PR92845

FLORENCE, Italy, Nov. 4, 2021 /PRNewswire=KYODO JBN/ --

The Menarini Group announced today that the U.S. Food and Drug Administration

(FDA) granted orphan drug designation (ODD) to SEL24/MEN1703 for the treatment

of Acute Myeloid Leukemia (AML). SEL24/MEN1703 is a first-in-class, orally

available, dual PIM/FLT3 inhibitor, in-licensed by Menarini from Ryvu

Therapeutics, and currently investigated in the DIAMOND-01 trial as a single

agent for the treatment of patients with relapsed/refractory AML.

ODD is granted by the FDA to therapies intended for the treatment of conditions

that impact fewer than 200,000 people in the US and provides companies with

several incentives to support the development of therapeutics and diagnostics

for rare diseases. Of note, ODD does not influence the process of regulatory

approval, and drugs for rare diseases go through the same rigorous scientific

review process established for any other drug.

"FDA orphan drug designation represents an important milestone for SEL24/MEN1703

program," said Elcin Barker Ergun, Chief Executive Officer of the Menarini

Group. "SEL24/MEN1703 is a first-in-class, orally available, dual PIM/FLT3

inhibitor that can contribute to finding new treatment paradigms for AML where

significant unmet needs exist especially as resistance develops in later lines.

We look forward to advancing the clinical development of SEL24/MEN1703 in AML

with a goal to ultimately provide patients with a new therapeutic option for

this hard-to-treat disease."

DIAMOND-01 (CLI24-001; clinicaltrials.gov identifier NCT03008187) is a

First-in-Human, Phase I/II, dose escalation and cohort expansion trial of

SEL24/MEN1703, investigated as a single agent for the treatment of patients with

relapsed/refractory AML.

In the dose escalation part of the DIAMOND-01 trial, SEL24/MEN1703 demonstrated

a manageable safety profile up to the recommended dose (RD) of 125 mg/day, along

with initial evidence of anti-leukemic activity as single agent. This evidence

has been confirmed in the cohort expansion part of the study, which also showed

preliminary single agent efficacy in relapsed/refractory AML, particularly in

patients with IDH mutant disease, either naive - or previously exposed - to IDH

inhibitors.

The trial is currently recruiting AML patients bearing IDH1 or IDH2 mutation, to

further investigate the activity of SEL24/MEN1703 in this molecularly defined

sub-population of patients.

About SEL24/MEN1703

SEL24/MEN1703 is a first-in-class, orally available, dual PIM/FLT3 inhibitor

in-licensed by Menarini from Ryvu Therapeutics. It is an investigational

compound, not approved for use by regulatory authorities, currently being

evaluated in the DIAMOND-01 trial (CLI24-001; clinicaltrials.gov identifier

NCT03008187) for the treatment of relapsed/refractory Acute Myeloid Leukemia.

About Menarini in Oncology

At Menarini, we understand that patients' hope for a longer and healthier life

is inextricably linked to the progress of scientific and medical research - that

is what drives us forward.

Menarini Group has a strong commitment to oncology research and development,

focused both on therapeutics and diagnostics. We invest in the development of

precision medicine through our pipeline of investigational drugs, which includes

both small molecules and biologics investigated for the treatment of hematologic

and solid tumors. We are also committed to developing innovative technologies

for the detection and analysis of circulating tumor cells through the work of

Menarini Silicon Biosystems.

The acquisition of Stemline Therapeutics, a New York-based biopharmaceutical

company in 2020, marked the entry of the Menarini Group into the U.S.

biopharmaceutical oncology market and, together with the license agreement

reached with Radius Health, strengthened Menarini's oncology portfolio with the

addition of both commercial and clinical-stage assets.

For further information about Menarini's pipeline, please visit the dedicated

page on our website at

https://www.menarini.com/en-us/innovation-research/our-pipeline-and-products

About Menarini

The Menarini Group is a leading international pharmaceutical and diagnostics

company, with a turnover of $4.2 billion and over 17,000 employees. Menarini is

focused on therapeutic areas with high unmet needs with products for cardiology,

oncology, pneumology, gastroenterology, infectious diseases, diabetology,

inflammation, and analgesia. With 18 production sites and 10 Research and

Development centers, Menarini's products are available in 140 countries

worldwide. For further information, please visit www.menarini.com

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Source: Menarini I.F.R.