Update for June 1, 2021

Regulatory Update
 
The FDA approved sotorasib (Lumakras, Amgen), on 5/28/2021, for the treatment of non-small cell lung cancer with a KRAS G12C mutation in patients who have received at least one prior systemic therapy. Amgen has set WAC at $17,900 per month for sotorasib.
 
The FDA approved infigratinib (Truseltiq, QED Therapeutics), on 5/28/2021, for the treatment of previously treated locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement.
 
An FDA review of teplizumab found that evidence to support efficacy came from a single small trial, but supported approval given the lack of treatments for a severe and rare disease. The analysis verified available data showing a delay in the onset of Type 1 diabetes in patients treated with teplizumab. The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 10-7 to recommend approval of teplizumab for the delay of Type 1 diabetes in at-risk patients. The AdComm had concerns regarding the small size of the study and the lack of follow-up after patients developed diabetes.
 
The FDA accepted the NDA for Mezzion’s udenafil to improve exercise capacity in patients with single ventricle heart disease who have undergone a Fontan operation and set a PDUFA date for 3/22/2022.
 
The FDA has accepted the BLA for ublituximab to be used in combination with umbralisib in the treatment of patients with chronic lymphocytic leukemia and small lymphocytic leukemia and set a PDUFA date for 3/25/2022.
 
The FDA accepted the NDA for palovarotene for the prevention of heterotopic ossification in fibrodysplasia ossificans progressiva (FOP) and set a PDUFA date for 11/30/2021.
 
The FDA informed Travere that interim data from the Phase III DUPLEX trial was not enough to support approval. Travere hopes to file an NDA in the first half of 2022 with full data from the DUPLEX trial. Travere plans to submit an MAA by the end of 2021.
 
The EMA’s CHMP recommended approval of odevixibat for the treatment of progressive familial intrahepatic cholestasis. 
 
Announced Research Updates
 
EQRx announced interim data from the Phase III GEMSTONE-301 trial, where treatment with sugemalimab prolonged progression-free survival compared to placebo in Chinese patients with locally advanced/unresectable Stage III NSCLC without disease progression after concurrent or sequential chemoradiotherapy. 
 
AB Science announced that in the 60-month, 580 patient, Phase III, AB12003 trial, treatment with masitinib plus docetaxel resulted in progression free survival at 12, 18 and 24 months of 32.0%, 27.6% and 23.1% compared to 19.6%, 14.6%, and 12.0% with docetaxel alone in patients with metastatic Castrate Refractory Prostate Cancer.
 
NGM announced that in the 24-week 171 patient, Phase IIb, ALPINE 2/3 trial, treatment with aldafermin did not improve liver fibrosis by one or more stages with no worsening of steatohepatitis in patients with non-alcoholic steatohepatitis and stage 2 or 3 liver fibrosis. NGM discontinued development of aldafermin for the treatment of non-alcoholic steatohepatitis after failure of the ALPINE 2/3 trial.
 
Published Research Updates
 
In the 332 patient, open-label, Chinese, Phase III, RATIONALE 304 trial, treatment with tislelizumab plus pemetrexed and either carboplatin or cisplatin resulted in PFS of 9.7 months compared to 7.6 months with pemetrexed plus carboplatin or cisplatin alone in patients with advanced non-squamous non-small cell lung cancer. 
 
In the 313 patient, Chinese, Phase III, ACTIVE trial, treatment with apatinib plus gefitinib resulted in progression-free survival of 13.7 months compared to 10.2 months with gefitinib alone in treatment-naive patients with stage IIIB/IV non-squamous non-small cell lung cancer with an EGFR-mutation.
 
In a six-week, 281 patient, Phase III trial, 53.9% of patients treated with sofpironium achieved a Hyperhidrosis Disease Severity Score (HDSS) score of 1 or 2 and a 50% or more reduction in total gravimetric weight of sweat compared to 36.4% with placebo in Japanese patients with primary axillary hyperhidrosis. All patients received sofpironium in a 52-week, 161 patient extension of the trial, where 57.4% of patients that switched from placebo to sofpironium achieved an HDSS score of 1 or 2 and a 50% or more reduction in total gravimetric weight of sweat compared to 58.2% that continued treatment with sofpironium.