On September 13, 2021 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM: HCM; HKEX: 13) reported that the Center for Drug Evaluation of China’s National Medical Products Administration ("NMPA") has granted Breakthrough Therapy Designation ("BTD") to amdizalisib (HMPL-689), a highly selective and potent PI3Kδ inhibitor, for the treatment of relapsed or refractory follicular lymphoma ("FL"), a subtype of non-Hodgkin’s lymphoma ("NHL") (Press release, Hutchison China MediTech, SEP 13, 2021, View Source [SID1234590538]).
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NMPA grants BTD to new drugs that treat life-threatening diseases or serious conditions for which there are no effective treatment options, and where clinical evidence demonstrates significant advantages over existing therapies. Drug candidates with BTD may be considered for conditional approval and priority review when submitting a New Drug Application (NDA). This indicates that the development and review of amdizalisib for relapsed or refractory FL may be expedited, to address patients’ unmet needs more quickly.
Christian Hogg, CEO of HUTCHMED, said, "The granting of BTD to amdizalisib by the NMPA underscores the promising clinical value of this highly selective and potent PI3Kδ inhibitor. There is a clear need for new therapies in this treatment setting, particularly with regard to specific toxicities and suboptimal efficacy with existing treatments across different lymphoma subtypes. We look forward to important clinical data on amdizalisib being presented at the ESMO (Free ESMO Whitepaper) Congress next week and are continuing to accelerate global development of this novel therapy."
Updated preliminary results from the ongoing Phase Ib expansion study in China will be presented as a Proffered Paper at the 2021 ESMO (Free ESMO Whitepaper) (European Society for Medical Oncology) Congress on September 20, 2021. To date, amdizalisib has been shown to be well tolerated, exhibiting dose-proportional pharmacokinetics ("PK"), a manageable toxicity profile, and single-agent clinical activity in relapsed/refractory B-cell lymphoma patients. Additional details may be found at clinicaltrials.gov, using identifier NCT03128164.
HUTCHMED has initiated an extensive, globally-focused clinical development pathway for amdizalisib. In April 2021, HUTCHMED initiated a Phase II registration study in China for amdizalisib in approximately 100 patients with relapsed or refractory FL and approximately 80 patients with marginal zone lymphoma ("MZL"). The trial is being conducted in over 35 sites in China. Additional details may be found at clinicaltrials.gov, using identifier NCT04849351.
Amdizalisib is also being evaluated in an ongoing Phase I/Ib study in the U.S. and Europe in patients with relapsed or refractory NHL (NCT03786926).
About PI3Kδ and NHL
PI3Kδ (phosphoinositide 3-kinase delta) is a lipid kinase that controls the activation of several important signaling proteins. Upon an antigen binding to B-cell receptors, PI3Kδ can be activated through the Lyn and Syk signaling cascade. The abnormal activation of B-cell receptor signaling is closely related to the development of B-cell type hematological cancers, which represent approximately 85% of all NHL cases. Therefore, PI3Kδ is considered a promising target for drugs that aim to treat certain hematologic cancers.
FL accounts for approximately 17% of NHL. In 2020, there were an estimated 16,000 and 13,000 new cases of FL in China and the U.S., respectively[i],[ii],[iii]. Patients with relapsed or refractory FL do not have curative treatment options and have a high unmet need for optimal therapeutic options.
About Amdizalisib
Amdizalisib (HMPL-689) is a novel, selective and potent oral inhibitor targeting the isoform PI3Kδ. Amdizalisib’s PK properties are favorable with good oral absorption, moderate tissue distribution and low clearance in preclinical PK studies, suggesting a low risk of drug accumulation and drug-to-drug interaction. Because of its high target selectivity and optimal PK profile, amdizalisib has the potential to demonstrate an optimal benefit-risk profile in this class.
HUTCHMED currently retains all rights to amdizalisib worldwide.