Cancer

Amgen Inc. (Thousand Oaks, Calif.)

Kyprolis

Carfilzomib

Relapsed or refractory multiple myeloma

Phase III ASPIRE showed Kyprolis plus Revlimid and dexamethasone (KRd) reduced the risk of death by 21% compared to Revlimid and dexamethasone (Rd) alone and extended overall survival (OS) by 7.9 months from a median of 40.4 months for Rd to a median of 48.3 months for KRd; in patients treated at first relapse, OS was improved by 11.4 months from a median of 35.9 months for Rd to 47.3 months for KRd

1/19/18

Beigene Ltd. (Beijing)

Tislelizumab

Anti-PD-1 antibody

Previously untreated advanced hepatocellular carcinoma

The first patient was dosed in a global phase III trial

1/3/18

Celsion Corp. (Lawrenceville, N.J.)

Thermodox

Heat-activated liposomal encapsulation of doxorubicin

Liver cancer

Reached 74% of the 550 patients it plans to enroll in the phase III OPTIMA study

1/19/18

Deciphera Pharmaceuticals Inc. (Waltham, Mass.)

DCC-2618

Pan-KIT and PDGFR-alpha inhibitor

Advanced gastrointestinal stromal tumors

Initiated a pivotal phase III study called Invictus

1/5/18

Diffusion Pharmaceuticals Inc. (Charlottesville, Va.)

TSC

Trans sodium crocetinate

Newly diagnosed inoperable glioblastoma multiforme

Dosed the first patient in the phase III Investigating Tsc Against Cancerous Tumors (INTACT) trial

1/31/18

Exelixis Inc. (South San Francisco) and Ipsen SA (Paris)

Cabozantinib

Multitarget tyrosine kinase inhibitor

Previously treated advanced hepatocellular carcinoma

A second interim analysis of phase III CELESTIAL data showed median overall survival of 10.2 months with it vs. eight months with placebo (p=0.0049); median progression-free survival (PFS) was more than doubled, at 5.2 months with it and 1.9 months with placebo (p<0.0001); objective response rates per RECIST 1.1 were 4% with it and 0.4% with placebo (p=0.0086); disease control (partial response or stable disease) was achieved by 64% of the cabozantinib group compared with 33% of the placebo group

1/18/18

Ipsen Biopharm-aceuticals Inc. (Basking Ridge, N.J., affiliate of Ipsen SA)

Onivyde

Encapsulated form of irinotecan

Metastatic pancreatic cancer

Subanalysis of phase III NAPOLI-1 data showed a numerical but not statistically significant difference in overall survival (OS) between patients who did have a dose reduction (patients = 34, OS = 9.3 months) or dose delay (patients = 49, OS = 8.4 months) vs. patients who did not require dose reductions (patients = 83, OS = 5.4 months) or dose delays (patients = 68, OS = 5.6 months)

1/18/18

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

Keytruda

Pembrolizumab

Resected high-risk melanoma

Interim analysis of the phase III EORTC1325/KEYNOTE-054 trial showed it increased the recurrence-free survival compared to placebo with a hazard ratio of 0.57

1/9/18

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

Keytruda

Pembrolizumab

Metastatic nonsquamous non-small-cell lung cancer

The independent data monitoring committee determined that the phase III KEYNOTE-189 trial met its dual primary endpoints with the combination with Alimta and cisplatin or carboplatin extending overall survival and progression-free survival compared to Alimta plus platinum chemotherapy alone

1/17/18

Pfizer Inc. (New York)

PF-05280586

Biosimilar to Rituxan/Mabthera

CD20-positive, low tumor burden follicular lymphoma

REFLECTIONS B3281006, a phase III comparative safety and efficacy study of it vs. Mabthera, met its primary endpoint, demonstrating equivalence in overall response rate for the first-line treatment of patients

1/25/18

Progenics Pharmaceuticals Inc. (New York)

1404 imaging

PSMA-targeted small-molecule SPECT/CT imaging agent

Prostate cancer

Completed enrollment in its phase III study

1/3/18

Cardiovascular

Amarin Corp. plc (Bedminster, N.J.)

Vascepa

Icosapent ethyl

Primary major adverse cardiovascular events

Its REDUCE-IT cardiovascular outcomes study reported and documented more than 90% of the targeted 1,612 primary major adverse cardiovascular events

1/24/18

Bellerophon Therapeutics Inc. (Warren, N.J.)

Inopulse

Pulsed, inhaled nitric oxide

Pulmonary arterial hypertension

Enrollment in its phase III INOvation-1 study exceeds 100 patients, more than half of the anticipated enrollment

1/30/18

Liquidia Technologies Inc. (Research Triangle Park, N.C.)

LIQ-861

Powder formulation of treprostinil

Pulmonary arterial hypertension

Initiated a phase III trial

1/4/18

The Medicines Co. (Parsippany, N.J.)

Inclisiran

RNAi targeting PCSK9

Atherosclerotic cardiovascular disease

Completed enrollment of more than 1,500 patients in the phase III ORION-11 trial

1/26/18

Dermatologic

Foamix Pharmaceuticals Ltd. (Rehovot, Israel)

FMX-101

Topical formulation of the antibiotic minocycline

Moderate to severe acne

Phase III data showed application site adverse events occurred in less than 2% of patients during the additional 40 weeks of open-label treatment with it; patient satisfaction at the end of the 52-week treatment was similar to scores at the end of the 12-week initial trial

1/5/18

Novartis AG (Basel, Switzerland)

Cosentyx

Secukinumab

Moderate to severe plaque psoriasis

After 12 weeks of treatment, 66.5% and 72.3% of patients treated with it in the CLARITY trial achieved PASI 90 and IGA mod 2011 0/1, respectively, compared to 47.9% and 55.4%, respectively, of patients treated with Stelara; it produced PASI 100 in 38.1% of the patients at week 12 compared to 20.1% of the patients who received Stelara

1/17/18

Endocrine/Metabolic

Ascendis Pharma A/S (Copenhagen, Denmark)

Transcon

Growth hormone

Pediatric growth hormone deficiency

Reached its target enrollment in the phase III Height Trial

1/5/18

Eddingpharm Co. Ltd. (Shanghai)

Vascepa

Icosapent ethyl

Severe hypertriglyceridemia

Started a clinical trial in mainland China

1/18/18

Gastrointestinal

Anthera Pharmaceuticals Inc. (Hayward, Calif.)

Sollpura

Non-porcine pancreatic enzyme replacement therapy

Exocrine pancreatic insufficiency

Completed the second, pre-specified interim futility analysis for the RESULT phase III study

1/23/18

Ardelyx Inc. (Fremont, Calif.)

Tenapanor

NHE3 inhibitor

Irritable bowel syndrome with constipation

Data from the safety extension portion of its phase III T3MPO program showed a mean tenapanor compliance rate of about 98%; it was well tolerated among the 240 patients treated; 9.2% reported experiencing diarrhea, with only 1.7% of patients discontinuing treatment due to diarrhea; the overall discontinuation rate was 2.1%

1/4/18

RDD Pharma Inc. (Tel Aviv, Israel)

RDD-1219

Nifedipine-based drug

Chronic anal fissure

Completed enrollment in its pivotal phase III trial

1/5/18

Tigenix NV (Leuven, Belgium)

Cx-601

Darvadstrocel

Complex perianal fistulas in Crohn's disease

Phase III ADMIRE-CD data showed single injection produced a statistically superior rate of combined remission of perianal fistulas at week 52 compared to placebo

1/17/18

Genitourinary/Sexual Function

Reata Pharmaceuticals Inc. (Irving, Texas)

Bardoxolone

Oral, once-daily activator of Nrf2

Type 2 diabetics with chronic kidney disease (CKD)

Post-hoc analyses from the discontinued BEACON trial showed there was an increase in eGFR compared to both baseline and placebo in patients treated with it for at least 48 weeks, which was sustained for four weeks after stopping treatment; increases and durability of eGFR responses were correlated with early improvements in eGFR; patients taking it were significantly less likely to experience a composite renal endpoint consisting of confirmed decline of 30% or more from baseline in eGFR, confirmed eGFR of less than 15 mL/min/1.73 m2 and end-stage renal disease events

1/22/18

Hematologic

Generon Corp. (Shanghai)

F-627

Benegrastim

Chemotherapy-induced neutropenia in breast cancer

Its phase III study met its primary endpoint to shorten the duration in days of grade 4 neutropenia in the first chemotherapy cycle; patients treated with it demonstrated significantly reduced duration of severe neutropenia compared to patents in placebo group (p<0.0001)

1/26/18

Rexgenero Ltd. (London)

REX-001

Autologous cell therapy

Critical limb ischemia (CLI) and diabetes mellitus

Treated the first patient in its phase III program

1/26/18

Immune

Allergy Therapeutics plc (Worthing, U.K.)

PQ Birch

Subcutaneous immunotherapy

Allergic rhinoconjunctivitis

Completed recruiting participants for its phase III trial

1/10/18

Infection

Astellas Pharma Inc. (Tokyo) and Vical Inc. (San Diego)

ASP0113

DNA vaccine

Cytomegalovirus (CMV) and complications in CMV-seropositive hematopoietic stem cell transplant recipients

It failed to meet primary or secondary endpoints of the phase III Helios study

1/23/18

Insmed Inc. (Bridgewater, N.J.)

ALIS

Amikacin liposome inhalation suspension

Refractory nontuberculous mycobacterial lung disease caused by Mycobac-terium avium complex

In the phase III INS-312 extension study, 28.4% of the 67 patients who received guideline-based therapy (GBT) only in the original trial culture converted after six months of ALIS plus GBT; of the 57 patients who received ALIS plus GBT who continued the treatment in the extension study, 12.3% achieved culture conversion

1/5/18

Inflammatory

Flexion Therapeutics Inc. (Burlington, Mass.)

Zilretta

Triamcinolone acetonide, extended-release injectable suspension

Osteoarthritis of the knee

Phase IIIb data showed it produced a clinical benefit by week 12 in 95% of patients with 90% of those patients having received a second administration between weeks 12 and 24 with three patients still pending repeat dosing; the overall safety profile has been similar to the profile observed in the single injection pivotal phase III trial

1/9/18

Musculoskeletal

Novartis AG (Basel, Switzerland)

Cosentyx

Secukinumab

Ankylosing spondylitis

Started the SURPASS trial comparing it to a proposed biosimilar to Humira

1/10/18

Neurology/Psychiatric

Avenue Therapeutics Inc. (New York)

IV tramadol

Synthetic dual-acting opioid

Moderate to moderately severe pain

Dosed the first patient in its phase III safety trial

1/3/18

Axsome Therapeutics Inc. (New York)

AXS-02

Disodium zoledronate tetrahydrate

Complex regional pain syndrome

The independent data monitoring committee said its phase III trial, CREATE-1, should not proceed

1/10/18

Eli Lilly and Co. (Indianapolis)

Solanezumab

Monoclonal antibody

Mild to moderate Alzheimer's disease

Results from the two failed phase III trials, EXPEDITION 1 and EXPEDITION 2 showed: in EXPEDITION 1, the modeled difference in the change from baseline between groups (solanezumab group minus placebo group) was -0.8 points for the ADAS-cog11 score and -0.4 points for the ADCS-ADL score; in EXPEDITION 2, the difference in ADAS-cog14 was -1.7 points and -1.5 points

1/26/18

GW Pharmaceuticals plc (London)

Epidiolex

Cannabidiol

Lennox-Gastaut syndrome

Phase III data showed after a two-week dose-escalation period and 12 weeks of maintenance treatment, patients taking it had a 44% median decrease in drop seizures compared to a 22% median reduction for patients taking placebo

1/26/18

Kempharm Inc. (Coralville, Iowa)

KP415

Extended-release formulation of d-methylphenidate

Attention deficit, hyperactivity disorder

Treated the first patient in its pivotal trial in children ages 6 to 12 years

1/10/18

Novartis AG (Basel, Switzerland) and Amgen Inc. (Thousand Oaks, Calif.)

AMG-334

Erenumab

Episodic migraine

Phase IIIb LIBERTY data showed patients taking it had at least a 50% reduction from baseline in their monthly migraine days as compared to placebo; it also reduced monthly migraine days, reduced days requiring acute medication, improved scores on the Migraine Physical Function Impact Diary tool, and increased the rate of 75% and 100% responders

1/24/18

Pain Therapeutics Inc., of Austin, Texas

Remoxy

Extended-release oxycodone

Pain

Results from a human abuse potential study showed among non-dependent, recreational opioid users, nasal administration of it had significantly lower abuse potential compared to immediate-release oxycodone; it showed lower peak concentrations and longer time to peak drug concentration after nasal administration

1/10/18

Semnur Pharmaceuticals Inc. (Mountain View, Calif.)

SP-102

Non-opioid corticosteroid

Lumbar radicular pain/sciatica

Started the pivotal Corticosteroid Lumbar Epidural Analgesia for Radiculopathy (CLEAR) phase III trial

1/10/18

Sunovion Pharmaceuticals Inc. (Marlborough, Mass.)

APL-130277

Apomorphine sublingual film

Parkinson's disease

Phase III participants taking it showed a statistically significant mean reduction in off episodes at the study's 12-week point, meeting the trial's primary endpoint; 35% of those treated reported being full "on" after treatment during a maintenance period vs. 16% in the placebo group

1/31/18

Takeda Pharmaceutical Co. Ltd. (Osaka, Japan) and Zinfandel Pharmaceuticals Inc. (Chapel Hill, N.C.)

Pioglitazone

Thiazolidinedione

Alzheimer's disease

Terminated the phase III TOMMORROW trial after a planned interim futility analysis showed an inadequate treatment effect for 0.8 mg SR in delaying the onset of mild cognitive impairment

1/26/18

Ocular

Kala Pharmaceuticals Inc. (Waltham, Mass.)

KPI-121 0.25%

Mucus-penetrating particle technology to enhance delivery of loteprednol etabonate

Dry eye disease

Statistical significance was achieved for the primary sign endpoint, conjunctival hyperemia at Day 15 in the intent-to-treat (ITT) population in the STRIDE 1 and 2 phase III trials (p<0.0001); statistical significance was achieved for the primary symptom endpoint, ocular discomfort severity at Day 15 in the ITT population in STRIDE 1 (p<0.0001); ocular discomfort severity at Day 15 in the ITT population showed improvement but did not reach statistical significance in STRIDE 2 (p=0.1298); statistical significance for ocular discomfort severity at Day 15 in patients with more severe baseline discomfort was achieved in STRIDE 1 (p=0.0008), with a trend toward a treatment effect (p=0.0799) in STRIDE 2

1/8/18

Ohr Pharmaceutical Inc. (New York)

OHR-102

Squalamine

Wet age-related macular degeneration

The phase III Mako study did not meet its primary endpoint of visual acuity gain when combined with monthly Lucentis

1/8/18

Other/Miscellaneous

Auris Medical Holding AG (Zug, Switzerland)

AM-111

Contains brimapitide

Sudden deafness

Despite missing the primary endpoint of a phase III trial, called Healos, post-hoc analyses of data revealed "a clinically and statistically significant" hearing improvement from baseline to day 28 in 98 patients with "profound hearing loss" who participated in the study

1/5/18

Rhythm Pharmaceuticals Inc. (Boston)

Setmelanotide

Melanocortin-4 receptor agonist

Pro-opiomelanocortin (POMC) deficiency obesity

Swapped the primary and secondary endpoints for its phase III trial; the primary endpoint will now be a categorical analysis of responders for weight, defined as patients achieving a 10% change from baseline, while mean percentage change in weight will be the first secondary endpoint

1/5/18

Rhythm Pharmaceuticals Inc. (Boston)

Setmelanotide

Melanocortin-4 receptor agonist

Leptin receptor deficiency obesity

Started a phase III trial

1/5/18

Respiratory

Astrazeneca plc (London)

PT-010

Budesonide, glycopyrronium and formoterol fumarate

Chronic obstructive pulmonary disease (COPD)

Phase III Kronos data showed it demonstrated significant improvement in eight out of nine lung function primary endpoints compared with dual combination therapies, based on forced expiratory volume in one second assessments in patients with moderate to very severe COPD

1/29/18

Teva Pharmaceutical Industries Ltd. (Jerusalem)

Cinqair/Cinqaero

Reslizumab

Asthma

A phase III trial testing it in 468 patients with uncontrolled asthma and elevated blood eosinophils didn't meet the primary endpoint of reducing the frequency of clinical asthma exacerbations; a parallel-group phase III study testing the drug in 177 patients with oral corticosteroid (OCS)-dependent asthma and elevated blood eosinophils also missed its primary endpoint of reducing daily OCS dose

1/23/18

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.