Cancer

Advaxis Inc. (Princeton, N.J.)

ADXS11-001

Axalimogene filolisbac

Persistent or recurrent metastatic squamous or non-squamous cell cervical carcinoma (PRmCC)

Phase II data showed a 12-month overall survival (OS) rate of approximately 35% (n = 38/109); median OS was 8.28 months in the monotherapy arm and 8.78 months in the chemotherapy (cisplatin) combination arm (p=non-significant); approximately 25% of patients (n = 27/109) reached the 18-month survival milestone, including three confirmed complete responses and one confirmed partial response

2/13/18

Astrazeneca plc (Cambridge, U.K.)

Imfinzi

Durvalumab

Recurrent or metastatic head and neck cancer

The phase II CONDOR trial indicated that it was tolerable among heavily pre-treated patients and had the potential to slow growth in tumors with low or negative expression of the PD-L1 protein; 17 patients experienced partial responses to treatment, for overall response rates (ORR) of 9.2% for durvalumab alone, 7.8% for durvalumab/tremelimumab and 1.6% for tremelimumab alone; median overall survival was 7.6 months for durvalumab/tremelimumab, 6.0 months for durvalumab and 5.5 months for tremelimumab

2/14/18

Aveo Oncology Inc. (Cambridge, Mass.) and Eusa Pharma Inc. (Hemel Hempstead, U.K.)

Fotivda

Tivozanib

Metastatic renal cell carcinoma

Preliminary results of the phase II portion of the TiNivo phase Ib/II trial testing it with Opdivo showed the combination produced an objective response rate of 64% and a disease control in 100% of the patients

2/13/18

Biocancell Ltd. (Cambridge, Mass.)

BC-819

Double-stranded bacterial DNA plasmid construct

Early stage bladder cancer

Phase II data showed: at median follow-up of 18 months, the overall median time to recurrence was not reached; at the three-month time point, the first assessment following the induction treatment, 95% of patients were recurrence-free and none had progressed; at six months, 78% were recurrence-free and 5% had progressed; the 24-month recurrence-free and progression rates were 54% and 24%, respectively

2/14/18

Biothera Pharmaceuticals Inc. (Eagan, Minn.)

Imprime PGG

Activates a cascade of innate immune system responses

Squamous cell carcinoma of the head and neck

Treated the first patient in a phase II trial testing it in combination with Keytruda

2/7/18

Churchill Pharmaceuticals LLC (King of Prussia, Pa.)

Yonsa

Abiraterone acetate

Metastatic castration-resistant prostate cancer

STAAR and STAAR-E data showed more than 90% of patients treated with it achieved absolute testosterone levels of <1 ng/dL at each time point, while nondetectable testosterone levels (≤0.1 ng/dL) were achieved in a higher percentage of patients treated with it vs. patients treated with Zytiga; 24-four-hour pharmacokinetic results comparing it to Zytiga showed no statistical difference between the two agents despite the lower dose of Yonsa

2/8/18

Exelixis Inc. (South San Francisco)

Cabozantinib

Targeted kinase inhibitor

Differentiated thyroid cancer

Phase II data showed 34 of 35 patients in the trial experienced a reduction in tumor size following treatment, and more than half experienced reductions in excess of 30%; partial response (i.e., greater than 30%) was achieved in 19 patients for an overall response rate of 54%

2/14/18

Innovation Pharmaceuticals Inc. (Beverly, Mass.)

Kevetrin

Modulates p53

Late-stage ovarian cancer

Closed its phase IIa trial testing following data released in December that showed the lowest dose modulated p53

2/9/18

Irx Therapeutics Inc. (New York)

IRX-2

Contains active cytokine components

Squamous cervical intraepithelial neoplasia 3 (CIN3) or vulvar intraepithelial neoplasia 3 (VIN3)

The first patient was dosed in a phase II trial

2/15/18

Kiadis Pharma NV (Amsterdam)

ATIR-101

Allodepleted T-cell immunotherapy

Hematologic malignancy

The last patient, who received a hematopoietic stem cell transplantation from a haploidentical donor, in its phase II CR-AIR-008 study received a single dose of the drug

2/1/18

Kura Oncology Inc. (San Diego)

Tipifarnib

Farnesyltransferase inhibitor

Head and neck squamous cell carcinoma with HRAS mutations

Phase II data showed confirmed partial responses in five of six evaluable patients as defined by standard RECIST criteria for an overall response rate of 83% (36% to 99.6%, 95% CI), including durable responses of more than 18 months in two patients; the sixth evaluable patient experienced tumor shrinkage and prolonged disease stabilization; it was generally well tolerated, and adverse events were consistent with the known safety profile

2/16/18

Merrimack Pharmaceuticals Inc. (Cambridge, Mass.)

MM-121

Seribantumab

Heregulin-positive, hormone receptor-positive and HER2-negative postmenopausal metastatic breast cancer

Dosed the first patient in its Sherboc phase II trial

2/27/18

Nohla Therapeutics Inc. (Seattle)

NLA-101

Stem and progenitor cell therapy

Acute myeloid leukemia

Initiated the phase II LAUNCH trial

2/14/18

Novartis AG (Basel, Switzerland)

Kymriah

Tisagenlecleucel

Relapsed or refractory B-cell acute lymphoblastic leukemia

Data from a phase II trial testing it in pediatric and young adult patients showed it demonstrated an overall remission rate of 81%; 60% of patients achieved complete remission (CR) and 21% of patients achieved CR with incomplete blood count recovery, with no minimal residual disease detected among all responding patients

2/2/18

Oncology Venture AB (Hoersholm, Denmark)

Liplacis

Targeted liposomal formulation of cisplatin

Metastatic breast cancer

Data from the phase II part of a phase I/II study showed a clinical response in seven of 10 evaluable patients, whereas conventional cisplatin treatment has a reported response rate of 10% in previously conducted trials; in patients identified using the DRP companion diagnostic, five of five experienced clinical benefit

2/1/18

Puma Biotechnology Inc. (Los Angeles)

PB-272

Neratinib

Tumors targeting HER2 or HER3 mutations

SUMMIT phase II data showed the HER2 cohort demonstrated clinical responses in tumors with S310, L755, V777, P780_Y781insGSP and A775_G776insYVMA mutations; when stratified by tumor type, responses were observed in patients with breast, cervical, biliary, salivary and non-small-cell lung cancers, which led to cohort expansions in those tumor types; no activity was observed in the HER3-mutant cohort

2/1/18

Rexahn Pharmaceuticals Inc. (Rockville, Md.)

RX-3117

Oral small-molecule nucleoside

Advanced urothelial cancer

Phase IIa data showed that, of the 21 evaluable patients, 33% experienced progression-free survival for two months or longer and 19% had stable disease for more than four months; four patients had a 15% or greater reduction in their tumor size

2/13/18

Sellas Life Sciences Group Inc. (New York)

GPS

Galinpepimut-S

Acute myeloid leukemia

The three-year overall survival rate in a phase II trial was 47.4%; the median disease-free survival from first complete response was 16.9 months

2/28/18

Tyme Technologies Inc. (San Francisco)

SM-88

Sirolimus, phenytoin, and methoxsalen

Nonmetastatic, biochemical-recurrent prostate cancer

Phase II data showed that 92% (12/13) have maintained radiographic progression-free survival (rPFS) with a median of 12 months since documented biochemical recurrence, and 10 months since starting treatment; all 12 patients who have maintained rPFS also exhibited meaningful reductions in circulating tumor cells (CTCs), while the one patient experiencing radiographic progression had a rise in CTCs; 85% (11/13) demonstrated rising or stable testosterone levels, with no drug-related serious adverse events observed

2/9/18

Xcovery Inc. (Palm Beach Gardens, Fla.)

X-396

Ensartinib

Advanced malignant melanoma harboring alterations in ALK

Started a phase II trial

2/8/18

Cardiovascular

Akcea Therapeutics Inc. (Cambridge, Mass.)

AKCEA-APO(a)-LRx

Ligand conjugated antisense (LICA) drug

High Lp(a) and established cardiovascular disease

Completed enrollment in a phase IIb study

2/8/18

Arca Biopharma Inc. (Westminster, Colo.)

Gencaro

Bucindolol hydrochloride

Atrial fibrillation (AF) with heart failure (HF) and reduced left ventricular ejection fraction (HFrEF)

In the phase IIb Genetic AF trial, it turned up only a trend for superior benefit vs. Toprol; in all patients, it demonstrated a similar treatment benefit compared to Toprol XL (143 total events, hazard ratio [HR] of 1.01) (95% confidence interval [CI]: 0.71, 1.42), which was associated with a predictive possibility of success (PPOS) of 14; the U.S. patient cohort came out markedly better (73 events, HR 0.70, [95% CI: 0.41, 1.19]), with the aforementioned PPOS, which beat the pre-specified criteria set by the company to move ahead with phase III development; the two non-U.S. countries exhibited HRs >1

2/27/18

La Jolla Pharmaceutical Co. (San Diego)

Giapreza

Angiotensin II

Shock

Treated the first patient in LJ501-CRH02, an open-label study testing it in pediatric patients, ages 2 to 17

2/22/18

Reata Pharmaceuticals Inc. (Irving, Texas)

Bardoxolone

Activator of Nrf2

Pulmonary arterial hypertension

LARIAT trial data showed the mean increase in eGFR, a measure of kidney function, was 10.7 mL/min/1.73 m2 from baseline after 56 weeks, which was maintained at a mean increase of 11.3 mL/min/1.73 m2 after 104 weeks of treatment

2/14/18

Dermatologic

Anaptysbio Inc. (San Diego)

ANB-020

Anti-IL-33 antibody

Moderate to severe adult atopic dermatitis

Phase IIa data showed that it was efficacious in all 12 patients enrolled, with each patient achieving at least EASI-50 on or before day 57 post-administration; rapid response was observed by day 15 post-administration, with nine of 12 patients (75%) achieving EASI-50, of which three patients (25%) also achieved EASI score improvement of 75% relative to baseline (EASI-75); day 29 results exceeded the primary efficacy objective, with 10 of 12 patients (83%) achieving EASI-50, of which four patients (33%) also achieved EASI-75

2/21/18

Athenex Inc. (Buffalo, N.Y.)

KX2-391

Dual Src kinase and tubulin polymerization inhibitor

Actinic keratosis lesions

Phase II data showed it achieved a higher overall 100% clearance at day 57 in the five-day treatment cohort vs. the three-day treatment cohort (43% vs. 32%); in the five-day treatment cohort, 23 of 44 subjects (52%) with actinic keratosis on the face and 13 of 40 (33%) on the scalp attained 100% clearance at day 57; there were no treatment-related serious adverse events or discontinuations

2/21/18

Bonti Inc. (Newport Beach, Calif.)

EB-001

Botulinum neurotoxin serotype E

Mohs micrographic surgery on the forehead

Started the phase II Scar Healing Improvement with Neurotoxin E (SHINE-1) trial

2/9/18

Brickell Biotech Inc. (San Diego)

Sofpironium bromide

Anticholinergic

Hyperhidrosis

The phase IIb trial met its primary efficacy endpoint of achieving at least a one-point improvement in the Hyperhidrosis Disease Severity Measure-Axillary score; statistically significant treatment responses were observed as early as day eight for both the 5% dose group (p=0.0009) and 15% dose group (p=0.0003), and the response was maintained throughout the treatment period; the proportion of subject responders at the end of treatment was 70.2% (p=0.0387) for the 5% dose group and 75.9% (p=0.0099) for the 15% dose group, compared to 54.4% for the vehicle group

2/21/18

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-543

Deuterium-modified analog of janus kinase inhibitor ruxolitinib

Moderate-to-severe alopecia areata

Started enrollment of the second cohort of its phase IIa trial

2/13/18

Dermira Inc. (Menlo Park, Calif.)

Lebrikizumab

Humanized monoclonal antibody

Moderate to severe atopic dermatitis

Started a phase IIb dose-ranging study

2/1/18

Menlo Therapeutics Inc. (Redwood City, Calif.)

Serlopitant

NK-1 receptor antagonist

Chronic pruritis

Phase II data demonstrated a statistically significant difference in the change from baseline in subjects treated with either 1 mg or 5 mg, compared with placebo (p<0.05 at weeks four, five and six), meeting the primary endpoint; at week six, the mean percent reduction from baseline visual analogue scale pruritus scores in the 1-mg and 5-mg groups were 41.4% and 42.5%, respectively, as compared with 28.3% in the placebo-treated group; it was well-tolerated

2/21/18

Sol-Gel Technologies Ltd. (Ness Ziona, Israel)

Twin and Vered

Benzoyl peroxide and tretinoin; benzoyl peroxide

Acne and subtype II rosacea

In phase II trials, Twin showed statistically significant improvements in all pre-defined co-primary and secondary efficacy endpoints, as compared to vehicle and that Vered showed statistically significant improvements in the IGA pre-defined co-primary efficacy endpoint and in the percent change in inflammatory lesion count at week 12, as compared to vehicle

2/20/18

Endocrine/Metabolic

Armagen Inc. (Calabasas, Calif.)

AGT-181

Enzyme replacement therapy

Mucopoly-saccharidosis type I

Phase II 52-week data suggest that it stabilized the neurocognitive development quotient and validate previous findings that demonstrated the ability to transport biopharmaceuticals across the blood-brain barrier and provide therapeutic benefit

2/9/18

Caladrius Biosciences Inc. (Basking Ridge, N.J.)

CLBS-03

Cell therapy

Type 1 diabetes

Completed enrollment of a trial in 110 children

2/21/18

Corcept Therapeutics Inc. (Menlo Park, Calif.)

Relacorilant

GR antagonist

Cushing's syndrome

Data from the first 17 patients enrolled in a phase II trial found no evidence of progesterone receptor affinity and no serious adverse events

2/26/18

Madrigal Pharmaceuticals Inc. (Conshohocken, Pa.)

MGL-3196

Thyroid hormone receptor beta-selective agonist

Heterozygous familial hypercholesterolemia

Phase II data showed it produced a placebo-corrected 18.8% decrease in LDL cholesterol (LDL-C) and a 21% decrease in patients on an optimal dose of MGL-3196; placebo-adjusted LDL-C was 28.5% lower in patients who did not tolerate high intensity statin doses; it also improved levels of apolipoprotein B, triglycerides, apolipoprotein CIII and lipoprotein(a)

2/9/18

Zealand Pharma A/S (Copenhagen)

Dasiglucagon

Glucagon analog

Type 1 diabetes

Phase IIa data showed patients treated with it and those treated with glucagon for reconstitution spent comparable time in different glycemic targets

2/16/18

Gastrointestinal

Abbvie Inc. (North Chicago)

Upadacitinib

Oral JAK1-selective inhibitor

Severely active Crohn's disease

Phase II CELEST showed that, among patients who responded to induction treatment at week 16, dose-related increases in rates of modified clinical remission were observed with the 3 mg, 6 mg and 12 mg twice-daily doses at week 52; also at the week 52 time point, numerically greater rates of clinical remission and endoscopic response were achieved by patients who received 12 mg twice daily compared with the other treatment dose

2/20/18

Azurrx Biopharma Inc. (Brooklyn, N.Y.)

MS1819-SD

Recombinant enzyme

Exocrine pancreatic insufficiency caused by chronic pancreatitis

Enrolled three new patients in its phase IIa trial

2/13/18

Celgene Corp. (Summit, N.J.)

Otezla

Apremilast

Active ulcerative colitis

Phase II trial data showed that a higher proportion of patients taking 30 mg twice daily achieved clinical remission compared to placebo; the primary endpoint of the study was Total Mayo Score (TMS) clinical remission at week 12, achieved by 21.8% of patients in the 40 mg arm vs. 13.8% in the placebo group; in the 30 mg arm, 31.6% of patients achieved clinical remission as measured by TMS at week 12 vs. 13.8% in the placebo group (n=58; nominally significant, P<0.05)

2/16/18

Durect Corp. (Cupertino, Calif.)

DUR-928

Endogenous small molecule

Primary sclerosing cholangitis

Commenced patient dosing in a phase IIa trial

2/27/18

Galmed Pharmaceuticals Ltd. (Israel)

Aramchol

Arachidyl amido cholanoic acid

HIV-associated lipodystrophy and non-alcoholic fatty liver disease (NAFLD)

A mid-stage trial testing it over 12 weeks found it made no apparent difference in liver fat for people with HIV-associated lipodystrophy and NAFLD

2/15/18

Gemphire Therapeutics Inc. (Livonia, Mich.)

Gemcabene

Oral therapy targeting known lipid metabolic pathways

Pediatric nonalcoholic fatty liver disease

Started a phase IIa proof-of-concept trial

2/2/18

Protalix Biotherapeutics Inc. (Carmiel, Israel)

OPRX-106

Recombinant human tumor necrosis factor receptor II fused to an IgG1 Fc domain

Ulcerative colitis

Treated the last patient in its phase II trial

2/16/18

Sterna Biologicals GmbH & Co. KG (Marburg, Germany)

SB-01

GATA-3-specific DNAzyme

Moderate to severe ulcerative colitis

Phase IIa SECURE data showed that patients' Mayo Scores dropped from 8.4 at baseline to 6.5 at day 28 and 5 at day 56, both of which were statistically significant; patients treated with placebo had Mayo Scores of 10 at baseline, 9 at day 28 and 9 at day 56, the differences of which weren't statistically significant

2/21/18

Hematologic

Akari Therapeutics plc (London)

Coversin

Complement C5a inhibitor

Paroxysmal nocturnal hemoglobinuria

Phase II COBALT data showed the final three patients in the trial had a median lactate dehydrogenase level of 1.5 times or less the upper limit of normal at day 28, day 60 and day 90; of the six patients who completed the trial that were transfusion-dependent prior to the trial, three didn't need transfusions while on it during the COBALT trial

2/7/18

Akari Therapeutics plc (London)

Coversin

Complement C5a inhibitor

Atypical hemolytic uremic syndrome

Started a phase II trial

2/7/18

Arch Therapeutics Inc. (Framingham, Mass.)

AC5

Topical hemostatic agent

Skin neoplasms

Data from a study of 46 patients undergoing dermatologic surgery showed it reduced the median time to hemostasis (TTH) by 41% compared to the control treatment; the median TTH for it was the same for patients regardless of whether they were taking an antiplatelet therapy, unlike the control treatment where the median TTH was 90 seconds and 40 seconds for the patients taking or not taking antiplatelet therapy, respectively

2/6/18

Imara Inc. (Cambridge, Mass.)

IMR-687

Inhibitor of phosphodiesterase-9

Sickle cell disease

Dosed the first patient in its phase IIa trial

2/14/18

Omeros Corp. (Seattle)

OMS-721

Fully human monoclonal antibody targeting MASP-2

Hematopoietic stem cell transplant-associated thrombotic microangiopathy

Phase II data showed patients treated with it had a median overall survival of 347 days compared to 21 days for matched historical controls; it also improved markers of thrombotic microangiopathy: mean platelet count, mean lactate dehydrogenase and mean haptoglobin levels

2/16/18

Ra Pharmaceuticals Inc. (Cambridge, Mass.)

RA101495 SC

Synthetic, macrocyclic peptide

Paroxysmal nocturnal hemoglobinuria

Phase II data showed that in 10 Soliris naive patients, it reduced lactate dehydrogenase levels, resulting in near-complete suppression of complement activity; half of the six Soliris naive patients who were transfusion-dependent prior to enrollment remained transfusion-free while on study; seven of the 11 patients who switched from Soliris that were transfusion-dependent at baseline had breakthrough hemolysis

2/13/18

Immune

Adamas Pharmaceuticals Inc. (Emeryville, Calif.)

ADS-5102

Amantadine extended-release capsules

Multiple sclerosis

Phase II data showed at week four it produced a 17% improvement in walking speed, as assessed by a timed 25-foot walk, from baseline in the 56 intent-to-treat patients; it also helped a greater proportion of treated patients improve their walking speed by at least 20% from baseline compared to placebo

2/5/18

Allergy Therapeutics plc (Worthing, U.K.)

PQ Grass

Aluminium-free immunotherapy

Allergic rhinoconjunctivitis due to grass pollen

Its phase II grass allergy study is fully enrolled

2/13/18

DBV Technologies SA (Montrouge, France)

Viaskin Milk

Epicutaneous immunotherapy patch

IgE-mediated cow's milk protein allergy

Data from the phase II portion of its phase I/II trial testing determined the 300 mcg dose to be the best to take forward in children ages 2 to 11 with a 1,322.4 mean change in cow's milk protein cumulative reactive dose (CRD) compared to a mean change of 565.6 in placebo-treated patients; the dose produced a responder rate of 57.9% compared to 32.5% for placebo in the children

2/28/18

Jazz Pharmaceuticals plc (Dublin)

Defitelio

Defibrotide

Acute graft-vs.-host disease after allogeneic hematopoietic stem cell transplant

The first patient has been enrolled in a phase II trial

2/26/18

Kadmon Holdings Inc. (New York)

KD-025

Rho-associated coiled-coil kinase 2 inhibitor

Chronic graft-vs.-host disease (cGVHD)

Updated data from cohort 2 of the phase II trial, in which 16 patients received a 200-mg dose twice daily showed an overall response rate (ORR) of 69%, as of a data cutoff of Jan. 3; responses in cohort 1, in which 17 patients received a 200-mg dose once daily remained the same, showing an ORR of 65%; responses were rapid and were observed across all affected organs; 83% of patients were able to reduce the dose of tacrolimus

2/26/18

Medicinova Inc. (La Jolla, Calif.)

MN-166

Ibudilast

Progressive multiple sclerosis

Phase IIb SPRINT-MS data showed it produced a 26% reduction in the risk of confirmed disability progression compared to placebo, as measured by the Expanded Disability Status Scale

2/5/18

TG Therapeutics Inc. (New York)

TG-1101

Ublituximab

Relapsing forms of multiple sclerosis

Phase II data showed a 99% median B-cell depletion at week four, which was maintained at week 24; T1 Gd-enhancing lesions were completely eliminated at week 24; patients taking it had a mean Expanded Disability Status Scale (EDSS) improvement from baseline of 0.3 points with 78% of patients showing improved or stable EDSS; one of the 40 patients reported a relapse, but the patient was initially randomized to placebo and relapsed 12 days after the first infusion of ublituximab

2/5/18

Infection

Cytodyn Inc. (Vancouver, Wash.)

PRO-140

Humanized CCR5 monoclonal antibody

HIV

A phase IIb/III CD02 trial testing it in combination with existing antiretroviral therapy (ART) in 52 patients failing their current HIV therapy showed that after one week of treatment, PRO-140 plus ART reduced the HIV-1 RNA viral load by greater than 0.5 log vs. patients who continued on ART

2/21/18

Medimmune (biologics arm of Astrazeneca plc, London)

MEDI-3902

Bispecific anti-PcrV/Psl monoclonal antibody

Pneumonia caused by Pseudomonas aeruginosa

Began enrolling patients in the Evade trial

2/26/18

Medimmune (biologics arm of Astrazeneca plc, London)

Suvratoxumab

Human immunoglobulin G1 kappa monoclonal antibody

Disease caused by Staphylococcus aureus

Began enrolling patients in the Saatellite trial

2/26/18

Myr Pharma GmbH (Burgwedel, Germany)

Myrcludex B

Entry inhibitor

Chronic hepatitis delta (HDV) infection

Completed the 48-week active treatment phase in MYR 203, part of a phase IIb program

2/23/18

Reviral Ltd. (London)

RV-521

Oral RSV fusion inhibitor

Respiratory syncytial virus (RSV)

Phase IIa data of 66 healthy adult subjects infected intranasally showed treatment reduced viral load to undetectable levels and also improved the mean total mucus weight and total symptom diary score compared to placebo

2/6/18

Vaxart Inc. (South San Francisco)

VXA-A1.1

H1 influenza oral tablet vaccine

Influenza

Phase II data showed it reduced the infection rate by 48% compared to 38% with injectable quadrivalent influenza vaccine (QIV); only 37% of subjects developed infection compared with 44% who received the QIV and 71% who received placebo; results for both vaccines were statistically significant compared to placebo, with "p" values of 0.001 for the Vaxart vaccine and 0.009 for QIV

2/2/18

Vical Inc. (San Diego)

VL-2397

Antifungal compound

Invasive aspergillosis in acute leukemia and allogeneic hematopoietic cell transplant

Started a phase II trial

2/21/18

Inflammatory

Symic Bio Inc. (San Francisco)

SB-061

Bioconjugate molecule inspired by the properties of aggrecan

Mild to moderate osteoarthritis of the knee

Phase II MODIFY2 data showed that, in the overall patient population, which included patients with and without effusions, treatment did not meet the primary endpoint of demonstrating a clinically meaningful improvement compared to control

2/8/18

Bone Therapeutics SA (Gosselies, Belgium)

Allob

Allogeneic cell therapy

Lumbar spinal fusion

Completed enrollment in its phase IIa trial

2/21/18

Musculoskeletal

Catabasis Pharmaceuticals Inc. (Cambridge, Mass.)

Edasalonexent

Inhibits NF-κB

Duchene muscular dystrophy (DMD)

Phase II MoveDMD data showed that through 60 weeks of treatment, patients receiving the 100 mg/kg/day dose showed consistent and clinically meaningful improvements in rates of decline compared to rates of change during the control period prior to treatment; all four assessments of muscle function – 10-meter walk/run, four-stair climb, time to stand and the North Star Ambulatory Assessment – improved

2/14/18

Summit Therapeutics plc (Oxford, U.K.)

Ezutromid

Utrophin modulator

Duchenne muscular dystrophy

Phase II Phaseout DMD analysis of the 24-week interim dataset showed a statistically significant decrease in muscle inflammation as measured by magnetic resonance spectroscopy transverse relaxation time T2 (MRS-T2)

2/27/18

Neurology/Psychiatric

Biogen Inc. (Cambridge, Mass.)

Tysabri

Natalizumab

Acute ischemic stroke

The phase IIb, dose-ranging Action 2 study did not yield improvement in clinical outcomes compared to placebo; both doses were generally well-tolerated and no new or important safety signals were observed

2/8/18

Boehringer Ingelheim GmbH (Ingelheim, Germany)

BI-409306

PDE9 inhibitor

Alzheimer's disease

The phase II trials did not meet their efficacy endpoints

2/12/18

Diamedica Therapeutics Inc. (Minneapolis)

DM-199

Recombinant human kallikrein

Acute ischemic stroke

Enrolled the first patient in its phase II Remedy trial

2/23/18

GW Pharmaceuticals plc (London)

GWP-42006

Cannabidivarin

Focal seizures

A phase IIa proof-of-concept study did not meet its primary endpoint; both active and placebo arms showed similar reductions in focal seizures of about 40%

2/23/18

Harvest One Cannabis Inc. (Vancouver, British Columbia; Satipharm AG)

Satipharm CBD

Cannabidiol capsules

Refractory, or treatment-resistant, epilepsy

A phase II trial by Phytotech Therapeutics Ltd. (Nedlands, Australia) showed that the capsules reduced monthly seizure frequency in children when added to current medications; treatment was generally well-tolerated, with a safety profile consistent with prior experience; six patients showed a reduction of 59% to 91% in mean monthly seizure frequency following 12 weeks of treatment

2/2/18

Recro Pharma Inc. (Malvern, Pa.)

Meloxicam

Long-acting, preferential COX-2 inhibitor

Pain following bunionectomy surgery

On the phase II primary efficacy endpoint of effect size based on the difference in Summed Pain Intensity Differences over the first 48 hours, both doses of I.V. meloxicam produced meaningful effect sizes compared to placebo, and both doses demonstrated statistically significant reductions in pain compared to placebo at all evaluated times and intervals

2/21/18

Vertex Pharmaceuticals Inc. (Boston)

VX-150

NaV1.8 inhibitor

Acute pain following bunionectomy

Phase II data showed that it provided statistically significant relief of acute pain vs. placebo; the Sum of the Pain Intensity Difference over the first 24 hours of treatment (SPID24) values for those treated with it and placebo were 36.14 and 6.64, respectively; the SPID24 value for treatment with hydrocodone+acetaminophen, representing a standard-of-care reference arm, was 40.16

2/15/18

Ocular

Aerpio Pharmaceuticals Inc. (Cincinnati)

AKB-9778

Inhibitor of VE-PTP

Moderate to severe nonproliferative diabetic retinopathy

Completed patient enrollment in its phase IIb TIME-2b study

2/12/18

Apellis Pharmaceuticals Inc. (Crestwood, Ky.)

APL-2

Complement C3 inhibitor

Geographic atrophy (GA) associated with age-related macular degeneration (AMD)

Eighteen-month data from a phase II trial found that it significantly reduced the growth of GA, with safety comparable to 12-month data

2/23/18

Eyegate Pharmaceuticals Inc. (Waltham, Mass.)

EGP-437

Dexamethasone phosphate

Post cataract surgery inflammation

Although it showed a higher rate of success compared to vehicle at all time points in a phase IIb study, the co-primary endpoints of proportion of subjects with an anterior chamber cell (ACC) count of zero at day seven and the proportion of subjects with a pain score of zero at day one did not show statistical significance – largely because the vehicle group outperformed its expected response

2/6/18

Genentech Inc. (South San Francisco; member of the Roche Group)

RG7716

Anti-VEGF/anti-angiopoietin-2 bispecific antibody

Vision loss from diabetic macular edema

Phase II BOULEVARD data showed the 6 mg dose produced an adjusted mean improvement of 13.9 chart letters from baseline using best corrected visual acuity, compared to 11.7 letters in patients treated with 1.5 mg , and 10.3 letters in patients treated with 0.3 mg of Lucentis

2/13/18

Respiratory

Kadmon Holding Inc. (New York)

KD-025

Rho-associated coiled-coil kinase 2 inhibitor

Idiopathic pulmonary fibrosis

Phase II showed it slowed the decline in lung function with a 48 mL median decline in forced vital capacity (FVC) at week 24, compared to a 175 mL median decline in the nine patients treated with best supportive care (BSC); patients taking it had an annualized decline of 32 mL, compared to 126 mL in the year prior to randomization

2/14/18

Synairgen plc (Southampton, U.K.)

SNG-001

Inhaled interferon-beta therapeutic

Chronic obstructive pulmonary disease

The first patients were dosed in its phase II trial

2/8/18

Vertex Pharmaceuticals Inc. (Boston)

VX-659 and VX-445

Next-generation correctors

Cystic fibrosis

Phase II data showed mean absolute improvements in percent predicted forced expiratory volume in one second (ppFEV1) of up to 13.3 and 13.8 percentage points from baseline through four weeks of treatment, dosed once daily as part of the triple-combination regimens with VX-659 (400 mg) or VX-445 (200 mg), respectively, in people with one F508del mutation and one minimal function mutation (F508del/Min)

2/2/18

Toxicity/intoxication

Acacia Pharma Group Ltd. (Cambridge, U.K.)

APD-403

Amisulpride injection

Chemotherapy-induced nausea (CINV)

Completed two phase II trials demonstrating it is well tolerated and effective at preventing acute and delayed CINV

2/6/18

Notes

The date indicated refers to the BioWorld issue in which the news item can be found.

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