Company Product Description Indication Status
Phase I

Deciphera Pharmaceuticals Inc., of Waltham, Mass.


CSF-1 antagonist

Tenosynovial giant cell tumor

Preliminary data from ongoing study in 3 individuals with diffuse-type TGCT enrolled in dose-escalation portion showed tumor reductions from baseline of 48%, 25% and 24%, respectively, with 1 confirmed partial response sustained for 9 months and ongoing who showed 84% tumor reduction from baseline to cycle 10 day 1; symptomatic improvements in mobility and reduced pain also reported

Molecular Templates Inc., of Austin, Texas, and Translational Research in Oncology, OF Edmonton, Alberta


EGFR modulator; Erbb2 tyrosine kinase receptor modulator

HER2-positive solid tumors

Enrolled first participant in study of MT-5111 as monotherapy in individuals with advanced disease to evaluate safety and tolerability and determine recommended phase II dose

VBI Vaccines Inc., of Cambridge, Mass., and Brii Biosciences Inc., of Durham, N.C.

BRII-179 (VBI-2601)

Recombinant, protein-based virus-like particle vaccine

Hepatitis B virus infection

Dosed first of expected 65 participants in phase Ib/IIa study assessing safety, tolerability and antiviral activity; initial data expected in second half of 2020

Phase II

Aadi Bioscience Inc., of Pacific Palisades, Calif.

Nab-sirolimus (ABI-009)

mTOR inhibitor

Perivascular epithelioid cell tumor

Based on 31 patients evaluable for efficacy, Ampect trial showed 39% confirmed independent radiology reviewed objective response rate in advanced PEComa; half the responders had ongoing response duration of 15.3 months or > and 67% of responders remain on treatment; 90% had best response of partial response or stable disease (SD), with disease control (confirmed response + SD ≥12 weeks) achieved in 71%

Arvelle Therapeutics GmbH, of Zug, Switzerland


Sodium channel inhibitor

Focal-onset epilepsy

Findings published in The Lancet Neurology showed drug, dosed at 100 mg, 200 mg and 400 mg/day, improved seizure control vs. placebo in those taking 1-3 anti-epileptic drugs and achieved responder rates during 12-week maintenance phase of 40% (p=0.036), 56% (p<0.001) and 64% (p<0.001), respectively, vs. 25% for placebo; during maintenance phase, 4% (not significant), 11% (p=0.002) and 21% (p<0.001) who received cenobamate 100 mg, 200 mg and 400 mg, respectively, reported 0 focal-onset seizures vs. 1% for placebo

Bellerophon Therapeutics Inc., of Warren, N.J.


Inhaled nitric oxide drug-device

Pulmonary hypertension associated with interstitial lung disease

In acute, dose-escalation study, pulmonary vascular resistance improved by 29% vs. baseline of 584 dynes × sec × cm-5; cardiac output improved by 16% vs. baseline of 3.5 L/min; mean pulmonary arterial pressure improved by 10% across doses vs. baseline of 34.3 mmHg; oxygen saturation remained stable across doses

Biomarin Pharmaceutical Inc., of San Rafael, Calif.


C-type natriuretic peptide ligand


Over 54 months, children in cohort 3 (n=10) of ongoing, open-label study, at dose of 15 µg/kg/day, achieved statistically significant (p<0.005) cumulative additional mean height gain of 9 cm compared to those, matched for age and gender, in natural history achondroplasia dataset (n=619); 2.2 cm of height gain occurred in most recent 12 months

Jaguar Health Inc., of San Francisco

Mytesi (crofelemer)

Botanical drug extracted from Croton lechleri; calcium activated chloride channel inhibitor


At interim analysis, Halt-D study evaluating Mytesi for prevention and prophylaxis of diarrhea in HER2-positive breast cancer patients on chemotherapy regimens was approved by data safety monitoring committee to enroll to completion

Trovagene Inc., of San Diego


Polo-like kinase 1 inhibitor

Metastatic castration-resistant prostate cancer

As of Oct. 28 data cutoff in combination trial with Zytiga (abiraterone acetate, Johnson & Johnson)/prednisone, 60% response rate (stable disease + partial response) observed among 15 participants who completed 3 months of treatment; 72% had decrease in PSA following 1 treatment cycle, including 5 who tested positive for AR-V7 variant; of those 5, primary efficacy endpoint (SD + PR) was achieved in 3 of 4 evaluable participants

Vistagen Therapeutics Inc., of South San Francisco


NMDA glycine antagonist

Major depressive disorder

In Elevate study of 199 participants, treatment arm did not differentiate from placebo on primary endpoint of change in Montgomery-Åsberg Depression Rating Scale total score compared to baseline

Zealand Pharma A/S, of Copenhagen, Denmark


Glucagon peptide analogue

Serious meal-induced hypoglycemia following bariatric surgery

Started proof-of-concept trial testing minidoses of the drug

Phase III

Acelrx Pharmaceuticals Inc., of Redwood City, Calif.

Sufentanil sublingual tablet 30 mcg (Dsuvia)

Opioid receptor mu agonist


Pooled dosing and efficacy data among multiple demographic subgroups, published in Journal of PeriAnesthesia Nursing, showed 3.9 as mean number of doses administered from 0-12 hours, dosing was less frequent for those ≥65 years compared to younger patients but similar among other subgroups; summed pain intensity difference to baseline over 12 hours was higher for study drug compared with placebo across subgroups

Bavarian Nordic A/S, of Copenhagen, Denmark

Jynneos (MVA-BN)

Smallpox vaccine

Smallpox infection prophylaxis

Data published in The New England Journal of Medicine showed Jynneos induced neutralizing antibody titers comparable with ACAM2000 at day 14; peak neutralizing antibodies induced by Jynneos were almost 2-fold higher on average than those stimulated by ACAM2000

Everest Medicine Inc., of New York


Oral sphingosine 1-phosphate receptor modulator

Moderately to severely active ulcerative colitis

Dosed first patient in trial assessing etrasimod for induction and maintenance therapy; about 330 patients will be enrolled in mainland China, South Korea and Taiwan

Neurocrine Biosciences Inc., of San Diego

Ingrezza (valbenazine)

Vesicular monoamine transporter 2 inhibitor

Moderate or severe tardive dyskinesia

Data from the Kinect 4 study published in the Journal of Clinical Psychopharmacology showed 90% and 89.2% of patients on 40 mg or 80 mg of Ingrezza, respectively, achieved ≥50% improvement from baseline in the Abnormal Involuntary Movement Scale total score after 48 weeks of treatment; 89%-95% of patients achieved a Clinical Global Impression of Change-TD or Patient Global Impression of Change response of "much improved" or "very much improved"

Phase IV

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

Acthar Gel (repository corticotropin)


Rheumatoid arthritis

After 12 weeks of treatment, Functional Assessment of Chronic Illness Therapy – Fatigue was reduced by 8.7 points, Health Assessment Questionnaire – Disability Index was reduced by 0.84 points, patient global assessment of disease activity was reduced by 35 points and patient global assessment of pain was reduced by 37.4 points (p<0.001 vs baseline for all)


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