Update for December 22, 2021

Regulatory Update
 
The FDA approved tezepelumab (Tezspire, Amgen & AstraZeneca), on 12/17/2021), as add-on maintenance treatment of severe asthma.
 
The FDA approved efgartigimod, on 12/17/2021, for the treatment of generalized myasthenia gravis that is anti-acetylcholine receptor antibody positive.
 
The FDA has placed a hold on patients under 18 years in trials evaluating lovotibeglogene autotemcel in the treatment of sickle cell disease, due to anemia in an adolescent patient that was treated 18 months ago. Enrollment of patients 18 and older is not affected.
 
The FDA placed a hold on fordadistrogene movaparvovec trials for the treatment of Duchenne muscular dystrophy,after the death of patient in a Phase Ib trial.
 
The FDA placed a hold on all islatravir HIV treatment trials due to the drop in T-cell counts.
 
Ipsen licensed world-wide marketing and development rights from Genfit for elafibranor in the treatment for primary biliary cholangitis.
 
Announced Research Updates
 
Compass Pathways announced that in a 3-week, 19 patient, open-label trial, treatment with a single dose of psilocybin 25 mg decreased the MADRS score by 14.9 points in patients with treatment-resistant depression, who were receiving an SSRI.
 
Sangamo announced that in the 11 patient, Phase I/II, ALTA trial, treatment with giroctocogene fitelparvovec results in an annualized bleeding rate of 0 after 1 year and 1.4 after 2 years with Factor VIII activity of 27.5% of normal in patients with severe hemophilia A.
 
Lilly announced that in the 618 patient, BRUIN trial, treatment with pirtobrutinib resulted in an overall response rate of 51% in 134 MCL patients and 68% in 252 chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) patients.
 
Actinium announced that in the 151 patient, Phase III, SIERRA trial, 100% of patients treated with Iomab-B achieved successful engraftment of allogeneic hematopoietic stem cell transplant compared to 17% that received salvage chemotherapy in patients with active, relapsed or refractory acute myeloid leukemia, eligible to receive a bone marrow transplant.
 
Sanofi announced that in the eight-month, 120 patient, open-label, Phase III, ATLAS-A/B trial, the ABR for bleeding episodes requiring treatment was 0 for fitusiran compared to 21.8 for treatment with on-demand factor therapy in patients with hemophilia A or B with no inhibitors. 50.6% of fitusiran patients experienced no bleeding of any type compared to only 5% with on-demand factor therapy. Sanofi announced that in the eight-month, 57 patient, open-label, Phase III, ATLAS-INH trial, the ABR for bleeding episodes requiring treatment was 0 for fitusiran compared to 16.8 for treatment with on-demand bypassing agents in patients with hemophilia A or B with inhibitors to factor VIII or IX.  65.8% of fitusiran patients experienced no bleeding of any type compared to only 5.3% with on-demand bypassing agents.
 
Kura announced that in a 65 patient, Phase II trial, treatment with tipifarnib resulted in a 56% overall response rate in patients with relapsed or refractory T-cell lymphoma. 
 
Galera announced that in the 455 patient, Phase III, ROMAN trial, 54% of patients treated with avasopasem experienced severe oral mucositis compared to 64% with placebo in patients with locally advanced head and neck cancer receiving seven weeks of radiotherapy plus cisplatin. Galera originally announced the results of the ROMAN trial were not significant, but after an error made by the contract research organization (CRO) was identified and corrected, the results achieved significance.
 
Lilly announced that in the 12-week, 1,044 patient, Phase III, LUCENT-2 trial, more patients receiving mirikizumab maintained remission compared to placebo in patients with moderate-to-severe active UC, who had achieved remission with mirikizumab in the LUCENT-1 trial.
 
Cidara announced that in the 30-day, 187 patient, Phase II, ReSTORE trial, treatment with once-weekly rezafungin was non-inferior to daily caspofungin for mortality at day 30 (23.7% vs 21.3%) and global cure at day 14 (59.1% vs 60.6%) in patients with candidemia and/or invasive candidiasis.
 
Johnson & Johnson announced that in the 97 patient, open-label, Phase Ib/II, CARTITUDE-1 trial, treatment with ciltacabtagene autoleucel resulted in an overall response rate (ORR) of 98% with a progression-free rate of 61% and overall survival rate of 74% at 21.7 months in heavily pre-treated patients with refractory or relapsed multiple myeloma. Johnson & Johnson announced 14.3-month results from 20 patients enrolled in Cohort A of the CARTITUDE-2 trial, where treatment with ciltacabtagene autoleucel resulted in an ORR of 95% in heavily pre-treated patients with multiple myeloma. The ORR was 95% after 10.6 months in 91 multiple myeloma patients enrolled in Cohort B who were in early relapse after initial therapy.
 
Novartis announced that in 2,119 patients enrolled in the 12-week, Phase III, PEARL 1 and 2 trials, treatment with ligelizumab decreased the Urticaria Activity Score (UAS) compared to placebo, but not omalizumab in patients with chronic spontaneous urticaria.
 
Summit Therapeutics announced that in the 30-day, 759 patient, Phase III, Ri-CoDIFy trial, treatment with ridinilazole did not improve the Sustained Clinical Response rate compared to vancomycin in patients with Clostridium difficile infection. Ri-CoDIFy combined 355 patients from the Ri-CoDIFy 1 trial with 404 patients from the Ri-CoDIFy 2 trial.
 
PhaseBio discontinued the Phase IIb, VIP trial evaluating pemziviptadil in the treatment of pulmonary arterial hypertension due to the impact of COVID-19 on drug manufacturing and trial enrollment.
 
Aldeyra announced that in the 12-week, 422 patient, Phase III, TRANQUILITY trial, treatment with reproxalap did not decrease ocular redness compared to placebo in patients with dry eye disease