Friday Five – The pharma week in review (23 January 2020)

Earnings season kicks off

As is the tradition, Johnson & Johnson led the charge for the latest quarterly earnings season- though it did not get off to the most auspicious start. The pharma reported strong sales in its vital oncology and immunology sectors, despite competition from AbbVie's Skyrizi (risankizumab). However, ongoing opioid litigation and headwinds from biosimilars- though milder than predicted, with little new encroachment into the Remicade (infliximab) franchise- contributed to a brief sell-off on Wednesday.

Fresh off updates from the J.P. Morgan Healthcare conference, during this earnings season analysts will once again be looking for Biogen to speak its piece on both Spinraza (nusinersen) sales and the latest comings and goings for its planned aducanumab submission. Gilead Sciences will be on the hook for any flickers of M&A activity, and analysts will be asking Roche for updates on the status of the clinical hold currently in place on its intrathecal delivery trials of Zolgensma (onasemnogene abeparvovec).

Viral outbreak drives biotech bottom line

An outbreak of coronavirus in China is generating concern over a potential pandemic as observers equate the largely unknown pathogen with prior outbreaks of SARS or even Ebola virus. While Chinese authorities have reportedly shut down large swaths of transport to quarantine the disease epicentre, the first incidence of coronavirus was reported in the US this week, further fomenting breakout concerns.

However, several biotechs were on hand to help dispel any panic- and bolster their own share price. Vaccine play Novavax got a boost and a follow-on offering based on its declarations that it was developing a vaccine to help contain the outbreak, a story replicated by follow small-cap companies NanoViricides and Inovio Pharmaceuticals.

Even larger players like Gilead got in on the antiviral action, with the company saying that it is exploring the use of remdesivir, an experimental Ebola therapy, in the coronavirus outbreak. Moderna was also in on the action, announcing that it would quickly get to work on using its mRNA platform to churn out a vaccine candidate, following up with collaborations culminating in a planned IND in the US.

A KRAS Revolution?

The ongoing battle between Amgen and Mirati Therapeutics' KRAS inhibitors has already provided hours of entertainment for oncology pundits. But an S-1 filing from Revolution Medicines provided a closer look at preliminary results from its platform, which uses natural products as a starting point to bind small molecules to otherwise intractable targets. That approach differs from that used by Amgen or Mirati, which are using the same fundamental mechanism to inhibit the KRAS protein. Rather than covalently binding to the cysteine residue of the inactive GDP-bound protein, Revolution is using its platform to target the GTP-bound, 'on' state of RAS protein.

Analysis - One to Watch: Revolution cues up next-gen KRAS platform

In an S-1 filing tied to its proposed initial public offering (IPO), Revolution disclosed pharmacokinetic data for RMC-4360, a SHP2 inhibitor partnered with Sanofi that it says offers particularly efficacious potential as a KRAS combination partner. Bernstein's Ronny Gal noted that the Phase I data showed "acceptable safety and hints of activity" in patients with RAS mutations, hopefully setting the stage for the eventual clinical studies in combination with its novel KRAS inhibitors. Its lead candidates targeting KRAS G12C mutations are still in preclinical stages.

Sarepta saga continues with CRL  disclosure

Sarepta Therapeutics is no stranger to controversy, but this time around the company got to share the limelight with the FDA. The agency released the complete response letter (CRL) tied to its August rejection of Vyondys 53 (golodirsen), as well as the company's appeal letter providing its counterarguments for the rejection as part of a post-approval data dump for the Vyondys 53 application.

Analysis - ViewPoints: FDA shines a light on Sarepta's Vyondys 53 CRL

The FDA came down hard on Sarepta for failing to initiate its confirmatory study for Exondys 51 (eteplirsen)- which has since been initiated- on the grounds that the data would have gone a long way to inform decision making for the next exon-skipping therapy. However, the agency also took on some heat for its arguably arbitrary decision-making, which saw two different FDA reviewers come to very different conclusions in response to similar data sets.

Roche keeps up the SMA pressure

Roche- partnered with PTC Therapeutics and the SMA Foundation- released an update from its risdiplam programme to treat spinal muscular atrophy (SMA), this time from the FIREFISH study in Type 1 patients. In these youngest and most severe patients, treatment with the oral RNA splicing therapy met the study's primary endpoint evaluating the proportion of infants sitting without support after 12 months of treatment.

FIREFISH data had already been included in the company's BLA submission for risdiplam, and further confirmatory evidence in the Type 1 setting could upset the apple cart at Novartis in particular. The pharma will be hoping that it maintains exclusive usage in the younger patient population, given that its label is limited to ages two and younger; however, key opinion leaders (KOLs) that spoke with FirstWord anticipated combination usage of risdiplam with all available treatments- assuming payer coverage.

Analysis - ViewPoints: Experts' enthusiasm for Roche's risdiplam grows as FDA grants priority review

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