PRAX Praxis Precision Medicines Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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PRAX-944
Essential Tremor
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Phase 2a
Phase 2a
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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PRAX-562
Healthy volunteers
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Phase 1
Phase 1
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Phase 2 trial to commence 2021. Phase 1 trial ongoing.
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PRAX-114
Major depressive disorder
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Phase 2a
Phase 2a
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IND for Phase 2/3 trial has been placed on clinical hold - November 9, 2020. Expects to initiate trial 1H 2021.
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Latest News
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Innovative collaboration to be implemented across multiple indications starting with SCN2A-DEE
CAMBRIDGE, Mass., Feb. 16, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal imbalance, today announced a collaboration with Ciitizen, a Palo Alto based healthcare technology company that helps patients get full control of their medical records so they can find better treatment options, participate in research and find clinical trials. Praxis will utilize the Ciitizen platform to provide deeper insights across disease populations through the analysis of real-world data, initially to inform the development of PRAX-222 and PRAX-562 in SCN2A developmental and epileptic encephalopathy (SCN2A-DEE).
"As a patient-guided company, this partnership with Ciitizen is well aligned with Praxis' core principles," said Marcio Souza, president and chief executive officer of Praxis. "By integrating the Ciitizen platform, we expect to improve and accelerate the drug development process through more informed clinical trial designs and endpoints, potentially getting new therapies to patients more efficiently. We also see its potential to inform key biomarkers and add to our understanding of the genetics underlying specific disorders through genotype-phenotype analysis."
"Our partnership with Praxis is designed to help guide the development of innovative therapies and deliver a holistic view of critical health data to patients, caregivers and their networks of doctors and providers," said Anil Sethi, chief executive officer of Ciitizen. "Enabling fully informed treatment decisions can benefit patients, and we can continue to improve care through additional insights into disease signs, symptoms and progression over time."
Patients in the U.S. with SCN2A-related disorders can sign up to participate in this collaboration at https://www.ciitizen.com/scn2a/. Within 30 days of signing up to the Ciitizen platform, a comprehensive view of the patient's medical records, pharmacy records, genomic data and imaging data will be made available for the caregiver and patient to use at their discretion. There is no cost to sign up and no onsite clinical visits are required. Patients will retain exclusive ownership of all the data. With patient consent, aggregated data will be provided to Praxis.
For additional information about SCN2A-related disorders, please visit https://scn2a.com/.
PRAX-222 and PRAX-562 have been granted rare pediatric disease designation and PRAX-222 has been granted orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of SCN2A-DEE. PRAX-562 is currently being evaluated in a Phase 1 clinical trial in adult healthy volunteers and PRAX-222 is currently being evaluated in IND-enabling studies.
About SCN2A-DEE
SCN2A-DEE is a rare developmental and epileptic encephalopathy caused by a variant in the SCN2A gene. The SCN2A gene is critical in the formation of sodium channel proteins in the brain, which control the flow of sodium ions into neurons. This movement of sodium ions is a major component of generating electrical signals called action potentials, the way in which the cells communicate. Patients suffer from recurrent, typically drug-resistant seizures which start as early as the first day of life. The seizures can be of multiple different types, up to dozens per day, with poor response to current treatment options. Patients with SCN2A-DEE may have moderate to severe cognitive and developmental impairments, other neurological disorders such as dystonia and chorea, and disturbances in other organ systems including gastrointestinal function.About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders. Praxis has established a broad portfolio, including five disclosed programs across multiple central nervous system disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates.About Ciitizen
Ciitizen is a leading consumer health technology company whose mission is to help patients get full control of their medical records so they can find better treatment options, participate in research and find clinical trials. As a patient-directed care platform, Ciitizen believes patients should have full control of directing the use of their data, which serves as one of the richest sources of real world evidence, unlocking new options for patients and the community. Please see https://www.ciitizen.com/research to learn more.Investor Contact: Alex Kane Praxis Precision Medicines [email protected] 617-300-8481 Media Contact: Ian Stone Canale Communications [email protected] 619-849-5388
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FDA grants RPD and ODD for PRAX-222 for treatment of SCN2A-DEE
FDA grants RPD for PRAX-562 for treatment of SCN2A-DEE and SCN8A-DEE
CAMBRIDGE, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal imbalance, today announced that the U.S. Food and Drug Administration (FDA) has granted both rare pediatric disease and orphan drug designations for PRAX-222 for the treatment of SCN2A developmental and epileptic encephalopathy (SCN2A-DEE). The FDA has also granted rare pediatric disease designation for PRAX-562 for the treatment of SCN2A-DEE and for the treatment of SCN8A developmental and epileptic encephalopathy (SCN8A-DEE).
"As a company deeply rooted in the genetic drivers of severe pediatric epilepsies, these designations are especially meaningful and validating," said Marcio Souza, president and chief executive officer of Praxis. "We are committed to drug development for both common and rare CNS diseases and look forward to progressing PRAX-562 and PRAX-222 in the clinic and ultimately to children who are in dire need of new treatment options."
PRAX-562 is a selective small molecule and is the first persistent sodium current blocker in development for the treatment of a wide range of rare CNS disorders. The clinical development plan for PRAX-562 encompasses exploring the broad potential for the mechanism of action in rare diseases through proof-of-concept trials in two rare types of cephalgia, and then expanding into a range of rare pediatric DEEs, including SCN8A-DEE and SCN2A-DEE. PRAX-562 is currently being evaluated in a Phase 1 clinical trial in adult healthy volunteers.
PRAX-222 is an antisense oligonucleotide that is designed to lower the expression levels of the protein encoded by the SCN2A gene in patients with SCN2A gain-of-function epilepsy. The program is ongoing under a three-way collaboration with Ionis Pharmaceuticals, Inc. and RogCon, Inc. PRAX-222 is currently being evaluated in IND-enabling studies.
"The potential broad utility of PRAX-562 in DEEs and other rare CNS disorders and the precision therapy approach of PRAX-222 represent two differentiated and potentially complementary treatment paradigms inspired by human genetics," said Steven Petrou, Ph.D., co-founder and chief scientific officer of Praxis. "The FDA granting these designations is an acknowledgement of the critical need to develop treatments for children living with these devastating diseases."
The FDA's rare pediatric disease designation program is designed for serious and life-threatening diseases primarily affecting children under the age of 18 with fewer than 200,000 people in the United States. Under the FDA's rare pediatric disease and voucher programs, subject to FDA approval of a product with such designation, a company may be eligible to receive a priority review voucher that can be redeemed to obtain priority review for any subsequent marketing application or sold or transferred to other companies.
The FDA's orphan drug designation program is designed to encourage and facilitate the development of investigational treatments for rare diseases that affect fewer than 200,000 people in the United States. The designation provides various development and commercial incentives, including tax credits for clinical research costs, waiver or partial payment of application fees and market exclusivity for seven years following FDA approval.
About SCN2A-DEE/SCN8A-DEE
SCN2A-DEE and SCN8A-DEE are rare developmental and epileptic encephalopathies caused by variants in the SCN2A and SCN8A genes, respectively. Both the SCN2A and SCN8A genes are critical in the formation of sodium channel proteins in the brain, which control the flow of sodium ions into neurons. This movement of sodium ions is a major component of generating electrical signals called action potentials, the way in which the cells communicate. Patients suffer from recurrent, typically drug-resistant seizures which start as early as the first day of life. The seizures can be of multiple different types, up to dozens per day, with poor response to current treatment options. Patients with SCN2A-DEE and SCN8A-DEE have significant cognitive disabilities, ranging from moderate to severe; often movement disorders, such as dystonia or ataxia; and problems in other body systems such as gastrointestinal or ocular. Those with SCN8A-DEE also may experience autonomic features such as increases or decreases in heart rate, abnormal breathing and cyanosis.About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders. Praxis has established a broad portfolio, including five disclosed programs across multiple central nervous system disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates.Investor Contact: Alex Kane Praxis Precision Medicines [email protected] 617-300-8481 Media Contact: Ian Stone Canale Communications [email protected] 619-849-5388
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CAMBRIDGE, Mass., Dec. 01, 2020 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance, today announced that Stuart Chaffee, Ph.D., will be transitioning from his current position as the Company's chief financial officer to a part-time role as a strategic advisor to Praxis. The Company has commenced a search for a full-time chief financial officer. Lauren Mastrocola, the Company's vice president of finance and principal accounting officer, has assumed the responsibilities of principal financial officer on an interim basis.
"I'm incredibly proud of what we've built at Praxis and consider it a privilege to have helped advance the company through critical milestones," said Dr. Chaffee. "I look forward to continuing to support Praxis in an advisory role as the company advances towards its next key milestones, while I evaluate opportunities to focus on my passion for building early-stage companies."
"On behalf of the leadership team and board of directors, I would like to thank Stuart for his contributions in helping to build Praxis into the company it is today," said Marcio Souza, president and chief executive officer of Praxis. "I wish him well in returning to his roots in early-stage company development."
About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders. Praxis has established a broad portfolio, including five disclosed programs across multiple central nervous system disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates.Investor Contact: Alex Kane Praxis Precision Medicines [email protected] 617-300-8481 Media Contact: Ian Stone Canale Communications [email protected] 619-849-5388
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CAMBRIDGE, Mass., Nov. 20, 2020 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance, today announced that management will participate in the following upcoming virtual investor conferences:
- Piper Sandler 32nd Annual Healthcare Conference
Prerecorded fireside chat available on Monday, November 23, 2020 at 10:00 a.m. ET; Institutional investor meetings to be conducted on Tuesday, December 1, 2020
- 3rd Annual Evercore ISI HealthCONx Conference
Fireside chat on Thursday, December 3, 2020 from 3:55 – 4:15 p.m. ET
The events will be available via webcast through the "Events & Presentations" page of the "Investors & Media" section of the company's website at www.praxismedicines.com. Replays of the webcasts will be available for 90 days following the events.
About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders. Praxis has established a broad portfolio, including five disclosed programs across multiple central nervous system disorders including, depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates.Investor Contact:
Alex Kane
Praxis Precision Medicines
[email protected]
617-300-8481Media Contact:
Ian Stone
Canale Communications
[email protected]
619-849-5388 - Piper Sandler 32nd Annual Healthcare Conference
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- Praxis expects to initiate Phase 2/3 clinical trial for PRAX-114 in MDD in 1H21 -
CAMBRIDGE, Mass., Nov. 17, 2020 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance, today announced that it has received comments from the U.S. Food and Drug Administration (FDA) on the full clinical hold of its Investigational New Drug (IND) submission for PRAX-114 for the treatment of major depressive disorder (MDD).
The IND for PRAX-114 was placed on clinical hold by the FDA pending the resolution of certain non-clinical pharmacology and toxicology matters. In its comment letter, the FDA proposed that the Company conduct further toxicological investigation of the effect of PRAX-114 and its metabolites on fertility, reproduction, and embryofetal development to support the planned trial. The Company believes that the results of its ongoing standard fertility and reproductive studies, expected to be completed in the first quarter of 2021, together with the available toxicology package will satisfy the FDA request. The FDA also requested updates to the Investigator's Brochure and to the requirements for contraception in the protocol.
The Company now expects to initiate the randomized, placebo-controlled Phase 2/3 clinical trial for PRAX-114 in MDD in the first half of 2021. The Company is in dialogue with the FDA and intends to explore potential options to accelerate the initiation of the Phase 2/3 clinical trial.
About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance. Praxis is applying insights into the genetic mutations that drive excitation-inhibition imbalance in diseases to select biological targets for severe pediatric epilepsies and more broadly for prevalent psychiatric diseases and neurologic disorders. Praxis has established a broad portfolio, including five disclosed programs across multiple central nervous system disorders including, depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates.
Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the Company's expectations regarding the timing in which it will receive additional communications regarding the clinical hold on its IND submission for PRAX-114 from the FDA. The words "may," "might," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "seek," "predict," "future," "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: the timing and outcome of the Company's planned interactions with regulatory authorities, including resolution of the current PRAX-114 clinical hold; the timing and anticipated results of our current preclinical studies and future clinical trials, strategy and future operations; the impact of COVID-19 on countries or regions in which we have operations or do business; the delay of any current preclinical studies or future clinical trials or the development of Praxis' drug candidates; the risk that the results of current preclinical studies may not be predictive of future results in connection with future clinical trials; Praxis' ability to successfully demonstrate the safety and efficacy of its drug candidates; and obtaining, maintaining and protecting its intellectual property. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Praxis' final prospectus related to its initial public offering, dated October 20, 2020, as well as discussions of potential risks, uncertainties, and other important factors in Praxis' subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Praxis' views only as of today and should not be relied upon as representing its views as of any subsequent date. Praxis explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Investor Contact: Alex Kane [email protected] 617-300-8481 Media Contact: Ian Stone [email protected] 619-849-5388
