PRAX Praxis Precision Medicines Inc.

16.76
-1.64  -9%
Previous Close 18.4
Open 17.84
52 Week Low 12.71
52 Week High 60.9505
Market Cap $751,782,823
Shares 44,855,777
Float 29,170,656
Enterprise Value $515,389,296
Volume 300,827
Av. Daily Volume 214,609
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Upcoming Catalysts

Drug Stage Catalyst Date
PRAX-944
Essential tremor
Phase 2a
Phase 2a
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PRAX-562
Healthy volunteers
Phase 1
Phase 1
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PRAX-114 (Acapella)
Major depressive disorder
Phase 2
Phase 2
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PRAX-114 Aria Study (Study 213)
Major Depressive Disorder
Phase 2/3
Phase 2/3
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PRAX-114
Essential Tremor
Phase 2
Phase 2
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PRAX-114
PTSD
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
PRAX-562
Rare Adult Cephalgias
Phase 2
Phase 2
Phase 2 trial to be initiated 4Q 2021.
PRAX-562
Developmental epileptic encephalopathies (DEEs)
Phase 2
Phase 2
Phase 2 trial to be initiated 1H 2022.
PRAX-944
Parkinson's Disease
Phase 2
Phase 2
Phase 2 trial to be initiated 1H 2022.
PRAX-114
Major Depressive Disorder and perimenopausal depression
Phase 2a
Phase 2a
Phase 2a Part B data released August 16, 2021. Mean decreases from baseline at Day 15 of 60% in frequency of moderate-to-severe hot flashes. Phase 2b trial plans to be disclosed 4Q 2021.

Latest News

  1. BOSTON, Nov. 24, 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 management will participate in a fireside chat at Piper Sandler's 33rd Annual Virtual Healthcare Conference on Thursday, December 2, 2021 at 10:30 a.m. ET.

    The fireside chat will be available via live webcast through the Events & Presentations page of the Investors + Media section of the company's website at www.praxismedicines.com. A replay of the webcast will be available on Praxis' website for 90 days following the event.

    About Praxis
    Praxis Precision…

    BOSTON, Nov. 24, 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 management will participate in a fireside chat at Piper Sandler's 33rd Annual Virtual Healthcare Conference on Thursday, December 2, 2021 at 10:30 a.m. ET.

    The fireside chat will be available via live webcast through the Events & Presentations page of the Investors + Media section of the company's website at www.praxismedicines.com. A replay of the webcast will be available on Praxis' website for 90 days following the event.

    About Praxis

    Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance. Praxis is applying insights from genetic epilepsies to broader neurological and psychiatric disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad portfolio, including multiple disclosed programs across CNS disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on LinkedIn and Twitter.



    Investor Contact:
    Alex Kane
    Praxis Precision Medicines
    
    617-300-8481
    
    Media Contact:
    Ian Stone
    Canale Communications
    
    619-849-5388

    Primary Logo

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  2. Enrollment on track for 1H22 topline results for PRAX-114 Phase 2/3 monotherapy MDD Aria Study and for PRAX-114 Phase 2 dose-ranging MDD Acapella Study

    PRAX-114 Phase 2 studies in post-traumatic stress disorder and essential tremor (ET), PRAX-562 Phase 2 study in rare adult cephalgias expected to initiate in 4Q21

    Preliminary results for PRAX-944 Phase 2a study for treatment of ET expected in 4Q21

    First patient enrolled in PRAX-944 Phase 2b Essential1 Study for treatment of ET; topline results expected in 2H22

    Cash and investments of $314.4 million as of September 30, 2021 supports runway into 2Q23

    Praxis to host Movement Disorder Day on December 17, 2021

    BOSTON, Nov. 03, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ…

    Enrollment on track for 1H22 topline results for PRAX-114 Phase 2/3 monotherapy MDD Aria Study and for PRAX-114 Phase 2 dose-ranging MDD Acapella Study

    PRAX-114 Phase 2 studies in post-traumatic stress disorder and essential tremor (ET), PRAX-562 Phase 2 study in rare adult cephalgias expected to initiate in 4Q21

    Preliminary results for PRAX-944 Phase 2a study for treatment of ET expected in 4Q21

    First patient enrolled in PRAX-944 Phase 2b Essential1 Study for treatment of ET; topline results expected in 2H22

    Cash and investments of $314.4 million as of September 30, 2021 supports runway into 2Q23

    Praxis to host Movement Disorder Day on December 17, 2021

    BOSTON, Nov. 03, 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 provided a corporate update and reported financial results for the third quarter ended September 30, 2021.

    "The advancements throughout our portfolio in recent months exemplify the potential of an unwavering team and an operational backbone that allows us to efficiently move a diverse set of programs forward in multiple CNS indications," said Marcio Souza, president and chief executive officer of Praxis. "As we look back on the progress in our first year as a public company and enter a catalyst-rich period with data readouts across each of our three clinical programs, we remain as determined as ever to bring CNS drugs to those in need."

    Recent Business Highlights and Upcoming Milestones:

    Psychiatry

    • Praxis expects topline results from the ongoing PRAX-114 Phase 2/3, placebo-controlled Aria Study for monotherapy treatment of Major Depressive Disorder (MDD) in the first half of 2022. The Aria Study is intended to serve as one of two trials required by the U.S. Food and Drug Administration (FDA) to demonstrate clinical efficacy to support registration of PRAX-114 for monotherapy treatment of MDD.



    • The Company expects topline results from the ongoing PRAX-114 Phase 2, placebo-controlled, dose-ranging Acapella Study in the first half of 2022. The Acapella Study is intended to provide additional understanding of the dose range and to evaluate the safety and efficacy of PRAX-114 at doses of 10, 20, 40 and 60 mg, primarily in adjunctive MDD participants.



    • Following the announcement of positive topline results from the PRAX-114 Phase 2a, proof-of-concept trial for treatment of perimenopausal depression, Praxis plans to continue investigation of PRAX-114 for treatment of women with menopausal and mood symptoms in a Phase 2b trial. Plans for the Phase 2b trial will be disclosed in the fourth quarter of 2021.



    • Praxis plans to initiate a PRAX-114 Phase 2, placebo-controlled study for treatment of post-traumatic stress disorder (PTSD) in the fourth quarter of 2021. Topline results are expected in the second half of 2022. The trial is designed to evaluate the safety, tolerability and efficacy of a nightly dose of 40 mg of PRAX-114 for 4 weeks in approximately 80 participants with PTSD, using the CAPS-5 total score as the primary endpoint.

    Movement Disorders

    • The Company has completed dosing in a two-part PRAX-944 Phase 1 study to explore a faster titration regimen. Topline results are expected in the fourth quarter of 2021. The Phase 1 study is designed to evaluate the safety, tolerability and pharmacokinetics (PK) of titrating PRAX-944 up to 120 mg in a 10-day regimen in participants aged 18 to 54 years (Part A) and 55 to 75 years (Part B).



    • Praxis is currently in the second of two cohorts of its PRAX-944 Phase 2a trial for treatment of essential tremor (ET), evaluating safety and efficacy in patients titrated up to 120 mg per day. Preliminary open-label safety, tolerability and efficacy data is expected in the fourth quarter of 2021, followed by complete open-label and placebo-controlled, randomized withdrawal results in the first half of 2022.



    • Praxis initiated the PRAX-944 Phase 2b Essential1 Study for treatment of ET in the third quarter of 2021 and has started enrolling participants. Topline results are expected in the second half of 2022. Essential1 is a placebo-controlled, dose-ranging clinical trial designed to evaluate the safety, tolerability and efficacy of PRAX-944 at 20, 60 or 120 mg per day.



    • Praxis plans to initiate a PRAX-114 Phase 2, placebo-controlled, crossover study for daytime treatment of ET to evaluate safety, PK and efficacy of 10 or 20 mg of PRAX-114 in the fourth quarter of 2021. Topline results are expected in the second half of 2022.



    • The Company intends to initiate a Phase 2 trial to evaluate the safety, PK and efficacy of PRAX-944 as a non-dopaminergic treatment for the motor symptoms of Parkinson's disease in the first half of 2022.



    • Praxis plans to host a Movement Disorder Day in New York City and virtually on Friday, December 17, 2021.

    Rare Disease

    • Praxis has completed the dosing and safety follow-up period for its single ascending dose (SAD) and multiple ascending dose (MAD) cohorts up to 150 and 120 mg, respectively, in its PRAX-562 Phase 1, healthy volunteer study. A summary of the findings is included below and in the slide deck accompanying our third quarter 2021 corporate update in the Investors + Media section of our website.



      • PRAX-562 was well-tolerated, with no clinically significant safety findings. The most common treatment emergent adverse events (TEAEs) were headache and dizziness. Almost all TEAEs were mild in severity. No drug-related serious adverse events or severe adverse events were observed.

      • The maximum observed concentration was observed between 2 and 3 hours after dosing. There was no impact of food intake on PK. The half-life of PRAX-562 is approximately 4-5 days across the dose range evaluated.

      • Dose-related changes were observed in the exploratory Auditory Steady-State Response (ASSR) electroencephalogram (EEG) translational biomarker. In the 120 mg dose group, a reduction in ASSR of greater than 50% was observed after 14 days QD as compared to baseline.

    • Based on the observed signal in the ASSR marker in the Phase 1, healthy volunteer study, Praxis has started dosing patients in the U.S. in a PRAX-562 Phase 1, placebo-controlled, two-cohort EEG study to validate the observed signal. Topline data is expected in the first half of 2022. The study is intended to evaluate ASSR as a biomarker for the PRAX-562 program to further support selection of therapeutic dose levels in Phase 2 studies.

    • The Company intends to initiate a PRAX-562 Phase 2 trial in the fourth quarter of 2021 in the U.S. for treatment of patients with rare adult cephalgias, including a cohort of participants with Short-lasting Unilateral Neuralgiform headache attacks with Conjunctival injection and Tearing (SUNCT) and Short-lasting Unilateral Neuralgiform headache with Autonomic symptoms (SUNA), and a cohort of participants with Trigeminal Neuralgia (TN).

    • Praxis plans to initiate a PRAX-562 Phase 2 trial for treatment of developmental epileptic encephalopathies (DEEs) in the first half of 2022.

    • Praxis has completed the Investigational New Drug (IND) enabling toxicology study for its lead antisense oligonucleotide (ASO) candidate, PRAX-222, and plans to initiate regulatory submissions in order to begin a PRAX-222 Phase 1/2 trial for treatment of SCN2A-DEE in the first half of 2022.

    Third Quarter 2021 Financial Results:

    As of September 30, 2021, Praxis had $314.4 million in cash, cash equivalents and marketable securities, compared to $296.6 million in cash and cash equivalents as of December 31, 2020. This increase of $17.8 million primarily reflects $98.4 million in net proceeds from the follow-on public offering of shares of our common stock in May 2021, partially offset by cash used in operations of $79.7 million during the nine months ended September 30, 2021. The company's cash, cash equivalents and marketable securities as of September 30, 2021 are expected to fund operations into the second quarter of 2023.

    Research and development expenses were $33.1 million for the three months ended September 30, 2021, compared to $12.8 million for the three months ended September 30, 2020. The increase in R&D expenses of approximately $20.4 million was primarily attributable to $10.9 million in increased expenses related to our clinical-stage programs, $5.2 million in increased personnel-related costs due to increased headcount and $2.9 million in increased expenses related to our discovery-stage programs.

    General and administrative expenses were $11.6 million for the three months ended September 30, 2021, compared to $3.4 million for the three months ended September 30, 2020. The increase in general and administrative expenses of $8.2 million was primarily attributable to $4.7 million in increased personnel-related costs due to increased headcount, $1.8 million in increased professional fees and a $1.7 million increase in other general and administrative expenses.

    Praxis reported net loss of $44.7 million for the three months ended September 30, 2021, including $6.5 million of stock-based compensation expense, compared to a net loss of $16.2 million for the three months ended September 30, 2020, including $1.0 million of stock-based compensation expense.

    As of September 30, 2021, Praxis had 44.8 million shares of common stock outstanding.

    About Praxis

    Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance. Praxis is applying insights from genetic epilepsies to broader neurological and psychiatric disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad portfolio, including multiple disclosed programs across CNS disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates. For more information, please visit https://praxismedicines.com/ and follow us on LinkedIn and Twitter.

    Forward-Looking Statements

    This press release may contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis' future expectations, plans and prospects, including, without limitation, statements regarding expectations, plans and timing for clinical data, the sufficiency of our cash, cash equivalents and marketable securities, the anticipated timing of our clinical trials and regulatory filings, and the development of our product candidates, including the design of our clinical trials and the treatment potential of our product candidates as well as other statements containing the words "anticipate," "believe," "continue," "could," "endeavor," "estimate," "expect," "anticipate," "intend," "may," "might," "plan," "potential," "predict," "project," "seek," "should," "target," "will" or "would" and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.

    The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials or to market products; whether Praxis' cash resources will be sufficient to fund Praxis' foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, uncertainties and assumptions regarding the impact of the continuing COVID-19 pandemic on Praxis' business, operations, strategy, goals and anticipated timelines, Praxis' ongoing and planned preclinical activities, Praxis' ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Praxis' timelines for regulatory submissions and Praxis' financial position; and other risks concerning Praxis' programs and operations are described in additional detail in its Annual Report on Form 10-K for the year ended December 31, 2020, its Quarterly Reports on Form 10-Q and other subsequent filings made with the Securities and Exchange Commission from time to time. Although Praxis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.



    PRAXIS PRECISION MEDICINES, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (Amounts in thousands)

    (Unaudited)

     September 30, 2021 December 31, 2020
    Assets 
    Cash and cash equivalents$165,679   $296,608  
    Marketable securities148,691     
    Prepaid expenses and other current assets4,969   5,718  
    Property and equipment, net625   82  
    Operating lease right-of-use assets4,028   754  
    Other non-current assets416   15  
    Total assets$324,408   $303,177  
    Liabilities and stockholders' equity   
    Accounts payable$7,544   $4,088  
    Accrued expenses16,529   10,869  
    Operating lease liabilities4,413   763  
    Common stock5   4  
    Additional paid-in capital553,975   437,007  
    Accumulated other comprehensive loss(25)    
    Accumulated deficit(258,033)  (149,554) 
    Total liabilities and stockholders' equity$324,408   $303,177  
              





    PRAXIS PRECISION MEDICINES, INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS 

    (Amounts in thousands, except share and per share amounts)

    (Unaudited)

     Three Months Ended



    September 30,
     Nine Months Ended



    September 30,
     2021  2020 2021 2020
    Operating expenses:       
    Research and development$33,139   $12,786   $76,746   $28,704  
    General and administrative11,634   3,431   31,929   7,552  
    Total operating expenses44,773   16,217   108,675   36,256  
    Loss from operations(44,773)  (16,217)  (108,675)  (36,256) 
    Other income:       
    Other income, net73   1   201   134  
    Total other income73   1   201   134  
    Loss before benefit from income taxes(44,700)  (16,216)  (108,474)  (36,122) 
    Benefit from (provision for) income taxes(5)     (5)  8  
    Net loss$(44,705)  $(16,216)  $(108,479)  $(36,114) 
    Accretion and cumulative dividends on redeemable convertible preferred stock   (3,943)     (8,046) 
    Gain on repurchase of redeemable convertible preferred stock         493  
    Net loss attributable to common stockholders$(44,705)  $(20,159)  $(108,479)  $(43,667) 
    Net loss per share attributable to common stockholders, basic and diluted$(1.00)  $(12.10)  $(2.61)  $(26.53) 
    Weighted average common shares outstanding, basic and diluted44,714,941   1,665,902   41,608,017   1,645,982  
                    





    Investor Contact:
    Alex Kane
    Praxis Precision Medicines
    
    617-300-8481
    
    Media Contact:
    Ian Stone
    Canale Communications
    
    619-849-5388

    Primary Logo

    View Full Article Hide Full Article
  3. BOSTON, Oct. 22, 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 it will provide a corporate update and report financial results for the third quarter 2021 on Wednesday, November 3, 2021, before the U.S. financial markets open. Management will discuss recent business and pipeline progress via a conference call and webcast at 8:30 a.m. ET.

    Conference Call and Webcast Details:
      
    US/Canada Toll Free:833-398-1037
      
    International:914-987-7735
      
    Conference ID:7687942
      
    Webcast:https://edge.media-server.com/mmc/p/8yk7hqak

    BOSTON, Oct. 22, 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 it will provide a corporate update and report financial results for the third quarter 2021 on Wednesday, November 3, 2021, before the U.S. financial markets open. Management will discuss recent business and pipeline progress via a conference call and webcast at 8:30 a.m. ET.

    Conference Call and Webcast Details:
      
    US/Canada Toll Free:833-398-1037
      
    International:914-987-7735
      
    Conference ID:7687942
      
    Webcast:https://edge.media-server.com/mmc/p/8yk7hqak
      

    A replay of the webcast will be available approximately two hours following its conclusion and will be archived for 30 days following the event, accessible through the Events & Presentations page of the Investors + Media section of Praxis' website at https://investors.praxismedicines.com/events-and-presentations.

    About Praxis

    Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance. Praxis is applying insights from genetic epilepsies to broader neurological and psychiatric disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad portfolio, including multiple disclosed programs across CNS disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates. For more information, please visit https://praxismedicines.com/ and follow us on LinkedIn and Twitter.



    Investor Contact:
    Alex Kane
    Praxis Precision Medicines
    
    617-300-8481
    
    Media Contact:
    Ian Stone
    Canale Communications
    
    619-849-5388

    Primary Logo

    View Full Article Hide Full Article
  4. CAMBRIDGE, Mass., Sept. 01, 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 management will participate in the ‘Emerging Therapies for Psychiatric Disorders' panel at Citi's 16th Annual BioPharma Virtual Conference on Wednesday, September 8, 2021 at 10:40 a.m. ET.

    The panel will be available via live webcast through the Events & Presentations page of the Investors + Media section of the company's website at www.praxismedicines.com. A replay of the webcast will be available on Praxis' website for 90 days following…

    CAMBRIDGE, Mass., Sept. 01, 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 management will participate in the ‘Emerging Therapies for Psychiatric Disorders' panel at Citi's 16th Annual BioPharma Virtual Conference on Wednesday, September 8, 2021 at 10:40 a.m. ET.

    The panel will be available via live webcast through the Events & Presentations page of the Investors + Media section of the company's website at www.praxismedicines.com. A replay of the webcast will be available on Praxis' website for 90 days following the event.

    About Praxis

    Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance. Praxis is applying insights from genetic epilepsies to broader neurological and psychiatric disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad portfolio, including multiple disclosed programs across CNS disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on LinkedIn and Twitter.



    Investor Contact:
    Alex Kane
    Praxis Precision Medicines
    
    617-300-8481
    
    Media Contact:
    Ian Stone
    Canale Communications
    
    619-849-5388

    Primary Logo

    View Full Article Hide Full Article
  5. Initiated PRAX-114 Phase 2 Acapella Study for adjunctive treatment of Major Depressive Disorder (MDD)

    Enrollment on track for 1H22 topline results in PRAX-114 Phase 2/3 Aria Study for monotherapy treatment of MDD

    PRAX-114 to advance in Phase 2b study in women with menopausal and mood symptoms

    PRAX-944 Phase 2b study for treatment of essential tremor to initiate in 3Q21; Phase 2a topline results expected by the end of 2021

    PRAX-562 Phase 1 healthy volunteer study completed with dose up to 150 mg and favorable safety profile

    Cash and investments of $339.2 million as of June 30, 2021 supports runway into 2Q23

    CAMBRIDGE, Mass., Aug. 16, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical…

    Initiated PRAX-114 Phase 2 Acapella Study for adjunctive treatment of Major Depressive Disorder (MDD)

    Enrollment on track for 1H22 topline results in PRAX-114 Phase 2/3 Aria Study for monotherapy treatment of MDD

    PRAX-114 to advance in Phase 2b study in women with menopausal and mood symptoms

    PRAX-944 Phase 2b study for treatment of essential tremor to initiate in 3Q21; Phase 2a topline results expected by the end of 2021

    PRAX-562 Phase 1 healthy volunteer study completed with dose up to 150 mg and favorable safety profile

    Cash and investments of $339.2 million as of June 30, 2021 supports runway into 2Q23

    CAMBRIDGE, Mass., Aug. 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 provided a corporate update and reported financial results for the second quarter ended June 30, 2021.

    "The second quarter marked another inflection point for Praxis as we make progress toward being a diversified, late-stage, CNS focused company," said Marcio Souza, president and chief executive officer of Praxis. "We expect to end the year with six controlled trials across our three clinical programs, including the ongoing registrational and Phase 2 studies in depression. As we know too well, the needs of people with CNS disorders are urgent and we are committed to bringing meaningful treatments to patients."

    Recent Business Highlights and Upcoming Milestones:

    Psychiatry

    • Praxis started dosing patients in the Acapella Study (Study 214), a PRAX-114 Phase 2 placebo-controlled study for adjunctive treatment of Major Depressive Disorder (MDD), in July 2021. Topline results from the Acapella Study are expected in the first half of 2022.

    • Praxis expects topline results from the ongoing PRAX-114 Phase 2/3 Aria Study for monotherapy treatment of MDD in the first half of 2022. The Aria Study is intended to serve as one of two trials required by the U.S. Food and Drug Administration (FDA) to demonstrate clinical efficacy to support registration of PRAX-114 for monotherapy treatment of MDD.

    • The Company reported topline results from the PRAX-114 Phase 2a Part B proof-of-concept trial for treatment of perimenopausal depression (PMD) and consolidated safety data across all three cohorts of the now completed Phase 2a study. Praxis intends to continue investigation of PRAX-114 for treatment of women with menopausal and mood symptoms in a Phase 2b trial. Plans for the Phase 2b trial will be disclosed by the end of 2021.



      • In Part B, participants treated with a single daily dose of PRAX-114 60 mg suspension formulation (n=6) for 14 days showed improvements in menopausal and mood symptoms that were rapid, marked and maintained throughout the two-week treatment period. Results trended toward baseline following discontinuation of PRAX-114, suggesting the need for continued treatment. PRAX-114 was well tolerated, with no change in the overall safety profile.

    • Praxis plans to initiate a PRAX-114 Phase 2 placebo-controlled study for treatment of post-traumatic stress disorder (PTSD) in the second half of 2021. Topline results are expected in the second half of 2022. The trial is designed to assess the efficacy of daily treatment of PRAX-114 in patients with PTSD for 4 weeks, using the CAPS-5 total score as the primary endpoint.

    Movement Disorders

    • The Company expects to initiate a PRAX-944 Phase 2b study for treatment of essential tremor (ET) in the US in the third quarter of 2021. Topline results are expected in the second half of 2022. The Phase 2b study is a randomized, double-blind, placebo-controlled, dose-range-finding clinical trial designed to assess the safety, tolerability and efficacy of PRAX-944 at three dose levels up to 120 mg per day.

    • Praxis is currently in the second of two cohorts of its PRAX-944 Phase 2a trial for treatment of ET, assessing patients titrated up to 120 mg/day of PRAX-944. Due to delays in recruitment in Australia, the company now expects preliminary topline open-label safety, tolerability and efficacy data by the end of 2021.

    • Praxis plans to initiate a PRAX-114 Phase 2 single-dose crossover study for treatment of ET to assess safety, pharmacokinetics (PK) and efficacy in the fourth quarter of 2021. Topline results are expected in the second half of 2022.

    • In the second quarter of 2021, the Company initiated a two-part PRAX-944 Phase 1 study to explore faster titration schemes for treatment of ET. Topline results are expected by the end of 2021. The Phase 1 study is intended to optimize the dosing regimen for PRAX-944 for treatment of ET and is designed to assess the safety, tolerability, and PK of titrating PRAX-944 up to 120 mg in a 10-day regimen in participants aged 18 to 54 years (Part A) and 55 to 75 years (Part B).

    Rare Disease

    • Praxis has completed the dosing and safety follow-up period for its single ascending dose (SAD) and multiple ascending dose (MAD) cohorts up to 150 mg and 120 mg, respectively, in its Phase 1 healthy volunteer study of PRAX-562. No dose-limiting toxicity has been observed. Dose-related changes were observed in the exploratory Auditory Steady-State Response (ASSR) electroencephalogram (EEG) translational marker, and exposures far exceeded those expected to be therapeutic in epilepsy-based animal models. Safety, tolerability, PK and preliminary biomarker data will be reported by the end of 2021. Based on the positive changes seen in the ASSR marker in this study, Praxis intends to initiate a dedicated PRAX-562 EEG study to validate the signal observed.

    • The Company expects to initiate an exploratory Phase 2 trial of PRAX-562 in the fourth quarter of 2021 for treatment of patients with rare adult cephalgia, including a cohort of participants with Short-lasting Unilateral Neuralgiform headache attacks with Conjunctival injection and Tearing (SUNCT) and Short-lasting Unilateral Neuralgiform headache with Autonomic symptoms (SUNA), and a cohort of participants with Trigeminal Neuralgia (TN).

    • Praxis plans to initiate a Phase 2 trial of PRAX-562 for treatment of developmental epileptic encephalopathies (DEEs), including SCN8A-DEE and SCN2A-DEE, in the first half of 2022.

    • Praxis plans to complete the ongoing Investigational New Drug (IND) enabling toxicology study by the end of 2021 for its lead antisense oligonucleotide (ASO) candidate, PRAX-222, and to initiate a Phase 1/2 trial of PRAX-222 for treatment of SCN2A-DEE in the first half of 2022.

    General Corporate Updates

    • In June 2021, Praxis entered into a collaboration with Ligand to discover and develop novel therapies for neurological disorders using the Icagen Ion Channel Technology platform.

    • In May 2021, Praxis completed a follow-on public offering of 5.75 million shares of common stock, including the exercise in full by the underwriters of their option to purchase up to 750,000 additional shares of common stock, at $18.25 per share, raising net proceeds of approximately $98.4 million after deducting underwriting discounts and commissions and other estimated offering expenses payable by Praxis.
    • In May 2021, Praxis announced the appointment of Tim Kelly as chief financial officer. Mr. Kelly brings more than 20 years of experience in the life sciences, pharmaceutical and biotechnology industries. Most recently, Mr. Kelly was the chief financial officer and head of corporate management at Foundation Medicine.

    Second Quarter 2021 Financial Results:

    As of June 30, 2021, Praxis had $339.2 million in cash, cash equivalents and marketable securities, compared to $296.6 million in cash and cash equivalents as of December 31, 2020. This increase of $42.6 million primarily reflects the addition of $98.4 million in net proceeds from the follow-on public offering of shares of our common stock, partially offset by cash used in operations of $55.7 million in the first half of the year. The company's cash, cash equivalents and marketable securities as of June 30, 2021 are expected to fund operations into the second quarter of 2023.

    Research and development expenses were $25.7 million for the three months ended June 30, 2021, compared to $9.1 million for the three months ended June 20, 2020. The increase in R&D expenses of $16.6 million was primarily attributable to $8.3 million in increased expenses related to our clinical-stage programs, $4.4 million in increased personnel-related costs due to increased headcount and $3.0 million in increased expenses related to our discovery-stage programs.

    General and administrative expenses were $10.8 million for the three months ended June 30, 2021, compared to $2.5 million for the three months ended June 30, 2020. The increase in general and administrative expenses of $8.3 million was primarily attributable to $4.6 million in increased personnel-related costs due to increased headcount, $2.2 million in increased professional fees and a $1.5 million increase in other general and administrative expenses.

    Praxis reported net loss of $36.4 million for the three months ended June 30, 2021, including $5.4 million of stock-based compensation expense, compared to $11.6 million for the three months ended June 30, 2020, including $0.3 million of stock-based compensation expense.

    As of June 30, 2021, Praxis had 44.7 million shares of common stock outstanding.

    About Praxis

    Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders (CNS) characterized by neuronal imbalance. Praxis is applying insights from genetic epilepsies to broader neurological and psychiatric disorders, using our understanding of shared biological targets and circuits in the brain. Praxis has established a broad portfolio, including multiple disclosed programs across CNS disorders including depression, epilepsy, movement disorders and pain syndromes, with three clinical-stage product candidates. For more information, please visit https://praxismedicines.com/ and follow us on LinkedIn and Twitter.

    Forward-Looking Statements

    This press release may contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis' future expectations, plans and prospects, including, without limitation, statements regarding expectations and plans for presenting pre-clinical and clinical data, projections regarding future revenues and financing performance, our long-term growth, cash, cash equivalents and marketable securities, the anticipated timing of our clinical trials and regulatory filings, the development of our product candidates and advancement of our preclinical programs, the timing, progress and success of our collaborations, as well as other statements containing the words "anticipate," "believe," "continue," "could," "endeavor," "estimate," "expect," "anticipate," "intend," "may," "might," "plan," "potential," "predict," "project," "seek," "should," "target," "will" or "would" and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.

    The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial; whether results from preclinical studies or earlier clinical studies will be predictive of the results of future trials; the expected timing of submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials or to market products; whether Praxis' cash resources will be sufficient to fund Praxis' foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, uncertainties and assumptions regarding the impact of the continuing COVID-19 pandemic on Praxis' business, operations, strategy, goals and anticipated timelines, Praxis' ongoing and planned preclinical activities, Praxis' ability to initiate, enroll, conduct or complete ongoing and planned clinical trials, Praxis' timelines for regulatory submissions and Praxis' financial position; and other risks concerning Praxis' programs and operations are described in additional detail in its Annual Report on Form 10-K for the year ended December 31, 2020, its Quarterly Reports on Form 10-Q and other subsequent filings made with the Securities and Exchange Commission from time to time. Although Praxis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.

    Investor Contact:

    Alex Kane

    Praxis Precision Medicines



    617-300-8481

    Media Contact:

    Ian Stone

    Canale Communications



    619-849-5388



    PRAXIS PRECISION MEDICINES, INC.

    CONDENSED CONSOLIDATED BALANCE SHEETS

    (Amounts in thousands)

    (Unaudited)

     June 30,



    2021
     December 31,



    2020
    Assets 
    Cash and cash equivalents$175,879   $296,608  
    Marketable securities163,305     
    Prepaid expenses and other current assets9,249   5,718  
    Property and equipment, net182   82  
    Operating lease right-of-use assets4,400   754  
    Other non-current assets496   15  
    Total assets$353,511   $303,177  
    Liabilities and stockholders' equity   
    Accounts payable$7,401   $4,088  
    Accrued expenses7,826   10,869  
    Operating lease liabilities4,512   763  
    Common stock5   4  
    Additional paid-in capital547,145   437,007  
    Accumulated other comprehensive loss(50)    
    Accumulated deficit(213,328)  (149,554) 
    Total liabilities and stockholders' equity$353,511   $303,177  

     

    PRAXIS PRECISION MEDICINES, INC.

    CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS 

    (Amounts in thousands, except share and per share amounts)

    (Unaudited)

     Three Months Ended



    June 30,
     Six Months Ended



    June 30,
     2021 2020 2021 2020
    Operating expenses:       
    Research and development$25,678   $9,050   $43,607   $15,918  
    General and administrative10,805   2,520   20,295   4,121  
    Total operating expenses36,483   11,570   63,902   20,039  
    Loss from operations(36,483)  (11,570)  (63,902)  (20,039) 
    Other income:       
    Other income, net82   5   128   133  
    Total other income82   5   128   133  
    Loss before benefit from income taxes(36,401)  (11,565)  (63,774)  (19,906) 
    Benefit from (provision for) income taxes   (3)     8  
    Net loss$(36,401)  $(11,568)  $(63,774)  $(19,898) 
    Accretion and cumulative dividends on redeemable convertible preferred stock   (2,039)     (4,103) 
    Gain on repurchase of redeemable convertible preferred stock         493  
    Net loss attributable to common stockholders$(36,401)  $(13,607)  $(63,774)  $(23,508) 
    Net loss per share attributable to common stockholders, basic and diluted$(0.88)  $(8.28)  $(1.59)  $(14.37) 
    Weighted average common shares outstanding, basic and diluted41,569,782   1,642,484   40,028,807   1,635,913  


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