Company Product Description Indication Status
Phase I
Adaptimmune Therapeutics plc, OF Oxfordshire, U.K. ADP-A2M4 Melanoma associated antigen 4 modulator Synovial sarcoma Updated data from 16 participants at Sept. 1, 2020, cutoff date showed 7 (44%) had confirmed partial responses (PRs) per RECIST criteria, with disease control in 15 (94%); median duration of response was 28 weeks (range: 12-72+ weeks), with 2 PRs ongoing >72 weeks at cutoff; 11 were alive at cutoff and median overall survival was not reached
Affimed NV, of Heidelberg, Germany AFM-13 CD30/CD16A innate cell engager Relapsed/refractory Hodgkin lymphoma Phase Ib data published in Blood showed combination with Keytruda (pembrolizumab, Merck & Co. Inc.) resulted in objective response rate of 88% at highest treatment dose, as well as a complete remission of 46%; that compares with Keytruda monotherapy, which demonstrated an ORR of 69% and a CR of 22.4% in the KEYNOTE-087 trial
Bio-Path Holdings Inc., of Houston BP-1002 BCL2 gene inhibitor Lymphoma; chronic lymphocytic leukemia First of 6 evaluable participants dosed; primary objectives include safety and tolerability of escalating doses, recommended phase II dose, pharmacokinetics and activity on BCL2 expression, with secondary endpoints including efficacy measurements of tumor response
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Icahn School of Medicine at Mount Sinai Ofev (nintedanib) Tyrosine kinase inhibitor COVID-19-related acute lung injury First of 120 participants enrolled in Endcov-I study; primary endpoint is percent change in FVC compared to baseline over 180 days; secondary endpoints include change from baseline FVC at 90 days, death within 90 days/180 days from enrollment due to respiratory or any cause, and qualitative/quantitative change in chest CT fibrosis score
Day One Biopharmaceuticals LLC, of South San Francisco DAY-101 Pan-RAF inhibitor Relapsed low-grade glioma Results from 9 patients under 18 showed, of 8 with RAF fusions, 2 achieved a complete response by Response Assessment for Neuro-Oncology, 3 had a partial response and 3 achieved prolonged stable disease; median time to response for those patients was 10.5 weeks
Elevar Therapeutics Inc., of Salt Lake City Rivoceranib VEGF-2 receptor antagonist Solid tumors Combination study with nivolumab (Opdivo, Bristol Myers Squibb Co.) in 30 people with locally advanced unresectable or metastatic  disease showed overall response rate of 13.3% and disease control rate of 76.7%; median progression-free survival was 7.2 months; partial response seen in 4 participants (13.3%)
GH Research Ltd., of Dublin GH-001 (5-methoxy-N,N-dimethyltryptamine) 5-HT 2a receptor agonist Depression Study in healthy volunteers showed candidate was safe and well-tolerated and identified dose range and dosing regimen for phase I/II trial
Nordic Nanovector ASA, of Oslo, Norway Betalutin (177Lu lilotomab satetraxetan) B-lymphocyte antigen CD20 stimulator; CD37 antagonist Follicular lymphoma  First participant enrolled in second safety cohort of Archer-1 (LYMRIT 37-07) combination trial with rituximab in second-line disease
Northsea Therapeutics BV, of Naarden, the Netherlands SEFA-1024 (structural engineered fatty acid) Lipid metabolism modulator Dyslipidemia First participant dosed in trial assessing study drug in 96 otherwise healthy volunteers with elevated plasma triglycerides
Oncolytics Biotech Inc., of San Diego Pelareorep  Oncolytic retrovirus Glioblastoma Investigator-sponsored phase Ib Reoglio trial showed evaluable participants treated at dose level-2 (3x1010 TCID50) had estimated median progression-free survival (PFS) of 9.4 months; those evaluable at dose level-1 (1x1010 TCID50) had estimated median PFS of 6.1 months; estimated median PFS of all evaluable patients was 7.8 months
Plus Therapeutics Inc., of Austin, Texas Rhenium nanoliposome (RNL) Nanoliposome-encapsulated radionuclide Glioblastoma In Respect trial, median survival in people who previously received bevacizumab (n=7) was 4.8 months while median and mean survival durations in bevacizumab-naïve (n=8) are currently 11 months (range 3.5–33) and 15.4 months, respectively, with 4 still alive
Y-mabs Therapeutics Inc., of New York Omburtamab CD276 antigen inhibitor Diffuse intrinsic pontine glioma Ongoing study suggested possibility of repeated convection enhanced delivery (CED) infusions into pediatric brainstem, showing that previous CED treatments did not negatively influence procedural work flow, technical application of targeted interface, accuracy of catheter placement or distribution capacity; 7 study participants underwent 2 or more sequential CED infusions
Ziopharm Oncology Inc., of Boston INXN-2001 + veledimex IL12 gene stimulator Glioblastoma Interim data from ongoing combination trial with PD-1 inhibitor nivolumab (Opdivo, Bristol Myers Squibb Co.) in recurrent disease showed median overall survival (mOS) for 10-mg veledimex cohorts (n=6; 83% unifocal, 67% low-dose steroids) was 16.9 months; mOS among all participants (n=21 across 10-mg and 20-mg doses) was 9.8 months
Ziopharm Oncology Inc., of Boston INXN-2001 + veledimex IL12 gene stimulator Diffuse intrinsic pontine glioma  Data from first participant in ongoing phase I/II study showed increase in serum recombinant IL-12 not detected after initial dosing at 5 mg per day but endogenous IFN-gamma was detected, peaking at day 3; circulating cytotoxic T-lymphocyte levels increased between days 7 and 28
Phase II
Acurx Pharmaceuticals LLC, of White Plains, N.Y. Ibezapolstat DNA polymerase III inhibitor Clostridium difficile infection In segment IIa of trial, 10 people with diarrhea caused by C. diff dosed with study drug at 450 mg orally for 10 days met primary and secondary efficacy endpoints of clinical cure at end of treatment and sustained clinical cure of no recurrence at 28-day follow-up visit; in segment IIb, about 64 additional people with CDI will be randomized 1-to-1 to ibezapolstat 450 mg every 12 hours or vancomycin 125 mg every 6 hours for 10 days and followed for 28 ± 2 days for recurrence
Astrazeneca plc, of Cambridge, U.K.  AZD-1222 COVID-19 Spike glycoprotein modulator COVID-19 infection Phase II component of ongoing phase II/III trial, published in The Lancet, showed candidate prompted equivalent immune response in healthy people age 70 to that seen in those ages 18 to 55, and older participants showed fewer side effects
CASI Pharmaceuticals Inc., of Rockville, Md. CNCT-19 (HY-001) B-lymphocyte antigen CD19 modulator Non-Hodgkin lymphoma Partner Juventas Cell Therapy Ltd. enrolled first participant in registration study in China in relapsed/refractory B-cell disease
Day One Biopharmaceuticals LLC, of South San Francisco DAY-101 Pan-RAF inhibitor Recurrent or progressive BRAF-altered low-grade gliomas Started single-arm, monotherapy, registration-enabling study in children and young adults
Gannex Pharma Co. Ltd., a unit of Shanghai-based Ascletis Pharma Inc., and Sagimet Biosciences Inc., of San Francisco ASC-40 (TVB-2640)  FASN inhibitor Nonalcoholic steatohepatitis Completed enrollment of the 30-patient study in China
Glaxosmithkline plc, of London, and Medicines for Malaria Venture Tafenoquine Succinate salt Plasmodium vivax infection Phase IIb Teach study in 60 children ages 6 months to 15 years showed no recurrence of P. vivax malaria in 95% of participants during 4 months of follow-up
Immutep Ltd., of Sydney, and Merck & Co. Inc., Kenilworth, N.J. Eftilagimod alpha and Keytruda (pembrolizumab) LAG-3 fusion protein First-line non-small-cell lung cancer Expanding the Tacti-002 study to include an additional 74 patients; plans to enroll first patient in the expanded trial by the end of 2020
Kintara Therapeutics Inc., of San Diego VAL-083 Bifunctional DNA-targeting agent MGMT-unmethylated glioblastoma multiforme Progression-free survival for 27 evaluable newly diagnosed patients taking VAL-083 as adjuvant therapy following treatment with radiation and TMZ was 10 months; in 43 evaluable patients who had failed first-line TMZ treated at the phase III dose, PFS was 8.5 months
Redhill Biopharma Ltd., of Tel Aviv, Israel Yeliva (opaganib/ABC-294640) Sphingosine kinase-2 selective inhibitor Hospitalized severe COVID-19 pneumonia Independent data and safety monitoring board recommended continuing the phase II/III study based on safety data from the first 70 patients treated for 14 days; futility interim analysis expected in the coming weeks; top-line data expected in the first quarter of 2021
Swedish Orphan Biovitrum AB, of Stockholm, and Apellis Pharmaceuticals Inc., of Waltham, Mass. Pegcetacoplan Targets C3 of the complement cascade Amyotrophic lateral sclerosis First of about 200 patients treated in the potentially registrational Meridian study; primary endpoint is the Combined Assessment of Function and Survival rank scores at week 52; secondary endpoints include measures of lung function, muscle strength and quality of life
VBI Vaccines Inc., of Cambridge, Mass. VBI-1901 Cancer vaccine immunotherapeutic Recurrent glioblastoma Disease control rate of 40% in 10 patients treated with VBI-1901 + GM-CSF, including 2 partial responses and 2 patients with stable disease; disease control rate of 56% in 9 patients treated with VBI-1901 + AS-01 adjuvant (Glaxosmithkline plc), all stable disease
Ziopharm Oncology Inc., of Boston Ad-RTS-hIL-12 plus veledimex Controlled IL-12 Recurrent glioblastoma  Median overall survival for patients treated with controlled IL-12 plus cemiplimab hasn't been reached, with mean a follow-up time of 6.5 months; median OS for 6 patients treated with controlled IL-12 with 10-mg veledimex plus nivolumab was 16.9 months; mean OS for all patients treated with controlled IL-12 with 10-mg or 20-mg veledimex plus nivolumab was 9.8 months
Phase III
Aravive Inc., of Houston AVB-500  Neutralizes GAS6 activity Platinum-resistant ovarian cancer Designing a study expected to enroll about 300-400 patients who have received 1 to 4 prior lines of therapy;  primary endpoint is progression-free survival; secondary endpoints include overall survival, objective response rate, duration of response, quality of life, clinical benefit rate and pharmacokinetic and pharmacodynamic profile
Ocuphire Pharma Inc., of Farmington Hills, Mich. Nyxol  Formulation of phentolamine mesylate Pharmacologically induced mydriasis Enrolled first patient in the Mira-2 study, the first of 4 upcoming late-stage studies
Pfizer Inc., of New York Lorbrena (lorlatinib) ALK tyrosine kinase inhibitor Previously untreated anaplastic lymphoma kinase-positive advanced non-small-cell lung cancer In the Crown study, Lorbrena reduced the risk of progression or death by 72% compared to Xalkori (crizotinib) (p<0.001); study is continuing for the secondary endpoint of overall survival
Sifi SpA, of Catania, Italy Polihexanide 0.08% Polymer Acanthamoeba keratitis infections Reached full enrollment of 135 patients; top-line results expected in the second half of 2021
Uniqure NV, of Lexington, Mass. Etranacogene dezaparvovec Gene therapy expressing Padua variant of factor IX Severe and moderately severe hemophilia B In the Hope-B study of 54 patients, mean factor IX (FIX) activity was 37% of normal at 26 weeks; mean annualized usage of FIX replacement therapy declined by 96% after dosing compared to the observational lead-in period; increases in FIX activity sustained for up to 18 months
Urovant Sciences Inc., of Irvine, Calif. Vibegron  Beta-3 adrenergic agonist Overactive bladder In the Empowur long-term extension study, at week 52, 61% of 143 patients treated with vibegron had a ≥75% reduction and 40.8% had a 100% reduction in urge urinary incontinence; 71.1% had ≥50% reduction in total incontinence episodes from baseline to week 52

Notes

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