KemPharm | May 16, 2022
Arimoclomol is an NDA-stage, revenue-generating investigational drug candidate being developed for the treatment of Niemann-Pick disease type C a rare progressive neurodegenerative disease
KemPharm plans to refile the New Drug Application (NDA) for arimoclomol in NPC with the U.S. Food and Drug Administration as early as the First Quarter of 2023
CELEBRATION, Fla., May 15, 2022 KemPharm, Inc. a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases, announced a definitive agreement with Orphazyme A/S to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC). NPC is a rare progressive neurodegenerative disease that impacts children, adolescents, and adults, and is characterized by an inability of the body to transport cholesterol and lipids inside of cells, which leads to the abnormal accumulation of these substances within various tissues of the body, including the brain. Arimoclomol is currently being made available to NPC patients in the U.S., France and Germany under Orphazyme’s Early Access Programs (EAP).
Under the terms of the agreement, KemPharm will purchase substantially all of the assets and operations of Orphazyme, including arimoclomol, for a cash payment of USD $12.8 million. The Company expects to finance the cash payment with a revolving line of credit secured by KemPharm’s balance sheet. KemPharm intends to retain the majority of Orphazyme’s current employees. In addition, KemPharm has agreed to assume an estimated reserve liability equal to approximately USD $5.2 million, which is an estimated future rebate due to the French regulatory authorities based on the revenue generated from the EAP in France. For the year ending December 31, 2022, the EAP is expected to generate at least USD $12 million in revenue based upon enrollment in France as of March 2022. The EAP is expected to remain in place until arimoclomol becomes commercially available in each of the current EAP markets. The transaction is expected to close on or before June 1, 2022, subject to customary closing conditions and approval by Orphazyme’s creditors and the Danish bankruptcy court. Canaccord Genuity LLC acted as a strategic advisor to KemPharm for the transaction.
“This strategic acquisition of arimoclomol is a transformative event that significantly expands our rare CNS disease development pipeline, bringing to KemPharm an NDA-stage, revenue-generating product upon which we intend to build commercial capabilities that allow KemPharm to create and retain value for the benefit of shareholders. Moreover, the financial structure of the acquisition combined with the revenue currently being generated by arimoclomol from the early access program in France affords us the opportunity to acquire the asset in a capital efficient manner that has the potential to create positive cash flow, while incurring no shareholder dilution.”
Richard Pascoe, Executive Chairman of KemPharm
Arimoclomol is administered orally and has been studied in ten Phase 1, four Phase 2, and three pivotal Phase 2/3 trials. Arimoclomol has received Orphan Drug Designation (ODD) for NPC in the United States and the European Union. Arimoclomol has received Fast-Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare Pediatric Disease Designation (RPDD) from the FDA for NPC. If approved in the U.S., arimoclomol would also be eligible to receive a Pediatric Priority Review Voucher. On June 17, 2021, Orphazyme received a Complete Response Letter (CRL) from the FDA regarding its NDA for arimoclomol for the treatment of NPC. Orphazyme also withdrew its European Marketing Authorisation Application (MAA) for arimoclomol for the treatment of NPC ahead of a final vote and opinion by the Committee for Medicinal Products for Human Use.
“The acquisition of arimoclomol aligns perfectly with our strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including our lead clinical candidate, KP1077 in idiopathic hypersomnia,” stated Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “We have carefully evaluated the CRL issued by the FDA and the minutes from the subsequent Type A meeting, as well as the data that has been generated from the development work performed to date. We believe the efficacy signal for arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission. KemPharm has significant experience with challenging regulatory situations, having successfully led or participated in three FDA product approvals, two of which followed an initial CRL. We welcome the opportunity to work with the FDA on the resubmission of the NDA for arimoclomol in NPC, which we expect to file as early as the first quarter of 2023.”
NPC is a rare progressive lysosomal storage disorder characterized by an inability of the body to transport cholesterol and lipids inside of cells. This leads to dysfunction in organs such as the brain, spleen and liver. NPC can range from a fatal disorder within the first few months after birth (neonatal period) to a late onset, chronic progressive disorder that remains undiagnosed well into adulthood. Disease progression is irreversible in all patients, and loss of neuro-cognitive function adversely impacts their daily life. The mean age of death is 13 years (Bianconi, 2019), and there are no approved treatments for NPC in the United States.
“NPC is an ultra-rare, inherited neurodegenerative disease that affects people of all ages from infancy to adulthood, and leads to progressive impairment of mobility, cognition, speech, and swallowing, culminating in premature death,” said Marc Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics at Mayo Clinic. “Therapies to treat NPC are desperately needed, and there is hope that a treatment such as arimoclomol could provide a solution to patients around the world who are living daily with the disease. It is encouraging that there is an opportunity to continue the regulatory process for arimoclomol with the FDA.”
KemPharm is a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system diseases through its proprietary LAT® platform technology. KemPharm utilizes its proprietary LAT® platform technology 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 prodrug product candidate pipeline is focused on the high need areas of idiopathic hypersomnia (IH) and other CNS/rare diseases. In addition, the U.S. Food and Drug Administration (FDA) has approved AZSTARYS®, a new once-daily treatment for ADHD in patients age six years and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), which is being commercialized by Corium, Inc. in the U.S., and APADAZ®, an immediate-release combination product containing benzhydrocodone, KemPharm’s prodrug of hydrocodone, and acetaminophen, which is being commercialized by KVK-Tech, Inc. in the U.S.
Titan Pharmaceuticals, Inc. | February 03, 2022
Titan Pharmaceuticals, Inc. announced that it has entered into a securities purchase agreement with a single institutional investor to purchase 3,374,242 shares of its common stock (or pre-funded warrants in lieu thereof) in a registered direct offering priced at-the-market under Nasdaq rules. In a concurrent private placement, Titan has also agreed to issue and sell to the investor 1,289,796 pre-funded warrants, at the same purchase price as in the registered direct offering. In addition, the Company has agreed to issue to the investor in a concurrent private placement unregistered warrants to purchase up to an aggregate of 4,664,038 shares of common stock. The combined offering price of each share or pre-funded warrant and each warrant will be $1.18. The warrants will be immediately exercisable, will expire on the five year and six month anniversary of the issuance date and will have an exercise price of $1.14 per share.
The aggregate gross proceeds to the Company of both offerings is expected to be approximately $5.5 million. The offerings are expected to close on or about February 4, 2022, subject to the satisfaction of customary closing conditions.
Maxim Group LLC is acting as the exclusive placement agent in connection with the offerings.
The shares of common stock in the registered direct offering are being offered pursuant to a shelf registration statement on Form S-3 which was declared effective by the United States Securities and Exchange Commission ("SEC") on April 24, 2019. The pre-funded warrants and warrants issued in the concurrent private placement and the shares issuable upon exercise of such warrants were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended and Regulation D promulgated thereunder and have not been registered under the Act or applicable state securities laws.
This press release does not constitute an offer to sell or the solicitation of an offer to buy, nor will there be any sales of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction. A prospectus supplement relating to the shares of common stock issued in the registered direct offering will be filed by Titan with the SEC.
About Titan Pharmaceuticals
Titan Pharmaceuticals, Inc. based in South San Francisco, CA, is a development stage company developing proprietary therapeutics with its ProNeura® long-term, continuous drug delivery technology. The ProNeura technology has the potential to be used in developing products for treating a number of chronic conditions, where maintaining consistent, around-the-clock blood levels of medication may benefit the patient and improve medical outcomes. Ultimate validation of the ProNeura® delivery system has been exemplified by approval of Probuphine in the US (FDA), EU and Canada.
BioMatrix Specialty Pharmacy | February 09, 2022
BioMatrix Specialty Pharmacy announced inclusion as a preferred provider in AscellaHealth's home infusion pharmacy network. AscellaHealth, a global specialty pharmacy and healthcare solutions company, has launched a site of care optimization strategy designed to cut costs for third-party payers without compromising quality of care. BioMatrix was included in the network to help the organization achieve its goals of improving drug affordability; increasing price transparency; reducing disparities in quality of care and safety; enabling care delivery in the highest-quality, lowest-cost care setting; and increasing patient convenience.
Determining the optimal site of care for patients to receive therapy is an important component of treatment success. BioMatrix Specialty Pharmacy provides robust specialty infusion services, including site of care coordination. Whether administering in-home with assistance from one of our home infusion nurses, in-office, or at an ambulatory infusion center, our clinical team works with patients, payers, and prescribers to make therapy administration as safe and convenient as possible. Using a nationwide network of highly trained specialty infusion nurses, BioMatrix coordinates over 1,200 home nursing visits every month.
"We are pleased to be included as a preferred provider in AscellaHealth's home infusion network. Our shared focus on positive outcomes for all patients, reduced cost, and price transparency will help AscellaHealth's members effectively manage their specialty infusion spend without compromising quality care."
Ken Trader, BioMatrix VP of Managed Markets
"By selecting top pharmacies for preferred status, we are taking an important step toward reducing the cost of specialty biologic medications and encouraging patients to move from higher-cost settings, like big hospitals and institutions, to lower-cost settings, such as physician's in-office infusion suites or stand-alone infusion centers," says Dea Belazi, president and CEO, AscellaHealth. "As part of our mission to help our clients better manage the high costs of specialty medication, this initiative exemplifies our efforts to increase care accessibility by encouraging the use of clinically appropriate, lower-cost care settings."
About BioMatrix Specialty Pharmacy
BioMatrix Specialty Pharmacy, an Inc. 5000 company, offers comprehensive, nationwide specialty pharmacy services and digital health technology solutions for patients with chronic, difficult to treat conditions. Our commitment to every patient is to provide individualized pharmacy services, timely access to care, and focused education and support. We offer a tailored approach for a wide range of therapeutic categories, improving health and empowering patients to experience a higher quality of life.
About AscellaHealth LLC
AscellaHealth is a global Specialty Pharmacy and Healthcare services organization serving patients, payers, life sciences and providers, offering a comprehensive portfolio of customized, tech-enabled specialty pharmaceutical and medical management services. An Inc. 5000 2021 winner, AscellaHealth's unique, patient-centric approach is built upon proprietary technology processes for innovative programs and services optimizing health outcomes and quality of life for patients with complex chronic conditions or rare diseases that require specialty medications and/or gene and cell therapies.
LexisNexis Reed Tech | April 27, 2022
Reed Technology and Information Services Inc. a leading provider of data management and analytics solutions for the life sciences industry, announced an alliance with Schlafender Hase®, the market leader for intelligent, automated proofreading software and services for regulated industries. Current and prospective customers of both companies can leverage exclusive benefits as part of this new alliance ecosystem. Pharmaceutical labelers who have regulatory deadlines for US FDA can benefit from the expert conversion and submission services offered by Reed Tech while ensuring reduced risk and greater accuracy in the proofreading process for drug labels and related documentation with Schlafender Hase's TVT®, the Text Verification Tool®.
"Schlafender Hase is already a trusted ally as part of our pharmaceutical SPL conversion process. By offering our UDI and pharma customers the benefits of TVT, we continue advancing our mission of providing innovation for the unique workflows of these specialty manufacturers. Ensuring the accuracy of labels, barcodes, graphics and IFU language is essential. With TVT®, regulatory team members around the world benefit from superior, automated proofreading in order to reduce risk and meet the compliance requirements of local health authorities,"
Arshad Rahman, General Manager of the Reed Tech Life Sciences division
"Schlafender Hase is pleased to recognize Reed Tech as an industry-leading, core SPL conversion service provider. It will be productive for our customers to have access to their deep understanding of regulatory submission requirements for drug manufacturers and distributors," said Peter Muller, Sales Director, North America at Schlafender Hase. Those manufacturers bringing new products to market or maintaining existing portfolios will benefit from the subject-matter expertise offered by Reed Tech."
Both the pharmaceutical and medical device industries are continually upgrading processes and preparing for multiple global health authority requirements. The complementary solutions and services between Reed Tech and Schlafender Hase offer critical efficiency gains in managing the product life cycle.
About LexisNexis Legal & Professional
LexisNexis® Legal & Professional provides legal, regulatory, and business information and analytics that help customers increase their productivity, improve decision-making, achieve better outcomes, and advance the rule of law around the world. As a digital pioneer, the company was the first to bring legal and business information online with its Lexis® and Nexis® services. LexisNexis Legal & Professional, which serves customers in more than 150 countries with 10,500 employees worldwide, is part of RELX, a global provider of information-based analytics and decision tools for professional and business customers.
Reed Tech serves manufacturers and distributors of medical device and drug products, and for those who support them in consulting and IT roles by delivering better outcomes for the collection, transformation, submission and analysis of regulatory data. Our solutions support the innovation ecosystem and enable you to achieve regulatory compliance and improved product data management contributing to impactful innovations to our world. The corporate mission is to advance humanity by delivering better outcomes to the innovation community. Reed Tech is a LexisNexis® company.
About Schlafender Hase
Schlafender Hase leads the way in intelligent, automated text and graphic proofreading solutions. With its flagship product, TVT®, the Text Verification Tool®, users can quickly compare source content with output formats, detecting changes and deviations prior to publication.
Schlafender Hase serves regulated industries across the globe. Clients include the world's leading pharmaceutical and medical device companies, as well as healthcare regulatory bodies. The continued success of Schlafender Hase is based on delivering quality products and a strong understanding of customer pain points. Easy to use, our products reduce proofreading workloads, assure the quality of printed and online materials, and mitigate the risk of costly errors.