Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Selecta Biosciences Inc. (Watertown, Mass.)

SEL-212

Immunotherapeutic from the synthetic vaccine particle platform

Chronic refractory and tophaceous gout

Completed patient visits for its phase Ia/b trials; combining SVP-Rapamycin and pegsiticase, ADAs can be prevented, enabling the pegsiticase enzyme to substantially and sustainably reduce serum uric acid levels for 30 days or longer

12/8/16

CANCER

Acea Biosciences Inc. (San Diego)

AC0010

Third-generation epidermal growth factor receptor tyrosine kinase inhibitor

Lung cancer

Phase I/II dose-escalation and expansion study data showed an overall response rate of 52%, with a disease control rate of 94%, in 48 patients treated with 300 mg twice daily; in the dose cohorts between 200 mg twice daily and 300 mg twice daily, the overall response rate was 50% and the disease control rate was 90%; the trial was conducted in China

12/19/16

Agenus Inc. (Lexington, Mass.)

INCAGN1949

Anti-OX40 agonist antibody

Advanced or metastatic solid tumors

The first patient has been dosed in a phase I/II trial

12/1/16

Alopexx Oncology LLC (Concord, Mass.)

DI-Leu16-IL2

A recombinant antibody fusion protein (immunocytokine) composed of interleukin-2 and a CD20-targeting monoclonal antibody

Tumors

Phase I data showed 15 of 18 patients receiving two or more cycles of therapy had tumor regression or stabilization including three complete and two partial responses

12/9/16

Argenx NV (Breda, the Netherlands)

ARGX-110

SIMPLE Antibody targeting CD70

Newly diagnosed, elderly acute myeloid leukemia

Started a phase I/II trial of ARGX-110 in combination with azacitidine

12/16/16

Ariad Pharmaceuticals Inc. (Cambridge, Mass.)

Brigatinib

Anaplastic lymphoma kinase (ALK) inhibitor

ALK-positive non-small-cell lung cancer and intracranial central nervous system metastases

Ongoing phase I/II and pivotal ALTA trial data showed the confirmed intracranial objective response rate (ORR) was 53% in the phase I/II trial, and the confirmed intracranial ORR was 67% in arm B (brigatinib 180 mg with seven-day lead-in at 90 mg once daily) in the ALTA trial; median intracranial progression-free survival in ALTA arm B was 18.4 months

12/6/16

Basilea Pharmaceutica Ltd. (Basel, Switzerland)

BAL101553

A small molecule oncology drug

Glioblastoma

Dosed the first patient in a new arm of its phase I/IIa trial; the new arm includes adult patients with recurrent or progressive glioblastoma who have been treated with prior radiotherapy, with or without chemotherapy

12/5/16

Bavarian Nordic A/S (Copenhagen)

CV301

Immunotherapy vaccine

Non-small-cell lung cancer patients who have failed prior therapy

Initiated a phase I/II trial combining it with Opdivo (nivolumab, Bristol-Myers Squibb Co.)

12/30/16

Beigene Ltd. (Beijing)

BGB-290

A poly ADP ribose polymerase inhibitor

Advanced solid tumors

Dosed the first patient in a phase I trial

12/23/16

Bluebird Bio Inc. (Cambridge, Mass.)

bb2121

Anti-BCMA CAR T cell product candidate

Relapsed/refractory multiple myeloma

Phase I data showed an overall response rate of 78%, and showed no dose-limiting toxicities to date

12/2/16

Bristol-Myers Squibb Co. (New York)

Opdivo

Nivolumab

Chemotherapy-naïve advanced non-small-cell lung cancer

Phase Ib trial, CheckMate-012, data of Opdivo monotherapy, or in combination with Yervoy (ipilimumab) showed the median progression-free survival in patients with PD-L1 expression ≥1% (n=46) was 12.7 months (95%) and was not reached in patients with PD-L1 expression >50%; the one-year overall survival rate was 100% in the pooled combination cohorts; the confirmed objective response rates in all treated patients was 43%, nearly double the response rate reported with Opdivo monotherapy (23%)

12/6/16

Bristol-Myers Squibb Co. (New York)

Opdivo

Nivolumab

Small cell lung cancer

Phase I/II data showed the confirmed objective response rate (primary objective) was 25% in patients who received Opdivo plus Yervoy and was 11% with Opdivo alone with additional follow-up

12/7/16

Calithera Biosciences Inc. (South San Francisco)

CB-839

Glutaminase inhibitor

Renal cell carcinoma

Out of 15 patients treated during a phase I study, 93% achieved disease control; one patient had a partial response, one patient had progressive disease and 13 patients had stable disease; the median progression-free survival at the cut-off date was 8.5 months

12/1/16

Cellectar Biosciences Inc. (Madison, Wis.)

CLR 131

Delivers cytotoxic radioisotope iodine-131 to tumor cells via the PDC delivery platform

Relapsed or refractory multiple myeloma

Phase I data showed that cohort 1 (12.5 mCi/m2 dose) and cohort 2 (18.75 mCi/m2 dose) patients have demonstrated post-treatment median survival of 11.9 months and 4.9 months, respectively. The median overall survival is not yet evaluable

12/2/16

Druggability Technologies Holdings Ltd. (Swatar, Malta)

DRGT-45

Super-API version of Zytiga (Johnson & Johnson); abiraterone

Prostate cancer

The version can eliminate the pharmacokinetic issues associated with the marketed drug, which requires high-dose administration under strict fasted conditions due to a significant food effect and high variability; DRGT-45 improved exposure fourfold and eliminated the food effect normally associated with the drug and significantly reduced patient variability during a phase I study

12/12/16

Fortress Biotech Inc. (New York)

MB-101

IL13R-alpha2-specific CAR T cell

Glioblastoma

Data from a case study showed treatment with MB-101 led to a regression of glioblastoma; the patient received intraventricular infusions that led to regression of all intracranial and spinal tumors and allowed him to temporarily return to normal life and work activities

12/29/16

Heat Biologics Inc. (Durham, N.C.) and Bristol-Myers Squibb Co. (New York)

HS-110

Utilizes ImPACT-modified lung cancer cells to stimulate a patient's immune system to activate a cytotoxic T cell response against a range of antigens

Non-small-cell lung cancer

One-year results from the first eight trial patients in a phase Ib showed that the HS-110/nivolumab combination was well tolerated with a safety profile consistent with single agent nivolumab

12/7/16

Hutchison Medipharma Co. Ltd. (Shanghai)

Epitinib

Selective oral endothelial growth factor receptor inhibitor

EGFR mutation positive non-small-cell lung cancer with brain metastasis

Phase I data showed it is safe and able to cross the blood-brain barrier; in the China study, the 160 mg dose demonstrated clinical activity in both extra- and intra-cranial and was well tolerated; the study did not reach maximum tolerated dose but determined 160 mg to be the recommended dose

12/8/16

Ignyta Inc. (San Diego)

RXDX-105

VEGFR-sparing RET inhibitor

Solid tumors

Phase I/Ib data showed it demonstrated clinical activity in patients harboring RET molecular alterations, with five out of nine patients who were RET inhibitor-naïve achieving a RECIST response, for a preliminary objective response rate of 56%

12/2/16

Immunogen Inc. (Waltham, Mass.)

IMGN853

Mirvetuximab soravtansine; antibody-conjugate targeting FRalpha

Folate receptor alpha (FRalpha)-positive platinum-resistant ovarian cancer

Phase I data demonstrate the potential benefit of the drug, including a 26% confirmed response rate and median progression-free survival (PFS) of 4.8 months; in a subset of 23 patients with low, medium or high FRalpha, who had received three or fewer prior lines of therapy, there was a 39% objective response rate and median PFS of 6.7 months

12/29/16

Innate Pharma SA (Marseille, France)

Monalizumab

Checkpoint inhibitors; an anti-NKG2A antibody

Ovarian cancer

Data showed that 18 patients with advanced, heavily pretreated ovarian cancer randomized to receive three dose levels of monalizumab during a phase I/II trial tolerated the drug with no apparent dose-limiting toxicities; no major differences in terms of safety were observed across the different dose levels; the most common adverse events reported include fatigue and headaches

12/1/16

Innocrin Pharmaceuticals Inc. (Research Triangle Park, N.C.)

Seviteronel

CYP17-lyase/androgen receptor inhibitor

Triple-negative or estrogen receptor-positive breast cancer

Results from the ongoing phase I/II CLARITY study showed that, of the initial 17 patients treated with 450 mg once daily, three of 11 women with ER+ breast cancer and three of six women with TNBC remained on study without progression for at least 147 days to 236 days and from 135 days to 159 days, respectively

12/14/16

Merck & Co. Inc. (Kenilworth, N.J.)

Keytruda

Pembrolizumab; company's anti-PD-1 therapy

Advanced small cell lung cancer and malignant pleural mesothelioma

Updated findings from the phase Ib KEYNOTE-028 study of Keytruda showed clinical activity and durable responses in some patients

12/7/16

Mustang Bio Inc. (New York; a Fortress Biotech Inc. company)

MB-101

IL13R-alpha-2-specific chimeric antigen receptor T-cell therapy

Glioblastoma

Reported a 7.5 month-long clinical response; infusions of MB-101, provided under a compassionate-use protocol, were well-tolerated and not associated with any toxic effects of grade 3 or higher

12/30/16

Nanobiotix SA (Paris)

NBTXR3

A radio-enhancer nanoparticle

Liver cancers

Phase I/II trial showed a good safety profile, with no serious adverse events recorded

12/15/16

Oncoceutics Inc. (Philadelphia)

ONC201

An orally active DRD2 small-molecule antagonist

Recurrent glioblastoma

Will expand the ongoing study of ONC201, enrolling up to 36 additional patients who have had their glioblastoma recur on temozolomide and radiotherapy

12/21/16

Redhill Biopharma Ltd. (Tel Aviv, Israel)

Yeliva

Sphingosine kinase-2 selective inhibitor

Refractory or relapsed multiple myeloma

The first patients have been screened and a first patient has been dosed in the phase Ib/II clinical study

12/20/16

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD33A

CD33-targeting antibody-drug conjugate

Acute myeloid leukemia

FDA put clinical holds on phase I and phase I/II trials in AML, though enrollment in the phase III CASCADE study continues; four deaths among six patients identified with liver toxicity sparked the action by U.S. regulators, who cited problems that included several cases of veno-occlusive disease

12/28/16

Tyme Technologies Inc. (New York)

SM-88

Designed to target only active cancer cells

Prostate cancer, with rising prostate-specific antigen non-
metastatic disease

Completed enrollment of the phase Ib portion of its open-label phase Ib/II trial

12/13/16

Zenith Epigenetics Ltd. (subsidiary of Zenith Capital Corp.)

ZEN-3694

N/A

Metastatic castration resistant prostate cancer

Initiated a phase Ib trial dosing ZEN-3694 in combination with Xtandi (enzalutamide)

12/15/16

CARDIOVASCULAR

Apellis Pharmaceuticals Inc. (Louisville, Ky.)

APL-2

Self-injected synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b

Paroxysmal nocturnal hemoglobinuria

Phase Ib open-label, dose-escalation trials show the drug reduces the breakdown of red blood cells when given as monotherapy or as an add-on to standard-of-care, intravenous Soliris (eculizumab; Alexion Pharmaceuticals Inc.); with APL-2 at 270 mg as monotherapy, three of three PNH patients achieved a reduction in lactate dehydrogenase levels to below the standard for control in PNH; as an add-on to Soliris, six of six previously transfusion-dependent PNH patients did not require transfusions during the study, and five of six achieved hemoglobin levels within the normal range

12/5/16

CSL Ltd. (Melbourne, Australia)

CSL312

Anti-factor XIIa Mab

Hereditary angioedema, and to prevent thrombosis

Advanced it into phase I in Australia

12/2/16

Edge Therapeutics Inc. (Berkeley Heights, N.J.)

EG-1962

N/A

Subarachnoid hemorrhage

Phase I/II NEWTON data showed all primary and secondary endpoints were achieved; 60% of patients treated with EG-1962 achieved a favorable outcome (scores of 6-8 as measured by the Extended Glasgow Outcome Score) at 90 days, compared to 28% of patients in the active control standard of care, oral nimodipine, arm who achieved a favorable outcome

12/12/16

Kiadis Pharma NV (Amsterdam)

ATIR201

Designed as an adjunctive immunotherapeutic on top of an hematopoietic stem cell transplant

Thalassemia

Obtained regulatory approval in the U.K. to start a phase I/II trial of ATIR201 for thalassemia; a total of up to 10 beta-thalassemia major patients will be enrolled in the study, with results expected in the second half of 2017

12/16/16

CENTRAL NERVOUS SYSTEM

AC Immune SA (Lausanne, Switzerland) and Genentech Inc. (unit of Roche Holding AG; Basel, Switzerland)

Crenezumab

Fully humanized IgG4 anti-A-beta monoclonal antibody

Alzheimer's disease

Genentech presented phase Ib data supporting the binding and increased dosing; the safety study and an exposure-response model predicted an improved outcome of the CREAD phase III trial in patients with prodromal-to-mild AD using the higher dose of 60mg/kg of crenezumab; no dose-limiting toxicities were observed at 30, 45 and 60mg/kg doses of crenezumab and no events of amyloid related imaging abnormality-edema were observed in the study

12/13/16

Axon Neuroscience SE (Bratislava, Slovakia)

AADvac1

Active tau vaccine

Mild-to-moderate Alzheimer's disease

Results of a first-in-human study showed that targeting tau pathology with immunotherapy did not induce abnormal accumulation of fluid and inflammation in the brain, commonly observed with anti-amyloid antibodies; treatment with AADvac1 induced a robust immune response in 29 of 30 vaccinated patients, and patient cognition remained stable on average for the duration of the study

12/13/16

Biogen Inc. (Cambridge, Mass.)

BIIB037

Fully human antibody, aducanumab

Alzheimer's disease

New data from its phase Ib PRIME study showed at 54 weeks, a statistically significant reduction of amyloid plaque was observed in all fixed-dose arms vs. placebo and in the titration arm

12/12/16

C2N Diagnostics Inc. (St. Louis)

ABBV-8E12

Formerly C2N-8E12; humanized anti-tau monoclonal antibody

Progressive supranuclear palsy

Results from its phase I study showed it was safe and well tolerated when administered intravenously in single doses of up to 50 mg/kg; no dose-limiting toxicities occurred, and adverse event frequency and severity did not vary by dose or when compared to placebo; no allergic reactions occurred in any of the study participants

12/12/16

Cognition Therapeutics Inc. (Pittsburgh)

CT1812

An oral, small-molecule therapeutic

Alzheimer's disease

Started U.S. clinical testing of CT1812; the first U.S. patient was dosed in a drug-drug interaction study, which will support Cognition's ongoing phase Ib trial testing CT1812 in Alzheimer's patients in Australia

12/14/16

Egalet Corp. (Wayne, Pa.)

Egalet-002

An abuse-deterrent, extended-release oxycodone product candidate

Severe pain

Top-line results from a category 3 intranasal human abuse potential study showed it hit the primary endpoint of reduced maximum drug liking, are promising

12/19/16

Ensysce Biosciences Inc. (San Diego)

PF614

Two-step extended-release oxycodone prodrug designed with abuse-deterrent properties

Pain

Treated the first two patients in a phase I trial

12/2/16

International Stem Cell Corp. (Carlsbad, Calif.)

ISC-hpNSC cells

Comprised of a neural stem cells derived from human parthenogenetic stem cells

Parkinson's disease

Treated the second patient in its Australian phase I trial

12/8/16

Karuna Pharmaceuticals Inc. (Boston)

Karxt

Xenomeline plus trospium chloride

Schizophrenia and Alzheimer's disease

Results from a tolerability proof-of-concept study found it to be generally well-tolerated and to have superior tolerability to xanomeline alone

12/16/16

Kempharm Inc. (Coraville, Iowa)

d-MPH

Experimental prodrug, extended release version of d-threo-methylphenidate

Attention deficit hyperactivity disorder

During a phase I proof-of-concept study, KP415, it demonstrated pharmacokinetic properties that produced earlier d-MPH exposure followed by a slower extended release of d-MPH than with Concerta (d-MPH, Johnson & Johnson)

12/15/16

Medicinova Inc. (La Jolla, Calif.)

MN-166

Ibuilast

Amyotrophic lateral sclerosis

Exploratory interim data from its ongoing phase Ib/IIa trial showed there were statistically significant decreases in muscle strength two weeks after stopping ibudilast for hip, leg and neck flexion; there was a non-statistically significant decrease two weeks after stopping ibudilast in the ALS Functional Rating Scale-Revised score, a measure of functional impairment, and vital capacity, a measure of respiratory function; ibudilast was well tolerated

12/12/16

Mitsubishi Tanabe Pharma Corp. (Osaka, Japan)

Edaravone

N/A

Amyotrophic lateral sclerosis

Data showed patients experienced significantly less functional loss as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) when treated with edaravone

12/12/16

Neuroderm Ltd. (Rehovot, Israel)

ND0701

Continuous, subcutaneously delivered apomorphine liquid formulation

Severe Parkinson's disease

Completed a pilot study in healthy subjects comparing the pharmacokinetics (PK) of ND0701 and commercial Apo-go (apomorphine, Britannia Pharmaceuticals Ltd.); study results demonstrate that ND0701 produced PK results that were comparable to those produced by the referenced drug

12/5/16

Spinalcyte LLC (Houston)

Cybrocell

Dermal fibroblast cell product

Degenerative disc disease

Received institutional review board approval to begin randomized, double-blind, phase I trials

12/14/16

Tetra Discovery Partners LLC (Grand Rapids, Mich.)

BPN14770

Selective PDE4D allosteric inhibitor

Alzheimer's disease

A pooled, post hoc analysis of elderly subjects in the low and mid-dose groups of two phase I studies showed significant improvement measures of working memory

12/20/16

Voyager Therapeutics Inc. (Cambridge, Mass.)

VY-AADC01

Gene therapy

Parkinson's disease

Phase I data showed that accurate magnetic resonance imaging-guided delivery of escalating doses of VY-AADC01 were well tolerated and resulted in increased coverage of the putamen (a structure in the brain where dopamine receptors are located), plus a boost in aromatic l-amino acid decarboxylase (AADC) enzyme activity, enhanced response to gold-standard PD therapy levodopa, and dose-related, clinically meaningful improvements in various measures of patients' motor function

12/9/16

Zynerba Pharmaceuticals Inc. (Devon, Pa.)

ZYN002

Cannabidiol gel

Epilepsy

Phase I data demonstrated that ZYN002 CBD gel is safe and well-tolerated in both healthy subjects and adult epilepsy patients with focal seizures across a wide range of doses and concentrations

12/6/16

DIABETES

Adocia SA (Lyon, France) and Eli Lilly and Co. (Indianapolis)

Biochaperone Lispro

Ultra-rapid formulation of insulin lispro

Diabetes

Completed an insulin pump study under their partnership; the first part of the study, which compared the products in the Roche Accu-Chek Spirit pump, did not demonstrate a clear advantage for Biochaperone Lispro; the second part of the study included three devices – Roche Accu-Chek Spirit pump, Medtronic Paradigm Veo pump and insulin syringe; Biochaperone Lispro U100 showed a statistically significant increase in insulin exposure over the first 30 minutes compared to Humalog in the two pumps tested: early exposure was increased by 33% in the Roche pump (primary endpoint, p=0.0007) and by 54% in the Medtronic pump (p<0.0001); when delivered as a bolus from syringes, on top of basal delivery with an insulin pump, Biochaperone Lispro also was associated with reductions in 2-hour blood glucose excursion vs. Humalog [-56% with the Roche pump (p=0.0008) and -61% with the Medtronic pump (p<0.0001)]

12/20/16

CSL Ltd. (Melbourne, Australia)

CSL346

Anti-VEGF-B Mab

Type 2 diabetes

Advanced it into phase I in Australia

12/2/16

GASTROINTESTINAL

Deciphera Pharmaceuticals Inc. (Waltham, Mass.)

DCC-2618

A pan-KIT and PDGFR-alpha-targeted tyrosine kinase inhibitor

Gastrointestinal stromal tumors and other KIT-driven diseases, such as systemic mastocytosis

Phase I data showed that partial metabolic responses were observed in 14 of 15 patients with KIT-mutant GIST, along with initial signs of decreases in circulating tumor DNA that codes for KIT; it was well tolerated with an encouraging safety profile

12/2/16

INFECTION

Ampliphi Biosciences Corp. (San Diego)

AB-SA01

Bacteriophage cocktail targeting Staphylococcus aureus infections

Chronic rhinosinusitis

Final results from its phase I trial showed it met the trial's primary endpoints of safety and tolerability and all nine patients enrolled in the study experienced a reduction in the quantity of S. aureus infecting their sinuses, with some patients showing complete eradication of the bacterial infection; all patients experienced a reduction in S. aureus bacterial load at the end of the study compared to baseline

12/20/16

Enyo Pharma SA (Lyon, France)

EYP001

A synthetic farnesoid X receptor agonist

Chronic hepatitis B infection

Started a phase I single and multiple ascending-dose trial testing EYP001 in healthy subjects, with the single dose-escalation completed, showing that EYP001 is safe and well-tolerated at all doses studied in 46 healthy subjects

12/19/16

Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.)

GLS-5700

DNA-based Zika vaccine

Zika virus

It generated robust antigen-specific antibody responses in a first-in-human, multi-center phase I trial

12/22/16

Micurx Pharmaceuticals Inc. (Hayward, Calif.)

MRX-4

Antimicrobial agent; a prodrug form of the oral antibiotic MRX-1

Infections caused by gram-positive bacteria

Started a phase I trial that will enroll 122 healthy subjects in nine single and four multiple ascending-dose cohorts

12/1/16

Poxel SA (Lyon, France)

EYP001

Synthetic farnesoid X receptor (FXR) agonist

Chronic hepatitis B

Said Enyo Pharma SA, of Lyon, France, reported the start of a phase I program

12/20/16

Spero Therapeutics LLC (Cambridge, Mass.)

SPR741

Potentiator molecule

Bacterial infection

Started a phase I clinical trial

12/20/16

Valneva SE (Lyon, France)

VLA15

Hexavalent, protein subunit-based vaccine targeting the outer surface protein A of Borrelia

Lyme disease

Progressed into phase I

12/12/16

Xbiotech Inc. (Austin, Texas)

514G3

Antibody therapy

Staphylococcus aureus infections

Enrollment has been completed in its randomized, placebo-controlled phase I/II study, which has received fast-track status from the FDA

12/7/16

INFLAMMATORY

CSL Ltd. (Melbourne, Australia)

CSL324

An anti-GCSFR Mab

Inflammatory diseases

Advanced it into phase I in Australia

12/2/16

Druggability Technologies Holdings Ltd. (Swatar, Malta)

DRGT-46 and DRGT-47

New compositions of celecoxib, a COX-2 selective nonsteroidal anti-inflammatory drug

Anti-inflammation

Phase I results showed very rapid absorption and the opportunity for immediate pain relief; the published effective concentration for onset of action (250 ng/ml) was reached within 12 minutes at both doses (100 and 200 mg) under fasted and fed (high fat) conditions

12/20/16

Eyegate Pharmaceuticals Inc. (Waltham, Mass.)

EGP-437

Iontophoretic; incorporates a reformulated topically active corticosteroid, dexamethasone phosphate, that is delivered into the ocular tissues through Eyegate's drug delivery system

Post-operative ocular inflammation and pain in cataract surgery

Positive data from its phase Ib/IIa trial demonstrated the most significant ACC improvement, with 40% of subjects in this cohort achieving an ACC count of 0 at day 14 which increased to 88% on day 28

12/6/16

MISCELLANEOUS

Abide Therapeutics Inc. (San Diego)

ABX-1431

A monoacylglycerol lipase inhibitor

Functional dyspepsia

Started dosing in a phase Ib study

12/15/16

Agios Pharmaceuticals Inc. (Cambridge, Mass.)

AG-519

Second pyruvate kinase-R activator

Pyruvate kinase deficiency

Company is no longer developing AG-519 and withdrew its IND following verbal notification of an FDA clinical hold; the decision followed a previously disclosed case of drug-induced cholestatic hepatitis occurring during the bioavailability portion of the phase I healthy volunteer study in the U.K.

12/19/16

Amicus Therapeutics Inc. (Cranbury, N.J.)

ATB200/AT2221

Consists of ATB200, a recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate, to enhance uptake, co-administered with AT2221, a pharmacological chaperone

Pompe disease

Disclosed positive preliminary data from a global phase I/II study that showed that 2/4 patients in cohort 1 (ambulatory enzyme replacement therapy-switch patients) showed early trends toward improvement on biomarkers at 14 weeks

12/9/16

Atyr Pharma Inc. (San Diego)

Resolaris

Physiocrine therapeutic

Limb-girdle muscular dystrophy 2B or facioscapulo-humeral muscular dystrophy

Reports from exploratory trials showed improved muscle strength in 50% to 78% of patients; top-line results from a phase Ib/II trial in adults showed that overall individualized neuromuscular quality of life (INQoL) questionnaire scores were relatively stable for the 10 GGMD2B patients, with overall equal proportions of patients with decreases in disease burden compared to increases in disease burden, while INQoL scores were relatively stable for the eight FSHD patients, with five of eight patients presenting with a small decrease in disease burden over the course of the 12-week trial

12/14/16

Cerenis Therapeutics SA (Toulouse, France)

CER-209

P2Y13 receptor agonist

Non-alcoholic fatty liver disease and non-alcoholic steatohepatitis

FDA cleared it to start phase I testing in healthy volunteers

12/19/16

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-543

A deuterium-modified analogue of JAK inhibitor ruxolitinib

Alopecia areata

Completed a phase I single and multiple ascending-dose trial; the company selected four doses (4 mg, 8 mg, 12 mg and 16 mg twice daily) to explore in its phase IIa trial expected to begin in the first quarter of 2017

12/15/16

Cytori Therapeutics Inc. (San Diego)

ECCS-50

An adipose-derived regenerative cell therapy

Raynaud's disease

A topline review of early three-year follow-up data from the SCLERADEC I trial shows sustained benefit in treated patients over baseline in major study endpoints

12/6/16

Diamedica Inc. (Minneapolis)

DM199

A recombinant human tissue kallikrein protein

Neurological and kidney diseases

Results of its most recent clinical trial identified a dose of DM199 via intravenous administration that produced pharmacokinetic and pharmacodynamic activity comparable to that produced by the reference drug, human urinary kallikrein (Kailikang [kallidinogenase], Techpool Bio-Pharma Co. Ltd.) approved in Asia

12/21/16

Gensight Biologics SA (Paris)

GS010

AAV2 containing the human wild-type ND4 gene

Leber's hereditary optic neuropathy

Additional data after 78 weeks of follow-up in its phase I/II trial confirm the safety and tolerability profile of GS010, while demonstrating sustainable visual acuity improvement

12/21/16

Opsona Therapeutics Ltd. (Dublin)

OPN-305

A humanized IgG4 monoclonal antibody against Toll-like receptor 2

Second-line lower-risk myelodysplastic syndrome

Preliminary results from its ongoing prospective, open-label, phase I/II study showed that of 15 patients evaluable for response – 75% of enrolled patients – hematological improvement was seen in 53% (eight of 15), with three patients (20%) achieving transfusion independence; to date, three (20%) patients were taken off study due to progression to acute myeloid leukemia and four (27%) due to no response all at the 5-mg/kg dose

12/21/16

Notes

Public biopharmaceutical company stock symbols can be found in the stock report located on the last two pages of this issue.

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 Thomson Reuters Cortellis.