Friday Five – The Pharma Week in Review (10 September 2021)

Sanofi snags Kadmon

  • Sanofi is acquiring Kadmon for $1.9 billion. It is the sixth largest pharma acquisition of 2021 to date.
  • In doing so, Sanofi gets its hands on Rezurock, a first-in-class agent recently approved by the FDA for chronic graft-versus-host disease (cGvHD).
  • Key opinion leaders have high hopes for the drug, which could become a blockbuster.
  • Sanofi remains pharma's most persistent acquirer, with this its fifth deal in the past year at a cost of $6.7 billion.

GT can't shake safety issues

  • The FDA's recent AdCom meeting to discuss safety issues relating to gene therapies was inauspiciously bookended by two clinical holds for agents in the field.
  • The latest of these – announced on Monday – is for BioMarin's BMN 307 in a Phase I/II study for phenylketonuria (PKU), and was prompted by a preclinical toxicity signal.
  • Prior to the AdCom, the FDA placed a clinical hold on Astellas' Phase I/II study of AT132 in X-linked myotubular myopathy (XLMTM).
  • Triggered by an abnormal liver function signal in a patient enrolled in the study, the path to recovery for Astellas could be more challenging.

A glimmer of hope in AD

  • Recently announced top-line data from a Phase II study of the anti-Tau agent semorinemab in mild-to-moderate Alzheimer's disease offer "a glimmer of hope" in the pursuit of disease modifying therapies, one key opinion leader told FirstWord this week.
  • Data from the study were mixed (with one endpoint met and one missed) and semorinemab has previously failed to hit the mark in a Phase II study of patients with prodromal-to-mild Alzheimer's disease.
  • Lack of efficacy in earlier-stage patients may not be a red flag, but it is hard to tell at this stage.
  • “The dynamics of Alzheimer's disease typically start with amyloid pathology, and after that the tau pathology picks up speed and creates the synaptic disruption. So maybe the later-stage population is more sensitive to tau-related mechanisms in a shorter timeframe.”

JAKs boxed in

  • Following on from new FDA safety guidance for all JAK inhibitors as treatments for rheumatoid arthritis, the drug class could be "irreparably damaged" by the agency's new labelling requirements, says one expert in the field.
  • Snap-polls we fielded to rheumatologists and dermatologists give an indication as to how much of an impact new safety guidance will have for the JAKs in rheumatoid arthritis and atopic dermatitis respectively.

 Can Farxiga follow Jardiance?

  • Eli Lilly and Boehringer Ingelheim's Jardiance is poised to become an important new therapy option for a group of harder-to-treat heart failure patients, which could result in a billions of dollars in additional revenues.
  • Data justifies a broader label for Jardiance than Novartis' competing treatment Entresto, our new cardiologist poll indicates, but one KOL told FirstWord that there is still room for other SGLT-2 inhibitors – specifically AstraZeneca's Farxiga – to further improve on Jardiance's impressive new data, if it can demonstrate a statistically significant mortality benefit.

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