Company

Product

Description

Indication

Status

Phase I

Bellus Health Inc., of Laval, Quebec

BLU-5937

Oral P2X3 antagonist

Refractory chronic cough

Results showed drug is well-tolerated, achieved dose levels required for optimal efficacy in preclinical models and, at anticipated therapeutic doses of 50 mg-100 mg BID, did not cause any loss of taste perception, with only 1 subject reporting transient taste alteration

Crinetics Pharmaceuticals Inc., of San Diego

CRN-01941

Somatostatin 2 receptor agonist

Neuroendocrine tumor

Initiated double-blind, randomized, placebo-controlled, single- and multiple-dose study to evaluate safety, pharmacokinetics and pharmacodynamics in up to 119 healthy volunteers

Debiopharm SA, of Lausanne, Switzerland

Debio-1143

X-linked inhibitor of apoptosis protein inhibitor

Advanced solid tumors

Enrolled first patient in SMARTPLUS-106 exploratory study investigating safety and efficacy, in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.), in indications such as small-cell lung cancer or squamous cell carcinoma of the head and neck, in individuals who progressed during or following anti-PD-1/PD-L1 treatment

Exuma Biotechnology Secz, of Grand Cayman, and affiliate Shanghai Perhum Therapeutics

CCT-301-38 and CCT-301-59

Axl tyrosine kinase receptor modulator and NKTR2 tyrosine kinase

Metastatic renal cell carcinoma

Interim data from umbrella trial of combination showed CAR T product blood exposure up to 80,000 copies/μg at 1 x 106/kg dose and early radiologic evidence of antitumor activity, with stable disease as best response; in heavily pre-treated population, 6 of 7 participants with recurrent or refractory stage IV mRCC were alive with median follow-up of 140 days; no dose-limiting toxicities observed to date, with no indications of on-target, off-tumor toxicity attributed to either product

Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa.

INO-4500

DNA-based vaccine

Lassa fever

Dosed first of 60 healthy volunteers in dose-escalation trial evaluating safety, tolerability and immune responses to prevent infection

Nordic Nanovector ASA, of Oslo, Norway

Betalutin (177Lu-satetraxetan-lilotomab)

CD37 antagonist

Follicular lymphoma

Following safety review of first participants in ongoing open-label, phase Ib Archer-1 trial in combination with rituximab in second-line disease, dose of the study drug was escalated to 15 MBq/kg for next patient cohort

Rainier Therapeutics Inc., of San Leandro, Calif.

Vofatamab

FGF3 receptor antagonist

Metastatic bladder cancer

Data from FIERCE-22 trial showed study drug up-regulated genes associated with inflammation in both wild-type and FGFR3 mutant tumors and, in combination with Keytruda (pembrolizumab, Merck & Co. Inc.), was associated with increased response rate

Theravance Biopharma Inc., of Dublin

TD-1473

Pan-JAK inhibitor

Ulcerative colitis

Data from exploratory phase Ib study showed 4 weeks of treatment produced signals of clinical, histologic and biomarker activity, suggesting achievement of localized target engagement in participants with moderate to severe disease; clinical response, defined as reduction in total Mayo score of ≥ 3 points and ≥ 30% with reduction in rectal bleeding subscore by ≥ 1 point or absolute rectal bleeding subscore of ≤ 1 point, was achieved by 2 of 10 patients in 20-mg cohort, 2 of 10 in the 80-mg cohort and 6 of 11 in 270-mg cohort, compared to 1 of 9 in placebo group; numerically higher rates of mucosal healing observed for study drug after 4 weeks, compared to placebo

Phase II

Amphera BV, of S-Hertogenbosch, the Netherlands

Mesopher

Autologous dendritic cells pulsed with allogeneic tumor lysate

Pancreatic cancer

Recruited first of 10 expected participants with surgically resected cancer who received adjuvant standard of care into open-label Reactive trial

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

Inopulse

Nitric oxide, inhaled

Pulmonary hypertension associated with interstitial lung disease

Additional data from cohort 1 of ongoing phase II/III INO-PF study showed minutes of moderate to vigorous physical activity improved by 34% (8% increase on INO vs. 26% decrease on placebo; p=0.04) and overall activity improved by 12% (stable on INO vs. 12% decrease on placebo; p=0.05); NT-ProBNP, a peptide marker of right ventricular failure where higher levels indicate disease worsening, improved by 27% (15% increase on INO vs. 42% increase on placebo); oxygen saturation improved by 20% (9% improvement on INO vs. 11% deterioration on placebo)

Eli Lilly and Co., of Indianapolis

Mirikizumab

Antibody targeting p19 subunit of IL-23

Moderately to severely active Crohn's disease

Patients in Serenity study achieved significant reductions in clinical and endoscopic measures of disease activity at 12 weeks; rates of response, defined as 50% reduction from baseline as measured by Simple Endoscopic Score for Crohn's disease, were 25.8%, 37.5% and 43.9% for patients in the 200-mg, 600-mg and 1,000-mg groups, respectively, vs. 10.9% for placebo

Oramed Pharmaceuticals Inc., of New York

ORMD-0801

Oral formulation of unmodified recombinant human insulin

Type 2 diabetes

Completed enrollment of nearly 300 participants in 90-day dose-ranging phase IIb study; 70% completed treatment; primary efficacy endpoint is reduction in HbA1c

Phase III

Array Biopharma Inc., of Boulder, Colo.

Braftovi (encorafenib), Mektovi (binimetinib), Erbitux (cetuximab)

BRAF inhibitor, MEK inhibitor, anti-EGFR antibody

Metastatic colorectal cancer

In Beacon CRC trial interim analysis, combo demonstrated a statistically significant improvement in objective response rate (26.1% vs. 1.9%, p<0.0001) and overall survival (median 9 months vs. 5.4 months, p<0.0001) vs. cetuximab plus irinotecan-containing regimens; Array intends to submit data for marketing approval in the second half of 2019

Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C.

BCX-7353

Plasma kallikrein inhibitor

Hereditary angioedema

In Apex-2 trial, once-daily, oral BCX-7353 achieved primary endpoint for both dose levels (110 mg and 150 mg), with 150-mg dose reducing HAE attack rate by 44% (p<0.001) vs. placebo

Clovis Oncology Inc., of Boulder, Colo.

Rubraca (rucaparib)

PARP1, PARP2 and PARP3 inhibitor

Ovarian cancer

In ARIEL3 study, mean quality-adjusted progression-free survival was significantly longer vs. placebo arm (12.02 vs. 5.74 months) for the intent-to-treat population and for patients with a BRCA mutation (15.28 vs. 5.92 months)

Heron Therapeutics Inc., of San Diego

HTX-011

Fixed dose of bupivacaine plus meloxicam

Pain

EPOCH 1 bunionectomy study results, showing a 27% reduction in pain intensity with HTX-011 vs. placebo (p<0.0001) and an 18% reduction vs. bupivacaine solution (p=0.0002) were published in Regional Anesthesia & Pain Medicine

Insmed Inc., of Bridgewater, N.J.

Arikayce (amikacin)

Inhaled formulation of amikacin

Mycobacteriaceae infection

In ongoing CONVERT study, among patients who achieved culture conversion by month 6, 80% of those receiving Arikayce plus guideline-based therapy (GBT) sustained culture conversion for up to 12 months of treatment after first dose that defined culture conversion vs. 30% of patients receiving GBT alone (p=0.0014)

Merck & Co. Inc., of Kenilworth, N.J.

Keytruda (pembrolizumab)

Anti-PD-1 therapy

Metastatic triple-negative breast cancer

Keytruda monotherapy for second- or third-line treatment missed primary endpoint of superior overall survival vs. chemotherapy (capecitabine, eribulin, gemcitabine or vinorelbine) in Keynote-119

Ocular Therapeutix Inc., of Bedford, Mass.

OTX-TP

Intracanalicular travoprost

Glaucoma

Top-line results showed the trial did not achieve its primary endpoint of statistically significant superiority in mean reduction of intraocular pressure compared with placebo at all nine time points

Vifor Pharma Group, of Zurich

Veltassa (patiromer)

Potassium binder

Heart failure

First patient treated in study evaluating potential to improve outcomes by enabling heart failure patients to be treated with renin-angiotensin-aldosterone system inhibitors therapy; primary endpoint is time to first occurrence of cardiovascular death or hospitalization; top-line results expected in 2022


Notes

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