Camp4 Therapeutics Corp
For the period ended Mar 31, 2025. Filed May 13, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2025 · Filed May 13, 2025
CAMP$4.06+9.1%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-21.
0001410578-24-002045
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-13.
0001736730-25-000039
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 109 previous paragraphs and 66 current paragraphs.
20 unchanged; 15 reworded; 30 added; 72 removed; 2 previous and 1 current paragraphs rearranged.
Tables are not compared: 48 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (30)
Current report
Overview
Current report
Based on our preclinical studies, we believe our clinical product candidate, CMP-CPS-001, has the potential to be the first disease-modifying therapy for the treatment of the most prevalent urea cycle disorders (“UCDs”). UCDs are a group of severe, inherited metabolic diseases caused by mutations in the genes that encode one or more of the eight enzymes and transporters necessary to convert ammonia into urea. The inability of the body to properly metabolize ammonia leads to the accumulation of toxic levels in circulation, ultimately resulting in severe health outcomes, such as neurologic disability, seizure and death. CMP-CPS-001 is designed to improve urea cycle activity by amplifying expression of carbamoyl phosphate synthetase 1 (“CPS1”), an enzyme that catalyzes the first step of the urea cycle, by binding to a CPS1-specific regRNA. Our preclinical studies have demonstrated that modulating the activity of the target regRNA increases expression of the CPS1 gene, resulting in increased CPS1 enzyme levels, which allows for more ammonia to be converted into urea, thereby lowering ammonia levels to normal, healthy ranges. These preclinical studies also demonstrated that CMP-CPS-001 can increase the level of, or upregulate, the production of multiple enzymes responsible for converting ammonia into urea, potentially allowing us to address more than 85% of patients with UCDs. We are evaluating CMP-CPS-001 in an ongoing Phase 1 clinical trial in healthy volunteers and have completed a planned interim analysis of safety and pharmacokinetic data from all four cohorts of the single ascending dose (“SAD”) portion of the clinical trial, from which no safety trends of concern were observed. We have also completed dosing of the first three cohorts from the multiple ascending dose (“MAD”) portion of the clinical trial. We expect to report data from the SAD and MAD portions of the clinical trial, including safety, pharmacokinetic and key pharmacodynamic biomarker data, in the fourth quarter of 2025. We have also submitted a clinical trial application in Europe for a Phase 1b expansion to enroll female participants who are heterozygous for a mutation of the OTC gene, pending regulatory clearance. We estimate that there are approximately 2,000 diagnosed female OTC heterozygotes in the United States who have inherited one copy of a gene with OTC-related changes and experience potentially addressable UCD symptoms.
Current report
We are also leveraging our RAP Platform to advance a preclinical program for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorders, a group of neurodevelopmental conditions caused by pathogenic variants in the SYNGAP1 gene that result in a haploinsufficient state, reducing SYNGAP protein levels by up to 50%. SYNGAP plays a critical role in cognitive development and synaptic function. Epilepsy is a common characteristic of these disorders and nearly all patients experience some degree of developmental delay and cognitive impairment. Incidence estimates vary significantly, ranging from one to 40 per 100,000 individuals. While we believe that SYNGAP1-related disorders remain underdiagnosed, we estimate that there are approximately 10,000 individuals living with these disorders in the United States. SYNGAP1-related disorders are reported to represent 0.5% to 1.0% of all intellectual disability cases, making them among the most common causes of intellectual disability in patients with epilepsy, and indicating that the patient population may be significantly larger than incidence estimates suggest. There are no FDA-approved, disease-modifying therapies for SYNGAP1-related disorders. Treatment is often limited to supportive physical, occupational and speech therapy. A combination of non-specific anti-seizure medications may be prescribed, though SYNGAP1-related disorders have proven difficult to control with available therapeutics. Up to 50% of patients do not adequately respond to medication, in which case implantable devices, such as those for vagus nerve stimulation, may offer incremental therapeutic benefit. We are advancing our CMP-SYNGAP program to address the significant unmet need by targeting the direct cause of SYNGAP1-related disorders, haploinsufficiency, which we believe is amenable to targeting through regRNAs. Our CMP-SYNGAP program is a novel approach that targets the SYNGAP1 gene at the transcriptional level to restore SYNGAP function and improve symptoms by utilizing an intrathecally delivered ASO. Upregulation of SYNGAP1 gene expression may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit. Our preclinical studies demonstrated a dose-dependent increase in SYNGAP1 mRNA levels accompanied by a reduction in SYNGAP1 expression. We have nominated a development candidate, CMP-SYNGAP-01, and expect to initiate GLP toxicology studies in 2025 to enable the filing of a clinical trial application. We also plan to present new preclinical data in an oral presentation at the 28th American Society of Gene and Cell Therapy Annual Meeting on May 16, 2025, demonstrating a meaningful increase in SYNGAP1 protein levels following intrathecal administration of CMP-SYNGAP-01 in non-human primates.
Current report
In addition, we have initiated a discovery program targeting a regRNA to increase GBA1 gene expression for the treatment of Parkinson’s disease (“PD”) caused by mutations in the GBA1 gene, and plan to initiate discovery programs in other CNS and metabolic indications utilizing our RAP Platform. Finally, we have entered into a strategic research collaboration with BioMarin Pharmaceutical Inc. (“BioMarin”) to advance novel therapeutics that increase protein levels by targeting regRNA sequences for two genetic targets.
Current report
•advance our lead product candidate, CMP-CPS-001, through clinical trials;
Current report
•finalize preclinical development for our program in SYNGAP1-related disorders;
Current report
•conduct preclinical studies and clinical trials for our GBA1 discovery program for the treatment of PD and any future product candidates we may develop;
Current report
•expand the capabilities of our RAP Platform and seek to identify and develop additional product candidates;
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•seek marketing approvals for any product candidates that successfully complete clinical trials;
Current report
•obtain, expand, maintain, defend and enforce our intellectual property portfolio;
Current report
•hire additional clinical, regulatory and scientific personnel;
Current report
•contract with third-party manufacturers for preclinical and clinical drug supply supporting any future product candidates we may develop, and commercial supply with respect to any such product candidates that receive regulatory approval;
Current report
•ultimately establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval; and
Current report
•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts, as well as to support our operations as a public company.
Current report
Research and Development Expenses
Current report
R&D expenses were $10.1 million for the three months ended March 31, 2025 compared to $9.7 million for the three months ended March 31, 2024. The increase of $0.4 million was primarily due to an increase of $0.1 million in clinical and preclinical expenses due to increases associated with the Company’s UCD clinical trial. In addition, there was an increase of $0.1 million in personnel-related expenses due to lower offsetting personnel reimbursements under one of our collaboration agreements. There was also an increase of $0.1 million in professional and consulting fees due to increased utilization of external support for clinical operations.
Current report
General and Administrative Expenses
Current report
The following table summarizes our G&A expenses for the three months ended March 31, 2025 and 2024 (in thousands):
Current report
G&A expenses were $3.8 million for the three months ended March 31, 2025 compared to $3.1 million for the three months ended March 31, 2024. The increase of $0.7 million was partially due to an increase of $0.4 million in personnel-related expenses due to annual merit-related salary increases and stock compensation expense and an increase of $0.1 million in facility-related and overhead expense due to increases in insurance premiums. In addition, there was an increase of $0.2 million in other expenses due to an increase in state franchise taxes.
Current report
Other Income, Net
Current report
As of March 31, 2025, we had cash and cash equivalents of $49.3 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of March 31, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our capital resources may not be sufficient to fund operations through at least the next twelve months from the date the accompanying unaudited condensed consolidated financial statements as of March 31, 2025 are issued based on our expected cash needs, which raises substantial doubt about our ability to continue as a going concern. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.
Current report
Contractual and Other Obligations
Current report
As of March 31, 2025, other than those disclosed within Note 4 and 5 to our condensed consolidated financial statements, there have been no material changes to our contractual obligations and commitments from those described under “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our 2024 Form 10-K.
Current report
Cash flows used in operations are primarily attributed to clinical and preclinical expenses, employee compensation, rent and variable rent expenses for our Cambridge and Boulder leases as well as professional fees. Our cash flows from operating activities are significantly affected by our use of cash for operating expenses and working capital to support the business.
Current report
Net cash used in operating activities for the three months ended March 31, 2025 was $14.3 million and consisted of net loss of $12.4 million, non-cash adjustments of $1.9 million and a net change in assets and liabilities of $3.7 million. Non-cash items primarily included stock-based compensation of $0.9 million, non-cash operating lease expense of $0.5 million, and depreciation and amortization of $0.4 million. The net change in assets and liabilities was primarily due to a decrease in accrued expenses due to the timing of payout related to annual employee bonuses and cash paid to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes from 2024.
Current report
Net cash used in operating activities increased by $3.1 million during the three months ended March 31, 2025, compared to the same period in 2024, primarily attributable to the timing of payout related to annual employee bonuses and cash paid to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes from 2024.
Current report
Our primary investing activities consist of capital expenditures for laboratory and computer equipment as well as software. Net cash used in investing activities for the three months ended March 31, 2025 was $0.3 million, due to purchases of laboratory equipment. Net cash used in investing activities increased by $0.3 million during the three months ended March 31, 2025, compared to the same period in 2024, primarily attributable to the timing of cash payments for laboratory equipment.
Current report
Our primary financing activities consist of principal payments on finance leases. Net cash used in financing activities for the three months ended March 31, 2025 was $0.2 primarily due to principal payments on finance leases. Net cash used in financing activities decreased by $0.1 million during the three months ended March 31, 2025, compared to the same period in 2024, primarily attributable to our completion and conversion of certain finance leases.
Current report
There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the three months ended March 31, 2025 compared to those disclosed in our 2024 Form 10-K.
Current report
See Note 2 to our condensed consolidated financial statements included elsewhere in this Quarterly Report for more information.
Removed paragraphs (72)
Previous report
Collaboration and License Agreements
Previous report
Below is a summary of the key terms and financial statement impact of certain of our license and collaboration agreements:
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In-License Agreements
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Whitehead Institute for Biomedical Research
Previous report
In October 2019, we entered into a patent license agreement with the Whitehead Institute for Biomedical Research (the “Whitehead Institute”), which was subsequently amended on December 14, 2021 (the “Whitehead First Amendment”), and on November 7, 2023 (the “Whitehead Second Amendment”). Under the agreement, we were granted a worldwide, royalty-bearing, sublicensable license under certain patent rights owned or controlled by the Whitehead Institute. As part of the agreement, we paid an initial $0.1 million license issuance fee, and de minimis additional fees in connection with each of the Whitehead First Amendment and Whitehead Second Amendment that were recorded as research and development expense in our condensed consolidated statement of operations and comprehensive loss. We are also obligated to pay annual license maintenance fees for the term of the agreement, pursuant to which we have paid an aggregate of $0.20 million through September 30, 2024. In addition, we are obligated to pay certain filing, prosecution and maintenance fees with respect to certain patent rights licensed to us under the agreement, pursuant to which we have paid an aggregate of $0.27 million through September 30, 2024. We are obligated to pay potential development milestone payments to the Whitehead Institute of up to an aggregate of $1.9 million under the terms of the agreement upon the achievement of certain specified contingent events. In addition, if we successfully commercialize a product under the agreement, we are also obligated to pay tiered royalties at percentage rates ranging from less than one percent to the mid-single digits of net sales or of running royalties of net sales, subject to specified reductions, until either the last-to-expire valid claim of a Whitehead Institute patent covering the product or seven years after the first commercial sale, in each case on a product-by-product and country-by-country basis. We incurred de minimis amounts of license maintenance and amendment issuance fees during each of the three and nine months ended September 30, 2024 and 2023 under the amended agreement and recorded the amounts in our research and development expense in our condensed consolidated statements of operations and comprehensive loss.
Previous report
Children’s Medical Center Corporation
Previous report
In April 2018, we entered into a development and license agreement (the “Children’s Medical Center Corporation Agreement”) with Children’s Medical Center Corporation (“CMCC”). The agreement allows us to use CMCC’s proprietary intellectual property to conduct research, development and commercialization of products utilizing CMCC’s proprietary intellectual property in return for specified payments. The proprietary intellectual property licensed pursuant to this agreement is related to certain legacy programs we are not pursuing and which were subsequently sublicensed to Fulcrum Therapeutics, Inc. (“Fulcrum”), as described below. As part of the agreement, we issued a total of 15,123 shares of common stock to CMCC and its affiliates based on the fair value of the common stock on the date of issuance.
Previous report
We are obligated to pay potential development milestone payments under the terms of the Children’s Medical Center Corporation Agreement of up to $7.7 million for the first licensed target, $3.9 million for the second licensed target and $1.9 million for the third licensed target upon the achievement of certain specified contingent events. If commercial sales of a licensed product commence, we will pay CMCC royalties at percentage rates ranging in the low- to mid-single digits on net sales of licensed products in countries where such product is protected by patent rights. We incurred less than $0.1 million of royalties owed to CMCC under the agreement during each of the three and nine months ended September 30, 2024 and 2023, and recorded the amounts in research and development (“R&D”) expense in the condensed consolidated statements of operations and comprehensive loss. We re-evaluate the likelihood of achieving future milestones at the end of each reporting period. As of September 30, 2024, we determined that the likelihood of achieving future milestones was not probable.
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Out-License Agreements
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Fulcrum Therapeutics, Inc.
Previous report
In July 2023, we entered into a license agreement with Fulcrum. Under this license agreement, we granted an exclusive license related to our related intellectual property and granted a non-exclusive sublicense for the intellectual property obtained through the Children’s Medical Center Corporation Agreement. In exchange for the license rights, Fulcrum paid us a $0.35 million upfront payment. In the event that Fulcrum achieves development and commercial milestones, Fulcrum will be obligated to pay us one-time milestone payments ranging from $1.0 million to $20.0 million (with respect to a Tier 1 Product, as defined in the agreement) or $0.6 million to $12.0 million (with respect to a Tier 2 Product, as defined in the agreement), depending on the milestone achieved. In addition, this license agreement includes both potential nominal minimum annual royalty payments as well as sales-based royalties upon commercialization of up to the low-double digits.
Previous report
During each of the three and nine months ended September 30, 2024, we did not record any license revenue pursuant to the Fulcrum Agreement. During the three and nine months ended September 30, 2023, we recorded $0.35 million of license revenue pursuant to the Fulcrum Agreement.
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Collaborative Arrangement
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Eli Lilly and Company
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In July 2023, we executed a Material Transfer Agreement (“MTA”) with Eli Lilly and Company (“Eli Lilly”), which was subsequently amended in July 2024. As part of the MTA, we and Eli Lilly agreed to perform research and development activities to generate up to three ASOs in accordance with a prescribed workplan. For the nine months ended September 30, 2024, we received $0.4 million from Eli Lilly related to the MTA. For the three and nine months ended September 30, 2024, we recorded $0.1 million and $0.3 million, respectively, as a reduction in R&D expense in the condensed consolidated statement of operations and comprehensive loss. For the three and nine months ended September 30, 2023, we recorded $0.2 million and $0.2 million, respectively, as a reduction in R&D expense in the condensed consolidated statement of operations and comprehensive loss.
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Research and Collaboration Agreement
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BioMarin Pharmaceutical, Inc.
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On September 26, 2024, we entered into a Collaboration and License Agreement (the “BioMarin Agreement”) with BioMarin Pharmaceutical Inc. (“BioMarin”), pursuant to which we and BioMarin agreed to collaborate with respect to the research and discovery of regulatory RNA-targeting ASOs using our proprietary RAP Platform to modulate the expression of two undisclosed genetic targets under two distinct programs. Under the terms of the BioMarin Agreement, BioMarin will pay an upfront, nonrefundable payment of $1.0 million, and will reimburse us for the research activities we will conduct for each program under an agreed research plan and budget. On a per-program basis, we will be eligible to receive up to $5.0 million in future contingent preclinical milestones, up to $75.0 million in future contingent development and regulatory milestones and up to $105.0 million in commercial sales milestones. We will be further eligible to receive tiered royalties at percentage rates ranging from the low single digits to the high single digits of net sales, subject to specified reductions, until either the last-to-expire valid claim of a patent covering the product, ten years after the first commercial sale, or the expiration of any applicable regulatory exclusivity obtained for the product, in each case on a product-by-product and country-by-country basis. The agreement may be terminated in its entirety or on a program-by-program basis for convenience by BioMarin. The agreement may also be terminated by either us or BioMarin under certain other circumstances, including material breach, as set forth in the agreement. The notice periods for termination provisions range from 30 days to 270 days depending on the reason for termination.
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During the three and nine months ended September 30, 2024, we did not record any research collaboration and license revenue pursuant to the BioMarin Agreement.
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Components of Our Results of Operations
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Revenue
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For the three and nine months ended September 30, 2023, we recognized $0.35 million in research collaboration and license revenue through our collaboration and license agreements. We did not recognize any research collaboration and license revenue during the three and nine months ended September 30, 2024. We have not generated any revenue from the sale of products, however, and do not expect to generate any revenue from the sale of products in the foreseeable future, if at all. If our or our collaborators’ development efforts for product candidates and any future product candidates are successful and result in regulatory approval, we may generate revenue in the future from product sales, payments from existing or potential future collaboration or license agreements with third parties, or any combination thereof.
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Operating Expenses
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Our operating expenses consist of (i) R&D expenses and (ii) general and administrative expenses.
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Research and development expenses consist primarily of external and internal costs incurred in performing clinical and preclinical development activities.
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Our R&D expenses consist of:
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We expense R&D costs as incurred. Certain third-party costs for R&D activities are recognized based on an evaluation of the progress to completion of specific tasks using information and data provided to us by our management and scientific personnel, vendors and third-party service providers. Non-refundable advance payments for goods and services that will be used over time for R&D are deferred and capitalized as R&D prepaid expenses on our consolidated balance sheets. The capitalized amounts are recognized as an expense as the goods are delivered or as the related services are performed. Since our inception, substantially all of our external costs were related to the development of product candidates. We use internal resources for platform development, early pipeline discovery, preclinical development, management of clinical development activities, technical operations and oversight of manufacturing partners. We do not track our research and development expenses on a program-by-program basis. Our third-party research and development expenses consist primarily of fees paid to outside consultants, CROs, CMOs and academic research laboratories in connection with our preclinical development, process development, manufacturing and clinical development activities. Our other R&D costs are internal costs primarily associated with our discovery efforts, laboratory supplies, and facilities, including depreciation, that are allocated across multiple programs.
Previous report
Although R&D activities are central to our business model, the successful development of any future product candidates is highly uncertain. There are numerous factors associated with the successful development of any product, including future trial design and various regulatory requirements, many of which cannot be determined with accuracy at this time based on our stage of development. In addition, future regulatory factors beyond our control may impact our clinical development programs. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and longer duration of later-stage clinical trials. As a result, we expect our R&D expenses will increase substantially in connection with our ongoing and planned clinical and preclinical development activities in the near term and in the future. At this time, we cannot accurately estimate or know the nature, timing and costs of the efforts that will be necessary to complete the preclinical and clinical development of our current product candidates and any future product candidates. Our future R&D expenses may vary significantly based on a wide variety of factors such as:
Previous report
A change in the outcome of any of these variables with respect to the development of our product candidates or any future product candidates could significantly change the costs and timing associated with the development of that product candidate.
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General and administrative expenses consist primarily of personnel-related expenses such as salaries and stock-based compensation and benefits for our personnel in executive, legal, finance and accounting, human resources and other administrative functions. General and administrative expenses also include legal fees relating to patent and corporate matters and professional fees paid for accounting, audit, consulting and tax services, as well as facilities-related costs not otherwise included in R&D expenses and other costs such as insurance costs and travel expenses.
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We anticipate our general and administrative expenses will increase substantially in the future as we expand our operations, including increasing our headcount to support our continued R&D activities and continue to advance the development of our product candidates. We also anticipate we will incur increased accounting, audit, legal, regulatory, compliance, director and officer insurance, and investor and public relations expenses associated with operating as a public company.
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Interest Income
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Interest income relates to interest earned on our invested cash and cash equivalent balances.
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Other (Expense)
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Other (expense) consists of miscellaneous items, such as foreign exchange gains and losses and other insignificant amounts.
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Research and development expenses were $9.7 million for the three months ended September 30, 2024 compared to $9.8 million for the three months ended September 30, 2023. The decrease of $0.1 million in R&D expenses for the three months ended September 30, 2024 as compared to the prior period was primarily due to a decrease of $0.5 million in workforce-related expenses, offset in part by an increase of $0.2 million in lab operations and information technology expenses, and an increase of $0.2 million in professional and consulting fees associated with preclinical, regulatory and clinical affairs and continued development of our lead product candidate.
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The following table summarizes our general and administrative expenses for the three months ended September 30, 2024 and 2023 (in thousands):
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General and administrative expenses were $3.8 million for the three months ended September 30, 2024 compared to $2.9 million for the three months ended September 30, 2023. The increase of $0.9 million in general and administrative expenses for the three months ended September 30, 2024 as compared to the prior period was primarily due to an increase of $0.5 million related to stock-based compensation associated with the forgiveness of promissory notes originally issued to certain executive officers, and an increase in accounting and consulting fees and fees paid to prosecute patents of $0.4 million.
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Results of Operations
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Comparison of the Nine Months Ended September 30, 2024 and 2023
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Reworded paragraphs (15)
Previous wording
The following discussion and analysis of our financial condition and results of operations and the unaudited interim condensed consolidated financial statements and related notes included in this Quarterly Report on Form 10-Q (“Quarterly Report) should be read in conjunction with the financial statements and related notes thereto as of and for the year ended December 31, 2023 contained in the prospectus (the “IPO Prospectus”) filed on October 11, 2024 pursuant to Rule 424(b) under the Securities Act of 1933, as amended (the “Securities Act”), with the Securities and Exchange Commission (the “SEC”). This discussion and analysis and other parts of this Quarterly Report contain forward-looking statements. Our actual results and the timing of selected events could differ materially from those described in or implied by these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the sections of this Quarterly Report titled “Special Note Regarding Forward-Looking Statements” and “Risk Factors,” under Part II, Item 1A.
Current wording
The following discussion and analysis of our financial condition and results of operations and the unaudited interim condensed consolidated financial statements and related notes included in this Quarterly Report on Form 10-Q (the “Quarterly Report”) should be read in conjunction with the audited financial statements and related notes thereto in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which was filed with the Securities and Exchange Commission (the “SEC”) on March 27, 2025 (the “2024 Form 10-K”). This discussion and analysis and other parts of this Quarterly Report contain forward-looking statements. Our actual results and the timing of selected events could differ materially from those described in or implied by these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the sections of this Quarterly Report titled “Special Note Regarding Forward-Looking Statements,” and those risk factors described in “Part I, Item 1A, Risk Factors” of our 2024 Form 10-K and in “Part II, Item 1A, Risk Factors” in this Quarterly Report.
Previous wording
We are a clinical-stage biopharmaceutical company pioneering the discovery and development of regulatory RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Regulatory RNAs (“regRNAs”) play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression. Our approach is designed to amplify messenger RNA (“mRNA”) expression by harnessing the power of regRNAs that form localized complexes with transcription factors and regulate gene expression. Our proprietary RNA Actuating Platform, or RAP Platform, allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes. Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel antisense oligonucleotide (“ASO”) candidates, which we also refer to as RNA Actuators. These ASOs are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way. We are initially focused on metabolic and central nervous system diseases with validated disease biology, and we believe our RAP Platform allows us to address a broad range of genetic diseases in which a modest increase in protein expression has the potential to be clinically meaningful.
Current wording
We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Regulatory RNAs (“regRNAs”) play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression. Our approach is designed to amplify messenger RNA (“mRNA”) expression by harnessing the power of regRNAs that form localized complexes with transcription factors and regulate gene expression. Our proprietary RNA Actuating Platform, or RAP Platform, allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes. Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel antisense oligonucleotide (“ASO”) candidates, which we also refer to as RNA Actuators. These ASOs are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way. We are currently focused on metabolic diseases and diseases of the central nervous system with validated disease biology, and we believe our RAP Platform allows us to address a broad range of rare and prevalent genetic diseases in which a modest increase in protein expression has the potential to be clinically meaningful.
Previous wording
Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, research and development activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including in our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through September 30, 2024, we have received gross proceeds of $188.3 million from the sale of our convertible preferred stock. In addition, through September 30, 2024, we have recognized $17.4 million in research collaboration and license revenue through our development and license agreements. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.
Current wording
Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including in our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through March 31, 2025, we have received net proceeds of $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock. In addition, through March 31, 2025, we have recognized $18.9 million in research collaboration and license revenue through our development and license agreements. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.
Previous wording
We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $13.5 million and $11.7 million for the three months ended September 30, 2024 and 2023, respectively, and were $38.5 million and $36.3 million for the nine months ended September 30, 2024 and 2023, respectively. As of September 30, 2024, we had an accumulated deficit of $198.5 million. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and, to a lesser extent, from general and administrative costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other research and development activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, following the closing of our IPO on October 15, 2024, we expect to incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and SEC requirements, director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. We anticipate that our expenses will increase substantially if and as we:
Current wording
We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $12.4 million and $12.5 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $224.2 million. Substantially all of our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, we expect to incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and requirements of the SEC, director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. We anticipate that our expenses will increase substantially if and as we:
Previous wording
We do not have any products approved for sale and have not generated any revenue from product sales. We will not generate revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for our current or future product candidates, which we expect will take a number of years and may never occur. As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through equity offerings, debt financings, or other capital sources, including current and potential future collaborations, license agreements, and other similar arrangements. However, we may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such agreements or arrangements as and when needed, we may delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise intend to develop and market ourselves, or even cease operations.
Current wording
We do not have any products approved for sale and have not generated any revenue from product sales. We will not generate revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for our current or any future product candidates, which we expect will take a number of years or may never occur. As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through equity offerings, debt financings, or other capital sources, including current and potential future collaborations, license agreements, and other similar arrangements. However, we may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such agreements or arrangements as, and when needed, we may delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise develop and market ourselves, or even cease operations.
Previous wording
As of September 30, 2024, we had cash and cash equivalents of $2.5 million. On October 15, 2024, upon the closing of the IPO, we received aggregate proceeds, net of underwriting discounts and commissions, of $69.8 million. In addition, on November 1, 2024, we received additional proceeds of $6.6 million pursuant to the partial exercise by the underwriters of their option to purchase additional shares in the IPO. Collectively, we received aggregate net proceeds of $76.4 million from the IPO and the subsequent partial exercise by the underwriters of their option to purchase additional shares (the “IPO Proceeds”). Based on our current operating plan, we estimate that our existing cash and cash equivalents as of the date of this Quarterly Report, including the IPO Proceeds, will be sufficient to fund our projected operating expenses and capital expenditure requirements through at least 12 months from the date of this Quarterly Report. We have based this estimate on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us. In addition, we could utilize our available capital resources sooner than we expect. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to Our Financial Position and Need for Additional Capital” included elsewhere in this Quarterly Report.
Current wording
As of March 31, 2025, we had cash and cash equivalents of $49.3 million. Based on our current operating plan, we estimate that our existing cash and cash equivalents will be sufficient to fund our projected operating expenses and capital expenditure requirements into the second quarter of 2026, which raises substantial doubt about our ability to continue as a going concern. See the sections titled “Liquidity and Capital Resources” included elsewhere in this Quarterly Report and “Risk Factors - Risks Related to our Financial Position and Need for Additional Capital” included in our 2024 Form 10-K.
Previous wording
We do not own or operate, and currently have no plans to establish, any manufacturing facilities. We rely, and expect to continue to rely, on third parties for clinical supply as well as commercial supply if we obtain marketing approval. In addition, we rely on third parties to package, label, store, and distribute our clinical supply and we intend to rely on third parties to conduct the same activities for our commercial products if we obtain regulatory approval. We believe that this strategy allows us to maintain a more efficient infrastructure by eliminating the need to invest in our own manufacturing facilities, equipment and personnel while also enabling us to focus our expertise and resources on the development of product candidates and continued enhancement of our RAP Platform.
Current wording
We do not own or operate, and currently have no plans to establish, any manufacturing facilities. We rely, and expect to continue to rely, on third parties for clinical supply as well as commercial supply if we obtain marketing approval. In addition, we rely on third parties to package, label, store, and distribute our clinical supply and we intend to rely on third parties to conduct the same activities for our commercial products if we obtain regulatory approval. We believe that this strategy allows us to maintain a more efficient infrastructure by eliminating the need for us to invest in our own manufacturing facilities, equipment, and personnel while also enabling us to focus our expertise and resources on the development of product candidates and continued enhancement of our RAP Platform.
Previous wording
We did not recognize any research and collaboration revenue during the three months ended September 30, 2024. We recognized $0.35 million in research and collaboration revenue during the three months ended September 30, 2023. The decrease of $0.35 million was due to revenue generated from the sublicense agreement with Fulcrum.
Current wording
We recognized $0.9 million in research and collaboration revenue during the three months ended March 31, 2025. We did not recognize any research and collaboration revenue during the three months ended March 31, 2024. The increase of $0.9 million was due to revenue recognized under our Collaboration and License Agreement with BioMarin.
Previous wording
Other income, net was less than $0.1 million for the three months ended September 30, 2024 compared to $0.6 million for the three months ended September 30, 2023. The decrease of $0.6 million was primarily due to a decrease in interest income on lower average cash equivalent balances in 2024.
Current wording
Other income, net was $0.7 million for the three months ended March 31, 2025 compared to $0.4 million for the three months ended March 31, 2024. The increase of $0.3 million was primarily due to an increase in interest income due to higher average invested cash equivalent balances during the three months ended March 31, 2025.
Previous wording
Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from operations. We expect to incur significant expenses and operating losses in the foreseeable future as we advance the development of product candidates. Through September 30, 2024, we have primarily funded our operations with proceeds from the sale of shares of our convertible preferred stock and revenues from our license and collaboration agreements. Through September 30, 2024, we have received aggregate gross proceeds of $188.3 million from the sale of shares of our convertible preferred stock. In addition, through September 30, 2024, we have recognized $17.4 million in research and collaboration revenue through our collaboration and license agreements. As of September 30, 2024, we had cash and cash equivalents of $2.5 million.
Current wording
Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from operations. We expect to incur significant expenses and operating losses in the foreseeable future as we advance the development of product candidates. Through March 31, 2025, we have primarily funded our operations with proceeds from the sale of our equity securities and revenues from our license and collaboration agreements.
Previous wording
We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we continue our development of, seek regulatory approval for, and potentially commercialize our product candidates and seek to discover and develop additional product candidates, conduct our ongoing and planned clinical trials and preclinical studies, continue our research and development activities, hire additional personnel, expand and protect our intellectual property, and incur additional costs associated with being a public company.
Current wording
We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we continue our development of, seek regulatory approval for, and potentially commercialize our product candidates and seek to discover and develop additional product candidates, conduct our ongoing and planned clinical trials and preclinical studies, continue our R&D activities, hire additional personnel, expand and protect our intellectual property, and incur additional costs associated with being a public company. The timing and amount of our funding requirements will depend on many factors for which there have been no material changes from those described under “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our 2024 Form 10-K.
Previous wording
Our operating plans and other demands on our cash resources may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than planned.
Current wording
Our operating plans and other demands for our cash resources may change because of many factors currently unknown to us, and we may need to seek additional funds sooner than planned.
Previous wording
The following table provides information regarding our cash flows for the nine months ended September 30, 2024 and 2023 (in thousands):
Current wording
The following table provides information regarding our cash flows for the three months ended March 31, 2025 and 2024 (in thousands):
Previous wording
We are an emerging growth company, as defined in the Jumpstart Our Business Startups Act of 2012, as amended (the “JOBS Act”), and we may remain an emerging growth company until December 31, 2029 or until such earlier time that we are no longer an emerging growth company. For so long as we remain an emerging growth company, we are permitted and intend to rely on certain exemptions from various public company reporting requirements, including not being required to have our internal control over financial reporting audited by our independent registered public accounting firm pursuant to Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements, exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and any golden parachute payments not previously approved and an exemption from compliance with the requirements regarding the communication of critical audit matters in the auditor’s report on financial statements.
Current wording
We are an emerging growth company, as defined in the Jumpstart Our Business Startups Act of 2012 (the “JOBS Act”), and we may remain an emerging growth company until December 31, 2029 or until such earlier time that we are no longer an emerging growth company. For so long as we remain an emerging growth company, we are permitted and intend to rely on certain exemptions from various public company reporting requirements, including not being required to have our internal control over financial reporting audited by our independent registered public accounting firm pursuant to Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements, exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and any golden parachute payments not previously approved and an exemption from compliance with the requirements regarding the communication of critical audit matters in the auditor’s report on financial statements.
Previous wording
We are also a “smaller reporting company,” meaning that the market value of our shares held by non-affiliates is less than $700 million and our annual revenue was less than $100 million during the most recently completed fiscal year. We may continue to be a smaller reporting company if either: (i) the market value of our shares held by non-affiliates is less than $250 million or (ii) our annual revenue was less than $100 million during the most recently completed fiscal year and the market value of our shares held by non-affiliates is less than $700 million. If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies. Specifically, as a smaller reporting company, we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Current wording
We are also a “smaller reporting company,” meaning that the market value of our shares held by non-affiliates is less than $700.0 million and our annual revenue was less than $100.0 million during the most recently completed fiscal year. We may continue to be a smaller reporting company if either: (i) the market value of our shares held by non-affiliates is less than $250.0 million; or (ii) our annual revenue was less than $100.0 million during the most recently completed fiscal year and the market value of our shares held by non-affiliates is less than $700.0 million. If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies. Specifically, as a smaller reporting company, we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
For the Three Months Ended March 31, 2025 and 2024
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
The following table summarizes our results of operations for the nine months ended September 30, 2024 and 2023 (in thousands):
Previous report — wording carried elsewhere
The timing and amount of our funding requirements will depend on many factors, including: