Squaring the circle on belantamab mafodotin’s place in MM landscape

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Fevipiprant flunks out

Novartis may have touted the experimental asthma treatment fevipiprant as a potential blockbuster as recently as May, but key opinion leaders FirstWord had spoken to were left largely unimpressed by the molecule, a DP2 receptor.

In this case, the experts were right to be sceptical, with Novartis confirming this week that it plans to terminate development of fevipiprant in asthma after top-line results from two Phase III studies showed that the drug failed to meet the clinically relevant threshold for reduction in rate of moderate-to-severe exacerbation compared to placebo. 

In October, Novartis had previously announced that two other studies, evaluating fevipiprant's effect on FEV1 improvement in patients with moderate asthma, had also failed.

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Amarin secures US label expansion for Vascepa

Late last week, the FDA expanded the labelling for Amarin's fish-oil-derived drug Vascepa to include its use as an adjunctive therapy to lower the risk of cardiovascular (CV) events in adults with triglyceride levels of 150 mg/dL or more. Patients must also have either established CV disease or diabetes and at least two additional risk factors for CV disease.

This approval is widely expected to reposition Vascepa from being a niche therapy (indicated to reduce triglyceride levels in adult patients with severe hypertriglyceridemia) to a potential multi-billion dollar product franchise. Sales of Vascepa have crept upwards over the course of 2019 since new clinical data to support its expanding labelling were first presented late last year.

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