KemPharm, Inc. (NASDAQ:KMPH), a clinical-stage specialty
pharmaceutical company engaged in the discovery and development of
proprietary prodrugs, today announced several key corporate and
clinical updates as a follow up to the Company’s second quarter
2016 business and financial update.
Ligand Activated Therapy (LAT) Prodrug Pipeline
Update:After a thorough evaluation of its entire drug
development pipeline, KemPharm has designated KP415, the Company’s
extended release (ER) d-threo-methylphenidate (d-MPH) product for
the treatment of attention deficit hyperactivity disorder (ADHD),
and KP201/IR, the Company’s single-entity, benzhydrocodone
hydrochloride (HCl) immediate-release (IR), abuse-deterrent product
candidate designed for the treatment of acute pain, as its two lead
product candidates. This decision was based on KemPharm’s
comprehensive review of the expected development timelines,
regulatory pathways and market economics for each product
candidate. As a result, KemPharm now anticipates the filing of New
Drug Applications (NDAs) for KP415 and KP201/IR in 2018, and for
the ER formulation of KP511, KemPharm’s prodrug of hydromorphone,
in 2019.
“We believe KP415 and KP201/IR represent substantial value
opportunities for KemPharm as each offers the potential to address
important patient, prescriber and market needs,” stated Travis C.
Mickle, Ph.D., President and Chief Executive Officer of KemPharm.
“Based on the strength of our existing data, as well as what we
believe are favorable developmental, regulatory and market
conditions, we are advancing KP415 and KP201/IR as our lead
pipeline candidates and expect to meet several clinical and
regulatory milestones for each product candidate prior to year-end
and over the course of the next 18 to 24 months.”
In preclinical studies of KP415, KemPharm observed features that
could provide significant benefits when compared with other
FDA-approved and widely prescribed methylphenidate products.
Pharmacokinetic data from preclinical studies suggest that the time
to maximum plasma concentration of methylphenidate after oral
administration of KP415 is approximately three times longer
compared to currently marketed IR methylphenidate. As a prodrug
that requires metabolism, KemPharm believes KP415 may be able to
deliver d-MPH more consistently than the current formulation based
approaches. This improved delivery may provide more consistent
therapy.
Dr. Mickle commented, “KemPharm expects to file the
Investigational New Drug (IND) application for KP415 prior to the
close of the third quarter of this year. Based on this
timeline, our plan is to initiate and complete proof-of-concept
studies before the end of 2016 with human clinical trials
potentially beginning during the first half of 2017. This
would put KemPharm on target to potentially file an NDA for KP415
in 2018 with the potential to introduce a differentiated product to
the ADHD market that could enable a more predictable therapy
compared with currently available, IR d-MPH products.”
As previously reported, KemPharm completed its End-of-Phase 1
(EOP1) meeting with the FDA for KP201/IR on June 23, 2016.
KemPharm had requested an EOP1 meeting for KP201/IR ahead of the
anticipated IND application based on the amount of data, both
nonclinical and clinical, that was generated from the development
of Apadaz™. KemPharm believes that data from its KP201.A03
study, which compared hydrocodone exposure following insufflation
of benzhydrocodone HCl (KP201) vs. hydrocodone bitartrate (HB),
aligns with the FDA Division of Anesthesia, Analgesia and Addiction
Products (Division) criteria for achieving abuse-deterrent product
labeling, and may potentially provide KP201/IR with a favorable
regulatory pathway.
Dr. Mickle remarked, “We expect to file the IND for KP201/IR
during the fourth quarter of 2016 with the potential to initiate
human clinical trials in the first quarter of 2017. As
previously announced, we guided towards a NDA filing for KP201/IR
by year-end 2017 and we are now targeting 2018 to file the KP201/IR
NDA due to the acceleration of the KP415 program as well as to
allow for current issues within the IR abuse-deterrent opioid space
to clarify. We believe this timeline would still enable
KP201/IR to potentially reach the market as the first
abuse-deterrent IR hydrocodone product as well as the first
hydrocodone-related product without acetaminophen.”
KemPharm will maintain the current investment plan to advance
the development of an abuse-deterrent, ER formulation of KP511
(KP511/ER). However, the need to add an external ER technology, as
well as the investigation of the potential for both overdose
protection and limitation of opioid induced constipation, has
required KemPharm to extend the original timeline for this product
candidate. KemPharm now anticipates filing a NDA for KP511/ER in
2019.
Dr. Mickle stated, “KP511/ER remains an important asset for
KemPharm, and we plan to continue its development as we believe a
differentiated product with potential abuse-deterrent and patient
benefits could capture a significant portion of the estimated $350
million hydromorphone market. However, we have made a strategic
decision to dedicate greater R&D and financial resources to the
development of KP415 and KP201/IR given what we believe to be the
developmental efficiencies and market potential of each product
candidate.”
Apadaz Regulatory and Commercial Update:As
previously announced, on August 3, 2016, KemPharm completed its End
of Review (EoR) meeting with the FDA for Apadaz (benzhydrocodone
and acetaminophen) after receiving a Complete Response Letter (CRL)
to the NDA. KemPharm has recently received and reviewed the
minutes from the EoR meeting and has notified the FDA that the
Company will elect to continue the regulatory review process with
the submission of a Formal Dispute Resolution Request (FDRR).
KemPharm anticipates up to twelve months may be required to
complete all parts of the FDRR process.
The intent of a FDRR submission is to provide a pathway by which
applicants seek to resolve scientific and/or medical policy
disputes that cannot be resolved at the Division level within the
FDA. If an issue is not resolved at the Division level, the
applicant may request that the matter be reviewed at the next
higher management level. KemPharm has elected to pursue a FDRR in
order to address and hopefully resolve certain disagreements with
the Divison’s interpretation of the “Guidance for Industry:
Abuse-Deterrent Opioids – Evaluation and Labeling” and the data
provided within the Apadaz NDA related to the abuse-deterrence
properties of the product candidate. The issues for the FDRR
are solely related to the abuse-deterrence properties of Apadaz and
what constitutes abuse-deterrence in the context of a label
claim.
As reported during the second quarter corporate and financial
update, the Apadaz EoR meeting provided clarity into the issues
identified by the FDA in the Apadaz NDA and involved discussions of
fundamental questions pertaining to abuse deterrence in relation to
the broader IR prescription opioid market and
hydrocodone-acetaminophen combination products, as well as
published industry guidance from the FDA concerning the evaluation
and labeling of abuse deterrent opioids. The EoR meeting
minutes have confirmed that the Division is focused primarily on
Category 3 data in determining whether an IR opioid product is
eligible for any abuse-deterrent label claims.
“Though we believe the data submitted in the Apadaz NDA
supported important elements of the Category 3 criteria, the data
did not meet the endpoints of greatest relevance to the Division,”
stated Dan Cohen, Executive Vice President of Government and Public
Relations. “Consequently, KemPharm and the Division could not agree
as to an appropriate label, resulting in the issuance of the CRL,
and now the next step is our submission of an appeal through the
FDRR process. By this petitioning for review at the next
levels within the FDA, we hope to find a path forward for an Apadaz
product label that could include abuse deterrence claims.”
Given the anticipated timeline for the FDRR and, if successful,
the time needed to submit any required elements to modify the
existing Apadaz NDA, KemPharm has decided to defer its commercial
operations and realign its financial resources and operational
priorities towards its product development pipeline. With
this strategic adjustment, KemPharm has eliminated the positions of
several managers and executives from the Company’s commercial team,
including that of Tracy M. Woody, Chief Commercial
Officer.
Dr. Mickle continued, “Due to the ongoing process with Apadaz,
we made the difficult decision to defer our commercial operations
and as a result have eliminated several commercial-oriented
positions, including that of Tracy Woody and other members of the
commercial team. I would like to express my sincerest
gratitude to Tracy and her team for their exceptional work and wish
them the very best in their future endeavors.”
Cash Position & Financial Update:As of June
30, 2016, total cash, cash equivalents, restricted cash, marketable
securities and long-term investments, was $102.6 million.
KemPharm continues to assess opportunities to strengthen its
balance sheet as it deems advantageous. Based on KemPharm’s
updated corporate, operational and clinical plans, KemPharm
anticipates that current cash on hand may fund the Company through
2018.
Conference Call Information:The Company will
host a conference call and live audio webcast on Thursday,
September 15, 2016, at 8:30 a.m. ET. Interested participants and
investors may access the conference call by dialing either:
- (866) 395-2480 (U.S.)
- (678) 509-7538 (international)
- Conference ID: 82561137
An audio webcast will be accessible via the Investor Relations
section of the KemPharm website
http://investors.kempharm.com/. An archive of the webcast
will remain available for 30 days beginning at approximately 9:30
a.m. ET, on September 15, 2016.
About KemPharmKemPharm is a clinical-stage
specialty pharmaceutical company focused on the discovery and
development of proprietary prodrugs to treat serious medical
conditions through its LAT platform technology. KemPharm
utilizes its LAT platform technology to generate improved prodrug
versions of FDA-approved drugs in the high need areas of pain, ADHD
and other CNS disorders.
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
expected features and characteristics of KP201/IR, KP415 and
KP511/ER and the timelines surrounding potential clinical trials
for KP415 and KP201/IR, the expected timing of filing of INDs for
KP201/IR and KP415, potential submissions of NDAs for KP201/IR,
KP415 and KP511/ER, the outcome of the FDRR process for Apadaz and
KemPharm’s ability to fund its operations through 2018. 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,
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 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 quarter ended June 30, 2016, 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.
Investor Contacts:
Jason Rando / Joshua Drumm, Ph.D.
Tiberend Strategic Advisors, Inc.
212-375-2665 / 2664
jrando@tiberend.com
jdrumm@tiberend.com
Media Contact:
Daniel L. Cohen
Executive VP, Government and Public Relations
KemPharm, Inc.
202-329-1825
dcohen@kempharm.com
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