Product Development

Prophylactic use of higher-dose heparin elicits interest in critically ill COVID-19 patients, but potential in earlier stages still an open question, experts say

Rising reports of thromboembolic cases among COVID-19 patients have prompted physicians and also several trials to evaluate higher doses of heparin than are typically used as an anticoagulant strategy, but experts noted there is still a lack of clarity on the optimal regimen. Additionally, they said, while all hospitalized patients stand to benefit from a prophylactic dose, whether to use the higher therapeutic dose in those patients is an open question.

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COVID-19 safety concerns lead BioMarin to allow tapering of immunosuppressive regimen doses in Phase III haemophilia A gene therapy trial, investigator says; other gene therapy sponsors consider protocol amendments

BioMarin Pharmaceutical (NASDAQ:BMRN) advised investigators earlier this month to start tapering the doses of the immunosuppressive regimen in its Phase III study of valoctocogene roxaparvovec in haemophilia A patients if they thought safety would be an issue in light of COVID-19, said investigator Dr Doris Quon, medical director, Orthopaedic Hemophilia Treatment Center, Los Angeles, California.

The company held a teleconference advising investigators, and provided guidance on how to taper the doses week by week, Quon said.

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Limitations of early data on mesenchymal stromal cells in ARDS due to COVID-19 prompt expert caution; patient heterogeneity a factor in upcoming studies

While COVID-19 data from a China-based study and Pluristem Therapeutics’ (NASDAQ:PSTI) compassionate use program focused on those with acute respiratory distress syndrome (ARDS) is encouraging to continue exploring mesenchymal stem cell (MSC) products, there are also several limitations with interpreting the results, experts noted. Readthrough to trials being planned by three main MSC players—Athersys (NASDAQ:ATHX), Mesoblast (ASX:MSB) and Pluristem—is unclear without more granular data and trial design details, they noted.

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Phase III investigator-led hydrocortisone trial for pneumonia redesigned to include COVID-19 patients; enrolment at 300 patients and expected to close in two weeks (as of 7th April), source says

An investigator-led Phase III trial studying hydrocortisone for community-acquired pneumonia has been redesigned to also recruit COVID-19 patients, according to a source familiar with the trial. The France-based study has so far recruited about 300 COVID-19 patients and enrolment is estimated to close in two weeks, he added. As of today (7 April), France has 98,984 confirmed cases and 8,911 deaths.

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Fulcrum’s losmapimod has unclear translation into functional benefit despite potential to achieve primary endpoint of DUX4 reduction in Phase II FSHD trial, experts say

Fulcrum Therapeutics’ (NASDAQ:FULC) losmapimod for facioscapulohumeral muscular dystrophy (FSHD) has some experts unsure if it could make meaningful gains in the Phase II trial’s function and muscle gain secondary endpoints. Despite losmapimod’s potential to meet the biomarker primary endpoint based on available data, some experts noted this may not translate to the more clinically relevant functional benefit endpoints.

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Scholar Rock’s SRK-015 Phase II trial unlikely to convince on potential in type 2 and 3 SMA even if benchmarks met, experts say

Scholar Rock’s (NASDAQ:SRRK) SRK-015 has experts questioning the value of the data set to come from its ongoing Phase II trial in spinal muscular atrophy (SMA), even if it were to meet clinically meaningful benchmarks.

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Novartis, Nektar’s Phase III combinations likely to struggle for a slice of 1L melanoma market due to tight Opdivo/Yervoy grasp; trial success not guaranteed, experts say

Phase III first-line (1L) melanoma treatment combinations are likely to face the daunting task of chipping away at Bristol-Myers Squibb’s (NYSE:BMY) Opdivo (nivolumab) plus Yervoy’s (ipilimumab) standard-of-care (SOC) stronghold due to its compelling long-term data, experts said.

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Amag’s Phase IIb/IIIa AMAG-423 efficacy confidence in pre-eclampsia lacks prospective data backing; operational hurdles keep promising EDLF-positive data at bay, experts say

Amag Pharmaceuticals’ (NASDAQ:AMAG) Phase IIb/IIIa success expectations with AMAG-423 (digoxin immune fab) in severe pre-eclampsia are clouded by lack of prior randomized data relevant to the trial’s primary endpoint, experts said.

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Mesoblast’s MPC-06-ID stem cell therapy for chronic low back pain lacks evidence for Phase III efficacy and safety confidence; access challenges expected, experts say

Mesoblast’s (ASX:MSB) MPC-06-ID (mesenchymal precursor cells) has experts unsure of Phase III success in chronic low back pain due to scant human evidence and a small sample size in the Phase II. Experts noted efficacy uncertainties, including cell survival, whether the precursor cells would turn into the requisite cells, whether disc repair would supersede puncture damage and regeneration translating into pain or functional benefit.

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Seattle Genetics’ tisotumab vedotin needs stronger response durability to make up for low ORR in cervical cancer; ADC mode favorable to TIL therapies for rapid use, experts say

Seattle Genetics’ (NASDAQ:SGEN) tisotumab vedotin needs to show longer responses in metastatic cervical cancer patients in a Phase II study than those previously seen in Phase I/II data in order to prove clinical significance and potentially support approval, experts said.

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