Product Development

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.

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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.

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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).

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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.

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Evofem’s Phase III Amphora contraceptive draws expert doubt on pregnancy-prevention potential with trial variables and compliance; more utility in STI prevention

Evofem Bioscience’s (NASDAQ:EVFM) Phase III Amphora (L-lactic acid, citric acid, and potassium bitartrate) has experts apathetic about its prospects in preventing pregnancies. The drug’s similarities to spermicide, which has questionable efficacy, is subduing confidence along with multiple variables and room for error with this trial design.

Experts remain concerned about how the drug’s user failure rate will translate to a Phase III US population, with early data already indicating a higher level of improper use in this cohort.

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Novavax’s RSV F vaccine has experts uncertain about Phase III trial outcome with insufficient primary endpoint and earlier results that cannot be extrapolated

Novavax’s (NASDAQ:NVAX) Phase III trial for respiratory syncytial virus (RSV) F vaccine for maternal immunisation has experts uncertain on expected trial outcomes due to the inadequacy of the primary endpoint and difficulty extracting conclusions from earlier data. Whilst three experts said the trial has targeted the appropriate population, another said it would have been better to target more specific subgroups.

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AbbVie/J&J’s Imbruvica has 90% seven-year PFS rate in low-risk CLL patients and 30%–40% for high-risk patients, source says

A 7.5-year follow-up of AbbVie (NYSE:ABBV) and Johnson & Johnson’s (NYSE:JNJ) Imbruvica (ibrutinib) for chronic lymphocytic leukemia (CLL) found that the seven-year progression-free survival (PFS) rate is over 90% for low-risk patients and 30%–40% for high-risk patients, a source with knowledge of the situation said.

Results are part of an abstract that is expected to be presented at the 2018 ASH meeting in December, the source added. Reached for comments, AbbVie's Pharmacyclics and Johnson & Johnson's (J&J's) Janssen Pharmaceutical did not respond.

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Akcea’s Phase II FPL trial underpowering, design flaws leave experts skeptical of AKCEA-ANGPTL3-LRx study success

Akcea Therapeutics’ (NASDAQ:AKCA) Phase II trial of AKCEA-ANGPTL3-LRx is insufficiently powered to meet its primary efficacy endpoint for familial partial lipodystrophy (FPL), experts said. Other design aspects as well as earlier Phase I/II data also do not give them confidence that the trial will succeed, they added.

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BMS’ Opdivo/Yervoy 1L maintenance therapy relevance to be clouded by Roche’s SOC-changing IMpower 133 results, experts say

Bristol-Myers Squibb’s (NYSE:BMY) bid for Opdivo (nivolumab) and Yervoy (ipilimumab) as a maintenance therapy following chemotherapy in small cell lung cancer (SCLC) will likely be thwarted by the expected addition of Roche’s (SIX:ROG) Tecentriq (atezolizumab) to the first-line (1L) setting, experts said.

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ObsEva’s Phase III IVF study of nolasiban draws expert skepticism on use of too many trial sites for clean data

ObsEva’s (SIX:OBSN) Phase III study of OBE001 (nolasiban) in improving pregnancy rates via in vitro fertilization (IVF) has experts question its use of greater than 40 trial centers. Inherent differences among physician techniques could result in challenges to demonstrating clean data, they added.

Further muddying the Phase III IMPLANT trial (NCT03081208) outcome is the drug’s failure to meet its Phase II primary endpoint and uncertainty on the oxytocin receptor targeting mechanism, experts added.

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