Company
(location)

Product

Description

Indication

Status

Date

AUTOIMMUNE

Ablynx NV (Ghent, Belgium) and Merck KGaA (Darmstadt, Germany)

M1095; ALX-0761

Bi-specific anti-interleukin-17A/F nanobody

Moderate-to-severe chronic plaque psoriasis

Phase Ib study showed a reduction in disease activity, as measured by the Psoriasis Area Severity Index (PASI) and improvement in static Physician Global Assessment was seen for all doses of M1095 vs. 0% for placebo; at day 85, all patients treated with 240 mg M1095 experienced a 75% reduction in disease activity (PASI 75) and had clear or almost clear skin (PASI 90), and 56% had clear skin (PASI 100)

3/7/17

Concert Pharmaceuticals Inc. (Lexington, Mass.)

CTP-543

Deuterium-modified analogue of JAK inhibitor ruxolitinib

Moderate-to-severe alopecia areata

Phase I showed it was well-tolerated across all dose groups, with no withdrawals or modifications; CTP-543 showed increased exposure with increasing doses and had a half-life of about 3.3 hours; in an open-label trial of 12 patients with moderate to severe alopecia areata, investigators reported that 20 mg of ruxolitinib administered orally twice daily resulted in significant hair regrowth in 75% of the patients

3/7/17

Galectin Therapeutics Inc. (Norcross, Ga.)

GR-MD-02

Complex carbohydrate drug that targets galectin-3

Severe and refractory atopic dermatitis

Preliminary results from a small, open-label study showed no serious adverse events; all three patients showed clinical response as determined by reduction of the Eczema Area and Severity Index (EASI) score at week 12 having received six every other week doses, with two patients achieving a 64% and 74% reduction in EASI, respectively, at six weeks after receiving only three doses

3/15/17

Sonoma Pharmaceuticals Inc. (Petaluma, Calif.)

Sebuderm

Topical hypochlorous acid gel

Mild to moderate facial and scalp seborrheic dermatitis

In a 25-patient study, no adverse effects were reported and overall treatment was well-tolerated by the subjects; the investigator's global assessment of efficacy improvement from baseline was 33% at day 14 and 52% at day 28; the subject global assessment of efficacy improvement from baseline was 62% through day 28

3/17/17

Nektar Therapeutics Inc. (San Francisco)

NKTR-358

Selectively stimulates the growth and activation of regulatory T cells

Autoimmune diseases and inflammatory disorders

Started dosing in a phase I study

3/28/17

CANCER

Advaxis Inc. (Princeton, N.J.)

Axalimogene filolisbac

Targeted Listeria monocytogenes-based immunotherapy

Persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRmCC)

Phase I data showed that nine patients who had documented disease progression after they had received curative treatments of chemotherapy and/or radiation with or without Avastin (bevacizumab, Roche Holding AG) were enrolled in the open-label, dose-determining study

3/28/17

Apexian Pharmaceuticals Inc. (Indianapolis)

APX-3330

Inhibitor of the APE1 protein

Cancer

Received FDA authorization to start a phase I study designed to affirm the safety, to measure the drug's inhibitory effect upon the APE1 protein and to confirm its ability to disrupt cancer cell signaling

3/2/17

Apexigen Inc. (San Carlos, Calif.)

APX-005M

CD40 agonistic antibody

Metastatic melanoma

Started patient enrollment in a phase I/II trial at the University of Texas MD Anderson Cancer Center, in combination with Keytruda (pembrolizumab)

3/22/17

Athenex Inc. (Buffalo, N.Y.)

Oratopo

Combination of topotecan and HM30181A

Numerous cancers

Received an FDA investigational new drug allowance to begin clinical testing

3/30/17

Aura Biosciences Inc. (Cambridge, Mass.)

AU-011

Light-activated targeted therapy

Ocular melanoma

Enrolled and dosed the first patient in its phase Ib trial

3/31/17

Aveo Oncology Inc. (Cambridge, Mass.)

Tivozanib

Oral, once-daily, vascular endothelial growth factor tyrosine kinase inhibitor

Advanced renal cell carcinoma

The first patient has been dosed in the phase I/II Aveo-sponsored TiNivo trial evaluating it in combination with New York-based Bristol-Myers Squibb Co.'s Opdivo (nivolumab)

3/23/17

Blueprint Medicines Corp. (Cambridge, Mass.)

BLU-667

RET inhibitor

Non-small-cell lung cancer and thyroid cancer

Dosed the first patient in a phase I trial in NSCLC, MTC and other advanced solid tumors that harbor a RET alteration

3/22/17

Briacell Therapeutics Corp.

Briavax

Genetically engineered whole-cell vaccine derived from a human breast tumor cell line

Advanced breast cancer

Following two phase I studies, the FDA cleared the company to begin an open-label phase I/IIa trial

3/16/17

CBT Pharmaceuticals Inc. (Santa Clara, Calif.)

Genolimz-umab (CBT-501)

Humanized IgG4 monoclonal antibody targeting the PD-1 membrane receptor

Advanced solid tumors

The first patient was enrolled in the phase I study in patients recurrent or refractory to standard of care

3/31/17

Celyad SA (Mont-Saint-Guibert, Belgium)

NKR-2 CAR-T cells

Autologous CAR-T cells

Five solid cancers and two hematological malignancies

The FDA authorized the initiation of a phase Ib trial to evaluate NKR-2 CAR-T cells in seven indications

3/9/17

Curelab Oncology Inc. (Boston)

Elenagen

Therapeutic DNA vaccine

Cancer

Phase I/IIa trials demonstrated both the first encouraging evidence of its clinical benefits for cancer patients as well as an excellent safety profile

3/29/17

Deciphera Pharmaceuticals Inc. (Waltham, Mass.)

DCC-3014

Selective small-molecule CSF1R inhibitor

Advanced malignancies

Started a phase I trial

3/7/17

Dynavax Technologies Corp. (Berkeley, Calif.)

SD-101

Intratumoral TLR9 agonist

Metastatic melanoma

Findings from the dose-escalation phase of an ongoing phase Ib/II study investigating SD-101 in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) showed that, in patients naïve to anti-PD-1 treatment, responses were observed in six out of seven patients, for an overall response rate of 86%, including two (29%) complete responses and four (57%) partial responses

3/7/17

Effector Therapeutics Inc. (San Diego)

eFT-508

Selective inhibitor of MNK1 and MNK2

B-cell hematological malignancies

Dosed the first subject in a phase I/II trial

3/10/17

Engeneic Ltd. (New York)

(EGFR)-EDV-doxorubicin

EDV nanocells

Recurrent glioblastoma multiforme

Began dosing patients in a U.S.-based open-label phase I trial

3/22/17

Galena Biopharma Inc. (San Ramon, Calif.)

GALE-301 (E39)

Immunotherapy

Prevention of cancer recurrence in ovarian and endometrial cancer

Final data from a prospective phase I/IIa trial showed the overall disease-free survival for the HLA-A2 positive vaccine group was 50% vs. 44% for an HLA-A2 negative control group (CG) (p=0.594)

3/20/17

Halozyme Therapeutics Inc. (San Diego)

PEGPH-20

Pegylated recombinant human hyaluronidase

Metastatic pancreas cancer

The SWOG phase Ib/II trial evaluating PEGPH20 plus modified FOLFIRINOX chemotherapy vs. modified FOLFIRINOX alone has been temporarily closed to enrollment, following a futility analysis, showing it was unlikely to demonstrate a statistically significant improvement in the primary endpoint of overall survival

3/31/17

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

HS-110

T-cell-stimulating therapeutic vaccine

Non-small-cell lung cancer

Achieved the primary endpoint in its phase Ib trial testing HS-110 in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.); five of 15 patients treated with the combination had 20% or greater tumor reduction

3/14/17

Herantis Pharma plc (Helsinki, Finland)

Lymfactin

Gene therapy

Secondary lymphedema

A data monitoring committee recommended proceeding to high-dose treatments in the phase I study

3/7/17

Hutchison China Meditech Ltd. (Hong Kong)

Sulfatinib

Oral angio-immunokinase inhibitor that selectively targets VEGFR, FGFR and CSF-1R

Advanced neuroendocrine tumors (NETs)

Data from the ongoing phase Ib/II trial in China showed that, as of Jan. 20, 13 patients had confirmed partial responses and 61 patients had stable disease, corresponding to an overall objective response rate of 16% (13/81), with 17.1% (7/41) in pancreatic NET and 15% (6/40) in extra-pancreatic NET, and an overall disease control rate of 91.4%; median overall progression-free survival (PFS) has not been reached, but is estimated to be 16.6 months (95% CI: 13.4, 19.4) with longer median PFS in pancreatic NET estimated at 19.4 months and shorter median PFS in extra-pancreatic NET estimated at 13.4 months

3/13/17

Immunogen Inc. (Waltham, Mass.)

Mirvetuximab soravtansine (IMGN-853)

FRalpha antibody-drug conjugate

Ovarian cancer

Data from a phase I biopsy expansion cohort demonstrated that archival tumor tissue can reliably identify patients with folate receptor alpha-positive, platinum-resistant ovarian cancer; of the 21 evaluable pre-treatment samples, 15 met the eligibility criterion for the biopsy expansion cohort, resulting in a 71% concordance with archival tumor tissues

3/14/17

Immunovaccine Inc. (Halifax, Nova Scotia)

DPX-Survivac

Cancer vaccine

Recurrent ovarian cancer

An interim analysis from an ongoing phase Ib study of DPX-Survivac in combination with epacadostat and low-dose cyclophosphamide showed signs of increased T-cell activity in the tumors of three out of the first four evaluable patients; three of four patients exhibited stable disease, while a fourth patient continued to progress and discontinued the trial

3/30/17

Innate Pharma SA (Marseille, France)

Lirilumab

Directed against the inhibitory killer-cell immunoglobulin-like receptors

Advanced refractory solid tumors

Said partner Bristol-Myers Squibb Co., of New York, amended the protocol for the ongoing phase I/II trial testing lirilumab in combination with Opdivo (nivolumab) to expand in scope to include additional cohorts of Opdivo plus lirilumab in solid tumors, including a randomized cohort exploring Opdivo with or without lirilumab in platinum-refractory recurrent or metastatic squamous cell carcinoma of the head and neck, and initial testing of the triplet combination of Opdivo, Yervoy (ipilimumab) and lirilumab in solid tumors

3/14/17

Kite Pharma Inc. (Santa Monica, Calif.)

Anti-CD19 CAR T-cell therapy

Chimeric antigen receptor (CAR) T-cell therapy

Relapsed/refractory non-Hodgkin lymphoma (NHL)

Results from a National Cancer Institute study showed objective responses (OR) were seen in 73% of patients, and complete remissions (CR) were observed in 55% of patients; among patients with aggressive B-cell NHL, OR and CR were 68% and 47%, respectively; reversible grade 3 or 4 neurotoxicity including confusion, dysphasia, encephalopathy and gait disturbances was observed in 55% of treated patients

3/16/17

Les Laboratoires Servier (part of Servier SAS; Suresnes, France), Pfizer Inc. (New York) and Cellectis SA (Paris)

UCART-19

Allogeneic, gene-edited cellular therapy

Relapsed/refractory acute lymphoblastic leukemia

The FDA cleared the IND to proceed with phase I clinical development

3/10/17

Madison Vaccines Inc. (Madison, Wis.)

MVI-118

Gene-based immunotherapy that uses plasmid DNA

Metastatic prostate cancer

The phase I trial is expanding to a third clinical site, The University of Washington, Seattle

3/22/17

Mateon Therapeutics Inc. (South San Francisco)

OXi-4503

Vascular targeting agent

Relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndrome

Preliminary data from the third dose cohort of its ongoing phase Ib study of the drug combined with cytarabine showed one patient with a high risk TP53 gene mutation had a complete remission; two other patients demonstrated evidence of AML blast reduction after one cycle; the fourth patient did not show a response and experienced progressive disease

3/16/17

Merrimack Pharmaceuticals Inc. (Cambridge, Mass.)

MM-310

Antibody-directed nanotherapeutic that encapsulates a taxane and targets the EphA2 receptor

Solid tumors

Enrolled the first patient in a phase I study

3/24/17

Netris Pharma (Lyon, France)

NP-137

Humanized monoclonal antibody targeting Netrin-1

Advanced/metastatic solid tumors

Started enrollment in its phase I study

3/22/17

Nordic Nanovector ASA (Oslo, Norway)

Betalutin

177Lu-satetraxetan-lilotomab; CD37 targeting antibody-radionuclide conjugate

Relapsed diffuse large B-cell lymphoma

The first patient has been dosed in its phase I study

3/20/17

Oncoceutics Inc. (Philadelphia)

ONC-201

Selectively binds to and antagonizes DRD2

Cancer

Phase data demonstrated it is well-tolerated at plasma concentrations that meet or exceed the targeted therapeutic threshold and is biologically active in advanced cancer patients when orally administered at the recommended phase II dose (RP2D) of 625 mg every three weeks

3/23/17

Oncoceutics Inc. (Philadelphia)

ONC-201

Selectively binds to and antagonizes DRD2

Prostate and endometrial cancer patients

A phase I trial demonstrated that it is well-tolerated and is biologically active when orally administered at the phase II dose of 625 mg every three weeks; no adverse events were observed

3/29/17

Oncolys Biopharma Inc. (Tokyo)

OBP-301

Telomelysin

Esophageal cancer

Submitted a clinical trial notification to Japan's PMDA regarding the first sponsor-initiated phase I trial in Japan

3/15/17

Oncolytics Biotech Inc. (Calgary, Alberta)

Reolysin

Pelareorep

Relapsing myeloma

Said Myeloma U.K. launched MUK eleven, a phase Ib trial to study Reolysin in combination with Summit, N.J.-based Celgene Corp.'s immunomodulatory drugs, Imnovid (pomalidomide) or Revlimid (lenalidomide)

3/17/17

Prima Biomed Ltd. (Sydney)

IMP-321

Antigen presenting cell activator

Unresectable or metastatic melanoma

A second cohort has been fully recruited for its TACTI-mel (Two ACTive Immunotherapeutics in melanoma) trial, in combination with Keytruda (pembrolizumab, Merck & Co. Inc.), being conducted in Australia

3/16/17

Samus Therapeutics Inc. (Boston)

PU-H71 and PU-AD

Anti-epichaperome candidates

Various cancers and neurodegenerative diseases

Launched an expanded development program and expects to file an IND and start various phase Ib trials later this year

3/31/17

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD33A

Vadastuximab talirine

Acute myeloid leukemia

The FDA lifted the clinical hold on phase I trials

3/7/17

Selvita SA (Krakow, Poland)

SEL-24

Dual PIM/FLT3 kinase inhibitor

Acute myeloid leukemia

The first patient was dosed in a phase I/II trial

3/20/17

Spotlight Innovation Inc. (Urbandale, Iowa)

Crotoxin

Snake venom toxin

Advanced cancer

Said its subsidiary, Celtic Biotech Iowa Inc., has begun part two of its phase I dose-escalation safety study

3/13/17

Sun Biopharma Inc. (Minneapolis)

SBP-101

Polyamine compound

Second-line pancreatic cancer

Interim data from its phase I dose-escalation phase showed that of seven patients dosed in the third and fourth cohorts, three showed stable disease at the eight-week conclusion of their first cycle of treatment, using RECIST criteria

3/31/17

CARDIOVASCULAR

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

EG-1962

Polymeric microsphere containing nimodipine suspended in a diluent of sodium hyaluronate

Aneurysmal subarachnoid hemorrhage

Randomized the first patient in its open-label study of intracisternal administration of EG-1962

3/14/17

Nuvox Pharma LLC (Phoenix)

NVX-208

Oxygen therapeutic

Acute ischemic stroke

The first patients were dosed in a phase Ib/II trial

3/7/17

Tigenix NV (Leuven, Belgium)

AlloCSCs

Donor-derived expanded cardiac stem cells

Acute myocardial infarction and left ventricular dysfunction

Top-line one-year results from the phase I/II CAREMI trial showed that all safety objectives were met, with no mortality or major cardiac adverse event (MACE) found at 30 days, meeting the primary endpoint; no mortality and MACE have been found at six months or 12 months of follow-up

3/14/17

CENTRAL NERVOUS SYSTEM

Alzheon Inc. (Framingham, Mass.)

ALZ-801

Amyloid-targeted prodrug of tramiprosate

Mild stage of Alzheimer's disease

In the phase Ib studies in healthy elderly volunteers, a tablet containing 265 mg of ALZ-801 administered twice daily demonstrated markedly improved pharmacokinetic properties, including steady target plasma levels with low intersubject variability, and sustained plasma concentrations

3/31/17

Asterias Biotherapeutics Inc. (Fremont, Calif.)

AST-OPC1

Composed of oligodendrocyte progenitor cells manufactured from its pluripotent embryonic stem cell platform

Spinal cord injury

Including the sixth and final patient in the AIS-A 10 million cell cohort in the ongoing Scistar phase I/IIa trial has further confirmed motor function improvements; the patient showed upper extremity motor function improvement at three months and further improvement at six months; all six patients in this cohort have now achieved at least a one motor level improvement over baseline on at least one side.

3/22/17

Herantis Pharma plc (Helsinki, Finland)

CDNF

Neurotrophic and neuroprotective factor

Parkinson's disease

The Medicines Agency of Sweden has authorized the company's first-in-human study

3/24/17

Intec Pharma Ltd. (Jerusalem)

AP-CBD/THC

Accordion Pill platform with the two primary cannabinoids contained in Cannabis sativa, cannabidiol and tetrahydrocannabinol,

Low back pain and fibromyalgia

Started a phase I trial

3/23/17

International Stem Cell Corp. (Carlsbad, Calif.)

ISC-hpNSC

Cells, intracranially transplanted

Parkinson's disease

The third patient in its Parkinson's disease trial was transplanted with ISC-hpNSC cells, without complications; the 12-patient phase I dose-escalation trial is testing three different dosing regimens of ISC-hpNSC, intracranially transplanted, with patients monitored for 12 months to evaluate safety and biologic activity

3/1/17

Kempharm Inc. (Coralville, Iowa)

KP-511

Prodrug of hydromorphone

Severe pain

Data from a phase I proof-of-concept study showed peak exposure (Cmax) was approximately 19% lower for KP511; similar reductions of approximately 15% and 17%, respectively, in dose-adjusted peak hydromorphone exposure were observed for the 4 mg and 16 mg doses of KP511 with dose-linear Cmax values across all three doses; median time to peak exposure (Tmax) was 0.5 hours for all treatments

3/17/17

Kempharm Inc. (Coralville, Iowa)

KP-201/IR

Prodrug of hydrocodone

Severe pain

Data from the KP201.A03 trial showed the peak hydrocodone plasma concentration was 36% lower, and total hydrocodone exposures (AUClast and AUCinf) were 20.3% and 19.5% lower, respectively, for intranasal (IN) KP201 compared to IN HB (p<0.0001)

3/17/17

Opiant Pharmaceuticals Inc. (Santa Monica, Calif.)

Naloxone

Nasal spray

Addiction and overdose

Completed a study evaluating two doses on the occupation of brain opiate receptors using PET imaging, demonstrating that the 4-mg dose results in a greater and more rapid occupancy of mu opioid receptors than the lower 2-mg dose

3/28/17

Renown Pharma Ltd. (London)

Apomorphine

Spray formulation

Parkinson's disease

In a phase I pilot study in 12 healthy volunteers with all subjects receiving a 2.5-mg subcutaneous injection of apomorphine on day one followed by escalating 10-mg, 15-mg, 20-mg and 25-mg doses of sublingual apomorphine spray on the subsequent four days, it was found that the 25-mg apomorphine spray dose closely replicated the characteristics of the subcutaneous injectable with very fast absorption and similar peak plasma concentrations

3/8/17

DIABETES

Protokinetix Inc. (St. Mary's, W. Va.)

PKX-001

AAGP (glycopeptide) islet cells

Type 1 diabetes

Started a phase I trial of the cells, used in conjunction with the Edmonton protocol

3/10/17

GASTROINTESTINAL

Topivert Pharma Ltd. (London)

TOP-1288

A narrow-spectrum kinase inhibitor

Ulcerative colitis

Dosed the first subjects in a phase I study

3/1/17

INFECTION

Lung Therapeutics Inc. (Austin, Texas)

LTI-01

Injectable, fibrinolytic drug

Loculated pleural effusions in patients with pneumonia-like symptoms

Enrolled the first patient in a phase Ia/Ib trial

3/15/17

Matinas Biopharma Holdings Inc. (Bedminster, N.J.)

MAT-2501

Orally administered formulation of the broad spectrum I.V.-only aminoglycoside antibiotic agent amikacin

Infection

Top-line data from its phase I study in healthy volunteers showed no serious adverse events, and they were mostly mild in severity and gastrointestinal (GI) in nature, and the incidence of GI adverse events significantly decreased when it was administered with food; oral administration at all three doses yielded blood levels of amikacin that were well below the labeled safety limits recommended for I.V.-administered amikacin

3/28/17

Vaccibody AS (Oslo, Norway)

VB10.16 immunotherapy

Vaccine

High-grade cervical intraepithelial neoplasia caused by human papillomavirus 16 HPV16

Vaccinated the first patient in the multicenter trial VB C-01, an exploratory, open-label, multicenter phase I/IIa study

3/24/17

MISCELLANEOUS

Aeglea Biotherapeutics Inc. (Austin, Texas)

AEB-1102

Engineered human arginase I enzyme

Arginase I deficiency

Topline data from a phase I open-label study showed that single, intermittent doses of AEB1102 were administered safely and tolerably, and resulted in the reduction of arginine in the blood to normal levels

3/24/17

Audentes Therapeutics Inc. (San Francisco)

AT-342

AAV8 vector containing a functional copy of the UGT1A1 gene

Crigler-Najjar syndrome

Started LUSTRO, a prospective study aimed at characterizing the disease course, and will evaluate subjects prior to potential enrollment in VALENS, the planned phase I/II study to evaluate the safety and preliminary efficacy of AT342

3/2/17

Avexis Inc. (Chicago)

AVXS-101

Gene therapy

Spinal muscular atrophy (SMA) type 1

Top-line results from the phase I trial showed it has a favorable safety profile, as of Jan. 20, with no new treatment-related or tolerability concerns identified; 12 of 12 patients (100%) in the cohort of patients who received the proposed one-time therapeutic dose of AVXS-101 (cohort 2) had reached 13.6 months of age event-free, where the expected event-free survival rate based on natural history of the disease is 25%; mean increases from baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) scores of 7.7 points in cohort 1 and 24.7 points in cohort 2 were observed

3/20/17

Biophytis SA (Paris)

Sarconeos

Based on the activation of the MAS receptor

Sarcopenia

Results of the SARA-PK study showed favorable pharmacokinetics and pharmacodynamics; the analyses confirmed the pharmacokinetic profile in healthy elderly volunteers, the therapeutic window of Sarconeos and also confirmed the dosages that will be tested in the phase IIb trial

3/3/17

Bone Therapeutics SA (Gosselies, Belgium)

Allob

Allogeneic cell therapy

Delayed-union fractures of long bones

Completed the recruitment of the first 16 patients in its phase I/IIa delayed-union study

3/10/17

Catalyst Pharmaceuticals Inc. (Coral Gables, Fla.)

Firdapse

Amifampridine phosphate; potassium channel blocker

Myasthenia gravis

Topline results from the investigator-sponsored trial showed both of the co-primary efficacy endpoints of change from baseline (CFB) in total Quantitative Myasthenia Gravis score (p=0.0003) and CFB in total Myasthenia Gravis Activities of Daily Living score (p=0.0006) were statistically and clinically significant in the seven-patient trial

3/16/17

Cesca Therapeutics Inc. (Rancho Cordova, Calif.)

Platelet-rich plasma (PRP)

Autologous PRP injections composed of thrombocytes, cytokines and various growth factors

Chronic non-healing ulcers

Results from the 24-patient patient study showed healing of the wound/ulcer was observed in patients as early as four weeks after the PRP treatment with a mean healing time of 8.2 weeks ±1.9; all patients demonstrated healing of the wound/ulcer, with 17 (70.8%) showing a 90% reduction in wound size and three (12.5%) showing an 80% to 90% reduction over the course of the 24-week follow-up

3/16/17

Cesca Therapeutics Inc. (Rancho Cordova, Calif.)

Autologous bone marrow cell concentrate

Automated point-of-care technology

Critical limb ischemia

Results from a feasibility study showed improvements in wound healing, rest pain and six-minute walking distance, along with a reduction in intermittent claudication pain following the treatment

3/21/17

Greenovation Biotech GmbH (Freiburg, Germany)

Moss-agal

Recombinant form of human alpha-galactosidase

Fabry disease

Dosed the first patient in a phase Ib study

3/16/17

Minoryx Therapeutics SL (Mataró, Spain)

MIN-102

Differentiated PPAR agonist

X-linked adrenoleukodystrophy

Phase I results showed it was generally safe and well tolerated and no serious adverse events were observed

3/22/17

Nightstarx Ltd. (London)

Gene therapy

Uses a viral vector known as adeno-associated virus to deliver a codon-optimized copy of the retinitis pigmentosa GTPase regulator gene into cells of the eye

X-Linked Retinitis Pigmentosa

Started enrollment and dosing of subjects in a phase I/II trial

3/21/17

Replicel Life Sciences Inc. (Vancouver, British Columbia)

RCH-01

Dermal sheath cup cells, an autologous cell therapy

Androgenetic alopecia

Completed its phase I study; at 24 months post-injection, the average hair density increase for the trial's seven best responders was 8.3% over baseline; the largest increase in hair density over baseline observed in the group was a 21% increase at 24 months

3/15/17

Replicel Life Sciences Inc. (Vancouver, British Columbia)

RCT-01

Type 1 collagen-expressing, hair follicle-derived fibroblasts

Achilles tendinosis

Data from its phase I/II study showed it met its goal of establishing a complete safety profile at six months and showed no serious adverse events; each of the treated participants showed numerous clinically important improvements by various measures, including tendon composition, blood supply, physical function and pain sensation

3/29/17

SRI International (Menlo Park, Calif.)

Zinc diethylene triamine pentaacetate (Zn-DTPA)

Agent that removes heavy metals from the body by sequestering and increasing rates of elimination of those radionuclides

Radiation exposure

Received FDA clearance for a phase I trial testing the safety, tolerability and pharmacokinetics of an oral formulation in healthy human volunteers

3/31/17

Umecrine Cognition AB (Solna, Sweden)

GR-3027

GABAA receptor modulating steroid antagonist

Hepatic encephalopathy

Treated the first patient in a clinical phase Ib/IIa study

3/20/17

RESPIRATORY

Galapagos NV (Mechelen, Belgium)

GLPG-3067

CF potentiator

Cystic fibrosis (CF)

Started a phase I study

3/23/17

Notes

Public biotech 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.