Praxis Precision Medicines, Inc.

Previous wording

Our platforms utilize a deliberate, pragmatic and patient-guided approach, leveraging a suite of translational tools, including novel transgenic and predictive translational animal models and electrophysiology markers, to enable an efficient path to proof-of-concept in patients. Through this approach, we have established a diversified, multimodal CNS portfolio with four clinical-stage product candidates across movement disorders and epilepsy.

Current wording

Our platforms utilize a deliberate, pragmatic and patient-guided approach, leveraging a suite of translational tools, including novel transgenic and predictive translational animal models and electrophysiology markers, to enable an efficient path to proof-of-concept in patients. Through this approach, we have established a diversified, multimodal CNS portfolio with four late-stage product candidates across movement disorders and epilepsy.

Previous wording

For our ulixacaltamide program within the Cerebrum™ platform, we announced positive topline results for the two studies in the Phase 3 Essential3 program in essential tremor, or ET, in October 2025. We submitted a New Drug Application, or NDA, for ulixacaltamide HCl for the treatment of ET to the FDA, and it was accepted for review with a target action date under the Prescription Drug User Fee Act, or PDUFA, of January 29, 2027. In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing.

Current wording

For our ulixacaltamide program within the Cerebrum™ platform, we submitted a New Drug Application, or NDA, for ulixacaltamide HCl for the treatment of essential tremor, or ET, to the FDA based on the positive results of our two studies in the Phase 3 Essential3 program in ET, and it was accepted for review with a target action date under the Prescription Drug User Fee Act, or PDUFA, of January 29, 2027. The mid-cycle meeting was successfully completed and the FDA has identified no major safety or efficacy concerns to date. The agency confirmed that no advisory committee meeting is planned. In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing. In July 2026, we entered into a research collaboration and license agreement with Remagine Labs, Inc., a developer of electronically controlled transdermal drug delivery systems, to develop an iontophoretic transdermal patch for the delivery of ulixacaltamide.

Previous wording

For our relutrigine program within the Cerebrum™ platform, in December 2025, we announced positive results from the registrational cohort of the EMBOLD study in SCN2A and SCN8A developmental and epileptic encephalopathies, or DEEs, after receiving a recommendation from the Data Monitoring Committee to stop the study early for efficacy. We submitted an NDA for relutrigine for the treatment of SCN2A-DEE and SCN8A-DEE to the FDA, and it was accepted for priority review with a target PDUFA action date of September 27, 2026. In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing. The EMERALD study, which is evaluating relutrigine in patients with broad DEEs, has completed recruitment, with topline results anticipated in the fourth quarter of 2026. Assuming approval of the NDA for relutrigine, we believe the EMERALD study, if positive, could serve as the basis for a supplemental NDA, or sNDA, submission in 2027.

Current wording

For our relutrigine program within the Cerebrum™ platform, we submitted an NDA for relutrigine for the treatment of SCN2A and SCN8A developmental and epileptic encephalopathies, or DEEs, to the FDA based on the positive results from the registrational cohort of the EMBOLD study in SCN2A-DEE and SCN8A-DEE, and it was accepted for priority review with a target PDUFA action date of September 27, 2026. In June 2026, the FDA notified us that it had extended by three months the review period for our NDA for relutrigine, setting an updated PDUFA action date of December 27, 2026. The mid-cycle meeting was successfully completed and the FDA has identified no major safety or efficacy concerns to date. The agency confirmed that no advisory committee meeting is planned. In anticipation of potential approval, commercial preparations and pre-launch activities are ongoing. The EMERALD study, which is evaluating relutrigine in patients with broad DEEs, has completed recruitment, with topline results anticipated in the fourth quarter of 2026. Assuming approval of the NDA for relutrigine, we believe the EMERALD study, if positive, could serve as the basis for a supplemental NDA, or sNDA, submission in 2027.

Previous wording

For our vormatrigine program within the Cerebrum™ platform, our ENERGY program is ongoing, which consists of five studies to generate patient eligibility, efficacy, safety and pharmacokinetics, or PK, data in patients with focal onset seizures, or FOS, or generalized epilepsy. Studies under the ENERGY program include EMPOWER, RADIANT, POWER1, POWER2 and POWER3. In the second half of 2025, we announced positive results from our RADIANT Phase 2 open-label study evaluating the PK, safety and efficacy of vormatrigine in patients with FOS or generalized epilepsy. We initiated the EMPOWER study, an observational study of vormatrigine in patients with epilepsy, in the third quarter of 2024. For our POWER1 study, a double-blind, placebo-controlled, 12-week study in FOS, we expect to announce topline results in the second quarter of 2026. POWER2, our third efficacy study, is enrolling patients, with completion expected in the second half of 2026 and topline results expected in 2027. We intend to initiate POWER3, a clinical trial evaluating vormatrigine as a single-agent treatment for FOS, in the first half of 2026.

Current wording

For our vormatrigine program within the Cerebrum™ platform, our ENERGY program is ongoing, which consists of five studies to generate patient eligibility, efficacy, safety and pharmacokinetics, or PK, data in patients with focal onset seizures, or FOS, or generalized epilepsy. Studies under the ENERGY program include EMPOWER, RADIANT, POWER1, POWER2 and POWER3. In the second half of 2025, we announced positive results from our RADIANT Phase 2 open-label study evaluating the PK, safety and efficacy of vormatrigine in patients with FOS or generalized epilepsy. We initiated the EMPOWER study, an observational study of vormatrigine in patients with epilepsy, in the third quarter of 2024. In June 2026, we announced that the primary endpoint was not met in our POWER1 study, a double-blind, placebo-controlled, 12-week study in FOS. We paused enrollment in POWER2, our third efficacy study, to reassess the vormatrigine program and determine potential modifications. We intend to restart enrollment of the POWER2 study and initiate the POWER3 study, a clinical trial evaluating vormatrigine as a single-agent treatment for FOS, in the fourth quarter of 2026.

Previous wording

For our most advanced product candidate under the Solidus™ platform, elsunersen, we announced positive topline results from the EMBRAVE Part A study evaluating the safety and efficacy of elsunersen versus sham procedure in April 2026 for treating SCN2A gain-of-function DEE. We are currently enrolling EMBRAVE3, a Phase 3 registrational study for SCN2A gain-of-function DEE, and expect to announce topline results in 2027.

Current wording

For our most advanced product candidate under the Solidus™ platform, elsunersen, we announced positive topline results in April 2026 from the EMBRAVE Part A study evaluating the safety and efficacy of elsunersen versus sham procedure for treating SCN2A gain-of-function DEE. In June 2026, we received FDA breakthrough therapy designation for elsunersen for the treatment of seizures associated with SCN2A DEEs caused by gain-of-function variants. We are currently enrolling EMBRAVE3, a Phase 3 registrational study for early-seizure-onset SCN2A DEE, and expect to announce topline results in 2027.

Previous wording

We also have three earlier stage novel ASOs with preclinical proof of mechanism. PRAX-080 is focused on PCDH19 mosaic expression, PRAX-090 is targeting SYNGAP1 loss-of-function, or LoF, mutations which is a leading cause of severe intellectual disability and epilepsy in DEEs, and PRAX-100 is targeting SCN2A LoF mutations, the predominant genetic link to de novo autism spectrum disorders, or ASD. We anticipate nominating a development candidate for PRAX-080, PRAX-090 and PRAX-100 in the first half of 2026.

Current wording

We also have three earlier stage novel ASOs with preclinical proof of mechanism. PRAX-080 is focused on PCDH19 mosaic expression, PRAX-090 is targeting SYNGAP1 loss-of-function, or LoF, mutations which is a leading cause of severe intellectual disability and epilepsy in DEEs, and PRAX-100 is targeting SCN2A LoF mutations, the predominant genetic link to de novo autism spectrum disorders, or ASD. We anticipate nominating several development candidates in 2026.

Previous wording

We are a development stage company and we have not generated any revenue from product sales. All of our product candidates are in preclinical and clinical development or awaiting regulatory approval. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our product candidates, if approved. We have incurred recurring operating losses since inception, including a net loss of $92.6 million for the three months ended March 31, 2026. As of March 31, 2026, we had an accumulated deficit of $1.2 billion. We expect to incur significant expenses for the foreseeable future as we expand our research and development activities and prepare for commercialization of our product candidates. In addition, our losses from operations may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities. We anticipate that our expenses will be maintained or increased in connection with our ongoing activities, as we:

Current wording

We are a development stage company and we have not generated any revenue from product sales. All of our product candidates are in preclinical and clinical development or awaiting regulatory approval. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our product candidates, if approved. We have incurred recurring operating losses since inception, including a net loss of $176.3 million for the six months ended June 30, 2026. As of June 30, 2026, we had an accumulated deficit of $1.3 billion. We expect to incur significant expenses for the foreseeable future as we expand our research and development activities and prepare for commercialization of our product candidates. In addition, our losses from operations may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and our expenditures on other research and development activities. We anticipate that our expenses will be maintained or increased in connection with our ongoing activities, as we:

Previous wording

Since our inception, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or for our earned research and development tax credits due to our uncertainty of realizing a benefit from those items. Income taxes are determined at the applicable tax rates adjusted for non-deductible expenses, research and development tax credits and other permanent differences. Our income tax provision may be significantly affected by changes to our estimates. There was no income tax provision recognized for the three months ended March 31, 2026 and 2025.

Current wording

Since our inception, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each year or for our earned research and development tax credits due to our uncertainty of realizing a benefit from those items. Income taxes are determined at the applicable tax rates adjusted for non-deductible expenses, research and development tax credits and other permanent differences. Our income tax provision may be significantly affected by changes to our estimates. There was no income tax provision recognized for the three and six months ended June 30, 2026 and 2025.

Previous wording

The $17.2 million increase in research and development expenses was primarily attributable to the following:

Current wording

The $6.4 million increase in research and development expenses was primarily attributable to the following:

Previous wording

◦a $1.7 million increase in activities for our earlier stage assets as we advance our portfolio; partially offset by,

Current wording

◦a $4.2 million increase in activities for our earlier stage assets as we advance our portfolio.

Previous wording

The $14.0 million increase in general and administrative expenses was primarily attributable to the following:

Current wording

The $14.4 million increase in general and administrative expenses was primarily attributable to the following:

Previous wording

•$9.2 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Current wording

•$2.6 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Previous wording

◦a $6.9 million increase in spend for our relutrigine program, primarily related to our EMERALD trial activities and manufacturing spend to support commercialization;

Current wording

◦a $7.6 million increase in spend for our relutrigine program, primarily related to our EMERALD trial activities and manufacturing spend to support commercialization;

Previous wording

◦a $6.9 million increase in spend for our vormatrigine program, primarily driven by spend for our ENERGY program;

Current wording

◦a $2.1 million increase in spend for our vormatrigine program, primarily driven by spend for our ENERGY program, partially offset by,

Previous wording

◦a $6.3 million decrease in spend for our ulixacaltamide program, driven primarily by decreased activity in the Essential3 study, partially offset by increased manufacturing costs to support commercialization and the PDUFA fee paid in the first quarter of 2026.

Current wording

◦a $13.0 million decrease in spend for our ulixacaltamide program, driven primarily by decreased activity in the Essential3 study, partially offset by increased manufacturing costs to support commercialization and the PDUFA fee paid in the first quarter of 2026.

Previous wording

•$9.8 million increase in personnel-related costs mainly due to increased stock-based compensation expense and increased headcount; and

Current wording

•$15.5 million increase in personnel-related costs mainly due to increased stock-based compensation expense and increased headcount; and

Previous wording

•$3.5 million increase in professional expenses, primarily attributable to increased commercial spend as we prepare for the potential launch of ulixacaltamide and relutrigine.

Current wording

•$11.3 million increase in professional expenses, primarily attributable to increased commercial spend as we prepare for the potential launches of ulixacaltamide and relutrigine.

Previous wording

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with Jefferies LLC, or Jefferies, to provide for the offering, issuance, and sale of up to an aggregate amount of $150.0 million of common stock in at-the-market offerings. In December 2024, we entered into an amendment to the 2024 Sales Agreement with Jefferies to provide for the offering, issuance and sale of up to an aggregate amount of $250.0 million of common stock from time to time in at-the-market offerings. During the three months ended March 31, 2025, we issued and sold an aggregate of 694,212 shares under the amended 2024 Sales Agreement for aggregate net proceeds of $54.9 million, after deducting commissions and offering expenses payable by us. The amended 2024 Sales Agreement was terminated in September 2025.

Current wording

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with Jefferies LLC, or Jefferies, to provide for the offering, issuance, and sale of up to an aggregate amount of $150.0 million of common stock in at-the-market offerings. In December 2024, we entered into an amendment to the 2024 Sales Agreement with Jefferies to provide for the offering, issuance and sale of up to an aggregate amount of $250.0 million of common stock from time to time in at-the-market offerings. During the six months ended June 30, 2025, we issued and sold an aggregate of 1,351,689 shares under the amended 2024 Sales Agreement for aggregate net proceeds of $83.3 million, after deducting commissions and offering expenses payable by us. The amended 2024 Sales Agreement was terminated in September 2025.

Previous wording

In September 2025, we entered into a Sales Agreement, or the 2025 Sales Agreement, with TD Securities (USA) LLC, or TD Cowen, to provide for the offering, issuance and sale of up to an aggregate of $250.0 million of common stock from time to time in at-the-market offerings. During the three months ended March 31, 2026, we did not issue or sell any shares of common stock under the 2025 Sales Agreement.

Current wording

In September 2025, we entered into a Sales Agreement, or the 2025 Sales Agreement, with TD Securities (USA) LLC, or TD Cowen, to provide for the offering, issuance and sale of up to an aggregate of $250.0 million of common stock from time to time in at-the-market offerings. During the six months ended June 30, 2026, we did not issue or sell any shares of common stock under the 2025 Sales Agreement.

Previous wording

During the three months ended March 31, 2026, net cash used in operating activities of $86.2 million was primarily due to our $92.6 million net loss and $10.3 million in changes in operating assets and liabilities primarily related to a decrease in accrued expenses, partially offset by $16.7 million of non-cash charges primarily related to stock-based compensation.

Current wording

During the six months ended June 30, 2026, net cash used in operating activities of $163.7 million was primarily due to our $176.3 million net loss and $15.1 million in changes in operating assets and liabilities primarily related to a decrease in accrued expenses, partially offset by $27.7 million of non-cash charges primarily related to stock-based compensation.

Previous wording

During the three months ended March 31, 2025, net cash used in operating activities of $53.0 million was primarily due to our $69.3 million net loss, partially offset by $8.4 million in changes in operating assets and liabilities primarily related to an increase in accounts payable and $7.9 million of non-cash charges primarily related to stock-based compensation.

Current wording

During the six months ended June 30, 2025, net cash used in operating activities of $107.7 million was primarily due to our $140.4 million net loss, partially offset by $17.8 million in changes in operating assets and liabilities primarily related to an increase in accounts payable and $14.9 million of non-cash charges primarily related to stock-based compensation.

Previous wording

During the three months ended March 31, 2026, net cash used in investing activities of $344.3 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

Current wording

During the six months ended June 30, 2026, net cash used in investing activities of $333.3 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

Previous wording

During the three months ended March 31, 2025, net cash used in investing activities of $51.1 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

Current wording

During the six months ended June 30, 2025, net cash used in investing activities of $33.2 million was primarily related to purchases of marketable securities offset by maturities of marketable securities.

Previous wording

During the three months ended March 31, 2026, net cash provided by financing activities of $609.4 million consisted primarily of net proceeds from our January 2026 follow-on offering.

Current wording

During the six months ended June 30, 2026, net cash provided by financing activities of $614.0 million consisted primarily of net proceeds from our January 2026 follow-on offering.

Previous wording

During the three months ended March 31, 2025, net cash provided by financing activities of $54.3 million consisted primarily of net proceeds from our at-the-market offerings.

Current wording

During the six months ended June 30, 2025, net cash provided by financing activities of $82.9 million consisted primarily of net proceeds from our at-the-market offerings.