Company Product Description Indication Status
Phase I
Imbrium Therapeutics LP, of Stamford, Conn., a subsidiary of Purdue Pharma LP IMB-115 Selective partial agonist for nociceptin/orphanin-FQ peptide receptors Insomnia disorder Results from 2 phase Ib studies showed minimal or no residual next-day somnolence or psychomotor impairment; data from both studies demonstrated dose-dependent, statistically significant and clinically meaningful enhancement in sleep efficiency (1 mg – 10 mg) and reduction in Wakefulness After Sleep Onset (0.5 mg – 10 mg); with the exception of Latency to Persistent Sleep and subjective Sleep Latency, data showed all other objective and subjective measures of sleep were statistically significantly improved at doses ≥3 mg vs. placebo
Kiadis Pharma NV, of Amsterdam, the Netherlands FC21-NK cells Haploidentical expanded natural killer cells Acute myeloid leukemia In 13 patients with primary refractory (n=5) or relapsed (n=8) disease and median of 5 prior therapies, including 9 with prior stem cell transplant, complete and overall response rates were 50% and 78.6%, respectively; median overall and disease-free survival after treatment were 231 and 186 days, respectively
Shattuck Labs Inc., of Durham, N.C. SL-172154 (SIRP-alpha-Fc-CD40L) CD40 ligand receptor agonist; CD47 antagonist Ovarian cancer Open-label, dose-escalation study initiated
Phase II
Bluebird Bio Inc., of Cambridge, Mass. Elivaldogene autotemcel (Lenti-D) ABCD1 gene stimulator Cerebral adrenoleukodystrophy Long-term results from phase II/III Starbeam study showed 20 of 23 evaluable participants who were free of major functional disabilities (MFDs) at 2 years remained MFD-free through last available follow-up, including all 10 who reached at least year 5 follow-up; 31 of 32 in phase II portion had stable neurologic function scores following treatment, including 24 with score of 0 at last available visit
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Lambda (peginterferon lambda-1a) + lonafarnib Interferon receptor modulator + farnesyl transferase inhibitor Hepatitis D virus In phase IIa Lift study in 26 adults, 96% of participants (n=25) achieved primary endpoint of > 2 log decline in HDV RNA, 58% were below limit of quantitation or undetectable and median HDV RNA decline was 3.2 log IU/mL (CI: 2.50-3.93, p<0.0001)
Embera Neurotherapeutics Inc., of Boston EMB-001 (metyrapone + oxazepam) Benzodiazepine receptor agonist + glucocorticoid receptor antagonist Cocaine use disorder First of 80 participants dosed; primary efficacy endpoint is abstinence in those who receive study drug vs. placebo during final 3 weeks of treatment
Mirum Pharmaceuticals Inc., of Foster City, Calif. Maralixibat ASBT inhibitor Progressive familial intrahepatic cholestasis In 5-year analysis of Indigo data, those who exhibited serum bile acid control on study drug had improved transplant-free survival (p=0.0006); 7 of 19 non-truncating patients with PFIC2 achieved response threshold
NGM Biopharmaceuticals Inc., of South San Francisco Aldafermin FGF-19 ligand Nonalcoholic steatohepatitis Additional analysis of cohort 4 data in participants with stage 3 (F3) liver fibrosis from 24-week study showed 30% dosed with study drug at 1 mg achieved fibrosis improvement >1 stage without worsening of NASH vs. 0% for placebo; responder analysis in F3 liver fibrosis patients who achieved ≥30% liver fat content reductions showed 46% treated with study drug had fibrosis improvement of ≥1 stage without worsening of NASH vs. 0% for placebo; enrollment also completed in phase IIb Alpine 2/3 study, with top-line data readout expected in second quarter of 2021
Novartis AG, of Basel, Switzerland LNP-023 Complement factor B inhibitor Paroxysmal nocturnal hemoglobinuria  10 patients who received study drug as add-on to eculizumab had reduced levels of lactate dehydrogenase and, compared with baseline values on eculizumab alone, increased hemoglobin (Hb) levels by 2.87 g/dL (p<0.001); all but 2 achieved Hb levels >12 g/dL in absence of red blood cell transfusions; after at least 6 months of stable LNP-023 add-on therapy, 7 discontinued eculizumab and remained on study drug as monotherapy, retaining Hb levels with no changes in biomarkers of disease activity or signs of breakthrough hemolysis
Orpheris, wholly owned subsidiary of Ashvattha Therapeutics LLC, of Redwood City, Calif. OP-101 Dendrimer-based acetylcysteine therapy COVID-19 infection First of about 24 adults with severe infection enrolled in Prana study; participants will be randomized to single intravenous infusion (2 mg/kg, 4 mg/kg or 8 mg/kg) or placebo, all in addition to standard of care; efficacy endpoints include resolution of fever, improvement in oxygenation, hospitalizations and death
Provention Bio Inc., of Oldwick, N.J. PRV-015 Anti-interleukin-15 monoclonal antibody Celiac disease Phase IIb Proactive study launched in about 220 adults with nonresponsive disease; primary endpoint is efficacy measured by Celiac Disease Patient-Reported Outcome
Vir Biotechnology Inc., of San Francisco, and Glaxosmithkline plc, of London VIR-7831 (GSK-4182136) COVID-19 Spike glycoprotein modulator COVID-19 infection First of about 1,300 people with mild to moderate disease dosed in phase II/III Comet-Ice study in those at high risk of hospitalization; primary efficacy endpoint is proportion whose infections worsen, defined by need for hospitalization or death, within 29 days of randomization
Phase III
Acasti Pharma Inc., of Laval, Quebec Capre (omega-3 phospholipid) Long-chain omega-3 phospholipid carrying EPA and DHA Hypertriglyceridemia Trilogy 2 study in 278 participants achieved 30.4% median reduction in triglycerides at 12 weeks and 38.4% at 26 weeks but missed statistical significance on primary endpoint, with placebo-corrected triglyceride reduction of 12.4% (p=0.19); no additional trials planned
Akari Therapeutics plc, of New York Nomacopan Dual complement C5 and leukotriene inhibitor COVID-19-related pneumonia Global program to include investigator-led U.S. study, randomized study in Brazil and U.K. study under framework of AGILE therapeutic development platform; planned trials call for 2-to-1 randomization of study drug plus standard of care (SoC) vs. SoC alone, with initial target of about 60 patients hospitalized on supportive oxygen in each setting; primary endpoint is time to normalization of oxygen, with secondary endpoint of need for intubation and mortality
Axsome Therapeutics Inc., of New York AXS-05 NMDA receptor antagonist and sigma-1 receptor agonist Alzheimer’s disease agitation Company plans to run 1 additional phase III study to support an NDA together with the Advance-1 study; new study will use a randomized-withdrawal design, which is scheduled to start in fourth quarter of 2020
Bluebird Bio Inc., of Cambridge, Mass. Elivaldogene autotemcel (Lenti-D) ABCD1 gene stimulator Cerebral adrenoleukodystrophy In ongoing ALD-104 study of therapy after myeloablative conditioning using busulfan and fludarabine, with median of 6.1 months of follow-up on 13 participants, all achieved neutrophil engraftment and 12 evaluable patients had platelet engraftment; no events of acute or chronic GvHD reported; 2 cases of pancytopenia, deemed by principal investigator as suspected unexpected serious adverse reactions and considered possibly related to therapy, were diagnosed about 2 months post-infusion in 2 patients 
Novan Inc., of Morrisville, N.C. SB-206 Nitricil nitric oxide-releasing polymer  Molluscum contagiosum infection Started the 750-patient B-Simple4 study testing the drug for 12 weeks with a follow-up at week 24; primary endpoint is proportion of patients with complete clearance of all treatable molluscum lesions at week 12
Regenerx Biopharmaceuticals Inc., of Rockville, Md. RGN-259 Thymosin beta 4 ligand  Dry eye syndrome After a delay due to the COVID-19 pandemic, enrollment and treatment of 700 patients in the Arise-3 study expected in November 2020
UCB SA, of Brussels Vimpat (lacosamide)  Dihydropyrimidinase related protein 2 modulator; sodium channel modulator Epilepsy Data, published in the Journal of Neurology, Neurosurgery & Psychiatry, showed study drug as adjunct for uncontrolled primary generalized tonic-clonic seizures (PGTCSs) in 242 participants met primary and secondary endpoints of lowering risk of developing second PGTCS during 24-week treatment (p<0.001) and higher rate of freedom from PGTCS during treatment period vs. placebo (31.3% vs. 17.2%, p=0.011)
VBI Vaccines Inc., Cambridge, Mass. Sci-B-Vac Tri-antigenic prophylactic hepatitis B vaccine HBV infection In the Constant study, Sci-B-Vac produced a geometric mean concentration of hepatitis B antibodies (anti-HBs) that was more than 7.5 times the titers generated by Engerix-B after 2 vaccinations (day 168) and more than 3 times after 3 vaccinations (day 196); seroprotection rate for Sci-B-Vac was 90.4% compared to 51.6% with Engerix-B after 2 doses; rate was 99.3% for Sci-B-Vac vs. 94.8% for Engerix-B after the third dose; in the Protect study, 80.8% of patients taking Sci-B-Vac had anti-HBs titers ≥ 100 mIU/mL compared to 60.7% of patients who received Engerix-B

Notes

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