New Drug Update for The Week Ending May 25

Regulatory Update

As we reported on Friday with our special update, the FDA approved onasemnogene abeparvovec (Zolgensma, Novartis) on 5/24/2019 for the treatment of patients under the age of two with bi-allelic mutations in the SMN1 gene. This would include babies with Type 1, 2 or 3 spinal muscular atrophy (SMA). Onasemnogene abeparvovec is administered as a single intravenous infusion. The drug was approved with a black box warning regarding acute serious liver injury. ICER released an addendum to the SMA review after the approval of onasemnogene abeparvovec based on additional clinical data. ICER estimated the drug cost at $1.1 to $1.9 million compared to best supportive care to reach the $100,000 to $150,000 cost effective quality-adjusted life year thresholds and $1.2 million to $2.1 million to be cost effective as the life-year gained threshold. Novartis priced onasemnogene abeparvovec at $2.125 million per dose with the total price paid over five annual installments of $425,000 The price is lower than the $4-5 million cost to treat an SMA patient for 10 years and similar to the higher ICER estimates. Onasemnogene abeparvovec will initially be available at 60 medical centers. The sites will send blood for genetic manipulation to Novartis, who will then deliver the re-engineered treatment to the center. 

The FDA approved alpelisib (Piqray, Novartis) on 5/24/2019to be used in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer following tumor advancement after endocrine-based regimens. The companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, was also approved to detect the PIK3CA mutation in a tissue and/or a liquid biopsy.

Myovant announced that results from the Phase III LIBERTY 2 trial, which is evaluating relugolix and norethindrone in women with uterine fibroids and heavy menstrual bleeding are expected in 3Q19. If like LIBERTY 1 the results are positive, Myovant plans to file an NDA in 4Q19.

Announced Research Updates

Biocryst announced that in a 24-week, 121 patient, Phase II trial, where the 150 mg dose of BCX7353 reduced angioedema attacks by 44% compared to placebo and the 110 mg dose reduced attacks by 30% compared to placebo in patients with Type I and II Hereditary Angioedema.

Lilly announced that in the 12-week, 180 patient, Phase II, SERENITY trial, more patients treated with mirikizumab had a 50% reduction in the severity in the Simple Endoscopic Score for Crohn's Disease (SES-CD) than placebo with 200 mg, 600 mg, and 1000 mg in 25.8%, 37.5%, and 43.8% of patients compared to 10.9% with placebo in patients with moderate to severe Crohn's disease.

ResTORbio announced that in a 16-week, 652 patient, Phase IIb trial, treatment with dactolisib reduced laboratory confirmed respiratory tract infections during flu season by 30.6% compared to placebo in elderly patients at high risk to develop respiratory tract infections. In a subset of 98 asthma patients who were 65 or older, 12% patients treated with dactolisib developed a respiratory tract infection compared to 40% with placebo.

Zynerba announced that in a 12-week, 20 patient, Phase II, open label trial, ZYN002 reduced the Anxiety, Depression, and Mood Scale (ADAMS) score by 15.3 points in patients with fragile X syndrome.

Published Research Updates

In a 48-week, 111 patient, open label extension of a Phase II trial, recombinant human pentraxin 2 continued to slow the decline in forced vital capacity in both patients that continued the drug from the first trial and patients that are switched from placebo.