Company (location) |
Product |
Description |
Indication |
Status |
Date |
Cancer | |||||
Aptevo Therapeutics Inc. (Seattle) |
Otlertuzumab |
Monospecific antibody targeting CD37 |
Peripheral T-cell lymphoma |
Commenced patient dosing in a phase II study |
1/9/18 |
Avelas Biosciences Inc. (San Diego) |
ABV-620 |
IV protease-activatable flourescent peptide |
Primary, nonrecurrent breast cancer |
Completed period 1 patient enrollment in its phase II trial |
1/5/18 |
Bergenbio ASA (Bergen, Norway) |
BGB324 |
Bemcentinib |
Advanced non-small-cell lung cancer |
The first efficacy endpoint was met in its phase II BGBC004 trial in patients who have progressed on an approved EGFR inhibitor; it plus Tarceva produced an overall disease control rate of 33% in nine patients who completed at least one cycle of treatment |
1/10/18 |
Bergenbio ASA (Bergen, Norway) |
BGB-324 |
Bemcentinib |
Triple-negative breast cancer and non-small cell lung cancer |
Interim safety data from three phase II trials in combination with Keytruda, as well as an AXL immunohistochemistry method, showed to clearly detect the presence of AXL on tumor and immune cells in patient samples |
1/30/18 |
Bergenbio ASA (Bergen, Norway) |
BGB-324 |
Bemcentinib |
Acute myeloid leukemia |
Analyses from a phase II trial showed a clear immunomodulatory effect as a result of selective AXL inhibition, as evidenced by increased immune activity characterized by diversification of patients' T-cell receptor repertoire |
1/30/18 |
Biolinerx Ltd. (Tel Aviv, Israel) |
BL-8040 |
Short peptide designed to function as a high-affinity antagonist for CXCR4 |
Metastatic pancreatic cancer |
Phase IIa COMBAT data showed it increases infiltration of T cells into the tumor in patients with metastatic pancreatic cancer; it was safe, well tolerated and induced an increase in the number of total immune cells in the peripheral blood while the frequency of peripheral blood regulatory T cells was decreased; analysis of available biopsies (n= 7) showed infiltration of various types of effector T cells into the tumor periphery and tumor microenvironment |
1/18/18 |
Biomarck Pharmaceuticals Ltd. (Durham, N.C.) |
BIO-11006 |
Inhibitor of MARCKS protein |
Non-small-cell lung cancer |
Started a phase II trial |
1/24/18 |
Bio-Path Holdings Inc. (Houston) |
Prexigebersen |
RNAi nanoparticle |
Accelerated and blast phase chronic myeloid leukemia |
Initiated a phase IIa study |
1/3/18 |
Bridgebio Pharma Inc. (Palo Alto) |
BGJ-398 |
Infigratinib |
Cholangiocarcinoma containing FGFR2 fusions |
Early phase II results found meaningful activity |
1/31/18 |
Bristol-Myers Squibb Co. (New York) |
Opdivo and Yervoy |
Nivolumab and ipilimumab |
DNA mismatch repair deficient or microsatellite instability-high metastatic colorectal cancer |
Phase II CheckMate-142 data showed the combination produced an objective response rate of 55%; with 13.4 months of follow-up, 94% of the responses were still ongoing; the overall survival (OS) rate at one year was 85%, while the median OS hadn't been reached yet |
1/23/18 |
Celgene Corp. (Summit, N.J.) |
Abraxane |
Nab-paclitaxel |
Locally advanced pancreatic cancer |
Phase II LAPACT data showed it plus gemcitabine produced a median time to treatment failure of 8.8 months, which exceeded the protocol-specified target of 6.6 months; the overall response rate was 32% with a median progression-free survival of 10.8 months; the estimated one-year overall survival was 72% |
1/23/18 |
Cellectar Biosciences Inc. (Madison, Wis.) |
CLR-131 |
Radiotherapeutic phospholipid-drug conjugate |
Relapsed or refractory B-cell hematologic cancers |
Enrolled the first patient in the diffuse large B-cell lymphoma (DLBCL) cohort of its phase II trial |
1/30/18 |
Iovance Biotherapeutics Inc. (San Carlos, Calif.) |
LN-145 |
Autologous tumor infiltrating lymphocytes |
Recurrent and/or metastatic squamous cell carcinoma of the head and neck |
Preliminary phase II C-145-03 data showed it produced a reduction of tumor size of at least 30% in three of eight patients |
1/26/18 |
Iovance Biotherapeutics Inc. (San Carlos, Calif.) |
LN-145 |
Autologous tumor infiltrating lymphocytes |
Recurrent, metastatic or persistent cervical carcinoma |
Preliminary phase II C-145-04 data showed one partial response and one patient with stable disease among two currently evaluable patients |
1/26/18 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Keytruda |
Pembrolizumab |
Advanced hepatocellular carcinoma |
Results of the phase II KEYNOTE-224 trial testing in patients who were previously treated with Nexavar showed that it produced an overall response rate of 16.3% with a disease control rate of 61.5% in 104 evaluable patients; median progression-free survival (PFS) was 4.8 months with a six-month PFS rate of 43.1%; six-month OS rate was 77.9% |
1/22/18 |
Merus NV (Utrecht, the Netherlands) |
MCLA-128 |
Full-length IgG bispecific antibody |
Metastatic breast cancer |
The first patient has been dosed in a phase II trial |
1/29/18 |
Nymox Pharmaceutical Corp. (Hasbrouck Heights, N.J.) |
Fexapotide |
Pro-apoptotic protein |
T1c prostate cancer |
Five-year results from its U.S. Study NX03-0040 showed that 15 mg single-dosage treatment resulted in 80% less surgery or radiotherapy associated with Gleason grade progression (p=0.0003), and that both doses (15 mg and 2.5 mg) were consistently effective (p=0.0003); 4.4% of patients in the entire group showed increase in their Gleason primary pattern grade in the five-year study, compared to controls where the incidence of grade 4 or higher primary pattern was 23.5%, a reduction of 81.3% (p=.0061) |
1/23/18 |
Oncosec Medical Inc. (San Diego) |
Immunopulse IL-12 |
DNA-based intratumoral immunotherapy |
Metastatic triple-negative breast cancer |
Preliminary OMS-I140 data showed two of the five patients treated with it were subsequently treated with Opdivo and experienced robust objective responses in both Immunopulse IL-12-treated and untreated lesions |
1/19/18 |
Rasna Therapeutics Inc. (New York) |
Act D |
Actinomycin D |
Acute myeloid leukemia |
Phase II data showed that four of out nine (44%) patients carrying the NPM1 gene mutation achieved complete remission (CR); three out of the four (75%) patients who obtained CR relapsed after three, five and seven months, respectively; one patient underwent haploidentical allogeneic peripheral blood stem cell transplantation at three months after CR achievement and is alive in molecular CR (MRD-negative) after 24 months; treatment was well-tolerated, with oral mucositis as the major toxicity |
1/4/18 |
Rexahn Pharmaceuticals Inc. (Rockville, Md.) |
RX-3117 |
Nucleoside compound |
Metastatic pancreatic cancer |
Final phase IIa data showed that of the 43 patients included in the efficacy analysis, 31% had progression-free survival of at least two months while 12% of patients had disease stabilization of more than four months |
1/23/18 |
Transgene SA (Strasbourg, France) |
TG-4010 |
Active immunotherapy against MUC1 tumor-associated antigen |
Advanced nonsquamous non-small-cell lung cancer |
Dosed the first patient in a phase II trial |
1/17/18 |
Trovagene Inc. (San Diego) |
PCM-075 |
Selective polo-like kinase 1 inhibitor |
Metastatic castration-resistant prostate cancer |
Started a phase II trial |
1/25/18 |
Tyme Technologies Inc. (New York) |
SM-88 |
Oral, tyrosine-based therapy |
Advanced metastatic or recurrent pancreatic cancer |
Phase II data demonstrated prolonged survival, with 40% (four of 10) of evaluable patients surviving more than 12 months, and 30% (three of 10) achieving a complete or partial response; all 10 reduced or maintained pain scores during their first cycle of therapy, in addition to gaining or maintaining weight; 80% showed improvement in ECOG performance status during the first treatment cycle, with 30% demonstrating an improvement of greater than 1.6 during that six-week period |
1/22/18 |
Cardiovascular | |||||
Akcea Therapeutics Inc. (Cambridge, Mass.) |
AKCEA-APOCIII-LRx |
Antisense drug |
Hypertriglyceridemia and established cardiovascular (CV) disease |
Started a phase IIb study |
1/9/18 |
Arca Biopharma Inc. (Westminster, Colo.) |
Gencaro |
Bucindolol hydrochloride |
Atrial fibrillation or atrial flutter |
Completed patient follow-up of its phase IIb GENETIC-AF trial |
1/9/18 |
Bellerophon Therapeutics Inc. (Warren, N.J.) |
Inopulse |
Pulsed, inhaled nitric oxide |
Pulmonary hypertension associated with interstitial lung disease |
The first patient was randomized in a phase IIb study |
1/4/18 |
Eiger Biopharma-ceuticals Inc. (Palo Alto, Calif.) |
Ubenimex |
Dual-inhibitor of aminopeptidase and leukotriene A4 hydrolase |
Pulmonary arterial hypertension |
The phase II LIBERTY study did not meet the primary efficacy endpoint of pulmonary vascular resistance and the secondary endpoint of 6-minute walk distance; the company will discontinue development |
1/17/18 |
Dermatologic | |||||
Xbiotech Inc. (Austin, Texas) |
MABp-1 |
Monoclonal antibody targeting Interleukin 1alpha |
Hidradenitis suppurativa |
The phase II study achieved its primary endpoint, showing significant treatment benefit using the HiSCR, or Hidradenitis Suppurativa Clinical Response, endpoint |
1/29/18 |
Endocrine/Metabolic | |||||
Caladrius Biosciences Inc. (Basking Ridge, N.J.) |
CLBS-03 |
Autologous cell therapy |
Type 1 diabetes |
Completed enrollment in the phase II trial: The Sanford Project: T-Rex Study |
1/19/18 |
Gemphire Therapeutics Inc. (Livonia, Mich.) |
Gemcabene |
Small molecule |
Severe hypertriglyceridemia |
Achieved its enrollment goal of 90 subjects in its phase IIb INDIGO-1 trial |
1/18/18 |
Minoryx Therapeutics SL (Mataro, Spain) |
MIN-102 |
PPAR gamma agonist |
Adrenomyelo-neuropathy |
Treated the first two patients in its phase II/III ADVANCE trial |
1/5/18 |
Zafgen Inc. (Boston) |
ZGN-1061 |
MetAP2 inhibitor |
Type 2 diabetes |
Completed enrollment in a phase II study |
1/8/18 |
Gastrointestinal | |||||
Cymabay Therapeutics Inc. (Newark, Calif.) |
Seladelpar |
Selective PPAR-δ agonist |
Primary biliary cholangitis |
Enrolled the first patients in its phase II/III extension study |
1/9/18 |
Protalix Biotherapeutics Inc. (Carmiel, Israel) |
OPRX-106 |
Oral anti-TNF |
Ulcerative colitis |
Interim data from the first 14 patients who completed to date the phase II trial showed that 57% achieved clinical response and 36% achieved clinical remission at week eight; in the rectal bleeding analysis, 79% showed an improvement, with 86% of those patients achieving an improvement in calprotectin and 64% with an improved Geboes score |
1/3/18 |
Redhill Biopharma Ltd. (Tel Aviv, Israel) |
RHB-102 |
Bekinda |
Diarrhea-predominant irritable bowel syndrome |
The phase II trial met its primary endpoint with it improving stool consistency by absolute difference of 20.7% vs. placebo |
1/17/18 |
Genitourinary/Sexual Health | |||||
Emotional Brain BV (Almere, the Netherlands) |
Lybrido and Lybridos |
Testosterone and sildenafil; testosterone and buspirone |
Female sexual interest and arousal disorder |
Phase IIb data showed Lybrido increased the satisfactory sexual events (SSEs) from baseline by 1.7 events compared to placebo and by 1.95 and 1.69 events compared to the sildenafil and testosterone monotherapies, respectively; Lybridos increased the SSEs by 0.99 events compared to placebo and by 1.52 and 0.98 events compared to the buspirone and testosterone monotherapies, respectively |
1/10/18 |
Enteris Biopharma Inc. (Boonton, N.J.) |
Ovarest |
Oral formulation of leuprolide |
Endometriosis |
Phase IIa data showed it enabled significant suppression of estradiol (E2), demonstrating a measurable pharmacodynamic effect that is correlated with efficacy in endometriosis |
1/4/18 |
Hansa Medical AB (Lund, Sweden) |
Ides |
Immunoglobulin G-degrading enzyme of Streptococcus pyogenes |
Chronic kidney failure |
Completed enrollment in its phase II Highdes trial |
1/5/18 |
Hematologic | |||||
Akebia Therapeutics Inc. (Cambridge, Mass.) |
Vadadustat |
Oral hypoxia-inducible factor stabilizer |
Anemia associated with dialysis-dependent chronic kidney disease |
Phase II results are consistent with findings from previous studies |
1/5/18 |
Beyondspring Inc. (New York) |
Plinabulin |
Non-granulocyte colony-stimulating factor (G-CSF) agent |
Prevention of docetaxel chemotherapy-induced neutropenia (CIN) |
The phase II portion of the phase II/III trial demonstrated that the duration of severe neutropenia (DSN) data for it is very similar to the DSN of Neulasta; incidence of grade 4 neutropenia was the same for Neulasta as for 20 mg/m2 plinabulin |
1/29/18 |
Catalyst Biosciences Inc. (South San Francisco) |
Marzeptacog alfa (activated) |
Subcutaneously administered factor VIIa therapy |
Hemophilia A or B |
Initiated enrollment of the phase II part of the phase II/III program |
1/5/18 |
Octapharma AG (Lachen, Switzerland) |
Octanate |
High-purity human Factor VIII / von Willebrand Factor (VWF) concentrate |
Severe hemophilia A |
Final data from a good clinical practice trial in previously untreated patients demonstrated that it is efficacious and associated with a low rate of inhibitor development in those patients, including those undergoing surgical procedures; five (9.8%) patients developed inhibitors, 4 (7.8%) of which were high titer; all inhibitors developed during on-demand treatment and all four high-titer inhibitors developed within the first 20 exposure days |
1/23/18 |
Immune | |||||
Cytori Therapeutics Inc. (San Diego) |
Habeo |
Cell therapy |
Impaired hand function due to scleroderma (systemic sclerosis) |
Completed enrollment in the phase II SCLERADEC II trial |
1/23/18 |
Eiger Biopharma-ceuticals Inc. (Palo Alto, Calif.) |
Ubenimex |
Inhibitor of leukotriene A4 hydrolase |
Primary and secondary lymphedema of the lower limb(s) |
Completed enrollment of the phase II ULTRA trial |
1/5/18 |
Kamada Ltd. (Rehovot, Israel) |
IV AAT |
Intravenous alpha-1 antitrypsin |
Lung transplant rejection |
Phase II data showed that of the 10 patients who received IV AAT plus standard of care (SOC), four died from common transplant-related complications; two of the six patients in the SOC arm died; of the six treatment patients who lived, two experienced the acute rejections, but their situations resolved |
1/10/18 |
Mapi Pharma Ltd. (Ness Ziona, Israel) |
GA Depot, a long-acting formulation of glatiramer acetate |
Long-acting formulation of glatiramer acetate |
Pprimary progressive multiple sclerosis |
Treated the first patient in a phase II trial |
1/31/18 |
Oryzon Genomics SA (Madrid, Spain) |
ORY-2001 |
Brain-penetrant drug that selectively inhibits LSD1 and MAOB |
Multiple sclerosis |
The first patient was included in the SATEEN phase IIa trial |
1/30/18 |
Infection | |||||
Aclaris Therapeutics Inc. (Malvern, Pa.) |
A-101 |
High-concentration hydrogen peroxide topical solution |
Verruca vulgaris |
Two phase II trials (WART-202 and WART-203) met all primary and secondary endpoints; in WART-203, where 159 patients were treated twice a week, it reduced the Physician's Wart Assessment (PWA) score on day 56 by 0.87 points compared to a reduction of 0.17 points for patients receiving placebo; in WART-202, where 157 patients were treated once weekly, it reduced the PWA score on day 56 by 0.77 points compared to a 0.23-point reduction in patients taking placebo |
1/10/18 |
Ait Therapeutics Inc. (New York) |
Nitric oxide |
Inhaled version |
Nontuberculous mycobacteria (NTM) |
Data at day 81, the end of its phase II NO-NTM Abscessus trial, showed the Mycobacterium abscessus load had a mean reduction of 65% vs. baseline; all patients in the trial were refractory to the standard of care for NTM abscessus and had underlying cystic fibrosis |
1/23/18 |
Chimerix Inc. (Durham, N.C.) |
Brincidofovir |
Nucleotide analog with antiviral activity |
Adenovirus |
Launched AdAPT (Adenovirus after Allogeneic Pediatric Transplantation), a comparative study designed to demonstrate the superiority of its antiviral effect in pediatric transplant recipients |
1/8/18 |
Emergent Biosolutions Inc. (Gaithersburg, Md.) |
FLU-IGIV |
IV influenza immune globulin |
Influenza A infection |
Started a phase II dose-ranging study |
1/5/18 |
Novan Inc. (Morrisville, N.C.) |
SB-208 |
Topical nitric oxide-based, silicone-based gel |
Fungal infections of the skin and nails |
Phase II data showed that 61.3% (p = 0.209) of patients treated with 2% gel, 80.6% (p = 0.002) of patients treated with 4% gel and 74.2% (p = 0.016) of patients treated with 16% gel achieved negative fungal culture at day 14 vs. 45.5% of patients treated with vehicle; the percentage of patients achieving mycological cure at the day 14 visit was 34.4% (p=0.305) of the patients treated with the 2% gel, 50% (p=0.009) with the 4% and 53.1% (p=0.010) with the 16% dose vs. 23.5% of patients treated with vehicle |
1/12/18 |
Novan Inc. (Morrisville, N.C.) |
SB-206 |
Nitric oxide product |
Molluscum contagiosum |
Treated the first patient in a phase II trial |
1/26/18 |
Paratek Pharmaceuticals Inc. (Boston) |
Omadacycline |
Antibiotic |
Urinary tract infection |
Started the first of two phase II trials |
1/4/18 |
Musculoskeletal | |||||
Acceleron Pharma Inc. (Cambridge, Mass.) |
ACE-083 |
Based on the naturally-occurring protein follistatin |
Facioscapulo-humeral dystrophy |
Released positive preliminary safety and tolerability data for the first two cohorts in part one of the phase II trial |
1/9/18 |
Neuraltus Pharmaceuticals Inc. (San Bruno, Calif.) |
NP-001 |
Regulator of macrophage activation |
Amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation |
All patients completed their last visit in the phase II trial |
1/5/18 |
Orphazyme A/S (Copenhagen) |
Arimoclomol |
Heat shock protein-based therapy |
SOD1 amyotrophic lateral sclerosis (ALS) |
Phase II data showed it was safe and well-tolerated at a dosage of 200 mg three times a day for up to 12 months; secondary endpoints, including survival, function based on ALS Functional Rating Scale Revised scores, vital capacity, and the combined assessment of function and survival, all favored it although they weren't powered to demonstrate efficacy |
1/26/18 |
Summit Therapeutics plc (Oxford, U.K.) |
Ezutromid |
Utrophin modulator |
Duchenne muscular dystrophy |
Interim phase II data showed treatment resulted in a reduction in muscle damage as measured by a 23% decrease in mean developmental myosin in muscle biopsies at 24 weeks compared to baseline (11.37% to 8.76%, 95% CI, -4.33, -0.90); 14 of 22 patients showed a decrease in developmental myosin, with five of those showing a greater than 40% reduction |
1/26/18 |
Neurology/Psychiatric | |||||
Anavex Life Sciences Corp. (New York) |
ANAVEX2-73 |
Agonist of the intracellular sigma-1 chaperone protein |
Mild to moderate Alzheimer's disease |
Post-hoc analysis of blood pressure data collected during the phase IIa study demonstrated that it seems to normalize systolic blood pressure (SBP) in a patient population with risk for hypertension; statistical analyses indicate a normalization of SBP from baseline of 143.3 mmHg to the subsequent readings after administration of 132.5 mmHg (p=0.048) at day 25 and of 135.3 mmHg (p=0.008) at day 36, respectively |
1/17/18 |
Antibe Therapeutics Inc. (Toronto) |
ATB-346 |
Hydrogen sulfide-releasing derivative of naproxen |
Acute and chronic pain |
The final subjects were enrolled in the phase IIb double-blind trial of healthy volunteers |
1/30/18 |
Axovant Sciences Ltd. (Basel, Switzerland) |
Intepirdine |
5-HT6 receptor antagonist |
Dementia with Lewy bodies |
Discontinued development after missing its primary efficacy endpoints in the phase IIb Headway trial as well as the pilot phase II gait and balance study in patients with various types of dementia |
1/9/18 |
Axovant Sciences Ltd. (Basel, Switzerland) |
Nelotanserin |
5-HT2A inverse agonist |
Dementia with Lewy bodies (DLB) |
Met its pre-specified primary safety endpoint of safety in the pilot phase II DLB visual-hallucination study and turned up a positive trend in efficacy in a pre-specified intent to treat (ITT) analysis of the motor function scale, the Unified Parkinson's Disease Rating Scale (UPDRS) Part III; it also showed an efficacy signal in a post-hoc subset analysis of patients with higher baseline scores on the Scale for the Assessment of Positive Symptoms in PD |
1/9/18 |
Intelgenx Corp. (Saint-Laurent, Quebec) |
Montelukast Versafilm |
CysLT1 antagonist |
Mild to moderate Alzheimer's disease |
Started a phase IIa proof-of-concept trial |
1/25/18 |
Neurotrope Inc. (New York) |
Bryostatin |
PKC epsilon activator |
Advanced Alzheimer's disease |
Phase II data showed evidence of improvement in cognition in patients receiving the 20 mcg regimen who did not receive memantine as background therapy; at week 15, non-memantine patients in the modified intent-to-treat group treated with 20 mcg (n=14) showed a Severe Impairment Battery (SIB) scale improvement of 5.88, while the placebo patients (n=11) showed a decline in their SIB scores of -0.05 (p=0.0576) |
1/8/18 |
PTC Therapeutics Inc. (South Plainfield, N.J.) |
RG-7916 |
Survival motor neuron 2 (SMN2) splicing modifier |
Spinal muscular atrophy |
Interim FIREFISH data showed the drug was safe and well-tolerated at all doses, and there have been no drug-related safety findings leading to withdrawal |
1/30/18 |
Recro Pharma Inc. (Malvern, Pa.) |
I.V. meloxicam |
COX-2 inhibitor |
Pain following dental surgery |
Phase II data showed a statistically significant difference in Summed Pain Intensity Difference over the first 24 hours (SPID24) compared with placebo at each dose level, and I.V. doses of 30 mg and 60 mg both demonstrated a statistically significant difference in SPID24 compared with oral ibuprofen 400 mg; it had a favorable safety and tolerability profile, with no serious adverse events or discontinuations due to adverse events reported |
1/18/18 |
ZZ Biotech LLC (Houston) |
3K3A-APC |
Genetically engineered variant of the naturally occurring activated protein C |
Acute ischemic stroke |
Phase II RHAPSODY data showed all doses were deemed safe and well-tolerated, yielding an maximally tolerated dose of 540 mcg/kg, the highest dose studied; total hemorrhage volume and hemorrhage incidence were substantially reduced and the incidence of any hemorrhage was reduced from 86.5% in placebo-treated patients to 67.4% in the combined treatment arms (p=0.046); total hemorrhage volume was reduced from an average of 2.1±5.8 mL on placebo to 0.8±2.1 mL in the combined treatment arms (p=0.066) |
1/30/18 |
Ocular | |||||
Aldeyra Therapeutics Inc. (Lexington, Mass.) |
ADX-102 |
Reproxalap |
Dry eye disease |
Enrolled the first patient in a phase IIb trial |
1/31/18 |
Kalvista Pharmaceuticals Inc. (Cambridge, Mass.) |
KVD-001 |
Intravitreally administered small-molecule plasma kallikrein inhibitor |
Diabetic macular edema |
Started a phase II proof-of-concept study |
1/8/18 |
Novaliq GmbH (Heidelberg, Germany) |
NOV03 |
Perfluorohexyloctane |
Dry eye disease |
Enrolled the first patients in its phase II SEECASE trial |
1/9/18 |
Ophthotech Corp. (New York) |
Zimura |
Avacincaptad pegol |
Autosomal recessive Stargardt disease |
Enrolled the first patient in a phase IIb trial |
1/17/18 |
Other/Miscellaneous | |||||
Innovation Pharmaceuticals Inc. (Beverly, Mass.) |
Brilacidin |
Defensin mimetic |
Oral mucositis (OM) |
Phase II trial in patients receiving chemoradiation for treatment of head and neck cancer showed a clear separation from placebo in delaying the onset of severe OM – particularly from about 28 to 42 days after the initiation of treatment, during which the incidence of severe OM rose strikingly in the placebo group while not in the group being treated; data measuring duration of severe OM showed it appeared to decrease the initial duration |
1/4/18 |
Rhythm Pharmaceuticals Inc. (Boston) |
Setmelanotide |
Melanocortin-4 receptor agonist |
Bardet-Biedl and Alström syndromes |
A phase II study of its once-weekly formulation demonstrated tolerability and pharmacokinetics that support further clinical development |
1/5/18 |
Saniona AB (Copenhagen) |
Tesomet |
Tesofensine/metoprolol |
Prader-Willi syndrome |
One of the three patients receiving placebo and four of the six patients who received it dropped out of the phase IIa study, preempting statistical evaluation; patients taking it lost 5% of their body weight after eight weeks compared to 0.46% for patients receiving placebo; after 13 weeks, the two remaining patients lost 6.76% of their body weight compared to 0.75% for the two patients receiving placebo |
1/10/18 |
Respiratory | |||||
Galapagos NV (Mechelen, Belgium) |
GLPG-2222 |
CFTR corrector |
Cystic fibrosis |
FLAMINGO phase II data showed it was well tolerated, with observed treatment-emergent adverse events being predominantly mild or moderate and typical; doses achieved the targeted exposure levels; a statistically significant dose-dependent decrease in sweat chloride concentration was observed with a maximum decrease of 18.3 mmol/L in the dose C cohort |
1/4/18 |
Galapagos NV (Mechelen, Belgium) |
GLPG-2737 |
C2 corrector |
Cystic fibrosis |
Dosed the first patient in the PELICAN phase II study |
1/4/18 |
Notes The date indicated refers to the BioWorld issue in which the news item can be found. For more information about individual companies and/or products, see Cortellis. |