Product Development

Capricor’s Phase I/II CAP-1002’s uncertain efficacy forecasts in DMD cardiomyopathy prompts future trial discussion – experts

Capricor Therapeutics’ (NASDAQ:CAPR) CAP-1002’s cardiac stem cell mechanism of action has unclear efficacy projections in its Phase I/II cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) trial, experts said. Given the lack of certainty regarding the therapy’s efficacy, experts discussed potential future trial designs that might elucidate an effect.

Some experts noted that cardiovascular (CV) cell therapies have previously struggled to demonstrate efficacy, with limited benefits largely derived from paracrine effects rather than myocardial regeneration.

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Kite’s success in DLBCL CAR-T rollout hinges on logistics, physician education, but uptake enthusiasm prevails - experts

Kite Pharma (NASDAQ:KITE) has delicate logistics and physician education hurdles to overcome if it is to be successful in launching axicabtagene ciloleucel (axi-cel) for aggressive non-Hodgkin’s lymphoma, experts said.

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AstraZeneca’s Lynparza AML/MDS side-effect rate not alarming, but concerns over future maintenance use remain - Special Report

AstraZeneca’s (LON:AZN) Lynparza (olaparib) is not exhibiting an alarming level of secondary malignancies, said experts based on an FDA adverse events (AEs) document this news service obtained through a Freedom of Information Act (FOIA) request.

The risk for secondary malignancies developing due to Lynparza treatment was a concern for the Advisory Committee that reviewed the drug’s maintenance treatment approval application.

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Longevity drugs face labelling and reimbursement quandaries with regulatory/payer reluctance, out-of-pocket payments possible – experts

Human longevity drugs may still face labelling and reimbursement challenges should they demonstrate evidence of extending a healthy life span, according to experts. As ageing is not considered a disease, regulators and payers may both take issue with labelling and paying for drugs for longevity. However, should generic drugs like metformin and rapamycin be repurposed for use as longevity agents, patients may just pay out-of-pocket given the drugs’ minimal cost, they added.

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Human longevity trials to focus on accumulation of age-related diseases rather than survival for extended life, QoL improvements – experts

Drug trials targeting human longevity will likely have endpoints focused on the accumulation of age-related diseases rather than survival to demonstrate an extended life span and improvements in health and quality of life (QoL), experts said. Trials designed around survival, however, would be challenging due to their expected high cost and likely long durations, they added.

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Humira biosimilars unlikely to launch before Amgen’s Amjevita or by 2018 - legal experts

The numerous biosimilars for AbbVie’s (NYSE:ABBV) Humira (adalimumab) are unlikely to reach the market prior to Amgen’s (NASDAQ:AMGN) approved biosimilar, Amjevita, and certainly not before 2020, legal experts agreed. AbbVie’s patent estate for Humira is far too complex and Amgen has enough of a head start advantage with its approval and ongoing litigation that it is not likely another biosimilar could surpass it to market, they said.

Further, legal experts agreed it is more likely a Humira biosimilar would reach the market in 2022.

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The Medicines Company/Alnylam’s Phase II inclisiran’s biannual dosing potential boosts hypercholesterolemia promise amid pending CVOT – experts

The Medicines Company (NASDAQ:MDCO)/Alnylam Pharmaceuticals’ (NASDAQ:ALNY) Phase II PCSK9 inhibitor inclisiran’s biannual dosing potential could substantially boost its hypercholesterolemia uptake prospects over competitors, experts agreed. Amgen’s (NASDAQ:AMGN) Repatha (evolocumab) and Regeneron Pharmaceuticals’ (NASDAQ:REGN) Praluent’s (alirocumab) approximate fortnightly dosing schedules are both less attractive than inclisiran’s, they said.

Also PCSK9 inhibitors, Praluent and Repatha were FDA approved in July and August, respectively.

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Novartis DLBCL CAR-T study closes recruitment amid enrollment backlog, faces long manufacturing wait times - source

Novartis' (VTX:NOVN) Phase II JULIET study of tisagenlecleucel-T in diffuse large B-cell lymphoma (DLBCL) is now closed for enrollment due to a large backlog of enrolled patients waiting to enter and receive treatment, a source said.

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Alkermes’ ALKS-5461 likely requires additional Phase III MDD trial for regulatory approvals based on past mixed results – experts

Alkermes’ (NASDAQ:ALKS) ALKS-5461 is likely to require an additional Phase III major depressive disorder (MDD) trial based on previous mixed clinical results before regulators will approve the therapy, experts said.

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Actelion’s independence pursuit strained to reap same R&D rewards of last two decades in PAH – analysis

• PAH portfolio an undeniable value driver with Opsumit and Uptravi
• Phase III C-Diff and MS drugs face market penetration hurdles
• Phase II pipeline lacks clinical evidence to back up differentiation

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