Update for June 28, 2022

Regulatory Update

Menarini and Radius submitted an NDA elacestrant for the treatment of ER+/HER2- advanced or metastatic breast cancer.

The EMA’s CHMP recommended approval of lenacapavir and valoctocogene roxaparvovec.

Announced Research Updates

Roche announced that in a 260 to 416 week, 252 patient, Phase II trial, treatment with crenezumab did not slow the decline in cognition in cognitively unimpaired individuals who have no clinical symptoms of Alzheimer’s disease and carry the PSEN1 E280A autosomal dominant mutation.

Ionis and AstraZeneca announced interim results at 35 weeks from the 66-week, 168 patient open-label, Phase III NEURO-TTRansform trial, where treatment with eplontersen reduced serum transthyretin (TTR) concentration and improved the modified Neuropathy Impairment Score +7 (mNIS+7) compared to a historical cohort of placebo patients from the inotersen NEURO-TTR trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).

PTC Therapeutics announced that in a 48-week, 359 patient, Phase III trial, treatment with ataluren the the decline in six-minute walking distance compared to placebo (53 m vs 67.4 m) in patients with Duchenne muscular dystrophy.

Galderma announced that in the 16-week, 274 patient, Phase III, OLYMPIA 2 trial, 38% of patients treated with nemolizumab achieved clearance or almost-clearance of skin lesions, when assessed using the investigator’s global assessment (IGA) score, compared to 11% with placebo in patients with moderate to severe prurigo nodularis. 56% of nemolizumab patients achieved at least a four-point reduction in itch, as measured by the peak-pruritus numerical rating scale (PP-NRS) score, compared to 21% with placebo.

Italfarmaco announced that in the 18-month, 179 patient, Phase III, EPIDYS trial, adding givinostat to corticosteroids resulted in a reduction in the decline of time required to climb four stairs compared to corticosteroids only in ambulatory DMD patients.

Gilead announced that in the 48-week, 150 patient, Phase III, MYR301 trial, undetectable levels of HDV RNA or a 2 log(10) IU/mL decrease and normalization of ALT levels was achieved in 45% of patients treated with bulevirtide 2 mg and 48% with 10 mg compared to 2% with placebo of untreated chronic HDV patients.

Published Research Updates

In the 454 patient, Phase III, PANTHER trial, adding pevonedistat to azactidine did not improve event-free survival compared to azactidine alone in patients with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia or low-blast acute myeloid leukemia.

In a 12-day, 62 patient, Phase IIa trial, vaccination with RSVpreF demonstrated 86.7% efficacy in preventing respiratory syncytial virus (RSV) compared to placebo in a challenge study of healthy adults 18 to 50.