WOODBRIDGE, Conn. (PRWEB) December 02, 2020
AlloMek Therapeutics, a privately held orphan disease-focused company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for its macrocyclic MEK kinase inhibitor, CIP-137401, for the treatment of Neurofibromatosis type 1 (NF1).
The FDA awards “orphan” status for treatments of diseases affecting fewer than 200,000 people in the U.S., for drugs to be used for the safe and effective treatment of rare diseases or for drugs that are not expected to recoup the costs of development and marketing. This designation qualifies AlloMek for incentives including certain tax credits and a new drug application fee waiver. It also entitles AlloMek to a period of market exclusivity after approval.
According to the Children’s Tumor Foundation, Neurofibromatosis-1 is one of the most common inherited neurological disorders, affecting about 1-in-3,000 people throughout the world.
AlloMek’s lead drug, CIP-137401, is a macrocyclic MEK kinase inhibitor with excellent pharmacokinetics and a profile optimized for potency and safety. The drug has been tested in an established preclinical model of NF1 and been found to dramatically reduce both absolute tumor numbers and the size of plexiform neurofibromas. The potential to administer this drug on a once-daily basis makes it an excellent candidate to combine with therapeutics with complementary mechanisms of action.
CIP-137401 has been shown in IND-enabling animal toxicological studies to be well tolerated with limited side effects which could potentially result in an improved therapeutic index (TI) and best-in-class profile. AlloMek is currently focused on developing the compound in rare diseases, including LMNA cardiomyopathy and Noonan Syndrome, along with NF1. The company is also seeking partnerships for trials in oncology, both as a single agent and in combinations.
The company plans to begin human testing in 2021.
AlloMek Therapeutics is a Woodbridge, CT based, privately held orphan disease-focused company, developing therapeutics using macrocycles. AlloMek is applying its expertise in macrocyclic chemistry and molecular modeling to design novel drug candidates to build a high-quality pipeline of drug candidates. For more information, please visit https://allomek.com.
This press release contains forward-looking statements, including statements regarding potential benefits associated with CIP-137401 and Orphan Drug designation. These statements involve known and unknown risks, uncertainties and other factors which may cause any actual benefits to be materially different from those expressed or implied by the forward-looking statements. These statements reflect our current views and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: Orphan Drug and Fast Track designations by the FDA may not lead to a faster development, regulatory review, or approval process with the FDA and does not increase the likelihood that CIP-137401 will receive marketing approval; and ongoing and future preclinical and clinical development of CIP-137401 may not have positive results.