Friday Five - The pharma week in review (14 August 2020)

Moderna secures large US vaccine deal

Moderna has reached an agreement to supply the US government 100 million doses of its experimental COVID-19 vaccine mRNA-1273 at a cost of up to $1.5 billion, with an option to supply a further 400 million doses.

Under the deal each dose of the vaccine will cost the US government around $15, compared to pricing of $32-$37 per dose under some smaller agreements. Moderna has previously received funding to support development of the vaccine worth $955m under the Operation Warp Speed programme and when taking this into account the vaccine is effectively costing the US government approximately $25 per dose.

It is reported that a lower price for mRNA-1273 will be charged if Moderna fails to secure emergency use or standard regulatory approval by the end of January 2021.

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Roche plays the price card with Evrysdi

Roche and PTC Therapeutics have received US approval for the spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) to treat all types of SMA in adult and paediatric patients aged two months and older. Availability of Evrysdi, which becomes the first available oral treatment for SMA and will go on sale within two weeks, is expected to pressurise sales of Biogen’s Spinraza.  

Roche said that the maximum price for the RNA splicing modifier will be capped at $340,000 annually, with pricing based on weight. The company noted that for patients from two months to two years of age, the annual cost was likely to be less than $100,000.

As predicted, Roche’s price point undercuts existing treatment options for SMA, most notably Spinraza which is priced at $750,000 for the first year of treatment followed by an annual cost of $375,000. Novartis’ Zolgensma is priced at $2.1 million for a one-time treatment, but the company argues that the duration of benefit for the gene therapy makes it cost effective relative to Spinraza.

More on the competitive dynamics here

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Mixed signals for Tecentriq in breast cancer

Elsewhere, Roche suffered a surprising setback by confirming that its cancer immunotherapy Tecentriq failed to significantly improve progression free survival (PFS) when used in combination with paclitaxel to treat PD-L1 positive triple negative breast cancer (TNBC) patients in the IMPassion131 study.

In a prior study (IMPassion130), the combination of Tecentriq with Bristol-Myers Squibb’s Abraxane had significantly prolonged PFS in PD-L1 positive patients with metastatic TNBC.

Commenting on the latest results, a leading expert told FirstWord that a lack of requirement to use steroids with Abraxane (which are necessary with paclitaxel) could explain the discrepancy between the two studies.

The KOL added that he does not expect the FDA to withdraw Tecentriq’s existing approval for metastatic TNBC in PD-L1 positive patients as a result of the IMPassion131 study but he does now perceive Merck & Co.’s competing immunotherapy Keytruda to potentially emerge as a favoured treatment for breast cancer patients, particularly when taking into account prior disclosure of positive top-line data in the neo-adjuvant setting.

More here

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A new player emerges in DMD

Sarepta Therapeutics is steadily building its leadership of an emerging market for Duchene muscular dystrophy (DMD) treatments but now faces competition following the US approval of NS Pharma’s Viltepso.

Viltepso has been approved for the treatment of DMD patients who have a confirmed mutation of the DMD gene amenable to exon 53 skipping, estimated to account for approximately 8% of the total DMD population. Sarepta’s Vyondys 53 was approved by the FDA for the same indication late last year.

The new approval marks the first time that Sarepta will be facing a marketed competitor, a unique challenge for the rare disease company. Moreover, launch of Vyondys 53 has been interrupted in relatively short order by the COVID-19 pandemic. Speaking on the company’s most recent earnings call, chief commercial officer Bo Cumbo told analysts that new patient starts had been limited in the current landscape, although Vyondys 53 still managed to meet the Street’s revenue expectations, in part due to a transition to in-home visits to keep patients on therapy.

More here

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Aducanumab gets priority review

Having confirmed that it had submitted a US regulatory application for the investigational Alzheimer’s disease treatment aducanumab in early July, Biogen provided a further update this week to confirm that the FDA has granted the application priority review status.

Aducanumab could therefore be approved by the FDA on or before March 7 2021, though an advisory committee meeting will be held to discuss Biogen’s application, the company also confirmed. Biogen also clarified that it has not used the priority review voucher it received upon approval of Spinraza in 2016 to accelerate regulatory review of aducanumab.

What analysts have described a ‘best case scenario’ for Biogen has inevitably put upward pressure on the company’s share price despite major question marks over the efficacy of aducanumab based on the data package submitted to the FDA.

Whilst Biogen must wait and see what conclusion that FDA arrives at, investors are now under the gun to make a decision on whether to buy in or sell out on the company’s AD candidate.

The potential upside is gigantic. Aducanumab has the potential to become a mega-blockbuster drug based on the prevalence of Alzheimer’s disease and massive unmet need, with a wide variety of peak sales projections that run the gamut from roughly $10 billion to more than twice that. On the flipside, the drug represents an enormous chunk of Biogen’s prospects for future growth, so rejection by the FDA would likely hit the company’s share price hard.

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