Product Development

Achillion’s Phase II C3 glomerulopathy trial of ACH4471 likely to be impacted by slow recruitment though experts optimistic about final results

Achillion Pharmaceuticals’ (NASDAQ:AHCN) 14-day Phase II ACH-4471 trial for complement (C3) glomerulopathy (C3G) is likely to face patient enrollment hurdles, affecting an on-time readout, experts said. Nonetheless, they earmarked the study for success, considering ACH-4471’s more targeted and upstream approach to modulating the complement system.

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Gilead/Spring Bank’s Phase II inarigivir/Vemlidy combo logical for HBV but inarigivir's modest data and unclear mechanism draws expert reservations

Gilead Sciences (NASDAQ:GILD) and Spring Bank Pharmaceuticals’ (NASDAQ:SBPH) Phase II trial investigating inarigivir in combination with Gilead’s nucleoside analogue Vemlidy (tenofovir) for hepatitis B virus (HBV) infection has a promising rationale due to the combination’s synergistic antiviral approach. However, inarigivir’s monotherapy efficacy impact is still unclear, drawing uncertainties to its potential contribution in a combination setting, some noted.

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Novartis’ CVOT and postmarket trial for Entresto likely to meet primary safety, NT-proBNP endpoints but expense will continue to curb access, experts say

Novartis’ (VTX:NOVN) heart drug Entresto (sacubitril/valsartan) will likely lead to a reduction in cardiovascular (CV) mortality events for a successful Phase III CV outcomes trial (CVOT), experts said, despite analyst misgivings of a hypotension risk. Experts said they also expect Entresto to lead to a reduction in NT-proBNP in the postmarket PIONEER-HF study due to significant data for heart failure (HF) patients with preserved ejection fraction (HFpEF) in the Phase II PARAMOUNT trial.

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Novartis’ Kymriah reimbursement continues to involve outcome-based provider contracts but payer engagement ambivalent with complicated reimbursement dynamics

Novartis’ (VTX:NOVN) outcome-based contracts (OBCs) for reimbursing its CAR-T therapy Kymriah (tisagenlecleucel) for acute lymphoblastic leukemia (ALL) remain a viable option for treatment centers, despite recent conflicting media reports saying otherwise, experts said. But private payer enthusiasm for engaging in OBCs and how the arrangement can overall reduce healthcare costs is unclear in light of nebulous financial logistics, they noted.

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Marinus’ IV ganaxolone holds efficacy promise for PPD Phase II based on Sage's success but suicide watch recommended; molecular structure-led differentiation debated, experts say

Marinus Pharmaceuticals’ (NASDAQ:MRNS) IV ganaxolone has experts generally optimistic for efficacy success in its Phase II trial for severe postpartum depression (PPD). Having competitor Sage Therapeutics’ (NASDAQ:SAGE) brexanolone, a similar IV-administered positive allosteric GABA-A receptor modulator, show positive Phase III efficacy as well as strong preclinical work in the drug class accounts for their optimism, they added.

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BridgeBio’s Phase II infigratinib trial for cholangiocarcinoma enrols 75-80% of 120 patients, sources say

BridgeBio Pharma’s Phase II trial investigating BGJ398 (infigratinib) for cholangiocarcinoma has enrolled 75-80% of its 120 patients, two sources familiar to the trial said.

The first source noted enrolment slowed down in the Phase II (NCT02150967) trial when Novartis (VTX:NOVN) out-licensed infigratinib to BridgeBio, but both sources noted the trial is back on track. This is thanks to the unmet need in this rare form of liver cancer, the second source explained.

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Nektar/Syndax NKTR-214 and entinostat combo for melanoma has logical mechanism but underwhelming entinostat data draws expert pause

Nektar Therapeutics’ (NASDAQ:NKTR) NKTR-214 potential combination with Syndax Pharmaceuticals’ (NASDAQ:SNDX) entinostat has drawn mixed expert response on its potential to deliver efficacy impact in metastatic melanoma.

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Bionomics’ BNC210 has experts question loose links to predict efficacy success in Phase II for PTSD; more assured on safety

Bionomics (ASX:BNO) has weak precedence to predict efficacy success in its Phase II trial for post-traumatic stress disorder (PTSD), experts said, disputing some industry analysts commenting that BNC210’s successes in other trials could spell primary efficacy success in the indication. Experts said the links are loose, while one expert noted that animal models showed strong results but translation into human benefit remains unproven.

Safety, however, seems to be of lower concern, with its mechanism of action known to have low sedation or impact on cognition.

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Alnylam’s patisiran, Ionis’s inotersen in hATTR amyloidosis have varied strengths and weaknesses to call market winner, experts say

If approved, Alnylam Pharmaceuticals' (NASDAQ:ALNY) patisiran and Ionis Pharmaceuticals’ (NASDAQ:IONS) inotersen, both for hereditary transthyretin-related (hATTR) amyloidosis, have market strengths and weaknesses, experts said, in contrast to an analyst’s prediction of patisiran’s market superiority.

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Nightstar's and AGTC’s gene therapies for X-linked retinitis pigmentosa have unclear efficacy potential but low safety concern in Phase I/II, experts say

Preclinical success for Nightstar Therapeutics' (NASDAQ:NITE) and Applied Genetic Technologies’ (NASDAQ:AGTC) gene therapies for X-linked retinitis pigmentosa (XLRP) has experts on the fence of translation into the clinic. While animal studies have shown strong protein expression and even visual function recovery, a lack of understanding of XLRP-led photoreceptor cell death precludes confidence for a benefit in clinical trials, they said.

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