Acadia Pharmaceuticals Announces Trofinetide New Drug Application for the Treatment of Rett Syndrome has been Accepted for Filing and Review by U.S. FDA

About Rett Syndrome

Rett syndrome is a rare genetic neurodevelopmental disorder that occurs primarily in females following a near normal development in the first two years of life.  It is caused by mutations on the X chromosome on a gene called MECP2.  Occurring worldwide in approximately one of every 10,000 to 15,000 female births and in the United States impacts 6,000 to 9,000 patients.  Children with Rett syndrome experience a period of developmental regression between 18-30 months of age, which is typically followed by a plateau period lasting years to decades. Rett syndrome is diagnosed based on clinical evaluation, typically by about three years of age

A complex and multisystem disorder, Rett syndrome causes profound impairment to central nervous system (CNS) function, including loss of communication skills, purposeful hand use, gait abnormalities, and stereotypic hand movements such as hand wringing/squeezing, clapping/tapping, mouthing and washing/rubbing automatisms.   People living with Rett syndrome may also experience a range of additional symptoms, such as gastrointestinal complications, skeletal abnormalities, neuroendocrine abnormalities, disruptive and anxiety-like behaviors, as well as mood dysregulation and sleep disturbances. Currently, there are no FDA-approved medicines for the treatment of Rett syndrome.

About Trofinetide

Trofinetide is an investigational drug. It is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function. Trofinetide is thought to stimulate synaptic maturation and overcome the synaptic and neuronal immaturities that are characteristic of Rett syndrome pathophysiology. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical for both normal development and for response to injury and disease. Trofinetide has been shown to inhibit the production of inflammatory cytokines, inhibit the overactivation of microglia and astrocytes, and increase the amount of available IGF-1 that can bind to IGF-1 receptors.

https://en.wikipedia.org/wiki/Trofinetide

Acadia Pharmaceuticals Inc.  announced   the U.S. Food and Drug Administration (FDA)  acceptance  for filing its New Drug Application (NDA) of trofinetide for the treatment of Rett syndrome. The FDA has granted a priority review and assigned a PDUFA (Prescription Drug User Fee Act) action date of March 12, 2023. The FDA has also informed the company that at this time they are not planning to hold an Advisory Committee meeting.

“We’re pleased that the FDA has accepted our NDA filing and we will be working closely with them to facilitate completion of the review in a timely manner,” said Steve Davis, Acadia’s Chief Executive Officer. “If approved, trofinetide will be the first drug available for the treatment of Rett syndrome, a rare and devastating condition for patients and their families. This milestone reinforces Acadia’s ongoing commitment to advancing research into high unmet needs in disorders affecting the central nervous system.”

Rett syndrome is a complex, multisystem neurodevelopmental disorder that includes a period of normal development followed by significant developmental regression with loss of language and hand function skills, impaired gait and development of hand stereotypes.1,2 It occurs worldwide in approximately one of every 10,000 to 15,000 female births.3

“Rett is a complex disease that can present with a diverse array of symptoms. In clinical trials, trofinetide demonstrated a significant improvement in a range of Rett syndrome symptoms,” said Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center, Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center and Phase 3 Lavender™ study investigator. “We look forward to the FDA’s review of this submission and the prospect of having access to the first approved treatment for Rett syndrome.”

The NDA is supported by results from the pivotal Phase 3 Lavender study evaluating the efficacy and safety of trofinetide versus placebo in 187 girls and young women aged 5-20 years with Rett syndrome. The study demonstrated a statistically significant improvement over placebo on the co-primary endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) total score change from baseline to 12 weeks (p=0.0175; effect size=0.37) and the Clinical Global Impression-Improvement (CGI-I) scale score (p=0.0030; effect size=0.47). The RSBQ is a caregiver assessment of the core symptoms of Rett syndrome, and the CGI-I is a global physician assessment of worsening or improving of Rett syndrome. In addition, the study also met its key secondary endpoint, the Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist–Social Composite Score (CSBS-DP-IT–Social) change from baseline to week 12 (p=0.0064; effect size=0.43), a caregiver assessment of ability to communicate.

In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited (ASX: NEU) for the development and commercialization of trofinetide for the treatment of Rett syndrome and other indications in North America. In addition to receiving priority review by the FDA, trofinetide has been granted Fast Track Status and Orphan Drug Designation for the treatment of Rett syndrome in the U.S. and has been granted Rare Pediatric Disease (RPD) designation by the FDA. Upon FDA approval of a product with RPD designation, the sponsor can receive a Priority Review Voucher, which can be used to obtain priority review for a subsequent application.

Sunovion Announces Acceptance by the U.S. FDA of the New Drug Application for Dasotraline for the Treatment of Adults with Moderate-to-Severe Binge Eating Disorder

In continuation of my update on dasotraline,

Sunovion Pharmaceuticals Inc.(Sunovion) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for dasotraline, a novel dopamine and norepinephrine reuptake inhibitor (DNRI), for the treatment of patients with moderate-to-severe binge eating disorder (BED). Dasotraline’s pharmacokinetic profile, characterized by an extended half-life, supports its potential for sustained control of moderate-to-severe BED symptoms.

The action date by the FDA under the Prescription Drug User Fee Act (PDUFA) is May 14, 2020.

Dasotraline is an investigational, once-daily medication that demonstrated significant efficacy for the treatment of moderate-to-severe BED in two 12-week, randomized, placebo-controlled studies, SEP360-221 and SEP360-321. Dasotraline was found to be generally well tolerated in clinical studies, including a long-term safety study, SEP360-322, that assessed patients with moderate-to-severe BED for up to one year.

“Binge eating disorder is a serious mental health condition for which limited treatment options exist. The disorder is often seen in association with other behavioral conditions such as depression, substance abuse and post-traumatic stress disorder, and it is often under-diagnosed and under-treated,” said Antony Loebel, M.D., President and Chief Executive Officer at Sunovion. “We are confident in the value dasotraline has shown in clinical trials to people living with BED and look forward to working with the FDA to advance this novel treatment option.”

According to the American Psychiatric Association in the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, (DSM-5),2,3 BED is characterized by recurrent and persistent episodes of binge eating, defined as consuming large quantities of food in a short period of time, perception of loss of control during the episode, and intense feelings of shame, guilt and embarrassment afterwards. Many individuals also suffer from depressive and anxiety symptoms, as well as a number of medical complications, leading to impaired functioning and quality of life.

https://en.wikipedia.org/wiki/Dasotraline

Ipsen Announces U.S. FDA Approval for Newly Designed Pre-Filled Syringe for Somatuline Depot (lanreotide)

 

Ipsen Biopharmaceuticals, an affiliate of Ipsen , announced today that the United States Food and Drug Administration (FDA) has approved a new pre-filled syringe for Somatuline Depot (lanreotide). The syringe includes updated features, such as larger flanges, designed to help make it easier for healthcare providers to administer the injection.1 The indications remain the same as those for the previous pre-filled syringe and include the treatment of adult patients with unresectable, well- or moderately-differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors (GEP-NETs) to improve progression-free survival; treatment of adults with carcinoid syndrome; when used, it reduces the frequency of short-acting somatostatin analog rescue therapy; and the long-term treatment of patients with acromegaly who have had an inadequate response to surgery and/or radiotherapy, or for whom surgery and/or radiotherapy is not an option. Please see Important Safety Information below and accompanying full Prescribing Information.

“The conditions of GEP-NETs and acromegaly can be associated with a number of uncomfortable and unpleasant symptoms, and innovation aimed at improving the injection process is a step forward,” said Daphne Adelman, Clinical Nurse Specialist, Northwestern University, Chicago, and one of the authors of the study.

Ipsen conducted five separate but complementary studies in partnership with patients, their caregivers, nurses and other healthcare professionals to better understand the current use of the existing Somatuline® Depot pre-filled syringe and to evaluate ways to improve the features of the device.1 The result of this collaboration is a redesigned delivery system intended to make it easy to grip the syringe and administer the injection. The new syringe features a needle shield removal system, more stable plunger and thermoform tray that has recessed areas designed to help prevent accidental plunger depression. The built-in safety system, which may help to prevent needle stick injury by locking in place following the administration, has not been changed.

“We consistently look for opportunities to respond to the needs of the communities we serve, and this approval would not have been possible without the direct involvement of nurses and the patients with GEP-NETs and acromegaly whom they treat,” said Bradley Bailey, SVP, and Franchise Head Oncology/Endocrinology Business Unit at Ipsen. “We listened and collaborated to enhance the existing pre-filled syringe, making it sturdier for healthcare providers when administering treatment, with the intention of improving the injection process. We look forward to bringing this innovation to healthcare providers for their patients soon.”

The new pre-filled syringe is for deep subcutaneous injection and is intended for administration by a healthcare professional. Healthcare providers can expect to receive the new syringe during Q3 2019. The device is approved for use in the U.S., EU and additional ex-U.S. markets.

Intellipharmaceutics Announces Resubmission of New Drug Application to the U.S. FDA for its Oxycodone ER

 In continuation of my update on Oxycodone ER

ntellipharmaceutics International Inc. (NASDAQ: IPCI, TSX: IPCI) ("Intellipharmaceutics" or the "Company"), a pharmaceutical company specializing in the research, development and manufacture of novel and generic controlled-release and targeted-release oral solid dosage drugs,  announced that it has resubmitted its New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") for its Oxycodone ER product candidate.

The Company's NDA for an abuse-deterrent version of Oxycodone ER was initially accepted for filing by the FDA in February 2017. Intellipharmaceutics resubmitted the NDA in response to a Complete Response Letter ("CRL") received from the FDA, which provided certain recommendations and requests for information. The FDA granted us an extension to February 28, 2019 to resubmit our NDA under section 505(b)(2) of the U.S. Federal Food, Drug and Cosmetic Act and the Company has now met this deadline.

There can be no assurance that Intellipharmaceutics will not be required to conduct further studies for Oxycodone ER, that the FDA will approve any of the Company's requested abuse-deterrent label claims or that the FDA will ultimately approve the NDA for the sale of Oxycodone ER in the U.S. market, or that it will ever be successfully commercialized.

About Intellipharmaceutics

Intellipharmaceutics International Inc. is a pharmaceutical company specializing in the research, development and manufacture of novel and generic controlled-release and targeted-release oral solid dosage drugs. The Company's patented Hypermatrix™ technology is a multidimensional controlled-release drug delivery platform that can be applied to a wide range of existing and new pharmaceuticals. Intellipharmaceutics has developed several drug delivery systems based on this technology platform, with a pipeline of products (some of which have received FDA approval) in various stages of development. The Company has ANDA and NDA 505(b)(2) drug product candidates in its development pipeline. These include the Company's abuse-deterrent oxycodone hydrochloride extended release formulation ("Oxycodone ER") based on its proprietary nPODDDS™ novel Point Of Divergence Drug Delivery System (for which an NDA has been filed with the FDA), and Regabatin™ XR (pregabalin extended-release capsules).

Cautionary Statement Regarding Forward-Looking Information

Certain statements in this document constitute "forward-looking statements" within the meaning of the United States Private Securities Litigation Reform Act of 1995 and/or "forward-looking information" under the Securities Act (Ontario). These statements include, without limitation, statements expressed or implied regarding our expectations regarding our plans, goals and milestones, status of developments or expenditures relating to our business, plans to fund our current activities, and statements concerning our partnering activities, health regulatory submissions, strategy, future operations, future financial position, future sales, revenues and profitability, projected costs and market penetration and risks or uncertainties related to our ability to comply with the Nasdaq and TSX continued listing standards and our ability to develop and implement a plan of compliance with the Nasdaq continued listing standards acceptable to a Nasdaq Panel. In some cases, you can identify forward-looking statements by terminology such as "appear", "unlikely", "target", "may", "will", "should", "expects", "plans", "plans to", "anticipates", "believes", "estimates", "predicts", "confident", "prospects", "potential", "continue", "intends", "look forward", "could", "would", "projected", "goals", "set to", "seeking" or the negative of such terms or other comparable terminology. We made a number of assumptions in the preparation of our forward-looking statements. You should not place undue reliance on our forward-looking statements, which are subject to a multitude of known and unknown risks and uncertainties that could cause actual results, future circumstances or events to differ materially from those stated in or implied by the forward-looking statements. Risks and uncertainties relating to us and our business can be found in the "Risk Factors" section of our latest annual information form, our latest Form 20-F, and our latest Form F-1 and Form F-3 (including any documents forming a part thereof or incorporated by reference therein), as amended, as well as in our reports, public disclosure documents and other filings with the securities commissions and other regulatory bodies in Canada and the U.S., which are available on www.sedar.com and www.sec.gov. The forward-looking statements reflect our current views with respect to future events and are based on what we believe are reasonable assumptions as of the date of this document and we disclaim any intention and have no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

https://www.drugbank.ca/drugs/DB00497

Intellipharmaceutics Announces Resubmission of New Drug Application to the U.S. FDA for its Oxycodone ER