Phase I

Adverum Biotechnologies Inc., of Menlo Park, Calif.


Gene therapy expressing aflibercept

Wet age-related macular degeneration

After a median follow-up of 34 weeks in the Optic study, no patients had anti-VEGF rescue injections; retinal anatomy improvements seen at 24 weeks were sustained; best corrected visual acuity was stable; 52-week data from first cohort and 24-week data from second cohort expected in first half of 2020; company plans to start third cohort in fourth quarter of 2019 and fourth cohort in the first quarter of 2020

Eyevensys SAS, of Paris


TNF-alpha inhibitor

Non-infectious uveitis

First patient treated in the lowest dose cohort had a >10 ETDRS letter improvement in best corrected visual acuity (BCVA); 2 patients treated in the highest dose cohort had at least a 12 ETDRS letter increase in BCVA and a significant reduction of macular edema

Forbius Inc., of Austin, Texas


TGF-beta 1 & 3 inhibitor

Advanced or metastatic solid tumor malignancies with no other treatment options

AVID200-03 trial is fully enrolled with 15 patients treated with 3 escalating doses

Kodiak Sciences Inc., of Palo Alto, Calif.


Anti-VEGF antibody biopolymer conjugate

Treatment-naïve wet age-related macular degeneration (AMD), diabetic macular edema (DME) and retinal vein occlusion (RVO)

Additional treatments weren't required for 4 months or longer in 80% of wet AMD-treated eyes and 78% of DME-treated eyes; 40% of patients improved diabetic retinopathy severity level within the first 12 weeks of treatment with the rest not worsening; more than half of the RVO patients didn't require treatments for 3 months or more after 3 loading doses

Regenxbio Inc., of Rockville, Md.


Gene therapy expressing an antibody fragment inhibiting VEGF

Wet age-related macular degeneration

In cohort 4 at 1.6x10^11 GC/eye, 5 of 12 patients remained anti-VEGF injection-free; mean injections through 6 months was 2.2, a 50% reduction from the mean annualized injection rate during the 12 months prior to treatment; best corrected visual acuity (BCVA) improved by 2 letters; mean change in central retinal thickness (CRT) of -42µm; in cohort 5 at 2.5x10^11 GC/ey, 9 of 12 patients remained anti-VEGF injection-free; mean injections through 5 or 6 months was 0.8, an 80% reduction from the mean annualized injection rate during the 12 months prior to treatment; BCVA improved by 4 letters; mean change in CRT was -68µm

Phase II

Concert Pharmaceuticals Inc., of Lexington, Mass.


JAK1 and JAK2 inhibitor

Moderate-to-severe alopecia areata

In the 8-mg twice-daily and 12-mg twice-daily cohorts, 47% and 58% of patients, respectively, achieved a ≥ 50% relative reduction in overall Severity of Alopecia Tool scores from baseline, compared to 9% for the placebo group (p<0.001 for both); 21% of the 4 mg cohort met the goal; 78% of patients taking 12-mg dose and 58% of patients taking 8-mg dose rated their alopecia areata as much improved or very much improved on the Patient Global Impression of Improvement scale at week 24 (p<0.001 for both compared to placebo)

Incyte Corp., of Wilmington, Del.


JAK inhibitor


At 52 weeks, 57.6% of patients taking 1.5% ruxolitinib cream twice daily achieved ≥50% improvement from baseline in the total vitiligo area severity index (T-VASI50); 51.5% achieved F-VASI75; 33.3% achieved F-VASI90; 21.2% of patients achieved Facial Physician Global Vitiligo Assessment scores of clear or almost clear; all scores were improvements over 24-week results

Matinas Biopharma Holdings Inc., of Bedminster, N.J.


Amphotericin B with lipid nanocrystal delivery

Fungal cryptococcal meningitis in HIV patients

Started the Enact study; part 1 will determine the maximum tolerated dose in patients with HIV but no fungal infection; part 2 will measure the safety, tolerability and efficacy of MAT-2203 in HIV-infected patients with cryptococcal meningitis, compared to IV-administered amphotericin B as an induction therapy

Opthea Ltd., of Melbourne, Australia


Vascular endothelial growth factor receptor 3

Wet age-related macular degeneration

In the subgroup with occult lesions, patients treated with OPT-302 plus Lucentis (ranibizumab, Roche Holding AG/Novartis AG) improved best corrected visual acuity by 16.2 letters, compared to 10.2 letters for Lucentis alone (p=0.0008); 55.8% of patients treated with combination gained ≥15 letters compared to 34% of patients taking Lucentis alone; minimally classic subgroup improved by 13.7 and 11.1 letters for combination and Lucentis alone, respectively; polypoidal choroidal vasculopathy subgroup improved by 13.5 and 6.9 letters for combination and Lucentis alone, respectively (p=0.0253)

Reneuron Group plc, of London

hRPC stem cell therapy

Retinal progenitor cells

Retinitis pigmentosa

At 90 days, 8 patients had a 6.1 mean letter improvement in best corrected visual acuity; at 180 days, 4 patients had an 18.5 mean letter improvement

Theratechnologies Inc., of Montreal


Binds and stimulates human GRF receptors

Nonalcoholic fatty liver disease

Data published in Lancet HIV showed liver fat in patients taking tesamorelin decreased by 32% compared to a 5% increase for placebo (p=0.02); 35% of patients taking tesamorelin had liver fat below 5% compared to 4% of placebo patients (p=0.007); 10.5% of patients taking tesamorelin had progression of liver fibrosis compared to 37.5% of patients receiving placebo (p=0.04)

Phase III

Allergan plc, of Dublin, Ireland


VEGF-A binder

Neovascular wet age-related macular degeneration

In the second year of these studies, four injections of Abicipar resulted in the maintenance of visual gains comparable to monthly ranibizumab (Lucentis, Roche Holding AG)

Cara Therapeutics Inc., of Stamford, Conn.

Korsuva (CR845/difelikefalin)

Mechanism of action is mediated by kappa receptors on peripheral sensory afference as well as on certain immune cells

Moderate-to-severe chronic kidney disease-associated pruritus 

Based on the independent data monitoring committee's recommendation, the size of the trial will be increased by approximately 20% from an original enrollment target of 350 patients to 430 patients, to maintain the pre-specified statistical power of 90% or greater on the trial's primary endpoint

Daiichi-Sankyo Co. Ltd., of Tokyo

Lixiana (edoxaban)

Selective inhibitor of factor Xa

Non-valvular atrial fibrillation

One-year outcomes showed low rates of potentially life-threatening bleeding and low cardiovascular events in elderly patients

Eli Lilly and Co., of Indianapolis

Taltz (ixekizumab)

Interleukin-17A binder

Moderate-to-severe psoriasis

Met primary endpoint, demonstrating that 89% of patients treated with Taltz achieved a 75% improvement from baseline to week 12 on their Psoriasis Area and Severity Index score and 81% of patients treated with Taltz achieved a static Physician's Global Assessment of clear or almost clear skin

Pfizer Inc., of New York


Janus kinase 1 inhibitor

Severe atopic dermatitis

Met all the co-primary and key secondary endpoints, which were related to skin clearance and itch relief compared to placebo

Roche Holding AG, of Basel, Switzerland

Mabthera/Rituxan (rituximab)

Targeter of CD20

Pemphigus vulgaris

The study met the primary endpoint at week 52 and demonstrated that Mabthera/Rituxan is superior to mycophenolate mofetil, with 40.3% of patients treated with Mabthera/Rituxan achieving sustained complete remission without the use of steroids for 16 consecutive weeks or more, compared to 9.5% in the comparator arm (p<0.0001)

Taiho Oncology Inc., of Princeton, N.J.

Lonsurf (trifluridine and tipiracil)

Oral nucleoside antitumor agent

Metastatic gastric or gastroesophageal junction adenocarcinoma

Treatment with Lonsurf was tolerable and prolonged survival vs. placebo regardless of prior gastrectomy


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


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