Cancer

Actinium Pharmaceuticals Inc. (New York)

Actimab-A

Antibody radio-conjugate that combines anti-CD33 antibody lintuzumab with radioisotope actinium-225

Relapsed or refractory acute myeloid leukemia

The Medical College of Wisconsin received clearance from the FDA for the previously announced IND for the phase I trial of Actimab-A in combination with CLAG-M

3/14/18

Adaptimmune Therapeutics plc (Philadelphia)

NY-ESO SPEAR T cells

T-cell therapy

Myxoid/round cell liposarcoma

Reported three partial responses (two confirmed and one to be confirmed), and one stable disease in the first four patients dosed; patients tolerated treatment well with cytokine release syndrome managed following standard treatment guidelines

3/16/18

Aduro Biotech Inc. (Berkeley, Calif.) and Merck & Co. Inc. (Kenilworth, N.J.)

Anti-CD27 antibody

Targets a functional epitope on CD27

Advanced solid tumors

Started a phase I trial alone and in combination with Keytruda (pembrolizumab)

3/7/18

Advaxis Inc. (Princeton, N.J.)

Axalimogene filolisbac (axal)

A product of Advaxis' live attenuated Listeria monocytogenes platform

Metastatic squamous or nonsquamous carcinoma of the cervix and metastatic HPV-associated squamous cell carcinoma of the head and neck

The FDA placed the open-label phase I/II study on a clinical hold after the death of a woman in a study combining Advaxis' axal with Astrazeneca plc's Imfinzi (durvalumab)

3/14/18

Aeglea Biotherapeutics Inc. (Austin, Texas)

AEB-1102

Pegzilarginase

Uveal and cutaneous melanoma

Dosed the first patients in its open-label phase I cohort expansions

3/9/18

Agios Pharmaceuticals Inc. (Cambridge, Mass.) 

AG-270

Methionine adenosyltransferase 2a inhibitor

Solid tumors or lymphoma with deletion of the metabolic gene methylthioadenosine phosphorylase

Dosed the first patient in a phase I study

3/20/18

Bergenbio ASA (Bergen, Norway)

Bemcentinib

Selective Axl kinase inhibitor

Non-small-cell lung cancer

Updated its ongoing U.S.-only phase Ib/II trial testing bemcentinib in combination with Tarceva (erlotinib, Roche Holding AG) in first- and second-line patients; to date, 33 patients have been enrolled in the trial and 10 have reported clinical benefit (four partial responses and six stable disease) from the combination

3/16/18

Bioatla LLC (San Diego)

BA-3011

Antibody-drug conjugate targeting AXL

Advanced solid tumors

Treated the first patient in the phase I/II BA3011-001 trial

3/1/18

Checkpoint Therapeutics Inc. (New York)

CK-301

Fully human anti-PD-L1 antibody

Recurrent or metastatic cancers

Completed the dose-escalation portion of the ongoing phase I trial, and initiated the first dose-expansion cohort, which is evaluating an 800-mg dose administered every two weeks

3/22/18

Ciclomed LLC (Kansas City, Mo.)

CPX-POM

Ciclopirox Prodrug

Advanced solid tumors

The first patient was dosed in a phase I trial

3/1/18

Cotinga Pharmaceuticals Inc. (London, Ontario)

COTI-2

Targets mutant forms of KRAS

Gynecological malignancies, head and neck squamous cell carcinoma, and solid tumors

Submitted a protocol amendment to the FDA for its ongoing phase I trial testing COTI-2 as a monotherapy; the amendment includes treating patients with gynecological malignancies with COTI-2 in combination with Avastin (bevacizumab, Roche Holding AG) and paclitaxel/doxorubicin and treating HNSCC patients with COTI-2 in combination with cisplatin

3/21/18

Etherna Immunotherapies NV (Niel, Belgium) 

ECI-006

Designed to boost the immune system through Etherna's Trimix platform

Metastatic melanoma

Completed enrollment of the low-dose cohort in its phase Ib study of ECI-006 as an adjuvant therapy

3/20/18

Fate Therapeutics Inc. (San Diego)

FATE-NK100

Allogeneic donor-derived natural killer cell cancer immunotherapy

Ovarian cancer resistant to, or recurrent on, platinum-based treatment

Initial data from the ongoing Apollo study showed no dose-limiting toxicity, after one infusion a subject showed stable disease, evidence of tumor reduction

3/29/18

Fate Therapeutics Inc. (San Diego) 

Protmune

Next-generation hematopoietic cell graft

Hematologic malignancies

Additional data from the phase I stage of its PROTECT trial showed that, of seven adult subjects undergoing matched unrelated donor hematopoietic cell transplantation who received Protmune, as of the Feb. 26 data cut-off, there have been no events of cancer relapse with a median time on study of 228 days; no serious adverse events have been reported

3/20/18

Five Prime Therapeutics Inc. (San Francisco)

FPA-150

Immune co-stimulatory protein B7H4 inhibitor

Solid tumor

Initiated dosing in patients with solid tumors, to be followed by dose expansion in pre-specified cohorts in tumor types based on B7-H4 expression levels

3/29/18

Humanigen Inc. (Brisbane, Calif.)

Lenzilumab

Humaneered recombinant monoclonal antibody designed to target GM-CSF

Previously treated chronic myelomonocytic leukemia

Completed patient enrollment in the ongoing phase I trial

3/16/18

Hutchison China Meditech Ltd. (London)

Epitinib

Oral EGFR inhibitor

Glioblastoma

Started a phase Ib/II trial testing in China

3/7/18

Immune Design Corp. (Seattle)

CMB-305

Prime-boost immunotherapy targeting NY-ESO-1+ cancers

Soft tissue sarcoma (STS)

Data from a trial in 25 NY-ESO-1+ STS patients, including 14 synovial sarcoma patients, showed the median overall survival across all STS patients was 23.7 months, while the median for the subset of synovial sarcoma patients has not been met; patients who developed anti-NY-ESO-1 immune responses had better survival

3/13/18

Immunogen Inc. (Waltham, Mass.)

Mirvetuximab soravtansine

Fra-targeting antibody drug conjugate

Platinum-resistant ovarian cancer

FORWARD II in combination with anti-PD-1 pembrolizumab (Merck & Co. Inc.) showed a confirmed overall response rate (ORR) of 63% with a median progression-free survival of 8.6 months, in a subset of 8 patients with medium or high folate receptor alpha expression levels; for all patients, ORR was 43% and median PFS of 5.2 months; combination demonstrates favorable tolerability

3/26/18

Immutep Ltd. (Sydney)

Eftilagimod alpha (IMP-321)

Antigen-presenting cell activator

Melanoma

Dosed the first patient in a new cohort of the phase I Two Active Immunotherapies in Melanoma (TACTI-mel) trial testing it with Keytruda (pembrolizumab, Merck & Co. Inc.)

3/23/18

Innate Pharma SA (Marseille, France) and Astrazeneca plc (Cambridge, U.K.)

Monalizumab

Anti-NKG2A monoclonal antibody

Metastatic colorectal cancer

Added an expansion cohort of patients with first- and second-line cancer to its ongoing phase I trial, testing monalizumab with Imfinzi (durvalumab)

3/26/18

JW Therapeutics (Shanghai) Co. Ltd. 

JWCAR-029

CD19-directed investigational therapy

B-cell malignancies, specifically relapsed and refractory diffuse large B-cell lymphoma

Entered a phase I study in China

3/12/18

Leap Therapeutics Inc. (Cambridge, Mass.)

DKN-01

Anti-DKK1 monoclonal antibody

Advanced esophagogastric cancer

Reported phase I data from 16 patients treated with DKN-01 as a monotherapy, with central imaging review identifying two patients (12.5%) with a best response of a partial response and five patients (31.3%) with stable disease, representing a total disease control rate of 43.8%

3/16/18

Mabvax Therapeutics Holdings Inc. (San Diego)

MVT-1075

Antibody-based radioimmunotherapy targeting CA19-9

Pancreatic, colon or lung cancer that express CA19-9

Results from the first cohort of three patients in its phase I trial showed it was reasonably well-tolerated and accumulated on tumor after the first dose as evidenced by dosimetry measurements; two of the three patients had stable disease as measured using RECIST 1.1 criteria

3/1/18

Medigene AG (Martinsried, Germany)

MDG-1011

TCR therapy

Acute myeloid leukemia, myelodysplastic syndrome or multiple myeloma

Started a phase I/II trial testing it in approximately 92 patients

3/23/18

Oncolys Biopharma Inc. (Tokyo)

Telomelysin

Oncolytic adenovirus

Esophageal cancer

Data Safety Monitoring Board authorized Oncolys to move forward with enrollment for Cohort 2, confirming safety in Cohort 1 of the trial in Japan

3/26/18

Polyphor AG (Allschwil, Switzerland)

POL-6326

Balixafortide; an antagonist of CXCR

Metastatic breast cancer

Data from a dose-escalation phase Ib trial the combination of balixafortide with Halaven (eribulin, Eisai Co. Ltd.) in 56 patients produced a 63% clinical benefit rate and a 38% overall response rate

3/26/18

Redx Pharma plc (Alderley Park, U.K.)

RXC-004

Porcupine inhibitor

Advanced solid tumor

Told the MHRA that it's temporarily interrupting patient accrual in its phase I/IIa study after "clinically significant adverse events" in first patient dosed

3/29/18

Seattle Genetics Inc. (Bothell, Wash.)

SGN-CD48A

Antibody-drug conjugate targeting CD48

Relapsed or refractory multiple myeloma

Treated the first patient in a phase I trial

3/8/18

Selecta Biosciences Inc. (Watertown, Mass.)

SEL-403

SVP-Rapamycin in combination with LMB-100, a recombinant immunotoxin

Malignant pleural or peritoneal mesothelioma

The first patient was dosed in a phase I trial in patients who have undergone at least one regimen of chemotherapy

3/13/18

Sorrento Therapeutics Inc. and subsidiary, TNK Therapeutics Inc. (San Diego)

Autologous anti-CEA CAR T cells

Cell therapy

Refractory CEA-positive liver metastases

Phase Ib HITM-SURE showed all three heavily pre-treated patients had reduction in post-treatment serum CEA, with an average reduction of 19 ng/mL; two patients have progressive disease, but both are still alive at 4.8 months and seven months; the third patient has no evidence of liver metastases at 11 months on PET scan

3/6/18

Surface Oncology Inc. (Cambridge, Mass.)

SRF-231

Antibody to CD47

Advanced solid tumors and hematologic malignancies

Treated the first patient in a phase I trial

3/21/18

Tapimmune Inc. (Jacksonville, Fla.)

TPIV-200

Multi-epitope T-cell vaccine targeting folate receptor alpha (FRa)

Ovarian and breast cancer

Phase I data showed that more than 90% of patients developed robust and durable antigen-specific immune responses against FRa without regard for HLA type; a retrospective analysis showed that among the subset of ovarian cancer patients who were vaccinated following a first remission (n=10), median progression-free survival was extended compared to published clinical results for a similar patient population that received standard-of-care chemotherapy

3/16/18

Taris Biomedical LLC (Lexington, Mass.)

TAR-2000 (Gemris)

Designed to release gemcitabine continuously in the bladder for multiple weeks

Muscle invasive bladder cancer

Treated the first patient in a phase I trial testing it in patients who are unfit for therapy with curative intent

3/15/18

Trovagene Inc. (San Diego)

PCM-075

Adenosine triphosphate competitive inhibitor

Acute myeloid leukemia

The first patient in its phase Ib/II trial completed the first cycle of treatment with it in combination with low-dose cytarabine; the treatment was well-tolerated, and by day 15, blood leukemic cells decreased from greater than 40% to less than 5%

3/6/18

Cardiovascular

Acesion Pharma ApS (Copenhagen)

AP-30663

SK channel inhibitor

Atrial fibrillation

Started a phase I trial testing the safety and tolerability in 48 healthy subjects

3/13/18

Dermatologic

Sienna Biopharmaceuticals Inc. (Westlake Village, Calif.)

SNA-125

Topical dual JAK3/TrkA inhibitor

Atopic dermatitis and associated pruritis

The first patient was dosed in its phase I/II proof-of-concept trial

3/13/18

Endocrine/Metabolic

Aeglea Biotherapeutics Inc. (Austin, Texas)

AEB-1102

Pegzilarginase

Arginase 1 deficiency

Data from its phase I/II trial showed that repeated I.V. dose of 0.04 mg/kg of pegzilarginase resulted in sustained lowering of plasma arginine and arginine-derived metabolites; one pediatric patient developed anti-drug antibodies

3/14/18

Akcea Therapeutics Inc. (Cambridge, Mass.; affiliate of Ionis Pharmaceuticals Inc.)

AKCEA-APOCIII-LRx

Antisense drug designed to reduce the production of apolipoprotein C-III

High cholesterol

Data from a phase I/II trial in 67 healthy volunteers and patients showed it reduced ApoC-III protein by up to 84% after six weeks of treatment, reduced triglycerides by up to 71%, reduced apolipoprotein B by up to 30% and increased high-density lipoprotein cholesterol by up to 100%; ApoC-III protein levels remained reduced by up to 50% for approximately 90 days after the last dose

3/14/18

Ascendis Pharma A/S (Copenhagen) 

Transcon PTH

Parathyroid hormone (PTH)

Hypopara-thyroidism

Phase I data showed that in healthy adults it demonstrated a dose-dependent response with a flat infusion-like profile; the pharmacokinetic profile also translated into a predicted pharmacodynamic response consistent with the preclinical data, suggesting the ability to titrate patients with hypoparathyroidism into the normal calcemic range with Transcon PTH

3/20/18

Synlogic Inc. (Cambridge, Mass.)

SYNB-1020

Probiotic engineered to convert ammonia into an essential amino acid arginine

Hyperammonemia associated with urea cycle disorders and cirrhosis

Data from its phase I study showed that, in healthy volunteers, SYNB-1020 was safe and well-tolerated in 52 subjects up to a maximum tolerated daily dose of 1.5x10^12 CFU for 14 days; as designed, the bacteria did not colonize and all subjects cleared SYNB-1020 from their systems within two weeks of the final dose

3/13/18

Ultragenyx Pharmaceutical Inc. (Novato, Calif.)

DTX-301

Gene therapy

Ornithine transcarbamylase deficiency

Data from the first dose cohort of a phase I/II trial testing the 2 ×10^12 GC/kg dose showed one of the three patients had a clinically meaningful change in rate of ureagenesis, increasing from 67% of normal at baseline to 134% of normal at week 24 at which time the patient stopped alternate pathway medications and has been doing well clinically in the three subsequent weeks

3/8/18

Vtv Therapeutics Inc. (High Point, N.C.)

TTP-399

Selective and orally available glucokinase activator

Type 1 diabetes

Results from the phase Ib portion of Simplici-T1, a phase Ib/II trial, showed the drug was well-tolerated and improved or maintained glycemic control while reducing or simplifying patients' insulin regimen

3/26/18

Gastrointestinal

Cara Therapeutics Inc. (Stamford, Conn.)

Korsuva

CR-845/difelikefalin

Chronic liver disease

Treated the first patient in a phase I trial

3/1/18

GT Biopharma Inc. (Washington)

GTP-011

Muscarinic receptor antagonist

Motion sickness

Study of transdermal drug is more than 50% enrolled

3/29/18

Madrigal Pharmaceuticals Inc. (Conshohocken, Pa.)

MGL-3196

Oral thyroid hormone receptor beta agonist

Nonalcoholic steatohepatitis

Top-line phase II data showed it prompted triglyceride reductions of 30.1%, LDL-C reductions of 12.9% and lipoprotein a reductions of 37.5%, all compared to placebo

3/28/18

NGM Biopharma-ceuticals Inc. (South San Francisco)

NGM-282

Engineered variant of recombinant human fibroblast growth factor 19 and cytochrome P450 7A1

Nonalcoholic steatohepatitis

In a subset of patients treated with 3 mg once daily through subcutaneous injection, eight participants (50%) improved by greater than one stage, and three improved by two stages

3/28/18

Genitourinary/Sexual Function

Biospecifics Technologies Corp. (Lynbrook, N.Y.)

Xiaflex

Collagenase clostridium histolyticum

Uterine fibroids

Presented data from a phase I trial, in which three patients were injected with Xiaflex and underwent a hysterectomy 24-96 hours after treatment while two patients received a higher dose of Xiaflex and underwent a hysterectomy 63 days after treatment; the treated tissue samples showed a significant reduction of collagen and disruption of the tissue pattern, which didn't extend beyond the capsule of any fibroid

3/14/18

Hematologic

Bio-Path Holdings Inc. (Houston)

Prexigebersen

Liposomal Grb2 antisense

Hematological malignancies

Favorable phase I data published in The Lancet Hematology

3/29/18

Immune

Affibody AB (Solna, Sweden)

ABY-039

FcRn-targeting protein drug

Autoimmune diseases

Dosed the first subject in its phase I trial in healthy volunteers

3/14/18

Amyndas Pharmaceuticals Inc. (Philadelphia)

AMY-101

C3-targeted complement inhibitor

Complement-mediated conditions

AMY-101 had a good safety profile in healthy male volunteers administered single and multiple doses; pharmacokinetic and pharmacodynamic characteristics support subcutaneous dosing every 48 hours

3/28/18

Aobiome Therapeutics Inc. (Cambridge, Mass.)

Ammonia oxidizing bacteria (AOB) product candidate

Single strain of beneficial AOB, Nitrosomonas eutropha, that converts naturally occurring ammonia to nitric oxide

Seasonal allergic rhinitis

Findings from part one of a phase Ib/IIa trial showed it demonstrated safety and tolerability when delivered intranasally to healthy volunteers (N=24, randomized 1-to-1-to-1 high dose AOB, low dose and vehicle) over a period of two weeks; based on those data, Aobiome initiated part two

3/22/18

GT Biopharma Inc. (Washington)

GTP-004

Combines pyridostigmine with ondansetron

Myasthenia gravis

Analyzed pharmacokinetic data from phase I and is accelerating into phase II in the second half of 2018

3/26/18

Santhera Pharmaceuticals Holding Ltd. (Pratteln, Switzerland)

Raxone

Idebenone

Primary progressive multiple sclerosis

Phase I/II data showed there was no difference between patients treated with it and those treated with placebo as measured by the CombiWISE scale and other clinical assessments and biomarkers,

3/6/18

Infection

Altimmune Inc. (Gaithersburg, Md.)

Heptcell

Targeted immunotherapy

Chronic hepatitis B infection

Showed it was well-tolerated at all doses, while unblinded T-cell immunogenicity results were inconclusive

3/27/18

Arrowhead Pharmaceuticals Inc. (Pasadena, Calif.)

ARO-HBV

Targeted RNAi molecule

Chronic hepatitis B virus (HBV) infection

In the phase I/II, study dosed healthy subjects to evaluate safety in single ascending doses in one portion of the trial and the pharmacodynamic effects of multiple ascending doses in patients with chronic HBV in another portion

3/27/18

Cidara Therapeutics Inc. (San Diego)

Rezafungin

Echinocandin antifungal

Candida infections

Results from a phase I QT study showed that rezafungin in single doses up to 1,400 mg I.V. had no significant effect on QT prolongation or on any of the other cardiac conduction parameters tested

3/22/18

Combioxin SA (Geneva)

CAL-02

Broad-spectrum antitoxin

Community-acquired pneumonia

Completed first-in-man trial, with the independent data monitoring committee supporting a positive safety profile at both dosing regimens in patients with severe CAPB due to Streptococcus pneumoniae

3/28/18

Inovio Pharmaceuticals Inc. (Plymouth Meeting, Pa.)

INO-1800

DNA immunotherapy

Hepatitis B virus

Interim results from a phase I trial in 90 adults showed treatment with INO-1800 generated virus-specific T cells, including CD8+ killer T cells with markers believed to be important for retention of the cells in the liver

3/15/18

Novavax Inc. (Gaithersburg, Md.)

Nanoflu

Recombinant influenza vaccine

Influenza

Results from its phase I/II trial in older adults showed that it induced significantly higher hemagglutination inhibition (HAI) antibody responses against homologous H1N1 and H3N2 strains and comparable HAI responses against the homologous B/Brisbane strain, and induced significantly higher HAI immune responses against historic and forward-drifted H3N2 strains; it also induced strong neutralizing antibody responses that correlate with HAI results against H3N2 strains

3/2/18

Seres Therapeutics Inc. (Cambridge, Mass.)

SER-262

Fermented microbiome therapeutic

Primary  Clostridium

difficile infection

Phase Ib data showed no drug-related serious adverse events; no relevant differences were observed in the relative risk of recurrence rate in patients administered it, as compared to placebo; a low C. difficile recurrence rate was observed in patients treated with it and vancomycin, as compared to those treated with it and metronidazole (4% vs. 31%, respectively; "p" value of 0.0049)

3/9/18

Valneva SE (Lyon, France)

VLA-1553

Live-attenuated vaccine candidate

Chikungunya virus

Started a phase I trial in the U.S. to evaluate the safety and immunogenicity

3/14/18

Valneva SE (Lyon, France) 

VLA-15

Vaccine 

Lyme disease

Interim phase I results showed the study met its primary endpoint with a favorable safety profile; VLA-15 was also immunogenic in all doses and formulations tested; OspA-specific IgG antibody responses were induced in all treatment groups and against all OspA serotypes, with significant dose responses seen between the lowest and the highest dose groups

3/20/18

Vaxart Inc. (South San Francisco)

VXA-G1.1-NN

Norovirus oral tablet vaccine

Norovirus

Data from two phase I trials showed it was well-tolerated and generated robust systemic and local intestinal immune response, including memory and local effector IgA B-cell responses

3/5/18

Inflammatory

Cellular Biomedicine Group Inc. (Shanghai) 

Allojoin

Allogeneic adipose-derived mesenchymal progenitor cell off-the-shelf therapy

Knee osteoarthritis

Reported 48-week phase I data, showing it demonstrated a good safety tolerance and early signs of efficacy in preventing cartilage deterioration; the total WOMAC scores (consisting of pain, stiffness and function scores of joints) as a primary endpoint showed a significant improvement at 12 weeks post Allojoin cell therapy and continued improvement at 48 weeks

3/19/18

Musculoskeletal

Acceleron Pharma Inc. (Cambridge, Mass.)

ACE-083

Locally acting engineered protein therapeutic

Facioscapulo-humeral dystrophy and Charcot-Marie-Tooth disease

Phase I data showed it generated dose-dependent mean total muscle volume increases of up to 14.5% in the rectus femoris and 8.9% in the tibialis anterior three weeks after the last dose

3/1/18

Anges Inc. (Tokyo)

NF-kB Decoy

NF-kappa B decoy oligo DNA

Chronic discogenic low back pain

Anges Inc., of Tokyo, treated the first patient in a phase Ib trial

3/1/18

Kadimastem Ltd. (Ness Ziona, Israel)

Astrorx

Cell-based treatment containing functioning cells differentiated from pluripotent stem cells

Amyotrophic lateral sclerosis

Received approval of the Ministry of Health's Supreme Committee to conduct a phase I/IIa trial testing Astrorx in 21 patients

3/14/18

Solid Biosciences Inc. (Cambridge, Mass.)

SGT-001

Gene therapy

Microdystrophin gene transfer in Duchenne muscular dystrophy (DMD)

Received notification from FDA that IGNITE DMD phase I/II clinical trial has been placed on clinical hold following a serious adverse reaction in the first patient dosed; the boy has since recovered

3/16/18

Solid Biosciences Inc. (Cambridge, Mass.)

SGT-001

AAV-microdystrophin 5 gene therapy

Duchenne muscular dystrophy

Submitted a response to the FDA regarding the partial clinical hold on the high dose of SGT-001 in IGNITE DMD

3/29/18

Neurology/Psychiatric

Affiris AG (Vienna)

PD-01A andPD-03A

Synthetically produced alpha-synuclein-mimicking peptides

Multiple system atrophy (MSA)

Of the 30 patients in the phase I trial, six in the active treatment group discontinued early; two died during the study and one died soon afterward, in line with the up to 10% mortality rate in other MSA trials; PD-01A produced an antibody response specific to alpha-synuclein, but there were no significant differences in the immunogenicity profile between PD-03A and placebo

3/5/18

Aptinyx Inc. (Evanston, Ill.)

NYX-2925

N-methyl-D-aspartate receptor modulator

Neuropathic pain

Phase I data showed linear, dose-proportional pharmacokinetics and minimal accumulation in 84 healthy adult volunteers; it was generally well-tolerated with no serious adverse events reported, and no subjects discontinuing its use due to adverse events; an evaluation of cerebrospinal fluid samples from study participants confirmed that it crosses the blood-brain barrier

3/9/18

Arena Pharmaceuticals Inc. (San Diego)

APD-371

Peripherally restricted, highly selective, full agonist of the cannabinoid 2 receptor

Pain

Phase I data of healthy volunteers proved it to be safe and well-tolerated; all adverse events were classified as mild, the most common of which were headache and nausea; one subject discontinued treatment due to adverse events and there were no serious adverse events

3/2/18

Asterias Biotherapeutics Inc. (Fremont, Calif.)

AST-OPC1

Oligodendrocyte progenitor cells

Severe cervical spinal cord injury

Phase I/IIa SciStar data showed a positive safety profile and no serious adverse events; 92% (11/12) of cohorts 3 and 4 subjects had MRI scans at six months consistent with the formation of a tissue matrix at the injury site; 75% (9/12) of cohorts 3 and 4 subjects recovered at least one motor level on at least one side through six months, and 17% (2/12) of subjects recovered two or more motor levels on at least one side through six months

3/2/18

Bioarctic AB (Stockholm)

SC-0806

Combination of a medical device and medicinal product

Complete spinal cord injury

Received approval from Norwegian regulators to include Norwegian patients in its phase I/II study

3/22/18

Cadent Therapeutics (Cambridge, Mass.)

CAD-1883

Positive allosteric modulator of the small conductance calcium-activated potassium channel

Possibly spinocerebellar ataxia and/or essential tremor

Started a phase I trial

3/13/18

Cognition Therapeutics Inc. (Pittsburgh)

Elayta (CT-1812)

Brain-penetrant small-molecule

Alzheimer's disease (AD)

Phase Ib/IIa results demonstrated the synaptoprotective mechanism of action; in the 28-day study (COG0102), mild to moderate AD patients treated with Elayta experienced significantly reduced cerebrospinal fluid concentrations of neurogranin and synaptotagmin-1 compared to patients treated with a placebo

3/15/18

Corium International Inc. (Menlo Park, Calif.)

Corplex Donepezil

Transdermal formulation of donepezil

Alzheimer's disease

The FDA completed a preliminary review of data from its pilot bioequivalence study comparing it to oral Aricept, concluding that it may be sufficient for demonstrating bioequivalence

3/2/18

Denali Therapeutics Inc. (South San Francisco)  

DNL-747

Small-molecule RIPK1 inhibitor 

Alzheimer's disease

Started dosing in a phase I healthy volunteer study 

3/20/18

International Stem Cell Corp. (Carlsbad, Calif.)

ISC-hpNSC

Stem cells

Parkinson's disease

Completed dosing of the second cohort of patients in its phase I trial testing 50 million cells

3/6/18

Ionis Pharmaceuticals Inc. (Carlsbad, Calif.)

RG-6042

IONIS-HTTRx

Huntington's disease

Phase I/II data showed an average reduction of 40% and as high as 60% in the mutant huntingtin protein (mHTT) observed in the cerebral spinal fluid of patients treated for three months at the two highest study doses, 90 mg (p<0.01) and 120 mg (p<0.01), with mHTT levels continuing to decline at the last study measurement

3/5/18

Kempharm Inc. (Coralville, Iowa)

KP-415

D-methylphenidate prodrug

Attention-deficit hyperactivity disorder

Top-line results from its ongoing pharmacokinetic study in children and adolescents showed exposure to d-methylphenidate was predictable and varied by body weight as expected

3/21/18

Voyager Therapeutics Inc. (Cambridge, Mass.) 

VY-AADC

Gene therapy expressing AADC

Advanced Parkinson's disease

Reported longer-term data from its phase Ib trial in 15 patients; patients in cohort 1 who received total doses of up to 7.5×10^11 vg had a durable 2.1-hour improvement in patient-reported diary on-time without troublesome dyskinesia from baseline to three years; cohort 2 patients, who received a total dose of up to 1.5×10^12 vg, had a durable 3.5-hour improvement from baseline to 18 months, while cohort 3 patients, who received a total dose of up to 4.5×10^12 vg, saw improvement from baseline to six months of 1.5 hours that plateaued from six to 12 months

3/12/18

XW Laboratories Inc.

XW-10172

Small-molecule drug

Narcolepsy

Began dosing in healthy patients in Australia

3/20/18

XW Laboratories Inc. (Wuhan, China)

XW-10172

Small molecule

Narcolepsy

Started a phase I single, ascending-dose study in healthy subjects in Australia

3/13/18

Other/Miscellaneous

Allakos Inc. (San Carlos, Calif.)

AK-002

Humanized Siglec-8 monoclonal antibody

Chronic allergic and inflammatory diseases

Phase I data showed that it was found to rapidly deplete blood eosinophils after a single dose; all dose groups recorded complete depletion of blood eosinophils by the first post-dosing timepoint (one hour); eosinophil levels were not significantly changed in the placebo group; duration of eosinophil depletion also increased with dose level and was sustained for at least 28 days and up to 84 days in the 0.3-mg/kg and 1-mg/kg dose groups, respectively

3/6/18

Arrowhead Pharmaceuticals Inc. (Pasadena, Calif.)

ARO-AAT

RNAi drug designed to reduce the liver production of Z-AAT protein

Alpha-1 antitrypsin deficiency

Treated the first patient in the phase I AROAAT1001 trial

3/13/18

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.