KemPharm and Genco Sciences Announce Technology Licensing

Agreement May Lead to Addition of New Drug Product Candidate and Indication to KemPharm’s Prodrug Development Pipeline

CORALVILLE, Iowa, Oct. 04, 2017 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NASDAQ: KMPH ), a clinical-stage specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, announced today that it has entered into a technology licensing and assignment agreement with Genco Sciences, LLC (Genco). 

Utilizing Genco’s early research-stage proprietary nano-particulate amphetamine technology, KemPharm will focus on devising an unique prodrug to be developed as a treatment for pediatric Tourette’s syndrome when accompanied by ADHD (attention deficit hyperactivity disorder). Genco previously received an Orphan Designation letter from the U.S. Food and Drug Administration’s (FDA) Office of Orphan Products Development for its product candidate for pediatric Tourette’s syndrome. 

“Today’s announcement of the license and assignment agreement with Genco provides KemPharm’s discovery and development team the opportunity to apply our prodrug expertise to a potential orphan-drug area of the ADHD market,” said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm.  “In a subset of pediatric Tourette’s syndrome patients, there is a recognized problem of ADHD that requires additional treatment concurrent to treating the symptoms of Tourette’s.”

Under terms of the agreement, KemPharm will be responsible for financing and managing all product development.  In exchange, KemPharm will retain full intellectual property and commercial ownership of any product developed as a result of this agreement, with Genco eligible to receive certain milestone and royalty-based or value share payments. Additional details about the agreement were not disclosed. 

“KemPharm’s success at creating unique prodrug product candidates in multiple treatment areas allows us to see real potential for a prodrug to be successful as the basis for treating this rare pediatric disease,” said Subhash Desai, Ph.D., Chief Executive Officer of Genco.  “By treating the underlying ADHD, we believe we can unlock the door to also treating tics and/or Tourette’s syndrome to provide an effective therapy for the unmet needs of those affected by this complex neurologic disorder.”

“Our goal is to discover and develop a prodrug-based therapy that can provide an appropriate and effective treatment for a rare pediatric disease while also successfully completing the FDA’s regulatory approval process, which may potentially allow us to seek a Priority Review voucher,” Dr. Mickle continued.  “Uniquely, KemPharm’s proprietary LAT prodrug platform provides an opportunity to receive a voucher under the current guidelines which is an example of another way that prodrugs can be used to create value. Our agreement with Genco both builds on this platform and potentially adds significant assets to our development pipeline in ADHD.”

About Tourette’s Syndrome and ADHD

Tourette’s syndrome is an inherited neuropsychiatric disorder with onset in childhood, characterized by the presence of motor and phonic tics – the treatment goal is managing symptoms to achieve optimum functioning, rather than eliminating symptoms; not all persons with Tourette's require treatment, and there is no cure nor universally effective medication. 

In 90% of cases, Tourette’s syndrome exists in conjunction with another disorder. The most frequent co-occurring condition in people with Tourette (63%) is ADHD, though the cause of this association is uncertain.

The Centers for Diease Control and Prevention published a series of statistics related to Tourette’s syndrome in its “A National Profile of Tourette Syndrome, 2011-2012.”  The CDC profile is linked as follows:  https://www.cdc.gov/ncbddd/tourette/data.html

About KemPharm

KemPharm is a clinical-stage specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs to treat serious medical conditions through its proprietary LAT ^TM (Ligand Activated Therapy) platform technology.  KemPharm utilizes its proprietary LAT ^TM platform technology to generate improved prodrug versions of FDA-approved drugs in the high need areas of pain, ADHD and other central nervous system disorders. KemPharm’s co-lead clinical development candidates are KP415 and KP484, both based on a prodrug of methylphenidate, but with differing extended-release profiles for the treatment of ADHD, Apadaz™, an immediate-release, abuse-deterrent acetaminophen/hydrocodone combination product candidate and KP201/IR, an acetaminophen-free formulation of the company’s immediate release abuse deterrent hydrocodone product candidate, KP201.  For more information on KemPharm and its pipeline of prodrug product candidates visit www.kempharm.com .

About Genco

Genco is a privately held company developing proprietary products for the treatment of ADHD and Tourette’s/Tics.

Caution Concerning Forward Looking Statements

This press release may contain forward-looking statements made in reliance upon the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended.  Forward-looking statements include all statements that do not relate solely to historical or current facts, and can be identified by the use of words such as “may,” “will,” “expect,” “project,” “estimate,” “anticipate,” “plan,” “believe,” “potential,” “should,” “continue” or the negative versions of those words or other comparable words. These forward-looking statements include statements regarding the ability of KemPharm to develop any product candidates under the terms of its license agreement with Genco, the expected features and characteristics of any such product candidate, and the potential for KemPharm to make any regulatory filings with the FDA, including without limitation the ability of KemPharm to seek any Priority Review voucher related thereto. These forward-looking statements are not guarantees of future actions or performance. These forward-looking statements are based on information currently available to KemPharm and its current plans or expectations, and are subject to a number of uncertainties and risks that could significantly affect current plans. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: KemPharm's financial resources and whether they will be sufficient to meet KemPharm's business objectives and operational requirements; results of earlier studies and trials may not be predictive of future clinical trial results; the protection and market exclusivity provided by KemPharm's intellectual property; risks related to the drug discovery and the regulatory approval process; the impact of competitive products and technological changes; and the FDA approval process under the Section 505(b)(2) regulatory pathway, including without limitation any timelines for related approval. KemPharm's forward-looking statements also involve assumptions that, if they prove incorrect, would cause its results to differ materially from those expressed or implied by such forward-looking statements. These and other risks concerning KemPharm’s business are described in additional detail in KemPharm's Quarterly Report on Form 10-Q for the period ended June 30, 2017, and KemPharm’s other Periodic and Current Reports filed with the Securities and Exchange Commission.  KemPharm is under no obligation to (and expressly disclaims any such obligation to) update or alter its forward-looking statements, whether as a result of new information, future events or otherwise.