Orphazyme A/S in connection with a court-ordered restructuring to sell substantially all of its assets and business operations to KemPharm, Inc.

Orphazyme A/S in restructuring
company announcement
No. 24/2022
Inside information
www.orphazyme.com
Company registration number 32266355

• KemPharm to acquire the assets of Orphazyme, including those related to the development and approval of arimoclomol, for a total of $12.8 million in cash and assumed liabilities estimated at approximately $5.2 million

• The majority of approximately 20 current Orphazyme employees will become KemPharm employees

• KemPharm intends to pursue approval of arimoclomol as a treatment option for NPC

Restructuring Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme” or the “Company”), a late-stage biopharmaceutical company developing arimoclomol for Niemann-Pick type C (NPC) disease, today announces have today signed an agreement to sell substantially all of the Company’s assets and business operations to KemPharm Denmark A/S, a wholly owned subsidiary of KemPharm Inc. (KMPH: NASDAQ, NY). KemPharm is a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare diseases of the central nervous system.

Under the agreement, KemPharm intends to retain substantially all of Orphazyme’s current employees, continue early access programs with arimoclomol, and pursue potential approval of arimoclomol as an option. treatment for NPC. KemPharm will pay Orphazyme a total of $12.8 million in cash and assume liabilities estimated at approximately $5.2 million. Completion of the transaction is expected to result in full or very high coverage of creditors with uncontested claims based on claims filed during the restructuring. The agreement is subject to the approval of Orphazyme’s creditors and the Danish bankruptcy court, and is expected to close no later than June 1, 2022.

“Given that Orphazyme is undergoing legal restructuring, the primary objective was to reach an agreement that satisfies our obligations to creditors, including our employees. We are pleased that this has been achieved. In addition, we have assured the pursuit of the development of arimoclomol in the hope of making it available to NPC patients, which has been our primary motivation since the creation of the company,” said Georges Gemayel, Chairman of the Board of Orphazyme.

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Travis C. Mickle, President and CEO of KemPharm, said, “NCP is a devastating disease and there is a profound need for an effective treatment to help patients. We believe that the efficacy signal for arimoclomol is compelling and that there is a viable regulatory pathway to obtain marketing regulatory approvals. KemPharm has gained significant experience in difficult regulatory situations, and we welcome the opportunity to work with the team to resubmit regulatory submissions and make arimoclomol available to all who could benefit from the treatment.

After completion of the agreement, Orphazyme will have no ongoing operational business activities.

For more information, please contact

Orphazyme A/S undergoing restructuring

Anders Vadsholt, Managing Director and Chief Financial Officer +45 2898 9055

John Sommer Schmidt, Restructuring Administrator +45 8620 7500

About KemPharm
KemPharm is a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases through its proprietary LAT^® (Ligand Activated Therapy). KemPharm uses its proprietary LAT^® technology platform to generate improved prodrug versions of FDA-approved drugs as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. KemPharm’s pipeline of prodrug product candidates is focused on high-need areas of idiopathic hypersomnia (IH) and other CNS diseases/rare diseases. Additionally, the U.S. Food and Drug Administration (FDA) has approved AZSTARYS^®a once-daily treatment for ADHD in patients six years of age and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), which is marketed by Corium, Inc. in the United States, and APADAZ^®an immediate-release combination product containing benzhydrocodone, KemPharm’s hydrocodone prodrug, and acetaminophen, which is marketed by KVK-Tech, Inc. in the United States. For more information on KemPharm and its portfolio of prodrug candidates, visit www.kempharm.com or join us on Twitter, LinkedIn, Facebook and Youtube.

About Orphazyme
Orphazyme is a late-stage biopharmaceutical company developing arimoclomol for Niemann-Pick type C (NPC) disease. Orphazyme is headquartered in Denmark. Orphazyme shares are listed on Nasdaq Copenhagen (ORPHA).

About arimoclomol
Arimoclomol is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins and improve lysosome function. Arimoclomol is taken orally and has now been studied in 10 phase 1, four phase 2 and three pivotal phase 2/3 trials. Arimoclomol has received Orphan Drug Designation (ODD) for NPC in the US and EU. Arimoclomol has received Fast-Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare Pediatric Disease Designation (RPDD) from the United States Food and Drug Administration (FDA) for NPC. On June 17, 2021, Orphazyme received a full response letter from the FDA regarding its New Drug Application for arimoclomol for the treatment of NPC. The company has requested that a Type B meeting be held early in the third quarter of 2022.

Forward-looking statement
This company announcement may contain certain forward-looking statements under the United States Private Securities Litigation Reform Act of 1995 and others, including forward-looking statements regarding the company’s sale of substantially all of its assets and business operations. to KemPharm Inc. Although the company believes that its expectations are based on reasonable assumptions, all statements other than statements of historical facts included in this company announcement regarding future events are subject to (i) change without notice and (ii) factors beyond the Company’s control, including by virtue of regulatory or judicial intervention. Except as required by law, the Company undertakes no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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