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. For our most advanced product candidate under the Cerebrum™ platform, ulixacaltamide (formerly known as PRAX-944), our Phase 3 Essential3 clinical trials in essential tremor, or ET, are ongoing. We are planning to conduct an interim analysis in the fourth quarter of 2024, with topline results anticipated shortly thereafter. Within our PRAX-628 program, we announced positive results from our Photo-Paroxysmal Response ("PPR") study in the first quarter of 2024, and plan to initiate four studies to generate patient eligibility, efficacy, safety and pharmacokinetics (PK) data for the program. We plan to initiate an observational study of PRAX-628 in patients with epilepsy in the third quarter of 2024, as well as three efficacy studies. The first efficacy study will be an open label eight-week study in patients with focal onset seizures or generalized epilepsy to be initiated in the second half of 2024, with topline results expected in the first half of 2025. We also plan to initiate a double-blind, placebo-controlled, 12-week study in focal onset seizures in the fourth quarter of 2024, with topline results expected in the second half of 2025, and plan to initiate a third efficacy study in the first half of 2025. Our relutrigine (formerly known as PRAX-562) Phase 2 EMBOLD study was initiated in the first quarter of 2023, with two cohorts in SCN2A-DEE and SCN8A-DEE; topline results are expected for both cohorts in the third quarter of 2024. For our most advanced product candidate under the Solidus™ platform, elsunersen (formerly known as PRAX-222), we shared results from Part 1 of the EMBRAVE study in the fourth quarter of 2023, and have initiated the first arm of our global confirmatory study in Brazil. We are currently completing multiple global regulatory interactions to further expand the pivotal phase of the program later in 2024.

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 clinical-stage product candidates across movement disorders and epilepsy. For our most advanced product candidate under the Cerebrum™ platform, ulixacaltamide, our Phase 3 Essential3 clinical trials in essential tremor, or ET, are ongoing. We are planning to conduct an interim analysis in the first quarter of 2025. Within our vormatrigine program (formerly known as PRAX-628), we announced positive results from our Photo-Paroxysmal Response ("PPR") study in the first quarter of 2024, and have initiated or plan to initiate four studies to generate patient eligibility, efficacy, safety and pharmacokinetics (PK) data for the program. We initiated an observational study of vormatrigine in patients with epilepsy in the third quarter of 2024, and iniatiated or plan to initiate three efficacy studies. The first efficacy study will be an open label eight-week study in patients with focal onset seizures or generalized epilepsy to be initiated in the fourth quarter of 2024, with topline results expected in the first half of 2025. We have also initiated a double-blind, placebo-controlled, 12-week study in focal onset seizures in the fourth quarter of 2024, with topline results expected in the second half of 2025, and plan to initiate a third efficacy study in the first half of 2025. Within our relutrigine program (formerly known as PRAX-562), we announced positive topline results from the first cohort of our EMBOLD study in the third quarter of 2024, and have initiated enrollment of the second cohort, with topline results expected in the first half of 2026. For our most advanced product candidate under the Solidus™ platform, elsunersen (formerly known as PRAX-222), we shared results from Part 1 of the EMBRAVE study in the fourth quarter of 2023, and have initiated the first arm of our global confirmatory study in Brazil. We are currently completing multiple global regulatory interactions to further expand the pivotal phase of the program later in 2024.

Previous wording

We are a development stage company and we have not generated any revenue from product sales, and do not expect to do so for several years, if at all. All of our product candidates are still in preclinical and clinical development. 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 $72.2 million for the six months ended June 30, 2024. As of June 30, 2024, we had an accumulated deficit of $726.2 million. We expect to incur significant expenses and operating losses for the foreseeable future as we expand our research and development activities. 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, and do not expect to do so for several years, if at all. All of our product candidates are still in preclinical and clinical development. 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 $124.1 million for the nine months ended September 30, 2024. As of September 30, 2024, we had an accumulated deficit of $778.1 million. We expect to incur significant expenses and operating losses for the foreseeable future as we expand our research and development activities. 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

•advance our PRAX-628 product candidate into efficacy clinical trials for focal onset seizures;

Current wording

•advance vormatrigine (formerly PRAX-628) into efficacy clinical trials for focal onset seizures or generalized epilepsy;

Previous wording

As of June 30, 2024, we had cash, cash equivalents and marketable securities of $433.8 million. We expect that our cash, cash equivalents and marketable securities as of June 30, 2024 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our research efforts and clinical trials into 2027. The analysis included consideration of our current financial needs and ongoing research and development plans. We have based this estimate on assumptions that may provide to be wrong, and we could exhaust our available capital resources sooner than we expect. See “—Liquidity and Capital Resources.”

Current wording

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $411.2 million. We expect that our cash, cash equivalents and marketable securities as of September 30, 2024 will be sufficient to fund our operating expenditures and capital expenditure requirements necessary to advance our research efforts and clinical trials into 2027. The analysis included consideration of our current financial needs and ongoing research and development plans. We have based this estimate on assumptions that may provide to be wrong, and we could exhaust our available capital resources sooner than we expect. See “—Liquidity and Capital Resources.”

Previous wording

We have not generated any revenue from the sale of products since inception and do not expect to generate any revenue from the sale of products for several years, if at all. As discussed in Note 7 to our condensed consolidated financial statements, we entered into an Option and License Agreement, or the Collaboration Agreement, with UCB Biopharma SRL, or UCB, in December 2022. We recognized $0.4 million and $0.8 million, respectively, of collaboration revenue from the Collaboration Agreement during the three and six months ended June 30, 2024, compared to $0.8 million and $1.5 million, respectively, during the three and six months ended June 30, 2023.

Current wording

We have not generated any revenue from the sale of products since inception and do not expect to generate any revenue from the sale of products for several years, if at all. As discussed in Note 7 to our condensed consolidated financial statements, we entered into an Option and License Agreement, or the Collaboration Agreement, with UCB Biopharma SRL, or UCB, in December 2022. We recognized $0.3 million and $1.1 million, respectively, of collaboration revenue from the Collaboration Agreement during the three and nine months ended September 30, 2024, compared to $0.5 million and $1.9 million, respectively, during the three and nine months ended September 30, 2023.

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 and six months ended June 30, 2024 and 2023.

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 nine months ended September 30, 2024 and 2023.

Previous wording

The $0.4 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022. Changes in revenue recognized each period reflect timing of research services provided and costs incurred.

Current wording

The $0.2 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022. Changes in revenue recognized each period reflect timing of research services provided and costs incurred.

Previous wording

The $1.6 million increase in research and development expenses was primarily attributable to the following:

Current wording

The $24.6 million increase in research and development expenses was primarily attributable to the following:

Previous wording

•$8.1 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Current wording

•$21.6 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Previous wording

◦a $8.6 million increase in spend for our ulixacaltamide program due to Essential3 study activity and Phase 1 trial spend, partially offset by prior year spend for our Essential1 study;

Current wording

◦a $15.7 million increase in spend for our ulixacaltamide program, primarily due to Essential3 study spend, as well as Phase 1 trial spend, partially offset by completion of our Essential1 study in the prior year;

Previous wording

◦a $0.4 million increase in spend for our PRAX-562 program related to our EMBOLD Phase 2 clinical trial;

Current wording

◦a $0.5 million increase in spend for our relutrigine program, primarily related to the EMBOLD Phase 2 clinical trial.

Previous wording

•$4.5 million increase in personnel-related costs, including stock-based compensation expense; and

Current wording

•$4.0 million increase in personnel-related costs, including stock-based compensation expense, due to increased headcount;

Previous wording

The $2.5 million increase in general and administrative expenses was primarily attributable to the following:

Current wording

The $6.5 million increase in general and administrative expenses was primarily attributable to the following:

Previous wording

•$3.7 million increase in personnel-related costs mainly due to increased stock-based compensation expense; and

Current wording

•$4.6 million increase in personnel-related costs, mainly due to increased stock-based compensation expense;

Previous wording

•$1.1 million decrease in professional expenses mainly due to decreased consulting and legal expense.

Current wording

•$1.4 million increase in professional expenses mainly due to increased legal, audit and other consulting expenses; and

Previous wording

•$0.4 million decrease in indirect expenses, none of which were individually significant.

Current wording

•$0.5 million increase in other expenses, none of which were individually significant.

Previous wording

Other income for the three months ended June 30, 2024 and 2023 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

Current wording

Other income for the three months ended September 30, 2024 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization. Other income for the three months ended September 30, 2023 was compromised of interest income on our cash and cash equivalents.

Previous wording

The $0.7 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022. Changes in revenue recognized each period reflect timing of research services provided and costs incurred.

Current wording

The $0.8 million decrease in collaboration revenue is associated with a decrease in the revenue recorded under the Collaboration Agreement with UCB that was executed in December 2022. Changes in revenue recognized each period reflect timing of research services provided and costs incurred.

Previous wording

The $3.1 million increase in research and development expenses was primarily attributable to the following:

Current wording

The $27.7 million increase in research and development expenses was primarily attributable to the following:

Previous wording

•$9.7 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Current wording

•$31.4 million increase in expense related to our Cerebrum™ platform, driven primarily by:

Previous wording

◦a $13.7 million increase in spend for our ulixacaltamide program, primarily due to clinical-related activities for our Phase 3 program in ulixacaltamide, partially offset by prior year Essential1 study activity;

Current wording

◦a $29.5 million increase in spend for our ulixacaltamide program, primarily due to clinical-related activities for our Phase 3 program in ulixacaltamide and Phase 1 trial spend, partially offset by prior year Essential1 study activity;

Previous wording

◦a $0.4 million decrease in activities for our earlier stage assets.

Current wording

◦a $1.6 million decrease in activities for our earlier stage assets.

Previous wording

•$8.2 million decrease in expense within our Solidus™ platform for elsunersen, driven by a prior year milestone payment of $6.9 million due to Ionis Pharmaceuticals Inc., or Ionis, upon initiation of our EMBRAVE study, as well as prior year EMBRAVE study activity and toxicology-related spend; and

Current wording

•$12.1 million decrease in expense related to our Solidus™ platform, driven by a prior year $6.9 million milestone payment due to Ionis Pharmaceuticals Inc., or Ionis, upon initiation of our elsunersen EMBRAVE study, as well as prior year study activity; and

Previous wording

The $0.5 million increase in general and administrative expenses is due primarily to increased personnel-related costs.

Current wording

The $9.1 million increase in general and administrative expenses was primarily attributable to the following:

Previous wording

Other income for the three months ended June 30, 2024 and 2023 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

Current wording

Other income for the nine months ended September 30, 2024 and 2023 was comprised of interest income on our cash, cash equivalents, and marketable securities and investment premium and discount amortization.

Previous wording

To date, we have financed our operations primarily with proceeds from the issuance of redeemable convertible preferred stock and from the sale of common stock through an initial public offering, common stock and pre-funded warrants through follow-on public offerings and common stock from at-the-market offerings under our shelf registration statement. From inception through June 30, 2024, we have raised $1.0 billion in aggregate cash proceeds from such transactions, net of issuance costs. As of June 30, 2024, we had cash, cash equivalents and marketable securities of $433.8 million.

Current wording

To date, we have financed our operations primarily with proceeds from the issuance of redeemable convertible preferred stock and from the sale of common stock through an initial public offering, common stock and pre-funded warrants through follow-on public offerings and common stock from at-the-market offerings under our shelf registration statement. From inception through September 30, 2024, we have raised $1.0 billion in aggregate cash proceeds from such transactions, net of issuance costs. As of September 30, 2024, we had cash, cash equivalents and marketable securities of $411.2 million.

Previous wording

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with 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. During the six months ended June 30, 2024, we issued and sold an aggregate of 17,662 shares under the 2024 Sales Agreement for aggregate net proceeds of $0.9 million, after deducting commissions and offering expenses payable by us. We did not make any sales under the 2024 Sales Agreement during the three months ended June 30, 2024.

Current wording

In March 2024, we entered into an Open Market Sale Agreement, or the 2024 Sales Agreement, with 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. During the nine months ended September 30, 2024, we issued and sold an aggregate of 42,851 shares under the 2024 Sales Agreement for aggregate net proceeds of $2.3 million, after deducting commissions and offering expenses payable by us.

Previous wording

During the six months ended June 30, 2024, net cash used in operating activities of $48.1 million was primarily due to our $72.2 million net loss, partially offset by $2.2 million in changes in operating assets and liabilities primarily related to increases in accounts payable and accrued expenses, and $21.9 million of non-cash charges primarily related to stock-based compensation.

Current wording

During the nine months ended September 30, 2024, net cash used in operating activities of $75.6 million was primarily due to our $124.1 million net loss, partially offset by $15.8 million in changes in operating assets and liabilities primarily related to increases in accounts payable and accrued expenses, and $32.7 million of non-cash charges primarily related to stock-based compensation.

Previous wording

During the six months ended June 30, 2023, net cash used in operating activities of $64.1 million was primarily due to our $71.8 million net loss and $6.3 million in changes in operating assets and liabilities primarily related to decreases in accrued expenses and accounts payable, partially offset by $14.0 million of non-cash charges primarily related to stock-based compensation.

Current wording

During the nine months ended September 30, 2023, net cash used in operating activities of $87.3 million was primarily due to our $96.4 million net loss and $11.0 million in changes in operating assets and liabilities primarily related to decreases in accrued expenses and accounts payable, partially offset by $20.1 million of non-cash charges primarily related to stock-based compensation.

Previous wording

During the six months ended June 30, 2024, net cash used in investing activities of $287.1 million was primarily related to purchases of marketable securities partially offset by maturities of marketable securities.

Current wording

During the nine months ended September 30, 2024, net cash used in investing activities of $237.6 million was primarily related to purchases of marketable securities partially offset by maturities of marketable securities.

Previous wording

During the six months ended June 30, 2023, net cash provided by investing activities of $39.0 million was related to maturities of marketable securities.

Current wording

During the nine months ended September 30, 2023, net cash provided by investing activities of $39.0 million was related to maturities of marketable securities.

Previous wording

During the six months ended June 30, 2024, net cash provided by financing activities of $399.1 million consisted primarily of net proceeds from our January 2024 and April 2024 follow-on public offerings, our at-the-market offerings and our collaboration and license agreement with Tenacia.

Current wording

During the nine months ended September 30, 2024, net cash provided by financing activities of $400.6 million consisted primarily of net proceeds from our January 2024 and April 2024 follow-on public offerings, our at-the-market offerings and our collaboration and license agreement with Tenacia.

Previous wording

During the six months ended June 30, 2023, net cash provided by financing activities of $87.7 million consisted primarily of net proceeds from at-the-market offerings and our June 2023 follow-on public offering.

Current wording

During the nine months ended September 30, 2023, net cash provided by financing activities of $87.7 million consisted primarily of net proceeds from at-the-market offerings and our June 2023 follow-on public offering.