Company Product Description Indication Status
Phase I
Adocia SA, of Lyon, France M1pram (ADO-09) Ready-to-use, fixed ratio co-formulation of pramlintide and A21G human insulin analogue Type 1 diabetes Preliminary results from extension part of phase Ib study showed treatment with individualized doses resulted in 69% decrease of glycemic excursion vs. Novolog (Novo Nordisk A/S) over first 4 hours after meal (primary endpoint; p=0.0266); pharmacological effect further confirmed by statistically significant decrease of glycemic excursion by more than 100% vs. Novolog over first 2 hours after meal (p<0.0001); over 23 days of treatment, average daily Time-in-Range as assessed by CGM, was statistically improved for patients treated vs. Novolog by 6% (p=0.0432); at end of treatment period, significant average weight loss of 1.6 kg vs. baseline observed vs. weight increase of 0.4 kg in Novolog group (p=0.0065)
Arbutus Biopharma Corp., of Warminster, Pa. AB-729 GalNAc-delivered RNAi compound Chronic hepatitis B virus infection Data from ongoing phase Ia/Ib study showed single subcutaneous injection of 90 mg resulted in mean HBsAg reduction of 1.23 log10 IU/mL at week 12
Atossa Therapeutics Inc., of Seattle AT-301 COVID-19 Spike glycoprotein inhibitor COVID-19 Interim data from first cohort of healthy subjects receiving drug administered via nasal spray showed positive safety profile to move on to next cohort
Atyr Pharma Inc., of San Diego, and Kyorin Pharmaceutical Co. Ltd., of Tokyo ATYR-1923 (KRP-R120) Immunomodulator Interstitial lung diseases Kyorin dosed first healthy subjects in Japan
Clinuvel Pharmaceuticals Ltd., of Melbourne, Australia Scenesse (afamelanotide) Melanocortin-1 receptor agonist Xeroderma pigmentosum Dosed first patient with XP under special access program
Codiak Biosciences Inc., of Cambridge, Mass. ExoIL-12 Designed to enhance dose control of IL-12 to localize to tumor microenvironment Early stage cutaneous T-cell lymphoma Initiated dosing; trial to enroll healthy volunteers and then transition to patients with early stage CTCL
Curasen Therapeutics Inc., of San Mateo, Calif. CST-2032 Brain-permeant, selective adrenoceptor modulator Neurodegenerative disease Dosed first subjects in multipart study in up to 70 healthy volunteers and patients with diseases such as Parkinson’s disease or mild cognitive impairment
Curevo Vaccine Inc., of Seattle CRV-101 Adjuvanted subunit shingles vaccine Shingles virus Antibody response data showed robust response against a key component (gE) of the shingles virus at 1 month after second vaccination, and at 1 year; plans to advance to phase IIb trial
Galecto Inc., of Boston GB-1211 Oral, small-molecule galectin-3 inhibitor Nonalcoholic steatohepatitis Drug was well-tolerated; phase IIa study planned
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole Inhalation Powder Next-generation, direct-to-lung, inhaled dry powder version of antifungal voriconazole Invasive pulmonary aspergillosis Top-line safety data found no clinically significant adverse events, laboratory test results, electrocardiograms, or ECGs, or vital signs in healthy volunteer study, according to data safety monitoring board; dosing also resulted in plasma levels greater than drug levels achieved in published reports for dose levels that were efficacious in IPA patients
Phase II
Aerpio Pharmaceuticals Inc., of Cincinnati Ocular formulation of razuprotafib Small-molecule inhibitor of vascular endothelial protein tyrosine phosphatase Open-angle glaucoma or ocular hypertension Completed patient enrollment in trial of patients with elevated intraocular pressure associated with OAG or OHT; data expected in fourth quarter of 2020
Opko Health Inc., of Miami Rayaldee (calcifediol) Extended-release oral formulation of prohormone of calcitriol COVID-19 Started Rescue study in mild to moderate COVID-19 infection; expected to enroll 160 subjects, many with stage 3 or 4 chronic kidney disease who are at higher risk for developing more severe illness 
Translational Research in Oncology, of Edmonton, Alberta, and Roche Holding AG, of Basel, Switzerland GDC-9545  Selective estrogen receptor degrader Metastatic breast cancer Enrolled first patient in trial sponsored by Roche to test combination with palbociclib in postmenopausal women with ER+ and HER2-negative untreated early breast cancer
Tyme Technologies Inc., of Bedminster, N.J. SM-88 Cancer metabolism-based therapy Prostate cancer Final results published in Investigational New Drugs showed drug had promising efficacy and safety outcomes for patients with nonmetastatic biochemical recurrent disease while sparing testosterone; from initial diagnoses of PSA rise, 100% of patients (23/23) remained free of metastatic progression and 87% of patients (20/23) have maintained radiographic progression-free survival with median duration of therapy of 6.5 months; all patients who have maintained meaningful reductions in circulating tumor cells (CTCs) on SM-88 were 100% free of any radiographic progression
Phase III
Axsome Therapeutics Inc., of New York AXS-05 Oral NMDA receptor antagonist Major depressive disorder Significantly improved patient-reported outcomes of depression in Gemini study; mean reductions from baseline in the Quick Inventory of Depressive Symptomatology Self-Report (QIDS-SR-16) total score of 7.8 points vs. 5.4 points for placebo, representing 48% and 34% reductions from baseline, respectively (p=0.001); rapidly and durably improved patient-reported depressive symptoms vs. placebo on the QIDS-SR-16 total score demonstrated at week 1 (p=0.016), the earliest time point assessed, at week 2 (p<0.001) and at all time points thereafter; clinical response on the QIDS-SR-16 (defined as ≥50% improvement) was statistically significantly greater vs. placebo at week 1 (p=0.048), at week 2 (p<0.001) and at every time point thereafter, being achieved by 53.4% of AXS-05 patients compared to 32.9% of placebo patients at week 6 (p<0.001)
Marinus Pharmaceuticals Inc., of Radnor, Pa. Ganaxolone Positive allosteric modulator of GABAA receptors CDKL5 deficiency disorder Top-line results from Marigold study in children and young adults showed median 32.2% reduction in 28-day seizure frequency vs. 4% for placebo achieving the primary endpoint (p=0.002); company plans to submit marketing applications to the FDA and EMA in 2021
Teva Pharmaceutical Industries Ltd., of Jerusalem Ajovy (fremanezumab-vfrm) CGRP receptor antagonist Chronic or episodic migraine Data from 52-week safety study testing monthly or quarterly injection showed most common adverse events (AEs) leading to discontinuation (3-5% of patients) included injection site erythema, injection site rash, injection site swelling, injection site pruritis and increased weight; no clinically significant patterns of AEs or serious AEs were seen; no treatment-emergent, clinically significant laboratory findings observed; results were published in Neurology


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