Company

Product

Description

Indication

Status

Phase I

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Lumasiran

Subcutaneous RNAi therapeutic targeting glycolate oxidase

Primary hyperoxaluria type 1

Updated results from the phase I/II study, as of data cut-off at Aug. 15, showed mean maximal reduction in urinary oxalate of 75% (43%-87% range) relative to baseline across cohorts dosed at 1 mg/kg monthly or 3 mg/kg monthly or quarter; mean relative reduction to baseline was 66% when measured 28 days post last dose; all patients achieved oxalate lowering to less than 1.5 times the upper limit of normal

Beyondspring Inc., of New York

Plinabulin

Small molecule that induces dendritic cell maturation and T-cell activation

Small-cell lung cancer

Investigator-initiated trial opened testing triple-combination therapy of plinabulin plus Opdivo (nivolumab, Bristol-Myers Squibb Co.) and Yervoy (ipilimumab, BMS); about 15 patients will be enrolled in the phase I portion, with an additional 40 in the phase II portion

Frequency Therapeutics Inc., of Woburn, Mass.

FX-322

Progenitor cell activation therapy

Hearing loss

Completed enrollment in the double-blind, placebo-controlled trial assessing safety of a single dose given by intratympanic administration in adults with stable sensorineural hearing loss who have a medical history consistent with either noise exposure or sudden hearing loss

Kandy Therapeutics Ltd., of Stevenage, U.K.

NT-814

Oral small-molecule dual antagonist of neurokinin-1/3 receptors

Alternative to hormone replacement therapy for symptoms of menopause

Data from phase Ib/IIa trial show NT-814 reduced symptoms of menopause, including vasomotor symptoms and night time awakenings; effects were immediate in onset, being both statistically significant and clinically relevant as soon as the first day of treatment; when taken once-daily orally for 2 weeks reduced hot flash frequency by 62% vs. 24% for placebo from baseline at week 1 (p<0.0014) and 84% vs. 37% for placebo at week 2 (p<0.0002); night time awakenings reduced 58% vs. 17% for placebo from baseline at week 1 (p<0.0022) and 81% vs. 32% for placebo from baseline at week 2 (p<0.0005)

Neurovive Pharmaceutical AB, of Lund, Sweden

Neurostat

Cyclosporine A

Severe traumatic brain injury

Completed biomarker analyses in CHIC study show time-based change in biomarker levels correlating with drug administration for all 4 biomarkers – GFAP, UCH-L1, NF-L and tau

Oramed Pharmaceuticals Inc., of New York

ORMD-0801

Oral insulin capsule

Nonalcoholic steatohepatitis

Enrolled the first patient in an exploratory trial; 3-month study will test effectiveness of ORMD-0801 in reducing liver fat content, inflammation and fibrosis

Poxel SA, of Lyon, France

PXL-770

AMPK activator

Nonalcoholic steatohepatitis

Data showed favorable pharmacokinetic, tolerability and safety in healthy subjects, as well as a favorable cardiac and ECG safety profile

Regenxbio Inc., of Rockville, Md.

RGX-314

Gene therapy

Wet age-related macular degeneration

Completed dosing of the fourth cohort of 6 patients; a total of 24 patients have been dosed in the trial

Sarepta Therapeutics Inc., of Cambridge, Mass.

AAVrh74.MHCK7.micro-dystrophin

Gene therapy

Duchenne muscular dystrophy

Updated results showed all 4 patients had robust expression of micro-dystrophin as measured by Western blot, with a mean of 74.3% compared to normal utilizing Sarepta's method, or 95.8% compared to normal pursuant to Nationwide Children's quantification of Sarepta's method that adjusts for fat and fibrotic tissue; for patient 4, microdystrophin positive fiber was 96.2% and the mean intensity of the fibers was 160% compared to normal control

Seqirus Ltd., of Maidenhead, U.K.

MF-59-adjuvanted trivalent influenza vaccine

Influenza vaccine

Influenza

Studies in those 65 and older showed immune response associated with increase in the dose, with 6% reduction in all-cause hospitalization among who live in U.S. nursing homes vs. those receiving traditional egg-based trivalent vaccine; repeat vaccination in young children showed improved response

Vedanta Biosciences Inc., of Cambridge, Mass.

VE-303

Oral, live biotherapeutic

Recurrent Clostridium difficile infection

Preliminary results from phase Ia/Ib study in healthy volunteers showed single and multiple doses, after vancomycin administration, ranging up to 1.1x10^11 total colony forming units, were safe and well-tolerated; abundant colonization of VE-303 strains lasted for at least 12 weeks, as detected at all doses; repeat dosing led to increased robustness of strain colonization; accelerated microbiota recovery after vancomycin administration in a dose-dependent manner

Phase II

Cidara Therapeutics Inc., of San Diego

Rezafungin

Antifungal

Candidemia and/or invasive candidiasis

Results from STRIVE study, which met all of its primary objectives, showed that once-weekly intravenous dosing at 2 dosing regimens was observed to be generally well-tolerated and safe; data also provide evidence of rezafungin efficacy, which was defined in the trial by clearance of Candida from the blood or other normally sterile sites, resolution of signs related to the infection and overall survival

Eli Lilly and Co., of Indianapolis

GIP/GLP-1 RA (LY-3298176)

Dual GIP and GLP-1 receptor agonist

Type 2 diabetes

6-month data showed average HbA1c reductions of up to 2.4 percentage points and an average weight reduction up to 11.3 kg (12.7%); results published in The Lancet

Ophthotech Corp., of New York

Zimura (avacincaptad pegol)

Complement C5 inhibitor

Geographic atrophy secondary to dry age-related macular degeneration

Completed patient recruitment in the phase IIb trial, with 286 patients enrolled; top-line data expected in the fourth quarter of 2019

Saniona AB, of Copenhagen

Tesomet

Combination of tesofensine and metoprolol

Prader-Willi syndrome

Company decided to keep recruitment of phase IIa study open for a few more weeks; trial is expected to be completed in early 2019, with top-line data available in the first quarter

Vaxart Inc., of South San Francisco

H1 influenza vaccine

Oral tablet vaccine

Influenza

Results showed significant expansion of mucosal homing receptor alpha4beta7 plasmablasts to about 60% of all activated B cells vs. 20% maintained with Fluzone; data confirm that, while the vaccine generated protective hemagglutinin inhibition antibodies in serum, it primarily protected through the mucosal mechanism, providing a 39% reduction in illness vs. placebo overall

Phase III

Adamas Pharmaceuticals Inc., of Emeryville, Calif.

Gocovri (amantadine)

High-dose 274-mg amantadine

Dyskinesia in Parkinson's disease

Data published in Parkinsonism from pooled phase III trials showed treated patients had a statistically significant improvement on 6 of the 10 activities of daily living: walking and balance (p<0.0001), eating tasks (p=0.0052), doing hobbies and other activities (p=0.0159), public and social settings (p=0.0165), exciting or emotional settings (p=0.0310) and speech (p=0.0494) vs. placebo-treated patients at week 12; difference between Gocovri and placebo was statistically significant in all 7 body regions, most markedly in the arms/shoulders (p≤0.0001), and for 3 of 4 ADL tasks at week 12: ambulation (p=0.0007), dressing (p=0.0074), and drinking (p=0.0112), as assessed by the UDysRS Part 3 and Part 4, respectively

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Lumasiran

Subcutaneous RNAi therapeutic targeting glycolate oxidase

Primary hyperoxaluria type 1

Initiated pivotal ILLUMINATE-A trial in children and adults with PH1; 30-patient study will measure reduction of urinary oxalate at 6 months as the primary endpoint; top-line results expected in late 2019 and, if positive, would support filings starting in early 2020

Amag Pharmaceuticals Inc., of Waltham, Mass., and Endoceutics Inc., of Quebec City

Intrarosa (prasterone)

Synthetic form of DHEA

Dyspareunia due to vulvar and vaginal atrophy

Data showed that while age and prior hormone therapy did not influence the overall efficacy, the greatest reduction of dyspareunia had a tendency to be observed in women who were 1-2 years post menopause, compared to 3-5 years and more than 6 years post menopause, although the difference did not reach statistical significance

Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Eli Lilly and Co., of Indianapolis

Empagliflozin

SGLT2 inhibitor

Type 1 diabetes

Data from EASE-2 trial published in Diabetes Care showed placebo-corrected mean change from baseline in A1C at week 26 was -0.54% and -0.53% for empagliflozin 10 mg and 25 mg, respectively; in EASE-3, placebo-corrected mean change from baseline in A1C at week 26 was 0.28%, -0.45% and -0.52% for 2.5 mg, 10 mg and 25 mg, respectively; empagliflozin treatment was effective on secondary endpoints, showing reductions in weight, decreases in blood pressure, and decreases in total daily insulin dose; drug was tested as adjunctive to insulin therapy

Genentech, of South San Francisco, a member of the Roche Group

Baloxavir marboxil

Antiviral designed to inhibit cap-dependent endonuclease protein

Influenza

CAPSTONE-2 study showed treatment significantly reduced time to improvement of influenza symptoms vs. placebo (median time of 73.2 hours vs. 102.3 hours; p<0.0001) in people at high risk of serious complications from the flu, which includes adults 65 or older, or those who have conditions such as asthma, chronic lung disease, morbid obesity or heart disease

Janssen Pharmaceutical Cos. of Johnson & Johnson, of New Brunswick, N.J.

Symtuza (darunavir 200 mg/cobicistat 150 mg/emtricitabine 200 mg and tenofovir alafenamide 10 mg)

Single-tablet HIV-1 regimen

HIV-1

Results from EMERALD study demonstrate that in adults who are virologically suppressed, switching to Symtuza resulted in maintained high virologic suppression (91%, 692/763) and low virologic failure (1%, 9/763) at week 96; low cumulative virologic rebound (3.1%, 24/763); and no resistance development, up to 96-weeks

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

Delstrigo (doravirine 100 mg/lamivudine 300 mg/tenofovir disoproxil fumarate)

Once-daily fixed-dose combination tablet

HIV-1 infection

DRIVE SHIFT data evaluating a switch to Delstrigo in adults who demonstrated virological suppression for at least 6 months on stable antiretroviral treatment regimen met its primary endpoint of noninferior efficacy as measured by the proportion of participants who switched and had plasma HIV-1 RNA levels <50 copies/mL at week 48, compared to the proportion of participants who continued baseline regimen and had HIV-1 RNA levels <50 copies/mL at week 24

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

Delstrigo (doravirine 100 mg/lamivudine 300 mg/tenofovir disoproxil fumarate)

Once-daily fixed-dose combination tablet

HIV-1 infection

DRIVE-AHEAD findings at week 96 were consistent with week 48 data, demonstrating noninferior efficacy vs. a fixed-dose combination of efavirenz, emtricitabine and TDF; proportion of participants achieving plasma HIV-1 RNA levels < 50 copies/mL was 77.5% in Delstrigo group vs. 73.6% in the EFV/FTC/TDF group

Zealand Pharma A/S, of Copenhagen

Glepaglutide

Long-acting GLP-2 analogue

Short bowel syndrome

Initiated registration trial designed to test efficacy and safety of once- and twice-weekly subcutaneous injections in patients on parenteral support; 129 patients will be enrolled; primary objective is reduction of parenteral support volume


Notes

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