Phase I

Imcyse SA, of Liège, Belgium


Modified peptide to drive generation of cytolytic CD4 T cells

Recent-onset type 1 diabetes

Treatment was deemed safe in phase Ib trial; drug-induced cytolytic CD4 T cells were detected; effector T cells decreased

Immunic Inc., of San Diego


Inverse agonist of ROR gamma t

Healthy volunteers (eventually psoriasis)

Dosed first volunteer in study testing the safety and pharmacokinetic profile of single and multiple ascending doses; study will also evaluate the effect of food

Onxeo SA, of Paris


Inhibitor of tumor DNA repair

Solid metastasized tumors

Results from the first group of patients in Driiv phase Ib study in combination with chemotherapy showed 2 of 3 demonstrated tumor control (stable disease, according to RECIST), since start of treatment (more than 4 and 5 months); company started second part of study to test combination of AsiDNA with carboplatin and paclitaxel in patients eligible for that treatment

Phase II

Alzprotect SAS, of Lille, France


Modulates the degradation of both APP and tau protein fragments

Progressive supranuclear palsy

Launched a 36-patient phase IIa study to test the tolerability and pharmacokinetics of 2 doses compared to placebo for 3 months, followed by a 3-month weaning observation period; 20-plus biomarkers will also be evaluated; data expected to be published in 2021

Antisense Therapeutics Ltd., of Melbourne, Australia


Inhibitor of CD49d expression

Duchenne muscular dystrophy

Preliminary data from first 6 patients treated for 24 weeks showed mean change from baseline in pinch and grip were 0.1 and 0.3, respectively

Apellis Pharmaceuticals Inc., of Crestwood, Ky.

APL-2 (pegcetacoplan)

Inhibits C3 and C3b of the complement cascade

Geographic atrophy secondary to age-related macular degeneration

Data from Filly study published in Ophthalmology showed the treatment produced a 29% reduction in the growth of GA lesion area from baseline to month 12 in the monthly treatment group (p=0.008) and a 20% reduction in the every other month (EOM) treatment group (p=0.067) compared to the pooled sham group; post-hoc analysis found reductions of 45% (p=0.0004) and 33% (p=0.009) for APL-2 monthly and EOM, respectively, compared to sham, over the last 6 months of treatment

Biogen Inc., of Cambridge, Mass., and Ionis Pharmaceuticals, of Carlsbad, Calif.

Spinraza (nusinersen)

Antisense oligonucleotide targeting survival motor neuron

Spinal muscular atrophy

Starting the 126-patient phase II/III Devote study comparing 2 loading doses of 50 mg 15 days apart followed by a maintenance dose of 28 mg every 4 months to the current regimen of 4 loading doses with 12-mg maintenance doses every 4 months; trial will also be used to determine how to safely and efficiently transition patients from the currently approved regimen to the higher dose

Bluebird Bio Inc., of Cambridge, Mass.


Gene therapy

Cerebral adrenoleukodystrophy

Updated data from phase II/III Starbeam study in boys 17 and under showed, of those who reached 24 months of follow-up, 88% (15/17) continue to be alive and major functional disability (MFD)-free; of 14 patients with less than 24 months follow-up, no evidence of MFDs

Graybug Vision Inc., of Redwood City, Calif.


Microparticle depot formulation of tyrosine kinase inhibitor sunitinib malate

Macular edema secondary to diabetic macular edema or retinal vein occlusion

Started phase IIa 20-patient study testing 2 doses of the drug; primary endpoint is safety; best corrected visual acuity, contrast sensitivity testing and time to rescue treatment will be measured as secondary endpoints

Hansa Biopharma AB, of Lund, Sweden


Cleaves IgG

Sensitized kidney transplant patients

Pooled data of 46 patients showed drug reduced donor-specific antibody (DSA) levels and all crossmatches were converted to negative; post-transplant, 33% of patients had antibody-mediated rejection episodes, but there was no strong correlation between DSA levels and rejections

Pulmatrix Inc., of Lexington, Mass.


Dry powder formulation of antifungal itraconazole

Allergic bronchopulmonary aspergillosis

Dosed first of about 64 patients in placebo-controlled study testing 3 dose levels of the drug for 28 days; primary endpoint is safety and tolerability; pharmacokinetics in plasma and sputum, effect on biomarkers of inflammation, pulmonary function, asthma symptoms and aspergillus burden in sputum will be measured as secondary endpoints

Saniona AB, of Copenhagen


Combination of tesofensine and metoprolol

Prader-Willi syndrome

3 adolescent patients in phase IIa trial lost an average of 2.6% over the 3-month period on 0.25-mg/day Tesomet compared to a weight gain of 2.3% during the 3-month period on 0.125-mg/day Tesomet and a weight gain of 2.2% in the 3-month period when 2 received placebo and 1 patient received 0.125-mg/day Tesomet; average hyperphagia score was reduced by 69% from baseline at the end of the period on 0.25 mg/day and 33% at the end of the period on 0.125-mg/day Tesomet

Vtv Therapeutics Inc., of High Point, N.C.


Glucokinase activator

Type 1 diabetes

In the Simplici-T1 study, 6 patients treated with drug had an 11% increase in time in range per day from baseline to the end of treatment and a 12% increase during waking hours, relative to the 9 patients on placebo; treatment reduced the total daily mealtime bolus insulin dose by 23% compared to 4% for placebo; patients on drug had fewer level 1 (≥54-70 mg/dl) and level 2 (<54 mg/dl) hypoglycemic events than patients in the placebo group

Zynerba Pharmaceuticals Inc., of Devon, Pa.

Zygel (ZYN-002)

Transdermal cannabidiol

Developmental and epileptic encephalopathy

Of the 33 patients with focal impaired-awareness seizures, median seizure frequency was reduced by 44% at month 2 and by 51% at month 6; in 11 patients with Lennox-Gastaut and Dravet syndromes, median seizure frequency was reduced by 6% at month 2 and by 51% at month 6

Phase III

Biogen Inc., of Cambridge, Mass., and Ionis Pharmaceuticals, of Carlsbad, Calif.

Spinraza (nusinersen)

Antisense oligonucleotide targeting survival motor neuron

Spinal muscular atrophy

In the Shine extension study, children with later-onset SMA (type 2 or type 3) experienced improvements or stabilization in 1 or more measures of motor function – Hammersmith Functional Motor Scale–Expanded, Upper Limb Module and 6-Minute Walk Test – for up to nearly 6 years

Poxel SA, of Lyon, France


Targets mitochondrial bioenergetics

Type 2 diabetes

Data showed Times 1 trial in Japanese patients achieved statistical significance (p<0.0001) for primary endpoint, defined as change in HbA1c level vs. placebo at week 24, with HbA1c placebo-corrected mean change from baseline of 0.87%; achieved statistical significance on main secondary endpoint of decrease from baseline in fasting plasma glucose (FPG) vs. placebo at week 24 (p<0.0001), with FPG placebo-corrected mean change from baseline of -19 mg/dL

Scynexis Inc., of Jersey City, N.J.

Ibrexafungerp (SCY-078)

Triterpenoid antifungal

Vulvovaginal candidiasis

Completed last patient visit in Vanish 303 study; top-line data expected by year-end

Phase IV

Novartis AG, of Basel, Switzerland


DPP-4 inhibitor

Type 2 diabetes mellitus

Results from Verify study show early combination with metformin met primary endpoint with statistically significant 49% reduction in relative risk for time to initial treatment failure (HbA1c >= 7% twice, consecutively, 13 weeks apart) vs. metformin alone (p<0·0001); patients treated with early combination also had lower HbA1c levels (below 6%, 6.5% or 7%) for 5 years vs. those receiving combo therapy only after metformin monotherapy failure; data published in The Lancet


For more information about individual companies and/or products, see Cortellis.


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