Company Product Description Indication Status
Phase I
Arch Biopartners Inc., of Toronto Metablock LSALT peptide Acute kidney injury in patients undergoing cardiac surgery Phase I completed dosing of all scheduled volunteers; met primary endpoints of safety and tolerability; no drug-related adverse events observed in 44 people
Chondrial Therapeutics, of Bala Cynwyd, Pa. CTI-1601 Recombinant fusion protein intended to deliver human frataxin Friedreich's ataxia First patients have been dosed in a phase I double-blind, placebo-controlled trial; FDA granted rare pediatric disease designation and fast track designation
Cullinan Oncology LLC, of Cambridge, Mass. VK-2019 First-in-class EBNA1 inhibitor  Epstein-Barr virus positive (EBV+) cancers  Dosing initiated; in preclinical models of EBV-associated cancers, VK-2019 eliminated EBV, resulting in tumor growth inhibition
Cullinan Oncology LLC, of Cambridge, Mass.  CLN-081 Epidermal growth factor tyrosine kinase inhibitor Non-small-cell lung cancer  Dosing initiated; it was engineered to inhibit EGFR variants with exon 20 insertion mutations, while sparing wild-type EGFR; CLN-081 is initially in development to target NSCLC driven by EGFR exon 20 insertion mutations
Rezolute Inc., of Redwood City, Calif. AB-101 Ultra-long basal insulin Type I and II diabetes Top-line phase I data showed a slow onset and sustained insulin levels and activity for more than 7 days when administered subcutaneously in three ascending dosing cohorts; at higher doses, the necessary drug volume was greater than anticipated; company intends to conduct additional formulation development before advancing
Solid Biosciences Inc., of Cambridge, Mass. SGT-001 Adeno-associated viral vector-mediated gene transfer Duchenne muscular dystrophy  Data from 2 patients dosed in the 2nd cohort of IGNITE DMD showed SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase function, providing evidence that SGT-001 has the potential to provide therapeutic benefit; also, a previously reported SAE experienced by a 3rd patient in the 2E14 vg/kg dose group has fully resolved; Solid received a clinical hold letter from the FDA and will continue working to address the  hold and determine a path forward
Zenith Epigenetics Ltd., of Calgary, Alberta ZEN-3694 BET inhibitor Metastatic castration-resistant prostate cancer Completed the phase Ib/II, ZEN-3694 plus enzalutamide combination trial in patients who had progressed on an androgen receptor (AR) antagonist; data showed it provided a significant and meaningful radiographic progression-free survival benefit of 44 weeks vs. 24 weeks for patients receiving only the AR antagonist
Zenith Epigenetics Ltd., of Calgary, Alberta ZEN-3694 BET inhibitor Triple-negative breast cancer Due to clinical activity in some patients dosed to date, Zenith is exploring the potential expansion to include additional tumor types that may respond to ZEN-3694 plus Pfizer Inc.'s talazoparib, a poly-ADP ribose polymerase inhibitor
Phase II
Avrobio, of Cambridge, Mass. AVR-RD-01 Gene therapy Fabry disease First patient was dosed using plato gene therapy platform, which is intended as a foundation for commercialization of the company's gene therapies; the patient is enrolled in FAB-201, a phase II trial
Chemiocare U.S.A. Inc., of New York CMIO-OLNZ 7-day matrix transdermal delivery system of olanzapine Poly-ADP ribose polymerase inhibitor induced nausea and vomiting First-in-woman study demonstrated it delivers target therapeutic levels of olanapine over 7 days, is well-tolerated to the skin, adheres to the skin over the dosing interval with easy removal, and has lower overall sedation side effect intensity when compared to Zyprexa oral; the product is ready to advance to phase III
Follica Inc., of Boston Follica hair follicle neogenesis Designed to stimulate new hair follicles; device enhanced with a topical compound Male androgenetic alopecia Top-line results from its safety and efficacy optimization study show it successfully met its primary endpoint, demonstrating a statistically significant 44% improvement of visible hair count after three months of treatment compared to baseline (p<0.001); overall improvement of visible hair count after 3 months was 29% compared to baseline (p<0.001); a phase III trial is expected in the first half of 2020
Inventiva, of Daix, France Odiparcil Orally-available small molecule Mucopolysaccharidosis type VI Phase IIa Improves study data showed the safety primary objective was met and that positive results were observed regarding efficacy, especially in hard-to-reach tissues; odiparcil has orphan drug designation in the U.S. and Europe, as well as rare pediatric disease designation in the U.S.
Minerva Neurosciences Inc., of Waltham, Mass. MIN-117 Antidepressant targeting adrenergic alpha 1a, alpha 1b, 5-HT1A, 5-HT2A receptors, serotonin and the dopamine transporter Moderate to severe major depressive disorder Failed to meet its primary and key secondary endpoints in a phase IIb trial, although it was generally well-tolerated with a safety profile comparable to placebo; neither dose showed a statistically significant separation from placebo on the reduction in the symptoms of MDD over the 6-week treatment period as measured by the change in the Montgomery Asberg Depression Rating Scale or on the reduction of symptoms of anxiety as measured by Hamilton Anxiety Rating Scale over the 6 weeks
Nicox SA, of Sophia Antipolis, France NCX-4251 Ophthalmic suspension of fluticasone propionate nanocrystals Acute exacerbations of blepharitis Met the primary objective of selecting the dose, 0.1% once daily, in its first-in-human, phase II trial, Danube; company plans to move into a larger phase IIb trial; dose demonstrated efficacy in reducing signs and symptoms of blepharitis and dry eye disease
Qu Biologics Inc., of Vancouver, British Columbia QBECO-SSI Site specific immunomodulator Moderate to severe Crohn's disease Completed enrollment for the first stage of the Restore phase II clinical trial; 20 patients will be treated for up to 52 weeks
Windmil Therapeutics, of Baltimore and Philadelphia, and Bristol-Myers Squibb Co., of Princeton, N.J. MILs and Opdivo Marrow-infiltrating lymphocytes and nivolumab, a PD-1 immune checkpoint inhibitor Locally advanced unresectable or metastatic non-small-cell lung cancer Parties entered into a new clinical research collaboration for an open-label, multicenter phase IIa trial of MILs alone and in combination with Opdivo; the primary endpoint is objective response rate
Phase III
Aldeyra Therapeutics Inc., of Lexington, Mass. ADX-2191 Inhibits cell growth diminishing scar formation To prevent proliferative vitreoretinopathy Enrolled the first patient in the phase III Guard trial; ADX-2191 has fast track designation and orphan drug designation in the U.S.
Selecta Biosciences Inc., of Waterton, Mass. SEL-212 ImmTOR + pegadricase; designed to control serum uric acid levels Chronic refractory gout Completed patient enrollment in the 6-month, head-to-head, phase II Compare clinical trial with 150 patients, comparing SEL-212 vs. a bi-weekly dose of pegloticase; primary endpoint is maintenance of serum uric acid levels of <6mg/dL at 3 and 6 months; top-line results are expected by mid-2020
Phase IV
Vivus Inc., of Campbell, Calif. Qsymia Phentermine and topiramate extended-release capsules CIV Obesity Phase IV post-marketing study in obese adolescents showed it had a favorable pharmacokinetic, efficacy and safety/tolerability profile when used for 8 weeks; both mid and top doses were found to be appropriate for longer-term study; PK analyses showed exposure was comparable to what was seen in adults; significant differences from baseline to day 56 were observed with respect to mean percentage change in weight for both Qsymia groups compared with placebo (-3.72%, -4.96%, and +1.06%), as well as for mean change in waist circumference and hunger scores

Notes

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

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