Company Product Description Indication Status
Phase I

Bicycle Therapeutics plc, of Cambridge, U.K.

BT-5528

Bicycle toxin conjugate targeting tumor antigen EphA2

Advanced solid tumors associated with EphA2 expression

Dosed first patient in phase I/II study

Phase II

Aivita Biomedical Inc., of Irvine, Calif.

AV-GBM

Patient-specific immunotherapy

Glioblastoma

Data from ongoing trial in newly diagnosed patients showed survival of 96% at 6 months and 91% at 12 months, with 3 patients followed for more than a year; a vast majority displayed appropriate immune response and decrease of tumor biomarkers

Cassiopea SpA, of Lainate, Italy

Clascoterone solution

Topical androgen receptor inhibitor

Androgenetic alopecia in females

Received approval from German authority BfArM and coordinating ethical committee for a phase II trial

Millendo Therapeutics Inc., of Ann Arbor, Mich.

Livoletide

Analogue of unacylated ghrelin

Prader-Willi syndrome

Phase IIb portion of pivotal phase IIb/III Zephyr study completed recruitment for patients ages 8 to 65; more than 150 patients enrolled; primary endpoint is an assessment impact on hyperphagia

Oncoceutics Inc., of Philadelphia

ONC-201

Imipridone

Glioblastoma

Results from second and third cohorts were published in Neuro-Oncology showing ONC-201 achieved intratumoral concentrations that exceeded preclinical efficacy thresholds and induced intratumoral biomarkers of pharmacodynamic signaling and apoptosis, indicating target engagement; radiographic regression of multifocal, multirecurrent disease in an adult recurrent H3 K27M-mutant glioblastoma patient remained durable for >1.5 years; no treatment modifications or discontinuations due to toxicity were observed, including those who underwent reresection

Palvella Therapeutics Inc., of Wayne, Pa.

PTX-022

Qtorin 3.9% rapamycin anhydrous gel

Pachyonychia congenita

Pivotal portion of Valo study is enrolling patients who met prespecified clinical response criteria during phase II portion; they will be assigned to 1 of 3 arms: placebo, twice-daily, or once-daily PTX-022

Remegen Ltd., of Yantai, China

RC-18 (telitacicept)

TACI-Fc fusion protein

Systemic lupus erythematosus

Phase IIb trial met primary endpoint of a greater than 4-point reduction in the SLE responder Index (SRI4); 75.8% of patients achieved clinically meaningful disease activity improvement (p<0.001), as compared to placebo (33.9%)

Samumed LLC, of San Diego

Lorecivivint

CLK/DYRK1A inhibitor that modulates the Wnt pathway

Knee osteoarthritis

Drug appeared safe and well-tolerated in pooled analysis of 3 trials that evaluated a single intra-articular injection

Therapix Biosciences Ltd., of Tel Aviv, Israel

THX-110

Combination of dronabinol (delta-9-tetrahydrocannabinol) and Cannamide (palmitoylethanolamide)

Obstructive sleep apnea

Top-line phase IIa results show of 9 patients who completed trial, 55% demonstrated significant improvement in Apnea-Hypopnea Index values (t-test; AHI mean difference 0.013, p<0.05)

Notes

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

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