RUSH UNIVERSITY MEDICAL CENTER

CEL-SCI Corporation plans to offer up to $150M of common, preferred, convertible preferred stock, rights, warrants, and units. The securities will be sold at prices and terms determined at or prior to the time of sale, considering market conditions. The offering includes reoffering shares held by certain shareholders. Specific terms will be detailed in accompanying prospectus supplements. CEL-SCI may sell securities directly, through agents, or underwriters. The securities are speculative and involve high risk, suitable only for those who can afford to lose their investment. CEL-SCI's common stock trades on the NYSE American under the symbol 'CVM'.

September marked a busy start to the hepatology conference season, with significant pipeline advancements in MASH and PBC treatments, including ALG-055009's liver fat reduction and CNP-104's disease-modifying potential. The European Commission's revocation of obeticholic acid's CMA for PBC and the FDA's negative advisory committee opinion on its clinical outcomes raised concerns. Research highlighted increased cancer risks associated with MASLD and MetALD, and the Revised REACH-B model improved HCC risk prediction in chronic hepatitis B. The GHAPP conference featured discussions on PBC treatment developments and the importance of nutrition in MASH and cirrhosis management.

Rush University celebrated four faculty members appointed to endowed positions, highlighting their contributions to research and education. The appointments include Mary Jo Fidler, Robert S.D. Higgins, Meenakshi Jolly, and Hannah J. Lundberg. These endowments, supported by philanthropic partners, signify commitment to Rush’s mission and future, providing financial resources to support leading faculty and recruit renowned experts.

FDA approves Zevra Therapeutics' arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC) in adults and children 2 years and older, marking the first FDA-approved drug for this condition. The approval is based on a 12-month trial showing arimoclomol, combined with miglustat, slowed NPC progression. The treatment comes with warnings for hypersensitivity reactions and is not recommended for pregnant women.

Miplyffa, an oral treatment for NPC, boosts TFEB and TFE3 proteins to regulate lipids, approved in combination with miglustat. Zevra plans to launch it at $85,000/month, with AmplifyAssist support. Arimoclomol, previously rejected by FDA, was acquired by Zevra. IntraBio and Azafaros also have NPC treatments in development.

Jason Mazzola, 24, with Fragile X, saw significant improvements in focus, confidence, and education after taking zatolmilast, an experimental drug. His mother, Lizzie Mazzola, notes his newfound independence, including working and managing daily tasks. A 2021 study found zatolmilast improved memory and language in 30 adult males with Fragile X, leading to ongoing larger studies to determine FDA approval.

The FDA has approved arimoclomol (Miplyffa) for treating Niemann-Pick disease type C (NPC) in patients aged 2 years and older, in combination with miglustat. This approval is based on a phase 2/3 trial showing arimoclomol met its primary outcome measure of improving the NPC Clinical Severity Scale score. Arimoclomol, an orally delivered medication, increases the activation of transcription factors EB and E3, leading to upregulation of lysosomal genes. Zevra Therapeutics plans to launch the therapy in the U.S. within 8 to 12 weeks.

FDA approves MIPLYFFA, the first treatment for Niemann-Pick disease type C (NPC), in combination with miglustat for neurological manifestations in adults and pediatric patients 2 years and older. Zevra Therapeutics receives a rare pediatric disease priority review voucher and launches AmplifyAssist™ patient support program. Conference call scheduled for Sept. 23, 2024.

FDA approves Miplyffa (arimoclomol) for Niemann-Pick disease type C (NPC), the first NPC drug. Indicated for neurological manifestations in adult and pediatric patients 2 years and older, in combination with miglustat. Zevra Therapeutics receives rare pediatric disease priority review voucher.