Company (location)

Product

Description

Indication

Status

Date

Cancer

Abbvie Inc. (Chicago)

Rovalpituzumab tesirine (Rova-T)

DLL3-targeting antibody-drug conjugate

Third-line small-cell lung cancer

Data from the phase II TRINITY study showed an objective response rate of 16% and a median survival of 5.6 months; the most common treatment-emergent adverse events were fatigue (38%), photosensitivity reaction (36%), pleural effusion (32%), edema peripheral (31%), decreased appetite (30%), nausea (26%), dyspnea (25%), thrombocytopenia (25%), constipation (22%), vomiting (17%), anemia (17%), hypoalbuminemia (16%) and cough (16%); Abbvie said it will not seek accelerated approval

3/23/18

Armo Biosciences Inc. (Redwood City, Calif.)

Pegilodecakin

Pegylated ilodecakin

Non-small-cell lung cancer (NSCLC)

Enrolled first patient in CYPRESS 2, a randomized IIb trial testing pegilodecakin (AM-0010) with nivolumab in second-line metastatic NSCLC

3/29/18

Benitec Biopharma Ltd. (Sydney)

BB-401

Recombinant DNA construct

Head and neck squamous cell carcinoma

Started a phase II study in Australia

3/6/18

Fidia Farmaceutici SpA (Abano Terme, Italy) 

Oncofid P-B

Paclitaxel-hyaluron bioconjugate targeted to cancer cells

Primary or recurrent Ta G1-G2 papillary bladder cancer

Results from a phase II trial showed the rate of complete response (CR) was 45% (27 CR/60 patients), with excellent safety and tolerability; a higher CR rate (57.1%; 16/28 patients) was observed in the subgroup of patients who had not previously been treated with chemo- or immunotherapy

3/20/18

G1 Therapeutics Inc. (Research Triangle Park, N.C.)

Trilaciclib

Short-acting CDK4/6 inhibitor

Extensive-stage small-cell lung cancer

Phase IIa data showed that it reduced clinically relevant consequences of chemotherapy-induced myelosuppression vs. placebo; it was well-tolerated, with no grade 3/4 treatment emergent adverse events reported; the trilaciclib arm showed favorable trends with reduced grade 3 anemia, red blood cell transfusions, and grade 3 thrombocytopenia vs. placebo; there was no grade 4 anemia or thrombocytopenia in either arm

3/6/18

Heat Biologics Inc. (Durham, N.C.)

HS-110

Viagenpumatucel-L

Non-small-cell lung cancer

Phase II overall response and disease control rates were 26% and 67%, respectively, for the evaluable intent-to-treat population; four of the nine patients with low levels of tumor-infiltrating lymphocytes had partial responses; of the nine patients with low expression of PD-L1, three responded

3/1/18

Immune Design Corp. (Seattle)

G-100

TLR4 agonist

Follicular lymphoma

Data from a phase II trial testing G-100 in combination with fractionated, low-dose radiation (XRT) with and without Keytruda (pembrolizumab, Merck & Co. Inc.) showed G-100 plus XRT produced an objective response rate (ORR) of 15% while G-100 plus XRT with Keytruda had an ORR of 45%; the eight patients with high TLR4 expression had an ORR of 75% after treatment with the G-100 plus XRT and Keytruda combination

3/13/18

Immunovaccine Inc. (Halifax, Nova Scotia)

DPX-Survivac

Survivin-based peptide antigens

Diffuse large B-cell lymphoma

Initiated patient dosing in investigator-sponsored trial in combination with pembrolizumab (Keytruda, Merck & Co. Inc.)

3/28/18

Kazia Therapeutics Ltd. (New South Wales, Australia)

GDC-0084

PI3K inhibitor

Glioblastoma

Started global phase II trial

3/29/18

Leap Therapeutics Inc. (Cambridge, Mass.)

DKN-01

Anti-DKK1 monoclonal antibody

Advanced endometrioid gynecologic malignancies

The first patient was enrolled in a phase II study testing DNK-01 as a monotherapy and in combination with paclitaxel

3/16/18

Merrimack Pharmaceuticals Inc. (Cambridge, Mass.)

MM-121

Seribantumab

Heregulin-positive non-small-cell lung cancer

Is expanding enrollment in its ongoing phase II SHERLOC trial testing MM-121 in combination with docetaxel, compared with docetaxel alone, in patients with who have progressed after a platinum-containing regimen

3/13/18

Moleculin Biotech Inc. (Houston)

Annamycin

Liposomal anthracycline

Relapsed or refractory acute myeloid leukemia

Enrollment has begun

3/27/18

Oncbiomune Pharmaceuticals Inc. (Baton Rouge, La.)

Proscavax

Combination of prostate cancer associated prostate specific antigen with the biological adjuvants interleukin-2 and granulocyte-macrophage colony-stimulating factor

Early stage prostate cancer

Received minimal comments from the scientific review committee responsible for its planned phase II trial, and the company plans to incorporate the suggested revisions in the upcoming days

3/23/18

Orsenix LLC (Atlanta)

ORH-2014

Oral form of arsenic trioxide

Acute promyelocytic leukemia

Completed an end-of-phase II meeting with the FDA and reached a general agreement on the key elements of the phase III program to support an NDA filing

3/21/18

Pharmacyte Biotech Inc. (Laguna Hills, Calif.)

Ifosfamide

Cell-in-a-box therapy

Locally advanced, non-metastatic, inoperable pancreatic cancer

Began a phase IIb trial

3/1/18

Progenics Pharmaceuticals Inc. (New York) 

Azedra 

Targeted, high-specific-activity radiotherapeutic candidate

Malignant, recurrent and/or unresectable pheochromocytoma and paraganglioma 

Biochemical tumor marker data from its pivotal phase II trial showed the overall tumor biomarker response correlated significantly with responder status with both the primary endpoint (r = 0.31, p = 0.011) and objective tumor response (r=0.35, p=0.006); Azedra was shown to have a significant positive impact on the cardiovascular symptoms associated with pheochromocytoma and paraganglioma, and also demonstrated objective antitumor effects as measured by RECIST criteria

3/20/18

Sellas Life Sciences Group Inc. (New York) 

Galinpepimut-S

Heteroclitic multivalent, multipeptide cancer immunotherapeutic agent

High-risk multiple myeloma

Data from an open-label phase II study showed patients had a median progression-free survival of 23.6 months, compared to historically inferior outcomes while on an immunomodulatory drug or proteasome inhibitor post-autologous stem cell transplant maintenance

3/20/18

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan)

Iclusig

Ponatinib; BCR-ABL1 inhibitor

Refractory chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL)

Data from the phase II Pace trial showed that, of the 267 chronic phase CML patients, 60% achieved major cytogenic response, including 54% who achieved complete cytogenic response, and 40% of patients achieved major molecular response and 24% achieved molecular response 4.5; for the 85 patients with advanced phase CML, major hematologic response (MaHR) was achieved by 61% of patients; the MaHR rates for 62 blast-phase CML patients and 32 patients with Ph+ ALL were 31% and 41%, respectively

3/23/18

Tesaro Inc. (Waltham, Mass.)

Niraparib

PARP inhibitor

Ovarian cancer

TOPACIO trial in combination with pembrolizumab showed overall response rate of 25% and disease control rate of 68%; ORR was 24% in the platinum-refractory population; response rates were not dependent on biomarker status

3/27/18

Tyme Technologies Inc. (New York)

SM-88

Combination therapy

Pancreatic cancer

The FDA accepted its IND for a phase II trial

3/15/18

Tyme Technologies Inc. (New York)

SM-88

Tyrosine derivative

Metastatic pancreatic cancer

The first site is open for enrollment

3/27/18

Cardiovascular

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

CLBS-12

CD34 cell therapy

No-option critical limb ischemia

Treated the first patient in a phase II trial in Japan

3/14/18

Miragen Therapeutics Inc. (Boulder, Colo.) and Les Laboratoires Servier SAS (Neuilly-sur-Seine, France)

MRG-110 (S95010)

Locked nucleic acid modified oligonucleotide inhibitor of microRNA-92

Heart failure

Initiated trial to evaluate safety and tolerability in a systemic dosing protocol

3/27/18

Myokardia Inc. (South San Francisco) 

Mavacamten (formerly MYK-461)

Oral, allosteric modulator of cardiac myosin

Symptomatic, obstructive hypertrophic cardiomyopathy

Data from the phase II PIONEER-HCM trial showed that both cohort A, in which patients received 10-mg, 15-mg and 20-mg doses, and cohort B, which tested 2-mg and 5-mg doses, met their primary endpoint of reduction in post-exercise left ventricular outflow tract gradient from baseline to week 12 with statistical significance

3/12/18

Reata Pharmaceuticals Inc. (Irving, Texas)

Bardoxolone methyl

Activator of Nrf2

Pulmonary hypertension in patients with interstitial lung disease in the form of either idiopathic pulmonary fibrosis or sarcoidosis

Top-line data from the phase II Lariat trial showed those who received bardoxolone for 16 weeks had a 38-meter increase in the six-minute walk test, compared to a 13-meter reduction in patients taking placebo; in the 25 patients with sarcoidosis, the patients taking bardoxolone for 16 weeks had a 17-meter increase, compared to a nine-meter increase in patients taking placebo

3/23/18

Savara Inc. (Austin, Texas)

Aironite

Sodium nitrate inhalation solution

Heart failure

Results from the phase II INDIE study in patients with heart failure with preserved ejection fraction showed it did not meet its primary endpoint of improvement in peak exercise capacity assessed by cardiopulmonary exercise testing or its secondary endpoints

3/15/18

Dermatologic

Follicum AB (Lund, Sweden)

FOL-005

Modified short version of osteopontin

Hair loss

Treated the first patient in its phase IIa trial

3/8/18

Inflarx NV (Jena, Germany)

IFX-1

Anti-human complement factor C5a antibody

Moderate or severe hidradenitis suppurativa

Enrolled the first patient in its phase IIb study

3/9/18

Endocrine/Metabolic

Caelum Biosciences Inc. (New York)

CAEL-101 (MAb 11-1F4)

Chimeric fibril-reactive monoclonal antibody

Relapsed or refractory amyloid light chain amyloidosis

Demonstrated correlation between sustained decrease in natriuretic peptide-proBNP and improvement in global longitudinal strain in cardiac population following CAEL-101 treatment in the phase Ib study

3/27/18

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

CLBS-03

Personalized autologous cell therapy

Recent-onset type 1 diabetes

Interim phase II analysis showed it continues to be well-tolerated and led to the conclusion that the study was non-futile as determined by pre-defined futility criteria for therapeutic effect

3/9/18

Eiger Biopharma-ceuticals Inc. (Palo Alto, Calif.)

Exendin 9-39

GLP-1 antagonist

Post-bariatric hypoglycemia

First patient dosed in PREVENT, with target enrollment of 20

3/26/18

Esperion Therapeutics Inc. (Ann Arbor, Mich.)

Bempedoic acid

Cholesterol biosynthesis blocker

Elevated low-density lipoprotein cholesterol

Study met primary endpoint with 30% additional LDL-C lowering (p<0.001); achieved a significantly greater reduction of 34% in high-sensitivity C-reactive protein; observed safe and well-tolerated when added to PCSK9 therapy

3/27/18

Jupiter Orphan Therapeutics Inc. (Jupiter, Fla.)

Jotrol

Trans-resveratrol product

Mucopoly-saccharidosis type I

Submitted an IND to the FDA

3/15/18

Laboratoris Sanifit SL (Palma, Spain)

SNF-472

Blocks pathological cardiovascular calcification progression

Calciphylaxis

Both the primary endpoint of wound healing and the secondary endpoint of reduction of pain were successfully met in the phase II trial, but the company is holding the full trial results for a scientific meeting in mid-2018

3/7/18

Opko Health Inc. (Miami)

OPK-88003

Once-weekly oxyntomodulin

Type 2 diabetes

Enrolled the first patient in its phase IIb dose-escalation trial

3/26/18

Stealth Biotherapeutics Inc. (Boston)

Elamipretide

Mitochondria-targeting peptide

Barth syndrome

Completed enrollment in the phase II/III TAZPOWER trial

3/7/18

Zafgen Inc. (Boston)

ZGN-1061

MetAP2 inhibitor

Type 2 diabetes

Phase II data from 57 patients treated for eight weeks showed that the 0.9-mg dose produced a statistically significant placebo-adjusted change in A1C of -0.57%

3/8/18

Gastrointestinal

Arena Pharmaceuticals Inc. (San Diego)

Etrasimod

Once-daily oral sphingosine 1-phosphate receptor modulator

Ulcerative colitis

New data from the phase II trial Oasis showed that patients dosed with 2 mg gained statistically significant improvements vs. placebo in all endpoints: primary, secondary and clinical remission, and the drug's profile appears not to require dose titration

3/21/18

Immuron Ltd. (Melbourne, Australia) 

IMM-124E

Oral antibody therapy targeting the endotoxin lipopolysaccharide (LPS) and other bacterial components

Nonalcoholic steatohepatitis

Data from its phase II trial showed that treatment for six months resulted in 64.29% of patients having a 15% or greater decrease in serum LPS levels compared to 34.48% of patients who received placebo; the drug also reduced serum ALT levels by at least 30% in 36.36% of patients compared to 13.64% of patients who received placebo; more patients receiving IMM-124E also reached their goal for serum CK-18 levels compared to placebo

3/12/18

NGM Bio Inc. (South San Francisco)

NGM-282

Engineered variant of the human hormone FGF19

Nonalcoholic steatohepatitis (NASH)

Confirmed results from an exploratory 12-week phase II trial, which revealed improvements in histological measures of disease; treatment improved fibrosis and NASH-related histology in patients with biopsy-confirmed NASH

3/13/18

Protagonist Therapeutics Inc. (Newark, Calif.)

PTG-100

Oral GI-restricted alpha-4-beta-7 integrin antagonist peptide

Moderate to severe ulcerative colitis

Discontinuing phase IIb PROPEL study after a Data Monitoring Committee deemed the trial futile based on an analysis of the primary endpoint of clinical remission

3/26/18

Protalix Biotherapeutics Inc. (Carmiel, Israel)

OPRX-106

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

Active mild to moderate ulcerative colitis

Results from a phase II trial showed treatment for eight weeks produced a clinical response in 67% of the patients; clinical remission occurred in 28% of the patients; rectal bleeding scores, fecal calprotectin levels and Geboes scores also improved

3/14/18

Redhill Biopharma Ltd. (Tel Aviv, Israel, and Raleigh, N.C.)

Bekinda

RHB-102

Diarrhea-predominant irritable bowel syndrome

Bekinda 12 mg successfully met its primary endpoint, improving stool consistency by an absolute difference of 20.7% vs. placebo (p=0.036)

3/26/18

Ritter Pharmaceuticals Inc. (Los Angeles)

RP-G28

Mitigates lactose intolerance symptoms by increasing lactose-metabolizing bacteria in the colon

Lactose intolerance

Microbiome data from a phase IIb study showed it modified the relative abundance of 28 bacterial species; specifically, a dramatic increase in species of Bifidobacterium was observed, including some known to metabolize lactose

3/13/18

Genitourinary/Sexual Function

AM-Pharma NV (Bunnik, the Netherlands)

Recap

Recombinant alkaline phosphatase

Sepsis-associated acute kidney injury

Phase II results showed it demonstrated a 40% reduction in mortality vs. placebo; treated patients showed a significant, progressive and sustained improvement in renal function in the 28 days of the trial, and overall, there was a significant and dose-dependent reduction in mortality in the treatment group compared to placebo; Recap did not improve kidney function in the first seven days of treatment, meaning the study failed to meet the primary endpoint

3/12/18

Gtx Inc. (Memphis, Tenn.)

Enobosarm

Selective androgen receptor modulator

Stress urinary incontinence

Phase II data showed it produced a statistically significant increase from baseline in mean levator ani muscle thickness, mean inner urethral muscle diameter and mean outer urethral muscle diameter, all measured by magnetic resonance imaging; in 11 of the 18 women who also had urge incontinence (UI), enobosarm produced a mean reduction in their UI episodes of 68%

3/6/18

Velicept Therapeutics Inc. (Malvern, Pa.) 

Solabegron

Selective beta-3 adrenoceptor agonist

Overactive bladder

Started the first of two phase IIb trials

3/20/18

Immune

Ablynx (Ghent, Belgium)

Vobarilizumab

Anti-IL-6R Nanobody

Systemic lupus erythematosus

Missed primary endpoint of dose response based on mBICLA at week 24, but favorable safety through week 58

3/26/18

Anaptysbio Inc. (San Diego)

ANB-020

Antibody that binds and inhibits IL-33

Peanut allergy

Top-line data showed six of 13 (46%) patients with moderate to severe symptoms at baseline improved peanut tolerance to cumulative 500 mg at day 14 after a single dose, compared with 0 placebo patients; concomitant allergy symptoms occurred in 80% of placebo, 7% of treated; therapy was well tolerated

3/27/18

Cytodyn Inc. (Vancouver, Wash.)

PRO-140

Humanized IgG4 monoclonal antibody directed against CCR5

Graft-vs.-host disease

The independent data monitoring committee for its phase II trial completed a planned interim analysis on the first 10 patients enrolled, and the company plans to amend the trial protocol and obtain accordance with the FDA

3/15/18

Flex Pharma Inc. (Boston)

FLX-787

Novel dual transient receptor potential A1/V1 ion channel activator

Multiple sclerosis

Statistically significant 27.3% reduction in frequency of cramps/spasms compared with control (p=0.001); a 1.4-day increase in cramp/spasm-free days per 14-day period (p=0.0457); clinician-rated improvement in spasticity better than control (p=0.01); well tolerated with no treatment-related serious adverse events

3/26/18

Geneuro (Geneva) and Servier (Paris)

GNbAC1

Monoclonal antibody designed to neutralize the pHERV-W protein

Multiple sclerosis

12-month data showed a significant, consistent positive impact on key neuroprotection markers known to be linked to disease progression; relative volume loss reductions of 31% and 72% in cortical and thalamic atrophy (p=0.045 and p=0.014); confirmed safety

3/26/18

Glenmark Pharmaceuticals Ltd. (Mumbai, India)

Ryaltris

Fixed-dose combination nasal spray

Seasonal allergic rhinitis

A phase II study testing it twice daily or once daily vs. twice-daily azelastine/fluticasone, olopatadine hydrochloride or placebo in a ragweed pollen environmental exposure chamber showed onset of action with twice-daily dose vs. placebo was observed at 10 minutes after the first dose (p=0.019) and was maintained at later time points except at 2.5 hours (p=0.06); onset of action could not be defined for it dosed once-daily

3/6/18

Merck KGaA (Darmstadt, Germany)

Evobrutinib

BTK inhibitor

Relapsing multiple sclerosis

The phase IIb study met the primary endpoint, demonstrating that treatment resulted in a clinically meaningful reduction of gadolinium enhancing T1 lesions measured at weeks 12, 16, 20 and 24 in comparison to patients receiving placebo

3/8/18

Morphosys AG (Planegg, Germany)

MOR-208

Antibody targeting CD19

Relapsed or refractory diffuse large B-cell lymphoma

Updated data from the phase II L-MIND trial testing MOR-208 plus Revlimid (lenalidomide, Celgene Corp.) in 81 patients who are not eligible for high-dose chemotherapy and autologous stem cell transplantation showed that, among the 68 evaluable patients, the overall response rate was 49%, with 31% of patients having a complete response

3/15/18

Infection

Ablynx NV (Ghent, Belgium)

ALX-0171

Inhaled Nanobody targeting the F-protein

Respiratory syncytial virus

Treated the first infant in a Japanese phase II trial

3/5/18

Aclaris Therapeutics Inc. (Wayne, Pa.)

A-101 45% topical solution 

High-concentration hydrogen peroxide topical solution 

Common warts (verruca vulgaris)

Data from the three-month, post-treatment, follow-up evaluation period of its twice-weekly placebo-controlled trial (WART-203) showed greater improvements in common wart reduction and clearance vs. placebo observed among subjects treated with A-101 45%

3/20/18

Allecra Therapeutics (Lorrach, Germany, and Saint Louis, France)

AAI101

Extended spectrum B-lactamase inhibitor

Complicated urinary tract infections, including acute pyelonephritis

Phase II CACTUS met all study objectives when used in combination with cefepime; it achieved target blood levels associated with efficacy and was safe and well tolerated

3/26/18

Altimmune Inc. (Gaithersburg, Md.)

Nasovax

Intranasal influenza vaccine

Influenza

Showed 100% seroprotection in the mid- and high-dose groups in 60 phase IIa patients

3/27/18

Bavarian Nordic A/S (Copenhagen, Denmark)

MVA-BN RSV

Universal vaccine

Respiratory syncytial virus

Phase II data showed patients receiving the vaccine had a 1.5-fold increase in IgA antibodies over baseline levels in their nasal mucosa, with peak increases occurring in the weeks after vaccination; patients with low levels of baseline mucosal IgA at the time of vaccination had a more pronounced boost in IgA antibodies

3/7/18

Cidara Therapeutics Inc. (San Diego)

Rezafungin

Echinocandin antifungal

Candida infections

Additional details from the phase II STRIVE trial show assessments confirm that both rezafungin dosing regimens were equally efficacious as predicted by the phase II pharmacokinetic data, which showed that both dosing regimens provided therapeutic exposure substantially above that needed to eradicate Candida infections

3/22/18

Cidara Therapeutics Inc. (San Diego)

Rezafungin acetate

Antifungal candidate

Candidemia and/or invasive candidiasis

Top-line results from the phase II STRIVE study showed the study met all of its primary objectives, as once-weekly intravenous dosing was observed to be generally well-tolerated and safe in patients

3/20/18

Hansa Medical AB (Lund, Sweden)

Ides

Imlifidase

IgG-degrading enzyme of Streptococcus pyogenes

Results from its phase II study showed it eliminated IgG in sensitized chronic kidney disease patients with unprecedented efficacy and no intact IgG could be detected less than one hour following Ides treatment; anti-HLA IgG antibody reactivity was substantially reduced in all patients and C1q binding to anti-HLA IgG was abolished

3/22/18

Imutex Ltd. (joint venture of Hvivo plc and Peptcell Ltd., both of London)

FLU-v

Influenza vaccine

Influenza

Preliminary analysis of the primary endpoint revealed a trend to stastistical significance; demonstrated a statistically significant reduction in overt flu symptoms in treated subjects in one arm of the trial (p=0.02)

3/26/18

Myr Pharma GmbH (Burgwedel, Germany)

Myrcludex B

Entry inhibitor

Chronic hepatitis delta virus

Completed its MYR 202 phase IIb trial

3/13/18

Savara Inc. (Austin, Texas)

Molgradex

Inhaled formulation of recombinant human GM-CSF

Nontuberculous mycobacterial lung infection

Started a phase IIa trial, dubbed OPTIMA

3/15/18

Spring Bank Pharmaceuticals Inc. (Hopkinton, Mass.)

Inarigivir soproxil

Antiviral agent

Chronic hepatitis B virus (HBV)

Data from part A of its ongoing phase II ACHIEVE trial showed that, of the 30 patients treated with 25 mg or 50 mg of inarigivir, eight patients had a potent antiviral response, defined as a greater than 1log10 reduction in HBV DNA and a greater than 3log10 reduction – or to undetectable – in HBV RNA at the end of 12 weeks; in the 11 HBeAg-negative patients, 55% had a potent antiviral response

3/15/18

Inflammatory

Mimedx Group Inc. (Marietta, Ga.)

Amniofix

Composite amniotic tissue membrane

Osteoarthritis of the knee

Enrolled first patient in prospective, double-blind, randomized, controlled phase IIb study

3/28/18

Musculoskeletal

Amo Pharma Ltd. (London)

AMO-02

Tideglusib

Congenital and childhood onset myotonic dystrophy type 1

Final data from its phase II study showed AMO-02 was safe and well-tolerated, with no early discontinuations or dose adjustments required; most patients showed clinical benefits, including improvements in cognitive function, levels of fatigue and the ability to perform daily tasks; there was a demonstrated greater response in patients who received 1,000 mg/day of AMO-02 vs. the 400-mg/day dose

3/16/18

Biophytis SA (Paris)

BIO-101

Sarconeos

Sarcopenia

Phase II data demonstrate its strong treatment and confirm the relevance of the SARA-INT international clinical study, in which the first patient will be enrolled soon

3/2/18

Reata Pharmaceuticals Inc. (Irving, Texas)

Omaveloxolone

Activator of Nrf2

Mitochondrial myopathies

In the phase II MOTOR trial, the 160-mg dose was selected as the optimal dose with robust Nrf2 induction and improvement in markers of mitochondrial function; that dose didn't improve peak work or six-minute walk distance vs. placebo – primary and secondary endpoints of the trial – but did improve heart rate and blood lactate levels compared to placebo in a submaximal exercise test; it produced a placebo-corrected reduction in heart rate of 12 beats per minute and blood lactate of 1.3 mM in 10 patients treated with 160 mg and 13 placebo-treated patients

3/5/18

Summit Therapeutics plc (Oxford, U.K.)

Ezutromid

Small molecule utrophin modulator

Duchenne muscular dystrophy (DMD)

Began enrollment for a planned additional group in the phase II PhaseOut DMD trial, allowing patients in the phase I trials of ezutromid who don't meet the entry criteria for the main cohorts to be enrolled in the additional group

3/8/18

Neurology/Psychiatric

Antibe  Therapeutics Inc. (Toronto)

ATB-346

Hydrogen sulfide-releasing derivative of naproxen

Acute and chronic pain

Results from a phase IIb trial comparing the gastrointestinal safety of ATB-346 to naproxen in 244 healthy volunteers showed that, at the end of the two-week treatment period, subjects taking ATB-346 had an ulceration rate of 2.5% compared to 42.1% for subjects on naproxen

3/21/18

Biogen Inc. (Cambridge, Mass.)

Spinraza

Nusinersen

Spinal muscular atrophy

Interim data from the ongoing phase II NURTURE trial testing Spinraza in 25 pre-symptomatic infants showed the patients had a mean CHOP INTEND score of 58.4 out of 64 at last visit, with all the infants achieving the age-expected World Health Organization motor milestone of sitting without support

3/13/18

Boehringer Ingelheim GmbH (Ingelheim, Germany)

Pradaxa

Dabigatran

Stroke prevention

Two-year safety data from the phase II GLORIA-AF trial in patients with atrial fibrillation showed there was major bleeding in 0.97% of patients taking Pradaxa while strokes occurred in 0.65% of the patients; in 412 patients taking Pradaxa who underwent a cardiovascular intervention, there was just one major bleed and one systemic embolic event

3/21/18

Cavion Inc. (Charlottesville, Va.)

CX-8998

Inhibitor of Cav3 channels

Generalized epileptic syndromes with drug-resistant absence seizures

Enrolled the first patient in its phase IIa T-WAVE trial in 30 adolescents and young adults

3/21/18

EIP Pharma LLC (Cambridge, Mass.)

Neflamapimod,

Inhibitor of p38 MAP kinase alpha

Early Alzheimer's disease

Phase IIa data showed that it improved episodic memory function as assessed by immediate recall and delayed recall composites of the relevant components of the Wechsler Memory Scale (WMS); individual patient's plasma drug concentration profiles were correlated with the level of improvement in combined WMS immediate and delayed recall; four of the 16 patients met the pre-defined responder criteria for a significant reduction in brain amyloid plaque load

3/8/18

Enterin Inc. (Philadelphia)

ENT-01

Orally administered synthetic derivative of squalamine

Parkinson's disease

Completed enrollment of a two-stage phase IIa study in 50 patients

3/27/18

Flexion Therapeutics Inc. (Burlington, Mass.)

Zilretta

Triamcinolone acetonide extended-release injectable suspension

Osteoarthritis knee pain

Analysis of three phase II/III trials showed that at 12 weeks after intra-articular treatment, pain reduction with it was statistically significant in the subgroup with no prior intra-articular corticosteroid (IACS) compared with saline-placebo (p=0.0002) and numerically favored it over placebo in the subgroup with prior IACS (p=0.0530)

3/7/18

Mimedx Group Inc. (Marietta, Ga.)

Amniofix

Injectable therapy

Plantar fasciitis

Demonstrated a clinically and statistically significant difference compared to patients in the control group in their reduction in the visual analog scale score for pain (p<0.0001) and Foot Function Index-Revised scores (p=0.0004) at three months compared to baseline

3/26/18

Nuvelution Pharma Inc. (San Francisco)

Austedo

Deutetrabenazine

Tourette syndrome

Enrolled the first patient in the phase II/III ARTISTS 1 trial

3/1/18

Tetra Discovery Partners Inc. (Grand Rapids, Mich.) 

BPN-14770

Selective small-molecule inhibitor of the phosphodiesterase type-4D enzyme

Fragile X syndrome 

The FDA accepted the IND 

3/19/18

Therapix Biosciences Ltd. (Tel Aviv, Israel)

THX-110

Cannabinoid candidate

Chronic low back pain

The FDA cleared the IND, and a phase IIa study is expected to begin in the second quarter

3/21/18

Ocular

Aerpio Pharmaceuticals Inc. (Cincinnati)

AKB-9778

Small molecule inhibitor of VE-PTP

Diabetic macular edema

Reported that preliminary results of a post-hoc analysis of data from the TIME-2 phase IIa study of AKB-9778 suggest a possible role for the drug in protecting the kidney vasculature in patients with diabetes

3/1/18

Ocugen Inc. (Malvern, Pa.)

OCU-310

Combination of brimondine tartrate and the corticosteroid loteprednol etabonate

Dry eye disease

Data from its phase II trial showed the tolerability of OCU-310 compared to placebo was similar through 12 weeks of treatment using a visual analogue scale, meeting the primary endpoint; patients treated with OCU-310 had better improvement on their Symptom Assessment Questionnaire in Dry Eye scores than patients treated with placebo

3/21/18

Opthea Ltd. (Melbourne, Australia)

OPT-302

VEGF-C/VEGF-D Trap therapy

Wet age-related macular degeneration

Began dosing patients in Europe in the ongoing phase IIb trial in combination with Lucentis

3/27/18

Realm Therapeutics plc (Malvern, Pa.)

PR-013

Topical ophthalmic solution

Allergic conjunctivitis

Its phase II study did not demonstrate efficacy, and the company decided to discontinue further development

3/13/18

Other/Miscellaneous

Lyric Pharmaceuticals Inc. (South San Francisco)

LP-101

Ulimorelin

Enteral feeding intolerance

Completed enrollment in its phase II PROMOTE trial testing in 120 critically ill patients

3/6/18

Novo Nordisk A/S (Bagsvaerd, Denmark) 

Semaglutide

Analogue of the GLP-1 hormone

Obesity

Phase II data showed that adults treated with semaglutide 0.4 mg, administered once daily via subcutaneous injections, lost up to 13.8% of their body weight after 52 weeks, significantly more than those treated with placebo who lost 2.3% of their body weight

3/20/18

Oragenics Inc. (Tampa, Fla.)

AG013

Oral rising solution designed to deliver Trefoil Factor I

Oral mucositis

Completed enrollment of the interim analysis cohort of 20 patients

3/23/18

Prometic Life Sciences Inc. (Laval, Quebec)

PBI-4050

Erythropoiesis stimulant

Alström syndrome

Observed a regression in cardiac and liver fibrosis in treated patients with the ultra-rare pediatric disorder

3/29/18

Respiratory

Verona Pharma plc

RPL-554

Dual phosphodiesterase 3 and 4 inhibitor

Cystic fibrosis

Preliminary phase II data showed that a single dose improved lung function and a sustained improvement in FEV1 of 6% vs. baseline; the improvements seen were statistically significant at both 1.5-mg (p<0.05) and 6-mg doses (p<0.01) at four-, six- and eight-hour time points; the effects were independent of patient genotype

3/5/18

Verona Pharma plc (London)

RPL554

Inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4

Chronic obstructive pulmonary disease

Met primary endpoint, clinically and statistically significant improvements in lung function at all dose levels (p<0.001); clinically relevant secondary endpoints also met; was well tolerated at all dose levels in the four-week 400-patient study

3/26/18

Toxicity/Intoxication

Medicinova Inc. (La Jolla, Calif.)

Ibudilast

Anti-inflammatory and neuroprotective agent

Amphetamine dependence

Missed the primary endpoint of methamphetamine abstinence confirmed via urine drug screens during the final two weeks of treatment (weeks 11 and 12)

3/29/18


Notes

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

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