Product Development

Enanta’s Phase II FXR agonist trial has poor clinical value for efficacy in NASH; Phase I data, structural difference limited success indicator, experts say

Enanta Pharmaceutical’s (NASDAQ:ENTA) Phase II ARGON-1 trial investigating its FXR agonist EDP-305 has limited clinical value to demonstrate efficacy in nonalcoholic steatohepatitis (NASH), experts said. In the coprimary endpoint change of alanine aminotransferase (ALT), such levels may not be high enough in all patients to demonstrate impact and level changes could be influenced by many unrelated factors, they said.

Continue reading →
Minerva’s MIN-117 for MDD raises expert doubts on efficacy, onset and safety as positive topline release appears to misrepresent Phase IIa results, experts say

Minerva Neurosciences (NASDAQ:NERV) has experts questioning MIN-117’s efficacy and safety profile in major depressive disorder (MDD) after discerning that positive topline results from two years ago do not accurately reflect non-statistically significant results emerging after a full analysis.

Continue reading →
Krystal’s KB103 splits experts' thoughts on potential for HSV-1 risk in dystrophic epidermolysis bullosa patients, but final Phase I/II efficacy assured

Krystal Biotech's (NASDAQ:KRYS) KB103 for dystrophic epidermolysis bullosa (DEB) patients has split expert opinions on whether the delivery vehicle involves a potential herpes simplex virus (HSV-1) risk.

Continue reading →
Celgene’s Revlimid ROBUST Phase III DLBCL trial draws mixed reviews on ABC subtype rationale; patient classification draws caution, experts say

Celgene’s (NASDAQ:CELG) Revlimid (lenalidomide) combo with R-CHOP has prompted mixed expectations on its potential in the diffuse large B-cell lymphoma (DLBCL) activated-B cell (ABC) subgroup, experts said, noting its failure to show superiority to R-CHOP in the overall DLBCL population in two Phase II trials.

Continue reading →
Loxo’s larotrectinib, LOXO-195 to face hurdles finding undefined NTRK-fusion patients; LOXO-195 should have lower success bar utility, but might not for all patients, experts say

Loxo Oncology (NASDAQ:LOXO) may face challenges to define its market for LOXO-101 (larotrectinib) and LOXO-195, as the prevalence of NTRK-fusion tumours is still difficult to assess, experts said. Genetic sequencing tests may not be as commonly used to readily select for this population, they added.

Continue reading →
Apellis’ APL-2 unlikely to resume AMD trial by YE18 due to time constraints and complex manufacturing investigation, investigators say

Apellis Pharmaceuticals (NASDAQ:APLS) is unlikely to resume its Phase III geographic atrophy (GA) trials by YE18 as there is not enough time to present all the required assurances to the FDA regarding the reported endophthalmitis cases, said two investigators on the sidelines of the ongoing American Academy of Ophthalmology annual meeting in Chicago, Illinois.

Continue reading →
Exelixis’ Cabometyx and Merck’s Keytruda approvable in second-line HCC but market obstacles set by available therapies, experts say

Exelixis’ (NASDAQ:EXEL) Cabometyx (cabozantinib) and Merck’s (NYSE:MRK) Keytruda (pembrolizumab), although FDA approvable, have less secure market success prospects in second-line hepatocellular carcinoma (HCC), experts said. This is due to both therapies having indistinguishable efficacy profiles with already approved treatments, experts said on the sidelines of the recent European Society of Medical Oncology Congress in Munich, Germany.

Continue reading →
UniQure's choice of higher dose in AMT-061's Phase IIb for hemophilia B draws mixed expert views on immune response risk, experts say

UniQure’s (NASDAQ:QURE) choice for a higher dose of AMT-061 in its Phase IIb hemophilia B study is drawing mixed expert views, with some concerned of an increased risk of an immune response. Others said that aiming higher could pay off, increasing the chance to achieve normal levels of factor IX (FIX).

The Phase IIb dose confirmation trial of AMT-061 (NCT03489291) is the first to test the second-generation product, an advancement of AMT-060, which obtained positive signals in Phase I/II. Analysts expect Phase IIb data at the end of 2018.

Continue reading →
Lexicon/Sanofi’s sotagliflozin divides experts on FDA approval prospects in T1D due to diabetic ketoacidosis risk

Lexicon Pharmaceuticals (NASDAQ:LXRX) and Sanofi’s (EPA:SAN) Zynquista (sotagliflozin) has split expert views on whether it will attain FDA approval as an adjunct therapy to insulin for type 1 diabetes (T1D).

Continue reading →
Sangamo’s Phase I SB-913 study in MPS II unlikely to see neurological disease changes but somatic manifestations have better chance, experts say

Sangamo Therapeutics’ (NASDAQ:SGMO) Phase I of SB-913 lacks the potential to treat mucopolysaccharidosis type II's (MPS II’s) significant neurological aspects, three experts said, noting preclinical data does not indicate the drug’s ability to cross the blood-brain barrier.

However, another expert said despite little to any enzyme passing into the brain, mice have demonstrated improvement in a measure of CNS function. Another said data indicated a small drug proportion had crossed the barrier and this may be enough to correct MPS II’s neural manifestation.

Continue reading →