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The Readout Damian Garde & Meghana Keshavan

Ovid’s drug for a rare, untreatable disease proves no better than placebo

Ovid Therapeutics’ experimental drug for a devastating rare disease failed in a pivotal clinical trial, the company said Tuesday, a bitter disappointment for families dealing with the untreatable condition.

The study enrolled 97 children with Angelman syndrome, a rare genetic disorder that causes seizures and severe cognitive impairment, and randomized them to receive either Ovid’s drug, OV101, or placebo. After 12 weeks, OV101 was no better than placebo at improving the symptoms of Angelman, which include impaired sleep, inability to speak, delayed motor development, and intellectual disability.

Ovid is pausing development of OV101 pending complete data and meetings with regulators. There are no approved treatments for Angelman, which affects just a few thousand patients in the U.S.

Read more.

Consider the nanoparticles that make Covid-19 vaccines work

As Covid-19 vaccines from Pfizer and Moderna speed toward global approvals, most of the scientific attention has focused on the synthetic messenger RNA that makes them effective. But neither would be possible if not for decades of research into nanotechnology, without which the vaccines could never reach their bodily targets and protect people from Covid-19.

In a sweeping new STAT Report, Elizabeth Cooney dives into the research behind lipid nanoparticles, which are used to deliver both vaccines to the right cells. The fatty molecular envelopes protect strands of mRNA from the body’s natural defenses, allowing them to their jobs and confer immunity.

Those particles are just one facet of the sprawling field that is nanotechnology, which is empowering novel approaches to medicine, repurposing of old drugs, and the clinical uses of CRISPR genome editing. It’s all explained in STAT’s latest comprehensive report, which you can purchase here.

Trump’s latest invitation puts biopharma in an awkward position

President Trump has invited a bunch of drug industry executives to the White House for a Covid-19 vaccine event that seems more self-congratulatory than informational. That puts them in an uncomfortable position, as most don’t want to do the lame-duck president a political favor, but no one wants to be alone in standing Trump up.

As STAT’s Lev Facher reports, the meeting is slated for Dec. 8, which is just two days before a panel of FDA advisers will publicly pore over the data supporting Pfizer’s Covid-19 vaccine. Among the invitees are executives from drug makers, distributors, pharmacies, and delivery services.

The agenda, according to people familiar with the meeting, is likely to be two-fold: allowing Trump to once more pressure the FDA to approve vaccines as quickly as possible, and giving him a chance to again take credit for their rapid development.

Read more.

Agios’s lead drug boosts hemoglobin in a key trial

Yesterday’s, Agios Pharmaceuticals’ most closely watched medicine met its main goal in a Phase 3 study, increasing hemoglobin levels in patients with a rare form of anemia.

As STAT’s Adam Feurstein reports, 40% of patients who to receive Agios’s drug, mitapivat, achieved a hemoglobin response, which was defined as an increase in hemoglobin of 1.5 grams per deciliter or higher from baseline to the end of the study. None of the patients given placebo had a hemoglobin response.

The study involved adults with pyruvate kinase deficiency, a form of anemia, but its success bodes well for mitapivat’s potential in other conditions where boosting hemoglobin would be beneficial, including sickle cell disease and two types of thalassemia.

Read more.

More reads

  • How society can make the most of Covid-19 vaccines — and not squander an opportunity. (STAT)
  • Nasdaq proposes board-diversity rule for listed companies. (Wall Street Journal)
  • A conversation on drug pricing, policy, and access. (STAT+)

Thanks for reading! Until tomorrow,

Wednesday, December 2, 2020


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