Product Development

MyoKardia’s mavacamten for obstructive HCM draws Phase III success pause due to theoretical safety red flags in available data, cumbersome study design, high success bar, experts say

MyoKardia’s (NASDAQ:MYOK) mavacamten for obstructive hypertrophic cardiomyopathy (HCM) draws muted expert enthusiasm for Phase III trial success based on available data’s long-term adverse event (AE) risk signal, experts noted. Adding to their hesitance over the drug's value is the Phase III dose-adjustment trial desin and the high success bar established by available highly efficacious one-off procedures.

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Lexicon/Sanofi's Zynquista outlook suffers from lack of direct comparison to SGLT2 inhibitors in type 2 diabetes; CKD results too short-term to determine Phase III outcomes

Lexicon Pharmaceuticals (NASDAQ:LXRX) and Sanofi’s (EPA:SAN) Zynquista (sotagliflozin) has experts reserving judgement on the drug’s value without a direct comparison to SGLT2 inhibitors following topline data in type 2 diabetes (T2D) patients with chronic kidney disease (CKD).

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Cylcerion’s Phase II praliciguat for HFpEF primary endpoint has debatable value in elderly; success bar challenging to reach for clinical relevance, experts say

Although some experts welcomed Cyclerion Therapeutics’ (NASDAQ:CYCN) Phase II praliciguat trial’s coprimary efficacy endpoint as clinically valuable, others questioned this choice of endpoint in elderly patients, who are the primary patients with heart failure with preserved ejection fraction (HFpEF). While change in VO2 from baseline endpoint relates to quality of life (QOL), it is measured via exercise capacity improvements and so may not be valuable to elderly patients with comorbidities, they said.

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Adaptimmune’s dose escalation strategy for lead MAGE-targeted TCR cancer therapies has nebulous efficacy value; prior data has interpretation limitations, experts say

Boosting the number of cells in Adaptimmune Therapeutics’ (NASDAQ:ADAP) lead T-cell receptor (TCR) therapies in ongoing Phase I oncology trials is unlikely to form the basis of enhancing its efficacy potential, experts said. While there is early positive data for both ADP-A2M4 (formerly MAGE-A4 TCR) and ADP-A2M10 (formerly MGE-A10 TCR), interpreting results across tumor types is challenging, they noted.

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Albireo’s Phase II LDL-C primary endpoint more pertinent in NASH versus Akcea/Ionis’ Phase IIb triglyceride reduction endpoint, experts say

Albireo Pharma’s (NASDAQ:ALBO) Phase II elobixibat trial’s cardiovascular disease (CVD)-related primary endpoint may be more relevant to nonalcoholic steatohepatitis (NASH) than Akcea Therapeutics (NASDAQ:AKCA)/Ionis Pharmaceuticals’ (NASDAQ:IONS) Phase IIb trial’s CV outcome, experts said. Albireo’s trial has a low-density lipoprotein cholesterol (LDL-C) reduction endpoint whilst the AKCEA-ANGPTL3-LRx trial has an outcome of fasting triglyceride decrease.

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Novartis’ Zolgensma has first-line potential in spinal muscular atrophy but durability questions linger without long-term data, experts say

Novartis’ (SIX:NOVN) Zolgensma (onasemnogene abeparvovec-xioi) has strong potential to be used as a first-line treatment in spinal muscular atrophy (SMA), but it is unclear how durable it will be despite being designed as a one-time treatment, experts said. Second-line options including Biogen’s (NASDAQ:BIBB) Spinraza (nusinersen) are being considered, but lack of clinical data attesting to post-Zolgensma use could cause payer resistance.

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Merck KGaA’s and Novartis’ Phase II drugs for MET exon 14 skipping mutation require diagnostic adjustments to spur use, experts say

Improving patient identification tools will be key to getting full market traction for Merck KGaA’s (ETR:MRK) tepotinib and Novartis’ (SIX:NOVN) capmatinib after an expected approval in non-small cell lung cancer (NSCLC) patients with MET exon 14 skipping mutations, experts said. Presentations at ASCO earlier in June on initial Phase II data for both drugs were impressive, however, with experts citing interest in using the drug in the first-line (1L) setting.

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Amgen’s AMG510 clinical data deemed encouraging for NSCLC on ASCO update with single-agent potential; CRC data disappointing but higher dose awaited, experts say

Amgen’s (NASDAQ:AMGN) Phase I/II AMG510 efficacy data in non-small cell lung cancer (NSCLC) improves expert outlook on its KRAS G12C inhibition mechanism’s potential as a single agent, experts said. However, some expert caution remains due to currently poor colorectal cancer (CRC) data in the same trial, and overall low patient numbers for both indications.

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Amarin’s Vascepa, AstraZeneca’s Epanova theoretical CVD risk reduction class effect possibly stifled by Epanova’s DHA ingredient, experts say

Although Amarin’s (NASDAQ:AMRN) Vascepa (icosapent ethyl) is likely to lock in FDA approval and swift uptake in cardiovascular disease (CVD) risk reduction, there could be future competition with AstraZeneca’s (LON:AZN) Epanova (omega-3-carboxylic acids), experts said. A potential class efficacy/safety effect is possible as both therapies are approved for hypertriglyceremia, but Epanova’s dissimilar ingredient list could be a key differentiating element, they added.

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PFIC patient variation to stifle SOC label domination by either Albireo’s odevixibat or Mirum’s maralixibat; differentiation still nebulous, investigators say

Heterogeneity among progressive familial intrahepatic cholestasis (PFIC) types 1 and 2 patients is likely to impede both Albireo Pharma’s Phase III odevixibat and Mirum Pharmaceuticals’ Phase III maralixibat in claiming standard-of-care designation for this rare liver disease, investigators said. With both odevixibat and maralixibat having a comparable mechanism and limited available data, it is unclear whether either have differentiating features to identify the subpopulations where they would flourish, they added.

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