CLR 131 is an investigational radioiodinated Phospholipid Drug Conjugate (PDC) drug candidate that delivers cytotoxic radiation directly and selectively to malignant cells.
There are currently no approved treatments or vaccines available for chikungunya, a virus transmitted through mosquito bites.
SCY-078 is an investigational antifungal agent that is a semi-synthetic derivative of the natural product enfumafungin.
The sNDA is accompanied by results from the MERIT-1 trial, a Phase 2 randomized, placebo-controlled, double-blind study which assessed the efficacy, safety and tolerability of macitentan 10mg in 80 patients with inoperable CTEPH.
Results showed that 14.3%, 20.7% (P=.0285), and 21.8% (P=.0129) achieved pain freedom in the placebo, ubrogepant 25mg dose, and 50mg dose groups, respectively.
The sBLA included clinical data from a Phase 4 study that enrolled nearly 2000 children and assessed the safety and immunogenicity of the 0.5mL dose.
XLRP represents the most severe form of retinitis pigmentosa (RP), an inherited retinal disease characterized by progressive retinal degeneration and vision loss that ends in complete blindness.
The BLA is supported by data from the global risankizumab Phase 3 psoriasis program evaluating more than 2,000 patients.
Results in the 202B and 202C trials showed that brexanolone achieved the primary endpoint of mean reduction from baseline in the Hamilton Rating Scale for Depression.
The Breakthrough designation was based on data from Phase 2 studies which assessed the safety and efficacy of the vaccine in this patient population.
The safety and efficacy of LentiGlobin were evaluated in 2 independent 2-year clinical studies: HGB-204 (Northstar, N=18), an open-label, single-dose, nonrandomized, multicenter Phase 1/2 study in patients with TDT, and HGB-205, an ongoing open-label, single-dose, nonrandomized, single-center Phase 1/2 study in patients with TDT and severe sickle cell disease.
Oraxol is a novel oral therapy that combines paclitaxel, a tubulin-stabilizing chemotherapeutic agent, with a non-absorbable gastrointestinal tract P-glycoprotein pump inhibitor.
The trial enrolled 152 patients with hemophilia A who were previously treated, either on-demand or as prophylaxis, with factor VIII therapy.
If approved, esketamine has the potential to be the first treatment for patients with major depressive disorder at imminent risk for suicide.
ALKS 5461 consists of a fixed-dose combination of buprenorphine, a partial mu-opioid receptor agonist and kappa-opioid receptor antagonist, and samidorphan, a mu-opioid receptor antagonist.
Results showed that 46% (n=6) of substudy patients improved their histological fibrosis stage, while 38% maintained (n=5), and 15% experienced 1 stage progression (n=2).
RP5063 is a new chemical entity with a novel mechanism of multimodal modulation of serotonin and dopamine signaling pathways.
Results showed that after 18-months, there was no significant statistical difference in cognitive or functional outcomes between those patients taking azeliragon and the placebo group.
At week 12, results showed 71% in the upadacitinib group had achieved ACR20, compared to 63% and 36% in the adalimumab and placebo groups, respectively.
The TAZPOWER study is a randomized, double-blind, placebo-controlled Phase 2/3 crossover study evaluating the safety and efficacy of elamipretide (daily subcutaneous injections) in 12 male patients (aged ≥12 years) with genetically-confirmed Barth syndrome.
Duvelisib is a first-in-class oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma.
Preclinical studies have demonstrated that RP6530 reprograms macrophages from an immunosuppressive M2-like phenotype (pro-tumor) to an inflammatory M1-like state (anti-tumor), which can potentially enhance the activity of checkpoint inhibitors or overcome resistance to these agents.
Omadacycline is an investigational, once-daily, broad-spectrum aminomethylcycline antibiotic that is related to tetracyclines.
REACH-2 was a randomized, double-blind, placebo-controlled trial that enrolled 292 patients with hepatocellular carcinoma (HCC) who were intolerant to, or had disease progression while on or following treatment with sorafenib and had a high alpha-fetoprotein (AFP-High) defined as AFP ≥400ng/mL.
Proteostasis Therapeutics is developing the combination treatment which includes a novel transmembrane conductance regulator (CFTR) amplifier (PTI-428), a third generation corrector (PTI-801) and a potentiator (PTI-808).
Results from the Phase 3 trial showed that the Quantitative Myasthenia Gravis (QMG) score at baseline to day 14 changed from 6.4 to 6.7 in the amifampridine group vs. 5.6 to 7.9 for the placebo group.
DFN-02 was evaluated in a multicenter, double-blind, randomized, placebo-controlled study (N=107) which showed it was effective in treating pain and associated symptoms during a migraine attack and in reducing attack-related functional disability.
The NDA submission included data from Phase 3, 12-week, randomized, double-blind, placebo-controlled, parallel-group study (CTH-300) that enrolled patients with levodopa-responsive Parkinson's disease complicated by OFF episodes.
Although they acknowledge the preliminary data is subject to further analysis, they say it demonstrates a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months.
Livantra may help rate of survival without transplantation or help improve survival until patients can receive a new liver.
NR supplementation showed reductions in mean systolic blood pressure (SBP; mean change -3.9mmHg) and diastolic blood pressure (DBP; mean change -2.0mmHg) for all patients; these findings were not statistically significant after adjusting for multiple comparisons.
ALIS has previously been designated orphan drug status, breakthrough therapy status and a Qualified Infectious Disease Product.
By inhibiting the activation of myostatin, SRK-015 may promote a clinically meaningful increase in muscle mass and strength.
Enfortumab vedotin is an investigational ADC composed of an anti-Nectin-4 monoclonal antibody attached to monomethyl auristatin E, a microtubule-disrupting agent.
Siponimod is an investigational, selective modulator of specific subtypes of the sphingosine-1-phosphate (S1P) receptor.
The Clinical Oncology Group Protocol ACCL0431 trial enrolled 125 pediatric patients newly diagnosed with a germ cell tumor, hepatoblastoma, medulloblastoma, neuroblastoma, osteosarcoma, or other malignancy.
The designation was supported by the Investigational New Drug application (IND) for the treatment of SSc-ILD and the anticipated safety and efficacy data from the Phase 3 double-blind, randomized, placebo-controlled SENSCIS trial.
A Phase 2 trial of OV101 in males with Fragile X syndrome aged 13 to 22 is planned for later in 2018. OV101 has previously been granted Orphan Drug status for Fragile X and Fast Track status for Angelman syndrome.
CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries.
Results - measured by the alkaline hematin method - found that at 6 months those in the combination group had 76.2% (P<0.001) clinical response compared to the elagloix along group, in which only 10.1% had a clinical response.
the Phase 3 'SOLUTION' study of Sollpura did not meet the primary endpoint of non-inferiority to an active-comparator in the coefficient of fat absorption (CFA).
In the Phase 3 SUNRISE 1 study, lemborexant achieved its primary and key secondary objectives vs. placebo and vs. zolpidem tartrate extended-release (active comparator) in patients aged ≥55 years with difficulty staying asleep through the night.
Dupixent is a human monoclonal antibody specifically designed to inhibit signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two important signaling proteins (cytokines) that contribute to Type 2 inflammation in moderate-to-severe asthma.
Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor.
The primary endpoint was the number of patients who required no platelet transfusion priory to primary invasive procedure and no rescue therapy for bleeding for 7 days post-procedure.
With regard to adverse events, 12.4% of AR101-treated patients discontinued treatment due to treatment-related side effects; these included gastrointestinal adverse events (6.7%) and systemic allergic hypersensitivity reactions (2.7%).
Inbrija (previously known as CVT-301) is a self-administered, orally inhaled levodopa; it is designed to deliver a precise dose of a dry powder formulation of L-dopa to the lung.
The full findings of the trials will not be released until later this year however the Company stated that the trials were a success, with the data already shared with the FDA in a pre-New Drug Application (NDA) meeting.
The disease can be life threatening. Phase 3 study results released last March demonstrated Rituxan substantialy improved pemphigus vulgaris remission rates and successful tapering.
In December, a Phase 3, randomized, double-blind, placebo-controlled trial (JADE) was initiated to evaluate the safety and efficacy of PF-04965842 in 375 patients aged ≥12 years with moderate to severe AD.
Jemdel, if approved, will be the first high-potency topical steroid for plaque psoriasis with dosing for 8 weeks. The most common adverse event in clinical trials of Jemdel was upper respiratory tract infection.
APECS is a randomized, placebo-controlled, parallel-group, double-blind Phase 3 clinical trial evaluating the efficacy and safety of verubecestat in people with prodromal AD.
The Breakthrough Therapy designation is intended to offer a potentially expedited development path and review for promising drug candidates.
The treatment was developed under the FDA's 'Animal Rule'; where efficacy is determined in animal studies and human studies determine safety and appropriate dosing. The FDA have stated a target action date for TPOXX of August 8, 2018.
A total of 1,327 migraine patients were treated in the study for a single migraine attack. At 2 hours after migraine attack, 19.2% (n=423) and 21.2% (n=448) of ubrogepant 50mg and 100mg were pain free, respectively.
Patisiran has been granted Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation for ATTR amyloidosis from the FDA.
The difference in score change for movement disorder from baseline to 30 minutes post-dose between the apomorphine and placebo group was 7.6 (P=0.0002).
A Phase 1 trial of PF614 compared the treatment to Oxycontin in healthy subjects and found that it demonstrated safety and extended-release characteristics of the prodrug delivery.
Aimovig is a fully human monoclonal antibody, designed to selectively block the calcitonin gene-related peptide (CGRP) receptor.
CNTX-4975 works by targeting the capsaicin receptor (TRPV1) to selectively and rapidly inactivate the local pain fibers transmitting signals to the brain.
The NDA submission was supported by data from two Phase 3 studies that met the primary efficacy endpoint of prevention of recurrence of uveitis at 6 months of follow-up (P<0.001).
The 'PROMISE 2' study included 1,072 patients who were randomized to receive eptinezumab (300mg or 100mg), or placebo once every 12 weeks.
If approved Inveltys would become the first twice-daily post-surgical topical treatment; all current treatments are approved for four-times-a-day dosing.
A 2016 Phase 3 trial (EPIC) including 609 patients with cUTI and acute pyelonephritis found plazomicin to be non-inferior to meropenum.
Eravacycline is a novel, fully-synthetic fluorocycline antibiotic being developed for the treatment of serious infections, including those caused by multidrug-resistant (MDR) pathogens that have been highlighted as urgent public health threats by both the World Health Organization and the U.S. Centers for Disease Control & Prevention (CDC).
Azedra, a high-specific-activity radiotherapeutic product, is a substrate for the norepinephrine reuptake transporter that is highly expressed on the cell surface of neuroendocrine tumors.
The study's primary objectives will be to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior and body weight in patients with Prader-Willi syndrome
The patient-the first to be treated with human polyclonal IgG harvested from Tc bovine-received infusions to combat the infection.
Angelman syndrome affects approximately 1 in 12,000 to 20,000 in the U.S. and symptoms include delayed development, intellectual disability, and seizures.
Results found that both groups had significantly greater improvements than placebo for the change from baseline to week 6 (P<0.05).
Results from the CM trial found that the fremanezumab arm experienced statistically significant reduction in the number of monthly headache days of at least moderate severity vs. placebo.
The FDA has granted Breakthrough Therapy designation for fingolimod for the treatment of children and adolescents 10 years of age or older with relapsing multiple sclerosis (MS).
Elamipretide has also been granted Fast Track designation for treating primary mitochondrial myopathy (PMM) - which gained Orphan Drug Designation in September - and Barth syndrome
Fitusiran, an RNAi therapeutic targeting antithrombin (AT), is designed to lower levels of AT in order to further sufficient thrombin generation to restore hemostasis and prevent bleeding.
For this group the risk of stroke was reduced by 42% (HR 0.58; 95% CI, 0.44-0.76; P<0.001), CV death by 22% (HR 0.78; 95% CI, 0.64-0.96; P=0.02) and heart attack by 14% (HR 0.86; 95% CI, 0.70-1.05; P=0.14).