Company Product Description Indication Status
Phase I
Anokion SA, of Lausanne, Switzerland ANK-700 Introduces antigen-specific tolerance to myelin-based autoantigens Relapsing remitting multiple sclerosis Started dosing of up to 40 patients in the Moves-it study; primary endpoint is safety and tolerability of ANK-700; secondary endpoint is pharmacokinetics of the drug
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Stem cell stimulator  Mild to severe sensorineural hearing loss In the FX-322-111 phase Ib study, 90 days after a single dose, 34% of patients achieved a 10% or greater absolute improvement in Word Recognition scores in the treated ear compared to the untreated control (p<0.05)
Ideaya Biosciences Inc., of South San Francisco IDE-196  Protein kinase C inhibitor Metastatic uveal melanoma Started dose expansion in the phase I/II study testing IDE-196 with Mektovi (binimetinib, Pfizer Inc.) with a target to enroll approximately 40 patients
Lineage Cell Therapeutics Inc., of Carlsbad, Calif. Opregen Allogeneic retinal pigment epithelium cells Dry age-related macular degeneration with geographic atrophy  In cohort 4 enrolling patients with less advanced disease, better baseline visual acuity and smaller areas of GA, 75% of the 12 patients' treated eyes were at or above baseline visual acuity at their last assessment, compared to 75% of the 12 patients' untreated eyes that were below baseline; best corrected visual acuity for treated eyes reached up to +19 letters
Precigen Inc., of Germantown, Md. PRGN-2012 Off-the-shelf Adenoverse immunotherapy Recurrent respiratory papillomatosis First patient dosed in the study testing PRGN-2012 as an adjuvant immunotherapy following standard-of-care surgical removal of visible papillomatosis disease; primary objective is to determine safety and tolerability and recommended phase II dose
Roche Holding AG, of Basel, Switzerland Glofitamab  CD20xCD3 T-cell engaging bispecific antibodies Relapsed or refractory B-cell non-Hodgkin lymphoma Data published in the Journal of Clinical Oncology showed glofitamab produced an overall response rate of 53.8%, including 36.8% complete responses, among all doses; ORR was 65.7%, including 57.1% CRs, in those dosed at the recommended phase II dose
Phase II
Akero Therapeutics Inc., of South San Francisco Efruxifermin  Fc-FGF21 fusion protein Nonalcoholic steatohepatitis, fibrosis stage 2 or 3 First patient randomized in the Harmony study comparing 2 dose levels of efruxifermin to placebo; primary endpoint is fibrosis regression at 24 weeks
Frequency Therapeutics Inc., of Woburn, Mass. FX-322 Stem cell stimulator  Mild to moderately severe sensorineural hearing loss Interim results from the phase IIa FX-322-20 study showed 4 injections produced no benefit on hearing compared to placebo; Word Recognition scores increased after the first dose, but repeated doses dampened the hearing benefit; placebo also improved more than expected
Neoimmunetech Inc., of Rockville, Md. NT-I7 (efineptakin alfa) Long-acting interleukin 7 Relapsed/refractory advanced solid tumors Treated first patient in the phase IIa portion of the phase Ib/IIa study testing NT-17 with Keytruda (pembrolizumab, Merck & Co. Inc.); study will enroll up to 150 patients testing the combination in patients who have been treated with a checkpoint inhibitor (CPI) for triple-negative breast cancer, non-small-cell lung cancer or small-cell lung cancer, and CPI-naïve patients with microsatellite stable colorectal cancer or pancreatic cancer
Omeros Corp., of Seattle  Narsoplimab  Monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 Critically ill COVID-19 Started dosing with narsoplimab in the adaptive platform I-Spy COVID-19 study sponsored by Quantum Leap Healthcare Collaborative
Sojournix Inc., of Waltham, Mass. SJX 653 Neurokinin 3 antagonist Moderate to severe vasomotor symptoms in postmenopausal women Decided to terminate study as anticipated target profile was not met
Phase III
Incyte Corp., of Wilmington, Del. Jakafi (ruxolitinib) JAK1/2 inhibitor COVID-19-associated acute respiratory distress syndrome Terminated Ruxcovid-Devent study in patients who require mechanical ventilation
Novartis AG, of Basel, Switzerland 177Lu-PSMA-617 Radioligand therapy targeting PSMA Progressive PSMA-positive metastatic castration-resistant prostate cancer The Vision study met both primary endpoints of overall survival and radiographic progression-free survival; data to be presented at an upcoming medical meeting
Oramed Pharmaceuticals Inc., of New York ORMD-0801 Oral insulin Type 2 diabetes Screened first patient for the ORA-D-013-2 study; primary endpoint is A1c over a 26-week treatment period; secondary endpoint is maintaining A1c over 52 weeks; enrollment in the concurrent ORA-D-013-1 study has surpassed 25%
Recordati SpA, of Milan, Italy Isturisa (osilodrostat) 11beta-hydroxylase inhibitor Cushing’s disease In the Linc 4 study, after 12 weeks of treatment, 77% of patients treated with Isturisa achieved normal mean urinary free cortisol (mUFC) compared to 8% of patients taking placebo (p<0.0001); at week 36, 81% of patients treated with Isturisa achieved normal mUFC
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Regen-Cov (casirivimab + imdevimab) Monoclonal antibody targeting SARS-CoV-2 High-risk non-hospitalized COVID-19 Drug reduced the risk of hospitalization or death by 70% and 71% at the 1,200-mg and 2,400-mg doses, respectively, compared to placebo (p=0.0024 and p<0.0001, respectively); study also met all secondary endpoints, including median time to resolution, which was 10 days for both doses, compared to 14 days for both placebo control groups (p<0.0001 for both)
Roche Holding AG, of Basel, Switzerland, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. Tominersen  Antisense drug targeting the huntingtin protein Huntington’s disease  Discontinuing dosing in the Generation HD1 study on recommendation of the independent data monitoring committee based on the benefit/risk profile; no new safety signals were identified; patients will continue to be followed; dosing paused in the open-label Gen-Extend extension study

Notes

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