Company (location)

Product

Description

Indication

Status

Date

Akcea Therapeutics Inc., of Boston, and Ionis Pharmaceuticals Inc., of Carlsbad, Calif.

Tegsedi (inotersen)

Antisense targeting TTR mRNA

Hereditary ATTR amyloidosis with polyneuropathy

Data from the open-label extension (OLE) study of the phase III Neuro-TTR study showed patients had benefit throughout the 104 weeks of total treatment; patients who switched from placebo to drug for the OLE portion had a stabilization of neurologic disease progression by mNIS+7 and Norfolk QoL-DN within 6 months, compared to an improvement of 17.1 points for mNIS+7 and 11.9 points for Norfolk QoL-DN in patients treated earlier with Tegsedi

5/7/19

Alder Biopharmaceuticals Inc., of Bothell, Wash.

Eptinezumab

CGRP-targeting monoclonal antibody

Migraine

Across phase II and III trials, drug, facilitated by 100% bioavailability at the end of infusion, showed rapid onset of migraine prevention, with response observed within day 1 and month 1, which was sustained through the first quarter following infusion, maintained or further increased through subsequent infusions and consistent across 4 trials; phase III highlights include 52.3% reduction in episodic migraine for patients with migraine on day 1 and treated with 100 mg and 54.9% reduction for those treated with 300 mg vs. 24.5% on placebo; in chronic migraine, 50.3% reduction in patients with migraine at day 1 treated at 100 mg and 51.6% in 300-mg group vs. 27.1% for placebo

5/6/19

Alder Biopharmaceuticals Inc., of Bothell, Wash.

Eptinezumab 

Monoclonal antibody targeting CGRP

Migraine prevention

In phase III Promise-2 study, 1 month after treatment, 45% and 57% of patients treated with 100 mg and 300 mg of eptinezumab, respectively, indicated their most bothersome symptom (MBS) was much improved or very much improved compared to 29% of patients taking placebo; 45% and 59% of patients reported the patients' global impression of change (PGIC) was much improved or very much improved for the 100 mg and 300 mg doses, respectively, compared to 32% of patients taking placebo; after 6 months, 57% of patients taking both 100 mg and 300 mg met the MBS goal compared to 42% of patients taking placebo; 60% of patients taking both 100 mg and 300 mg met the PGIC goal compared to 41% of patients taking placebo

5/8/19

Alterity Therapeutics Ltd., of Melbourne, Australia

PBT-434

Small-molecule inhibitor of alpha-synuclein aggregation

Synuclein-opathies such as Parkinson's disease and multiple system atrophy

Interim data from phase I trial in healthy volunteers show drug was well-tolerated in 3 multiple-dose cohorts; PBT-434 crosses the blood-brain barrier in humans and achieved concentrations in the brain exceeding those associated with efficacy in animal models of the disease

5/6/19

Avexis Inc., a unit of Basel, Switzerland-based Novartis AG

Zolgensma (onasemnogene abeparvovec-xioi)

Gene therapy

Spinal muscular atrophy

Interim data from ongoing trials showed positive results across broad spectrum of patients; data from phase I Strong trial showed motor function gains and milestone achievements in patients with type 2 disease via intrathecal delivery; data from phase III Strive trial continued to show prolonged event-free survival, increases in motor function and milestone achievement consistent with phase I Start trial; data from phase III Sprint trial showed motor milestone achievement consistent with normal development in patients treated pre-symptomatically

5/5/19

Avexis, a unit of Novartis AG, of Basel, Switzerland

Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101)

Gene therapy

Spinal muscular atrophy

Long-term follow-up of 10 patients in cohort 2 of the phase I Start study for a mean of 3.7 years showed all patients were alive and had no additional requirements for ventilatory or nutritional support; 2 of 4 patients who required bilevel positive airway pressure support at the beginning of the long-term follow-up study no longer required it regularly

5/7/19

Biogen Inc., of Cambridge, Mass.

Spinraza (nusinersen)

Antisense oligonucleotide

Spinal muscular atrophy

Data from Endear-Shine study showed increased average CHOP INTEND score by 16.8 points after nearly 3 years of treatment vs. 8.2 points over 1.5 years for those in Endear sham control arm; Shine extension study showed patients continue to achieve motor milestones, with 60% of younger (=5.42 months) and 38% of older (>5.42 months)able to sit without support; at nearly 3 years follow-up, infants in younger group showed greater improvements in CHOP INTEND score (19.4 vs. 13.8 points); patients in Endear and Shine also showed longer period of survival without need for permanent ventilation (median 75 weeks) vs. event-free survival in Shine of 22.6 weeks; among infants who initiated treatment in SHINE and were alive without permanent ventilation at baseline, 58% remained alive without permanent ventilation at data cutoff

5/6/19

Biogen Inc., of Cambridge, Mass.

Vumerity (diroximel fumarate, BIIB-098, formerly ALKS-8700)

Monomethyl fumarate prodrug

Relapsing multiple sclerosis

Data from 696 patients in open-label, 2-year phase III EVOLVE-MS-1 study that plans to enroll 1,000 participants showed 79% reduction in annualized relapse rate over 1 year compared to baseline and 82% in newly diagnosed patients; mean number of gadolinium-enhancing lesions was reduced by 77% compared to baseline in total population and by 96% in newly diagnosed patients; no unexpected safety findings were observed during 15 months of follow-up and no opportunistic infections occurred

5/7/19

Celgene Corp., of Summit, N.J.

Ozanimod

S1P receptor modulator

Relapsing multiple sclerosis

Post-hoc analysis of data from part B of phase III RADIANCE trial showed that ozanimod reduced cortical grey matter volume loss compared to first-line treatment, Avonex (interferon beta-1a), in adults ages 18 to 55, including those in youngest age group (18 to 25), who had greater brain volume at baseline but more active disease

5/7/19

Cytokinetics Inc., of South San Francisco

CK-2127107

Fast skeletal muscle troponin activator

Amyotrophic lateral sclerosis

Results showed phase II Fortitude-ALS study did not achieve statistical significance for pre-specified dose-response relationship in primary endpoint of change from baseline in slow vital capacity (SVC) after 12 weeks of dosing (p=0.11); similar analyses of ALSFRS-R and slope of the Muscle Strength Mega-Score yielded "p" values of 0.09 and 0.31, respectively; patients on all dose groups, however, declined less than patients on placebo for SVC and ALSFRS-R, with larger and clinically meaningful differences emerging over time

5/5/19

Eli Lilly and Co., of Indianapolis

Emgality (galcanezumab-glnm)

CGRP-targeting monoclonal antibody

Migraine

Pooled subgroup analyses from phase III Evolve-2 and Evolve 2 trials showed reduction in monthly migraine headache days vs. placebo in patients with low- and high-frequency episodic migraine

5/6/19

Eli Lilly and Co., of Indianapolis

Lasmiditan

Selective serotonin 5-HT1F agonist

Acute migraine

Pooled data from the phase III Samurai and Spartan studies showed rates of pain relief and freedom from most bothersome symptom (MBS) were both higher starting as early as 30 minutes post-dose in the lasmiditan 100 mg and 200 mg treated groups when compared with placebo (p<0.05); 200-mg dose also produced higher rates of freedom from photophobia and phonophobia than placebo starting as early as 30 minutes post-dose (p<0.05); freedom from pain was improved for 100 mg and 200 mg doses starting at 60 minutes (p<0.05 and p<0.001, respectively); a post-hoc analysis of patients with prior triptan exposure showed patients taking lasmiditan had higher rates of freedom from pain, freedom from MBS and pain relief vs. placebo, regardless of prior experience with triptans; interim results from the open-label Gladiator extension study showed long-term use produced similar rates of freedom from pain and freedom from MBS as the Samurai and Spartan studies

5/8/19

Engage Therapeutics Inc., of Summit, N.J.

Staccato alprazolam

Benzodiazepine

Epilepsy

Data from part 1 of phase IIb States study in patients who experience a predictable pattern of seizures showed 5 of 8 (62.5%) responded with cessation of seizure activity within 2 minutes of treatment and had no recurrence of seizure activity within 2 hours; responses were observed irrespective of seizure type, BMI or concurrent use of CYP450 inducers; enrollment of additional 115 patients in part 2 of study underway

5/6/19

Genentech, a unit of Roche Holding AG, of Basel, Switzerland

Risdiplam

SMN2 gene splicing modulator

Spinal muscular atrophy

Data from dose-finding part 1 of pivotal phase II/III FIREFISH trial showed infants with type 1 SMA achieved key motor milestones after 1 year of treatment; among those who received dose for confirmatory part 2 (n=17), 7 (41.2%) were able to sit without support for at least 5 seconds, 11 (64.7%) were able to sit with or without support and 9 (52.9%) achieved upright head control after 12 months of treatment; 1 infant achieved milestone of standing by 12-month time point; among all 21 infants enrolled in part 1, with median duration of treatment of 14.8 months, event-free survival was 18 (85.7%) overall and 15 out of 17 (88.2%) among those on study drug

5/7/19

Genentech, a unit of Roche Holding AG, of Basel, Switzerland

Risdiplam

SMN2 gene splicing modulator

Spinal muscular atrophy

Exploratory analysis of motor function using Motor Function Measure-32 (MFM32) scale was conducted for dose-finding part 1 of pivotal phase II/III SUNFISH trial in participants 2 to 25 years old with type 2 or 3 SMA (n=51); among those for which MFM32 scale was completed at all visits up to month 12 (n=43), 58% showed improvement of at least 3 points from baseline, including 71% among those 2 to 11 years old and 42% ages 12 to 25; change from baseline in total MFM32 score serves as primary efficacy endpoint in ongoing part 2 (n=180) of trial

5/7/19

Genentech, a unit of Roche Holding AG, of Basel, Switzerland

Ocrevus (ocrelizumab)

Monoclonal antibody targeting CD20

Multiple sclerosis

Pharmacokinetic, pharmacodynamic and exposure analyses in patients with relapsing multiple sclerosis (RMS) showed that higher exposure to the drug was correlated with lower B-cell levels and lower rates of the risk of 24-week confirmed disability progression (CDP); in patients with primary progressive multiple sclerosis (PPMS), there was a reduced risk of 24-week CDP at all exposure levels compared with placebo; the drug reduced T1 gadolinium-enhancing and new/enlarging T2 MRI lesions to nearly undetectable levels in RMS and PPMS patients and reduced annualized relapse rates to low levels (0.13-0.18) in RMS patients across all exposure segments; in the phase III Opera open-label extension (OLE) trial, the proportion of RMS patients with 48-week CDP was 10.4% for those treated for 5 years compared to 15.7% for patients who received 2 years of interferon beta-1a and 3 years of Ocrevus (p=0.004); in the Oratorio OLE, the proportion of PPMS patients with 48-week CDP was 43.7% for patients taking drug for 5 years, compared to 53.1% for patients who switched to drug after the 120-week double-blind period (p=0.03)

5/8/19

Merck & Co. Inc., of Kenilworth, N.J.

Belsomra (suvorexant)

Dual orexin 1/2 receptor antagonist

Insomnia in mild to moderate Alzheimer's disease

Phase III study met primary efficacy endpoint, improving mean total sleep time by 28.2 minutes vs. placebo (n=135 vs. n=139, respectively; p<0.005), corresponding to mean increase from baseline of 73.4 minutes with study drug and mean increase from baseline of 45.2 minutes with placebo

5/7/19

Neurocrine Biosciences Inc., of San Diego

Opicapone

Once-daily, oral, selective, peripherally acting catechol-O-methyltransferase inhibitor

Parkinson's disease

Analysis from 2 phase III trials showed treatment added to levodopa resulted in significant and sustained increase in ON time without dyskinesia; more than 60% of patient treated with 50 mg achieved >/= to 1-hour increase from baseline in total ON time at week 14/15

5/5/19

Neurocrine Biosciences Inc., of San Diego, and Voyager Therapeutics Inc., of Cambridge, Mass.

VY-AADC

Gene therapy

Parkinson's disease

Phase I results from 8 patients in open-label trial showed treatment improved good ON time by 1.7 hours from baseline and reduced OFF time by 2.2 hours at 12 months from baseline; exploratory analyses in 4 of 8 patients with low or no dyskinesia or absence of impulse control disorder at baseline showed greater improvement in motor function, including 3.2-hour improvement in good ON time from baseline to 12 months; results also show posterior trajectory is an additional surgical delivery route

5/5/19

Novartis AG, of Basel, Switzerland

Mayzent (siponimod)

Targets S1P1 and S1P5 receptors

Secondary progressive multiple sclerosis

In the phase III Expand study, patients treated with Mayzent experienced sustained improvement in Symbol Digit Modalities Test (SDMT) compared to those taking placebo (p=0.0131); patients treated earlier in their disease course with less cognitive impairment benefited most from Mayzent treatment with a significantly higher proportion experiencing meaningful improvement over the course of the study and follow up (p=0.0126 for those with relapses and p=0.0094 for those with an initial SDMT score greater than or equal to the median); drug also helped delay deterioration in SDMT for those with cognitive impairment at baseline and for those with an initial SDMT score less than the median (p=0.0269 and p=0.0071, respectively)

5/8/19

Retrotope Inc., of Los Altos, Calif.

RT-001

Oral, deuterated polyunsaturated fatty acid

Late-onset Tay-Sachs disease

Findings from expanded access study showed administration to a 44-year-old patient appears to have generated improvements in variety of patient-reported outcomes (choking coughs, strength, falls) and in objective measures of performance (timed walks, quantified speech measures, Archimedes spiral); effects were measured in daily or weekly diaries and test sessions at home

5/6/19

Sanofi SA, of Paris

Lemtrada (alemtuzumab)

CD52-targeting monoclonal antibody

Relapsing-remitting multiple sclerosis

Interim data from 2-year, ongoing, real-world Pro-Act study showed patients who had completed Treatment Satisfaction Questionnaire for Medication assessments at baseline and 12 months after starting Lemtrada reported improvements in global satisfaction (primary endpoint) and effectiveness; side effects score decreased and convenience score was stable; interim data from ongoing, 1-year, real-world Promis study showed patients reported significant and clinically meaningful improvements in health-related quality of life, as well as statistically significant improvements in functional health

5/6/19

TG Therapeutics Inc., of New York

Ublituximab (TG-1101)

Glycoengineered B-lymphocyte antigen CD20 modulator

Relapsing multiple sclerosis

Data from phase II and 45 patients from the study enrolled in open-label extension (OLE) through 48 weeks showed ublituximab remained well-tolerated at median duration of follow-up of 97.5 weeks; no participants discontinued due to drug-related adverse event during phase II or the OLE

5/7/19

Notes

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