GlycoMimetics rallies on renewed hopes for sickle cell drug

Thinly traded micro cap GlycoMimetics (GLYC +45.1%) jumps on more than a 6x surge in volume in reaction to its post hoc analysis of the failed Phase 3 RESET trial evaluating rivipansel for the treatment of vaso-occlusive crisis (VOC) in hospitalized sickle cell disease (SCD) patients that, it says, showed a statistically significant improvement in the time readiness for discharge, the primary endpoint, in participants who received rivipansel within ~26 hours of the onset of pain.

The results, along with new biomarker data, will be presented in late September at the Foundation for Sickle Cell Disease Research meeting.

The company plans to review the data with the FDA to determine next steps, if any, in a registration path.

In early August 2019, shares plummeted after licensee Pfizer announced that RESET failed to achieve the primary endpoint. It returned all rights to the pan-selectin inhibitor to GLYC four months ago.

Data on GMI-1687, a subcutaneously administered E-selectin inhibitor called uproleselan that company believes could be a more effective approach to treating acute VOC in SCD will also be presented at the September meeting.

https://seekingalpha.com/news/3582708-glycomimetics-rallies-on-renewed-hopes-for-sickle-cell-drug

Moderna mouse study shows positive result

Preliminary data show mice receiving one dose of Moderna’s (NASDAQ:MRNA) COVID-19 vaccine were sufficiently protected against the virus, without increasing the risk of more severe disease.
https://seekingalpha.com/news/3582742-moderna-mouse-study-shows-positive-result

FDA OKs expanded use of ViiV HIV med Tivicay

The FDA approves ViiV Healthcare’s Tivicay (dolutegravir) for the treatment of HIV-1 infection in children as young as four weeks old and weighing at least 3 kg, in combination with other antiretroviral treatments.

The agency approved the HIV-1 integrase strand transfer inhibitor in August 2013 for HIV-1-positive patients at least 12 years old and weighing at least 40 kg.

ViiV Healthcare is the HIV-focused joint venture between GlaxoSmithKline (GSK -0.3%), Pfizer (PFE +0.3%) and Shionogi (OTCPK:SGIOY +1.3%).

https://seekingalpha.com/news/3582747-fda-oks-expanded-use-of-viiv-hiv-med-tivicay

Principia’s rilzabrutinib shows positive effect in rare skin-blistering disorder

Principia Biopharma (NASDAQ:PRNB) announces positive results from Part B of a Phase 2 clinical trial, BELIEVE-PV, evaluating oral BTK inhibitor rilzabrutinib in patients with pemphigus, a group of rare autoimmune disorders characterized by blisters or sores on the skin. The data were virtually presented at the American Academy of Dermatology Annual Meeting.

At week 24, the complete remission rate was 40% while the median corticosteroid dose was reduced to 6 mg. 60% and 87%, respectively, of patients achieved control of disease activity (CDA) by week 4 and week 12.

A Phase 3 study is underway.

https://seekingalpha.com/news/3582781-principias-rilzabrutinib-shows-positive-effect-in-rare-skin-blistering-disorder

CDC warns on another round of lockdowns

Speaking at a press briefing, the CDC’s Jay Butler says “mitigation efforts” similar to March, i.e. economic shutdown, may be necessary should cases “dramatically” rise.

At the moment there are a number of report of rising cases in reopened areas – by itself not too much of an issue if the rise correlates to a rise in testing (though a Morgan Stanley report yesterday suggests otherwise). But there are also reports of rising hospitalizations – again, by itself not a huge issue if that correlates to folks headed back to hospitals for elective surgeries.

https://seekingalpha.com/news/3582749-cdc-warns-on-another-round-of-lockdowns

Lilly’s lebrikizumab shows encouraging action in dermatitis study

Eli Lilly (LLY -0.3%) announces new data from a Phase 2b clinical trial evaluating lebrikizumab in patients with moderate-to-severe atopic dermatitis (AD). The results were virtually presented at the American Academy of Dermatology Annual Meeting. Key findings:

Itch by day 2 improved to week 16.

Sleep by the first on-treatment assessment at week 1 which improved to week 16.

Disease severity improved by the first on-treatment assessment at week 16.

Quality of life measures improved.

Five Phase 3 studies are underway.

Lebrikizumab is an injectable humanized monoclonal antibody that binds to (inhibits) a pro-inflammatory protein called interleukin-13 (IL-13).

Lilly obtained the rights to the candidate via its $1.1B acquisition of Dermira in February.

https://seekingalpha.com/news/3582761-lillys-lebrikizumab-shows-encouraging-action-in-dermatitis-study

Roche’s risdiplam shows treatment benefit in SMA studies

Roche (OTCQX:RHHBY) unit Genentech announces positive results from two clinical trials evaluating risdiplam in patients with spinal muscular atrophy (SMA).

An exploratory analysis of the Phase 2/3 SUNFISH study in patients with type 2 or 3 SMA showed that risdiplam significantly improved motor function after 24 months of treatment.

Preliminary 12-month data from the Phase 2 JEWELFISH trial in patients with all SMA types showed that treatment with risdiplam led to rapid and sustained increases in survival motor neuron (SMN) protein levels.

No new safety signals were observed.

The company’s U.S. marketing application is currently under FDA review with an action date of August 24.

Risdiplam is a survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the central nervous system and in peripheral tissues.

Related ticker: PTC Therapeutics (NASDAQ:PTCT)

https://seekingalpha.com/news/3582789-roches-risdiplam-shows-treatment-benefit-in-sma-studies