Phase I

Beigene Ltd., of Beijing


Anti-PD-1 antibody

Advanced solid tumors

In 300 patients across 8 cohorts of tumor types in phase Ib/IIa trial, overall response rate ranged from 8% to 19%

Cstone Pharmaceuticals Co. Ltd., of Suzhou, China


Anti-PD-L1 antibody

Microsatellite instability-high/deficient mismatch repair (MSI-H/dMMR) solid tumors

In the MSI-H/dMMR cohort of the Gemstone-101 phase Ib study, 8 of 21 (38.1%) evaluable patients achieved a partial response; disease control rate was 57.1%; median duration of response hasn't been reached

Mustang Bio Inc., of New York


CAR T targeting prostate stem cell antigen

Metastatic PSCA-positive castration-resistant prostate cancer

Started study of up to 33 patients; primary purpose is to evaluate safety and to determine the best dose; expansion and persistence of PSCA CAR T cells, clinical response based on Prostate Cancer Working Group 3 criteria, survival outcomes and serum cytokine profiles in peripheral blood pre- and post-therapy will be measured as secondary endpoints

TG Therapeutics Inc., of New York


Glycoengineered monoclonal antibody targeting an epitope on the CD20 antigen found on mature B-lymphocytes

Relapsed or refractory chronic lymphocytic leukemia or Richter's transformation

Ublituximab plus umbralisib and Keytruda (pembrolizumab, Merck & Co. Inc.) produced an overall response of 91% in 11 patients with a median of 2 prior therapies; ORR was 83% in 6 BTK-refractory CLL patients; ORR of 38% in 8 RT patients

Phase II

Acadia Pharmaceuticals Inc., of San Diego


Selective serotonin inverse agonist and antagonist preferentially targeting 5-HT2A receptors

Parkinson's disease patients with depressive symptoms

In the open-label, 47-patient study, 17-item Hamilton Depression Rating Scale (HAMD-17) total score improved from baseline to week 8 (p<0.0001); improvement seen as early as week 2 (p<0.0001); 60% of patients had an improvement of at least 50% on the HAMD-17 total score at week 8; 44.4% of patients reached remission (HAMD-17 ≤7); improvements were also seen in the Clinical Global Impression-Severity scale (baseline to week 8, p<0.0001) and the SCOPA-Global Sleep Quality scale (baseline to week 8, p<0.0001)

Aveo Oncology Inc., of Cambridge, Mass., and Eusa Pharma Inc., of Hemel Hempstead, U.K.

Fotivda (tivozanib)

VEGF receptor tyrosine kinase inhibitor

Advanced or metastatic renal cell carcinoma

Fotivda plus Opdivo (nivolumab, Bristol-Myers Squibb Co.) produced an overall median progression-free survival of 18.9 months in 25 patients treated at the study's full dose and schedule; median PFS for 12 previously untreated patients was 18.5 months, while median PFS for 13 previously treated patients had not yet been reached; objective response rate was 56%, including 1 complete response in a naïve patient; disease control rate was 96%

Beigene Ltd., of Beijing


Anti-PD-1 antibody

Advanced lung cancer

Tislelizumab plus a doublet chemotherapy produced overall response rate in 36 patients of 66.7%; ORR was 43.8% (7/16) in patients with nonsquamous non-small-cell lung cancer (NSCLC), 80% (12/15) in patients with squamous NSCLC (cohort A), 66.7% (4/6) in patients with squamous NSCLC (cohort B) and 76.5% (13/17) in patients with small-cell lung cancer (SCLC); median progression-free survival was 9 months in patients with nonsquamous NSCLC, 7 months in squamous NSCLC (cohort A), 6.9 months in SCLC, and in squamous NSCLC (cohort B) the median PFS had not yet been reached

Beigene Ltd., of Beijing


Anti-PD-1 antibody

Advanced esophageal, gastric or gastroesophageal junction carcinoma

Patients treated with tislelizumab plus cisplatin and fluorouracil had an overall response rate of 46.7%, including 7 of 15 patients with a partial response; median duration of response was 12.8 months; median progression-free survival was 10.4 months; median overall survival had not been reached

Beyondspring Inc., of New York


Sequesters tubulin heterodimers

Breast cancer

Increasing Neulasta (pegfilgrastim, Amgen Inc.) doses caused a statistically significant dose-dependent increase in thrombocytopenia (p<0.0001 for all grade thrombocytopenia); at 6 mg of Neulasta, the frequency of grades 1, 2 and 3 thrombocytopenia were 56%, 19% and 19%, respectively, compared to 20%, 7% and 0%, respectively for patients treated with plinabulin (20mg/m2)

Can-Fite Biopharma Ltd., of Petach Tikva, Israel


Binds A3 adenosine receptor

Second-line advanced hepatocellular carcinoma

Median overall survival wasn't improved for whole study population; in the subpopulation of 56 patients with Child Pugh B7 cirrhosis, median OS was 6.8 months for patients treated with namodenoson compared to 4.3 months for placebo; partial response rate was 9% for namodenoson and 0% for placebo; 1-year survival was 44% for namodenoson and 18% for placebo (p=0.028)

Cerevel Therapeutics LLC, of Boston


Selective partial agonist of the dopamine D1 and D5 receptors

Early stage Parkinson's disease

In 57 patients, mean change from baseline at week 15 in the MDS-UPDRS Part III score was -9 for tavapadon and -4.3 for placebo (p=0.0407); 50% of patients treated with tavapadon reported being "much improved" or "very much improved" on the PGI-C scale, compared with 25% in the placebo group; daytime sleepiness on the Epworth Sleepiness Scale at weeks 9 and 15 were similar for tavapadon and placebo

Entera Bio Ltd., of Jerusalem

Oral hPTH(1-34)

Parathyroid hormone


Twice-daily, 3 times daily and 4 times daily produced a dose-dependent increase in 1,25-dihydroxyvitamin D; 4 times daily dose also increased serum albumin-corrected calcium and decreased serum phosphate and urinary calcium

Mitsubishi Tanabe Pharma Corp., of Jersey City, N.J.


Continuous delivery of carbidopa/levodopa via subcutaneous infusion

Parkinson's disease

Secondary, patient-reported outcomes showed those in the continuous 24-hour treatment group reported statistically significant change in United Parkinson Disease Rating Scale ADL score vs. baseline (p=0.02); quality of life also improved in that group (p=0.02) and patients reported improvement in 6 of 8 Parkinson's disease Questionnaire quality of life domains

Neurana Pharmaceuticals Inc., of San Diego


Blocks voltage-gated sodium and calcium channels

Acute muscle spasms of the back

In the 415-patient Star study, the 600-mg dose improved subject-rated pain due to acute back spasm using a Numerical Rating Scale (p=0.004); plans to start a phase III study in 2020

Ovid Therapeutics Inc., of New York


Inhibits cholesterol 24-hydroxylase

Rare developmental and epileptic encephalopathies

In the Endymion extension study, 6 patients had an 84% reduction in seizure frequency from baseline for weeks 25-36; 4 patients had a 90% reduction in seizure frequency from baseline for weeks 37-48

TG Therapeutics Inc., of New York


Glycoengineered monoclonal antibody targeting an epitope on the CD20 antigen found on mature B-lymphocytes

Chronic lymphocytic leukemia intolerant to prior BTK or PI3K delta inhibitor therapy

Estimated median progression-free survival of 50 evaluable patients was 23.5 months; overall survival had not been reached at a median follow-up of 15.7 months; 58% of patients have been on umbralisib for a duration longer than their prior BTK or PI3k inhibitor

Phase III

Gensight Biologics SA, of Paris


Gene therapy

Leber hereditary optic neuropathy

Results from week 96 of Rescue trial testing single intravitreal injections in 39 patients whose vision loss due to 11778-ND4 LHON began up to 6 months prior to treatment showed best-corrected visual acuity sustaining clinically meaningful improvement over nadir; treated eyes regained more than two-thirds of initial loss occurring in most acute phase of the disease; that improvement from nadir (-0.498 LogMAR mean improvement, or +24.9 ETDRS letters equivalent) corresponds to 5 lines of Snellen acuity, above the 3-line threshold commonly accepted as clinically meaningful level of visual improvement

Ipsen SA, of Paris


Injectable form botulinum neurotoxin type A

Upper and lower limb spasticity

Results from Engage study showed improvement in patients' voluntary movement as measured by composite active range of motion outcome; 72.1% were classified as responders

Mallinckrodt plc, of Staines-upon-Thames, U.K.


Regenerative tissue

Deep partial-thickness thermal burns

Top-line results showed pivotal trial met both primary endpoints; data showed significantly smaller area of burn wounds treated with Stratagraft required autografting by 3 months vs. those treated exclusively with autograft (p<0.0001); proportion of Stratagraft-treated wounds achieved durable wound closure at 3 months, exceeding predefined threshold for statistical significance; BLA expected to be submitted in second half of 2020

Rigel Pharmaceuticals Inc., of South San Francisco, and Kissei Pharmaceuticals Co. Ltd., of Tokyo

Fostamatinib disodium hexahydrate

SYK inhibitor

Chronic immune thrombocytopenia

Kissei started trial in Japan


For more information about individual companies and/or products, see Cortellis.


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