Syndax Pharmaceuticals Inc
For the period ended Sep 30, 2024. Filed Nov 5, 2024.
View on SEC.govAbout this filing
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.
Sep 30, 2024 · Filed Nov 5, 2024
SNDX$9.54+1.8%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-06-30; filed 2024-08-01.
0000950170-24-089241
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2024-09-30; filed 2024-11-05.
0000950170-24-121439
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 122 previous paragraphs and 127 current paragraphs.
67 unchanged; 30 reworded; 29 added; 25 removed; 0 previous and 1 current paragraphs rearranged.
Tables are not compared: 5 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 (29)
Current report
We are a commercial-stage biopharmaceutical company with one commercially approved product and are developing an innovative pipeline of cancer therapies. NIKTIMVO™ (axatilimab-csfr) is our approved product and revumenib is our lead product candidate.
Current report
Our commercially approved product, Niktimvo, received approval on August 14, 2024 from the U.S. Food and Drug Administration, or FDA, in the United States, for the treatment of chronic graft versus host disease, or cGVHD, after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs), with a recommended dosage of 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks until progression or unacceptable toxicity. While Incyte leads U.S. commercialization efforts, we are co-promoting Niktimvo with Incyte in the U.S. To facilitate patient dosing and limit product waste, following the FDA’s approval of Niktimvo, we, along with Incyte, have submitted to the FDA for the approval of two smaller vial sizes. We anticipate launching Niktimvo in the U.S. no later than early first quarter 2025. Incyte has exclusive commercialization rights for Niktimvo outside the U.S.
Current report
Business Update
Current report
We announced that data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in adult and pediatric patients with R/R KMT2Ar AML and ALL have been published in the Journal of Clinical Oncology.
Current report
We announced that a larger data set and longer follow-up from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in R/R KMT2Ar acute leukemia will be presented at the upcoming 66th American Society of Hematology, or ASH, Annual Meeting. Analysis of the larger efficacy population (n=97), which includes the 57 patients from the previously reported interim efficacy analysis plus an additional 40 patients. Consistent with previously reported data, the updated analysis shows that revumenib provides durable responses and robust rates of overall response, minimal residual disease, or MRD, negativity, and hematopoietic stem cell transplantation, or HSTC. With seven months of additional follow-up, the median duration of CR/CRh extended to 13 months among the 13 CR/CRh responders included in the interim analysis presented at ASH 2023.
Current report
INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial. Preliminary results from the first eight mNPM1 patients treated with revumenib will be presented at the upcoming 66th ASH Annual Meeting.
Current report
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Current report
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Current report
Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break Through Cancer, a collaboration between leading U.S. cancer research centers.
Current report
Niktimvo™ (axatilimab-csfr)
Current report
Niktimvo received FDA approval for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). We anticipate that Niktimvo will be launched in the U.S. no later than early first quarter 2025. In the U.S., Niktimvo will be co-commercialized by Incyte and us.
Current report
We announced Niktimvo was added to the latest NCCN Clinical Practice Guidelines in Oncology, or NCCN Guidelines®, as a category 2A recommendation for the treatment of GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Treatments are classified as category 2A when there is uniform NCCN consensus that the intervention is appropriate, based on lower level evidence.
Current report
We announced that results from the pivotal Phase 2 AGAVE-201 trial of Niktimvo in adult and pediatric patients with recurrent/refractory active cGVHD who had received at least two prior lines of systemic therapy were published in the New England Journal of Medicine.
Current report
We announced a secondary analysis of overall and organ-specific responses from the pivotal Phase 2 AGAVE-201 trial of Niktimvo in adult and pediatric patients with recurrent/refractory active cGVHD who had received at least two prior lines of systemic therapy will be presented at the 66th ASH Annual Meeting. The data demonstrated rapid responses and symptom improvement in inflammatory and fibrotic manifestations of cGVHD in heavily pretreated patients.
Current report
Our partner, Incyte, is now recruiting patients for a Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥12 years of age with newly diagnosed chronic GVHD (NCT06388564). A Phase 3 trial of axatilimab in combination with steroids for the treatment for chronic GVHD is currently in preparation.
Current report
Corporate
Current report
We announced a $350 million royalty funding agreement with Royalty Pharma Development Funding, LLC, or Royalty Pharma, based on U.S. net sales of Niktimvo. Under the agreement, we received $350 million in exchange for a 13.8% royalty on U.S. net sales of Niktimvo. Royalty payments to Royalty Pharma will cease upon reaching a multiple of 2.35x.
Current report
Revenue for the three months ended September 30, 2024, consisted of a $12.5 million milestone earned as a result of the approval of Niktimvo.
Current report
Revenue for the nine months ended September 30, 2024, consisted of a $12.5 million milestone earned in the third quarter of 2024 as a result of the approval of Niktimvo and $3.5 million of revenue recognized in the second quarter of 2024 under the license agreement with Eddingpharm.
Current report
The increase in costs related to axatilimab for the three and nine month period ended September 30, 2024, compared to the prior year periods, was primarily driven by the continuation of the IPF trial, pre-commercial manufacturing activities and a $15.0 million milestone payment to UCB as a result of the approval of Niktimvo in the third quarter of 2024.
Current report
The decrease in other research and development programs for the three and nine month periods ended September 30, 2024, compared to the prior year periods, was related to a decrease in clinical trial expense, not related to the revumenib and axatilimab programs.
Current report
The increase in personnel costs and other expenses, and stock-based compensation, which includes salaries, overhead, and related expenses, for the three and nine month periods ended September 30, 2024, compared to the prior year periods, was related to support of the on-going clinical trials and NDA/sNDA activities.
Current report
The increase in commercial-related expenses for the three and nine month periods ended September 30, 2024, compared to the prior year periods, was primarily driven by and increase in commercial readiness activities for Niktimvo and revumenib.
Current report
The increase in other selling, general, and administrative expenses for the three and nine months ended September 30, 2024, compared to the prior year periods, was primarily due to an increase in IT, recruiting, and legal costs.
Current report
The increase in personnel costs and other expenses and stock-based compensation, which includes salaries, overhead, and related expenses, for the three and nine months ended September 30, 2024, compared to the prior year periods, was primarily due to increased headcount in preparation for commercial readiness activities.
Current report
Interest Expense and Interest Income
Current report
Other Income (Expense)
Current report
Net cash used in operating activities increased from $119.3 million for the nine months ended September 30, 2023 to $217.5 million for the nine months ended September 30, 2024, primarily due to an increase in operating net loss, prepaid expenses, and collaboration receivables, and a decrease in accounts payable.
Current report
our ability, along with Incyte, to successfully launch and generate sales of Niktimvo;
Removed paragraphs (25)
Previous report
Clinical Developments
Previous report
We presented updated data from the AUGMENT-102 trial evaluating the combination of revumenib with fludarabine and cytarabine in patients with R/R acute leukemias at the EHA 2024 Congress. Treatment with the combination in R/R mNPM1, NUP98-rearranged, or NUP98r, or KMT2Ar acute leukemia patients resulted in a 52% (14 of 27 pts) CRc rate.
Previous report
Axatilimab
Previous report
The Biologics License Application, or BLA, for axatilimab, an anti-CSF-1R antibody, for patients with cGVHD after failure of at least two prior lines of systemic therapy, is under FDA Priority Review with a PDUFA action date of August 28, 2024.
Previous report
Our partner, Incyte, plans to initiate two combination trials with axatilimab in earlier lines of treatment for chronic GVHD in the second half of 2024, including a Phase 2 combination trial with ruxolitinib and a Phase 3 combination trial with steroids.
Previous report
We presented additional positive data from the AGAVE-201 pivotal trial evaluating axatilimab monotherapy in patients with refractory chronic GVHD at the EHA 2024 Congress. Responses were noted in all fibrosis-dominant organs and the clinical activity was supported by clinician-reported and patient-reported changes in organ-specific symptoms, such as improvements in swallowing, shortness of breath, skin and joints, and sclerotic skin.
Previous report
Corporate Updates
Previous report
In May 2024, we announced the appointment of Aleksandra Rizo, M.D., Ph.D to our Board of Directors. Dr. Rizo has extensive clinical development experience and a track record of successfully leading the development of several oncology drugs from discovery through commercialization.
Previous report
The current inflationary environment may materially affect our business and operating results by increasing our overhead costs, including the costs of our clinical trial materials and supplies. High interest rates could make it more difficult for us to obtain traditional financing on acceptable terms, if at all. Additionally, the ongoing recession risk together with the foregoing, could result in further economic uncertainty and volatility in the capital markets in the near term and, as a result could negatively affect our operations. Furthermore, such economic conditions have produced downward pressure on share prices. Although we do not believe that inflation has had a material impact on our financial position or results of operations to date, additional high inflation could increase our operating costs, including our labor costs and research and development costs. These costs may also be negatively impacted due to supply chain constraints, global geopolitical tensions as a result of the ongoing wars between Russia and Ukraine and the war in Gaza, worsening macroeconomic conditions and employee availability and wage increases, which may result in additional stress on our working capital.
Previous report
In September 2021, we granted Incyte an exclusive worldwide license to develop and commercialize axatilimab. For the three and six months ended June 30, 2024, no revenue has been recognized under the Incyte Agreements.
Previous report
For the three and six months ended June 30, 2024, revenue increased $3.5 million, or 100%, from the comparable quarter in the prior period. The increase is solely due to achievement of certain milestones under our license agreement with Eddingpharm related to the approval of entinostat in China.
Previous report
An increase of $3.0 million and $3.5 million, for the three and six months ended June 30, 2024, respectively, in axatilimab-related costs due to continuation of the IPF trial and pre-commercial manufacturing activities offset by lower registrational trial costs.
Previous report
An increase of $4.2 million and $10.4 million, for the three and six months ended June 30, 2024, respectively, in personnel costs, including non-cash stock-based compensation, and other expenses related to increases in headcount to support on-going clinical trials, NDA and sNDA, activities, and includes salaries and related expenses and overhead expenses (e.g. information technology and facilities).
Previous report
The following table summarizes the internal and external research and development expenses for the three and six months ended June 30, 2024 and 2023:
Previous report
We expect research and development expenses to fluctuate from quarter-to-quarter depending on the timing of clinical trial activities, clinical manufacturing, and other development activities.
Previous report
An increase of $5.8 million and $10.1 million, for the three and six months ended June 30, 2024, respectively, in commercialization-related expenses is due to commercial readiness activities in anticipation of expected regulatory marketing approvals for revumenib and axatilimab.
Previous report
An increase of $0.5 million and $2.0 million, for the three and six months ended June 30, 2024, respectively, related to increases in information technology, human resource and legal expenses.
Previous report
An increase of $7.8 million and $13.1 million, for the three and six months ended June 30, 2024, respectively, in personnel costs, including non-cash stock-based compensation, and related costs related to increases in headcount to support a growing research and development organization and preparation of commercial launches.
Previous report
Interest Income and Interest Expense
Previous report
For the three and six months ended June 30, 2024, interest expense increased by $10 thousand, or 29%, and $25 thousand, or 33%, respectively, from the comparable period in the prior year. The increase is primarily due to our operational and capital leases.
Previous report
Other Income (Expense) , net
Previous report
Net cash used in operating activities for the six months ended June 30, 2024, was $155.2 million and consisted of our net loss of $140.5 million adjusted for non-cash items, which include stock-based compensation of $18.8 million, an investment accretion of $7.5 million, a net decrease in operating assets and liabilities of $26.5 million and an increase in operating lease and depreciation expenses of $0.5 million. The increase in net loss was primarily due to increased clinical trial activities and manufacturing expenses. The net decrease in operating assets and liabilities primarily consisted of decreased accrued expenses and other liabilities of $13.6 million, other receivables of $9.6 million, collaboration payable of $4.1 million, prepayments and deposits of $0.9 million, partially offset by accounts payable of $1.6 million.
Previous report
Net cash used in operating activities for the six months ended June 30, 2023, was $74.6 million and consisted of our net loss of $85.7 million adjusted for non-cash items, which include stock-based compensation of $14.3 million, an investment accretion of $7.7 million, a net increase in operating assets and liabilities of $4.3 million, and an increase in operating lease and depreciation expenses of $0.2 million. The increase in net loss was primarily due to increased clinical trial activities and manufacturing expenses. The net increase in operating assets and liabilities primarily consisted of accounts payable of $6.6 million, increased other payables of $4.0 million and increased prepaids and deposits of $0.5 million, partially offset by decreased accrued expenses and other liabilities of $6.8 million.
Previous report
Net Cash (Used in) Provided by Investing Activities
Previous report
Net cash provided by financing activities for the six months ended June 30, 2023, was $3.8 million and was primarily due to proceeds from the exercise of stock options of $3.4 million and employee participation in our ESPP of $0.4 million.
Reworded paragraphs (30)
Previous wording
We are a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Our two lead product candidates are revumenib and axatilimab. We are developing revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, or KMT2Ar, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including acute lymphoblastic leukemia, or ALL, and acute myeloid leukemia, or AML, and nucleophosmin1, also known as NPM1, mutant AML. We are also exploring the use of revumenib as a treatment in solid tumors, specifically its activity in metastatic microsatellite stable, or MSS, colorectal cancer, or CRC. We are developing axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 receptor, or CSF-1R, in chronic graft-versus-host disease, or cGVHD, as well as idiopathic pulmonary fibrosis, or IPF. We plan to continue to leverage the technical and business expertise of our management team and scientific collaborators to license, acquire and develop additional therapeutics to expand our pipeline.
Current wording
We are developing revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, or KMT2Ar, also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including acute lymphoblastic leukemia, or ALL, and acute myeloid leukemia, or AML, and nucleophosmin1, also known as NPM1, mutant AML. We are also exploring the use of revumenib as a treatment in solid tumors, specifically its activity in metastatic microsatellite stable, or MSS, colorectal cancer, or CRC. We continue to develop axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 receptor, or CSF-1R, for idiopathic pulmonary fibrosis, or IPF.
Previous wording
We have no products approved for commercial sale and have not generated any product revenues from product sales to date. We continue to incur significant research and development and other expenses related to our ongoing operations. Except for 2021, we have not been profitable and have incurred losses in each period since our inception in 2005. For the six months ended June 30, 2024 and 2023, we reported a net loss of $140.5 million and $85.7 million, respectively. As of June 30, 2024, we had an accumulated deficit of $1.0 billion. As of June 30, 2024, we had cash, cash equivalents and short- and long-term investments of $454.6 million.
Current wording
We plan to continue to leverage the technical and business expertise of our management team and scientific collaborators to license, acquire and develop additional therapeutics to expand our pipeline. We have not generated any product revenues from product sales to date. We continue to incur significant research and development and other expenses related to our ongoing operations. Except for 2021, we have not been profitable and have incurred losses in each period since our inception in 2005. For the nine months ended September 30, 2024 and 2023, we reported a net loss of $224.6 million and $136.9 million, respectively. As of September 30, 2024, we had an accumulated deficit of $1.1 billion. As of September 30, 2024, we had cash, cash equivalents and short- and long-term investments of $399.6 million.
Previous wording
Our New Drug Application, or NDA, for revumenib, a highly selective menin inhibitor, for the treatment of adult and pediatric relapsed or refractory, or R/R, KMT2A-rearranged, or KMT2Ar, acute leukemia was granted Priority Review and is being reviewed by the United States Food and Drug Administration, or FDA, and is being reviewed under the FDA’s Real-Time Oncology Review, or RTOR, Program. On July 29, 2024, we announced that the FDA extended the Prescription Drug User Fee Act, or PDUFA, target action date for the revumenib NDA from September 26, 2024 to December 26, 2024 to provide FDA additional time to conduct a full review of supplemental information provided by us in response to the FDA’s requests.
Current wording
The New Drug Application, or NDA, for revumenib, an oral menin inhibitor, for the treatment of adult and pediatric relapsed or refractory, or R/R, KMT2A-r acute leukemia was granted Priority Review and is being reviewed under the U.S. FDA’s Real-Time Oncology Review, or RTOR, Program with a Prescription Drug User Fee Act, or PDUFA, target action date of December 26, 2024.
Previous wording
We expect to report topline data from the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin, or mNPM1, acute myeloid leukemia, or AML, in the fourth quarter of 2024. Positive data could support a supplemental NDA, or sNDA, filing for revumenib in R/R mNPM1 AML in the first half of 2025.
Current wording
We expect to report topline data from the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin, or mNPM1, AML in the fourth quarter of 2024. Positive data could support a supplemental NDA, or sNDA, filing for revumenib in R/R mNPM1 AML in the first half of 2025.
Previous wording
Multiple Phase 1 combination trials of revumenib in mNPM1 and KMT2Ar acute leukemias are ongoing across the treatment landscape. These trials include:
Current wording
Multiple trials evaluating the potential to expand revumenib use across the mNPM1 and KMT2Ar acute leukemia treatment landscape are ongoing. These trials include:
Previous wording
BEAT AML: Evaluating the combination of revumenib with venetoclax and azacitidine in front-line AML patients. This trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML® Master Clinical Trial. We presented updated positive data from the trial at the European Hematology Association, or EHA, 2024 Congress, showing a 96% (23 of 24 pts) composite complete remission, or CRc, rate in patients with newly diagnosed mNPM1 or KMT2Ar AML. The BEAT AML trial is expanding to validate the recommended Phase 2 dose of the combination of revumenib with venetoclax and azacitidine.
Current wording
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in front-line AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML® Master Clinical Trial. We presented updated positive data from the trial at the European Hematology Association, or EHA, 2024 Congress, showing a 96% (23 of 24 pts) composite complete remission, or CRc, rate in patients with newly diagnosed mNPM1 or KMT2Ar AML. The BEAT AML trial is expanding to validate the recommended Phase 2 dose of the combination of revumenib with venetoclax and azacitidine. We anticipate that an update on the trial will be available in the fourth quarter of 2024.
Previous wording
SAVE: Evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from MD Anderson Cancer Center. The trial is expanding to validate the recommended Phase 2 doses, with additional data expected in the second half of 2024.
Current wording
SAVE: A Phase 1 trial evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from MD Anderson Cancer Center. We announced that updated data showing an 88% ORR (23 of 26 pts) in R/R patients with KMT2Ar, mNPM1, or NUP98r leukemias will be presented at the upcoming 66th ASH Annual Meeting. In addition to the R/R cohort, a frontline cohort is now enrolling patients.
Previous wording
Intensive chemotherapy: Evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias. The Phase 1 trial is designed to identify the recommended Phase 2 dose for this combination to support further development.
Current wording
Intensive chemotherapy: A Phase 1 trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias. The trial is designed to identify the recommended Phase 2 dose for this combination to support further development.
Previous wording
We announced that we advanced into the Phase 1b portion of its Phase 1/2 proof-of-concept trial of revumenib in patients with R/R metastatic microsatellite stable, or MSS, colorectal cancer, or CRC, based on initial data from the Phase 1a portion of the trial.
Current wording
We are evaluating revumenib in patients with R/R metastatic MSS colorectal cancer. The trial is currently enrolling patients in the Phase 1b portion of its Phase 1/2 proof-of-concept trial.
Previous wording
Enrollment is ongoing in a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis, or IPF.
Current wording
Enrollment is ongoing in a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with IPF, now referred to as the MAXPIRe trial (NCT06132256). We expect to report topline data from the trial in 2026.
Previous wording
Additionally, we are subject to other challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of our late-stage product candidate; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements.
Current wording
We are subject to challenges and risks specific to our business and our ability to execute on our strategy, as well as risks and uncertainties common to companies in the pharmaceutical industry with development and commercial operations, including, without limitation, risks and uncertainties associated with: obtaining regulatory approval of our late-stage product candidate; identifying, acquiring or in-licensing additional products or product candidates; pharmaceutical product development and the inherent uncertainty of clinical success; the challenges of protecting and enhancing our intellectual property rights; and complying with applicable regulatory requirements.
Previous wording
To date, we have not generated any product revenues. Our ability to generate revenue depends upon our ability to obtain marketing approval of, and successfully commercialize our product candidates. Our revenue for the three and six months ended June 30, 2024 was solely derived from the achievement of milestones in connection with our license agreement with Eddingpharm Investment Company Limited, or Eddingpharm. We generated no revenue during the three and six months ended June 30, 2023.
Current wording
Our ability to generate sales revenue depends upon our ability to obtain marketing approval of, and successfully commercialize our product candidates. Our first commercially approved product, Niktimvo, received approval from the FDA in August 2024. We anticipate launching Niktimvo in the U.S. no later than early first quarter 2025. Our revenue for the three and nine months ended September 30, 2024 was derived from the achievement of milestones in connection with our license agreements with Incyte and Eddingpharm Investment Company Limited, or Eddingpharm. We did not generated any product sales revenue during the three and nine months ended September 30, 2024.
Previous wording
Selling, general and administrative expenses consist primarily of pre-commercialization costs as well as employee-related expenses, including salaries, benefits, non-cash stock-based compensation and travel expenses, for our employees in executive, finance, human resources, business development and support functions. Other selling, general and administrative expenses include facility-related costs not otherwise allocated to research and development expenses and accounting, tax, legal, information technology and consulting services. We anticipate that our selling, general and administrative expenses will further increase in the future as we continue to increase our headcount to support our continued research and development. Additionally, in anticipation of expected regulatory approvals of revumenib and axatilimab, we have begun preparation for commercial launches of both drug candidates and, as result, expect an increase in employee-related as well as sales and marketing expenses and other commercial expenses.
Current wording
Selling, general and administrative expenses consist primarily of pre-commercialization costs as well as employee-related expenses, including salaries, benefits, non-cash stock-based compensation and travel expenses, for our employees in executive, finance, human resources, information technology, business development and support functions. Other selling, general and administrative expenses include facility-related costs not otherwise allocated to research and development expenses and accounting, tax, legal, information technology and consulting services. We anticipate that our selling, general and administrative expenses will further increase in the future as we continue to increase our headcount to support our continued research and development and anticipated commercialization of revumenib. Additionally, we expect an increase in employee-related and sales and marketing expenses and other commercial expenses as a result of Niktimvo’s anticipated commercial launch and in anticipation of revumenib’s expected regulatory approval.
Previous wording
Comparison of the three and six months ended June 30, 2024 and 2023:
Current wording
Comparison of the three and nine months ended September 30, 2024 and 2023:
Previous wording
The following table summarizes the research and development expenses for the three months ended June 30, 2024 and 2023:
Current wording
The following table summarizes the research and development expenses for the three and nine months ended September 30, 2024 and 2023:
Previous wording
For the three and six months ended June 30, 2024, our total research and development expenses increased $13.9 million, or 40% and $36.3 million, or 53%, respectively, from the comparable quarter in the prior period. The increase is primarily due to:
Current wording
For the three and nine months ended September 30, 2024, our total research and development expenses increased $31.9 million and $68.2 million respectively, from the comparable prior year period. The increase was primarily due to:
Previous wording
An increase of $6.4 million and $22.8 million, for the three and six months ended June 30, 2024, respectively, in revumenib-related costs was primarily driven by continuation of the registrational trials, preparation for 1L/combo trials, costs related to the development of a companion diagnostic, pre-commercial manufacturing activities and medical affairs activities to prepare for commercialization.
Current wording
The increase in costs related to revumenib for the three and nine month period ended September 30, 2024, compared to the prior year periods, was primarily driven by the continuation of the registrational trials, preparation for front-line / combination trials, companion diagnostic development costs, pre-commercial manufacturing activities, medical affairs activities in preparation for commercialization, and an $8.0 million milestone paid to Vitae in the first quarter of 2024, related to the successful completion of the first pivotal trial in the first indication.
Previous wording
The following tables summarizes the selling, general and administrative expenses for the three and six months ended June 30, 2024 and 2023:
Current wording
The following tables summarizes the selling, general and administrative expenses for the three and nine months ended September 30, 2024 and 2023:
Previous wording
For the three and six months ended June 30, 2024, our total selling, general and administrative expenses increased $14.1 million, or 95% and $25.2 million, or 94%, respectively, from the comparable quarter in the prior period. The increase primarily is due to:
Current wording
For the three and nine months ended September 30, 2024, our total selling, general and administrative expenses increased $13.8 million and $39.0 million, respectively, from the comparable prior year period. The increase primarily is due to:
Previous wording
For the three and six months ended June 30, 2024, interest income increased by $1.1 million, or 21%, and $3.3 million, or 32%, respectively, from the comparable period in the prior year. The increase is primarily due to higher interest rates and increased average balance on cash equivalents and short and long-term investments.
Current wording
For the three and nine months ended September 30, 2024, interest income, net of interest expense, increased from the comparable prior year period. The increase was primarily due to higher interest rates and an increased average balance on cash equivalents and short and long-term investments.
Previous wording
For the three and six months ended June 30, 2024, total other income (expense) , net decreased from the comparable prior period primarily due to a decrease in foreign currency losses on short and long-term investments.
Current wording
For the three and nine months ended September 30, 2024, other income (expense) decreased from the comparable prior year period primarily due to the decrease in foreign currency losses on short and long-term investments.
Previous wording
Net cash provided by investing activities for the six months ended June 30, 2023 was $93.8 million and was due to $236.4 million from the maturities of available-for-sale securities, partially offset by the purchase of $142.6 million of available-for-sale securities.
Current wording
Net cash provided by investing activities for the nine months ended September 30, 2024, was $49.2 million and was related to $229.9 million from the maturities of available-for-sale securities, offset by the purchase of $180.7 million of available-for-sale securities.
Previous wording
Net cash used in investing activities for the six months ended June 30, 2024 was $37.6 million and was due to the purchase of $167.4 million of available for sale securities, partially offset by $129.7 million in proceeds from the maturities of available-for-sale securities.
Current wording
Net cash provided by investing activities for the nine months ended September 30, 2023, was $133.9 million and was related to $359.2 million from the maturities of available-for-sale securities, offset by the purchase of $225.3 million of available-for-sale securities.
Previous wording
Net cash provided by financing activities for the six months ended June 30, 2024, was $2.8 million and was primarily due to proceeds from the exercise of stock options of $2.3 million and employee participation in our 2015 Employee Stock Purchase Plan, or ESPP, of $0.5 million.
Current wording
Net cash provided by financing activities for the nine months ended September 30, 2024, increased by $0.3 million from the comparable prior year period primarily due to proceeds from the exercise of stock options and employee participation in our 2015 Employee Stock Purchase Plan, or ESPP.
Previous wording
Since our inception, our operations have been primarily financed by net proceeds from our public stock offerings, and revenue from our license agreements. We believe that our cash, cash equivalents and short- and long-term investments, totaling $454.6 million. as of June 30, 2024, will fund our projected operating expenses and capital expenditure requirements for at least the next 12 months. In addition to our existing cash, cash equivalents, short and long-term investments, we are eligible to receive research and development funding and to earn milestone and other contingent payments for the achievement of defined collaboration objectives and certain development, regulatory and commercial milestones, and royalty payments under our collaboration agreements. Our ability to earn these milestone and contingent payments and the timing of achieving these milestones is primarily dependent upon the outcome of our collaborators’ research and development activities and is uncertain at this time.
Current wording
Since our inception, our operations have been primarily financed by net proceeds from our public stock offerings, and revenue from our license agreements. We believe that our cash, cash equivalents and short- and long-term investments as of September 30, 2024, will fund our projected operating expenses and capital expenditure requirements for at least the next 12 months. In addition, we received a $350.0 million upfront payment in November 2024 pursuant to the purchase and sale agreement, or Royalty Agreement, with Royalty Pharma and are eligible to receive future aggregate payments up to $822.5 million. We are also and are eligible under our collaboration agreements to receive royalty payments, research and development funding, and milestone and other contingent payments for the achievement of defined collaboration objectives and certain development, regulatory and commercial milestones, which will continue to fund our future operations. Our ability to earn these milestone and contingent payments and the timing of achieving these milestones is primarily dependent upon the outcome of our collaborators’ research and development activities and is uncertain at this time.
Previous wording
the cost of establishing sales, marketing and distribution capabilities for our product candidates if either candidate receives regulatory approval and we determine, in the case of revumenib to commercialize it ourselves, or in the case of axatilimab to co-commercialize it with Incyte;
Current wording
the cost of establishing sales, marketing and distribution capabilities for our products and product candidates, including Niktimvo, which we are co-commercializing with Incyte, and revumenib, provided it receives regulatory approval and if we determine to commercialize it ourselves;
Previous wording
the outcome, timing and cost of seeking and obtaining regulatory approvals from the U.S. Food and Drug Administration, or FDA, and comparable foreign regulatory authorities, including the potential for such authorities to require that we perform more trials than we currently expect;
Current wording
the outcome, timing and cost of seeking and obtaining additional regulatory approvals from the FDA and comparable foreign regulatory authorities, including the potential for such authorities to require that we perform more trials than we currently expect;
Previous wording
We have no products approved for commercial sale and have not generated any product revenues from product sales to date. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings and additional funding from license and collaboration arrangements. Except for any obligations of our collaborators to reimburse us for research and development expenses or to make milestone or royalty payments under our agreements with them, we will not have any committed external source of liquidity. Our material contractual obligations and commitments as of June 30, 2024, primarily relate to our maturities of operating leases for office space and equipment and capital leases for office equipment. As of June 30, 2024, we have $1.1 million payable within 12 months.
Current wording
We have one commercially approved product, Niktimvo, which we anticipate launching in the U.S. no later than early first quarter 2025. We otherwise have no products approved for commercial sale and have not generated any product revenues from product sales to date. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings and additional funding from license and collaboration arrangements. Except for payments received under the Royalty Agreement or any obligations of our collaborators to reimburse us for research and development expenses or to make milestone or royalty payments under our agreements with them, we will not have any committed external source of liquidity. Our material contractual obligations and commitments as of September 30, 2024, primarily relate to our maturities of operating leases for office space and equipment and capital leases for office equipment. As of September 30, 2024, we have $0.8 million payable within 12 months.
Previous wording
We have incurred losses and cumulative negative cash flows from operations since our inception, excluding the year ended December 31, 2021. As of June 30, 2024, we had an accumulated deficit of $1.0 billion. We anticipate that we will likely continue to incur significant losses for at least the next couple of years. We expect that our research and development and selling, general and administrative expenses will continue to increase. As a result, we will need additional capital to fund our operations, which we may raise through a combination of the sale of equity, debt financings, or other sources, including potential collaborations. To the extent that we raise additional capital through the future sale of equity or debt, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing common stockholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or drug candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market drug candidates that we would otherwise prefer to develop and market ourselves.
Current wording
We have incurred losses and cumulative negative cash flows from operations since our inception, excluding the year ended December 31, 2021. As of September 30, 2024, we had an accumulated deficit of $1.1 billion. We anticipate that we will likely continue to incur significant losses for at least the next couple of years. We expect that our research and development and selling, general and administrative expenses will continue to increase. As a result, we will need additional capital to fund our operations, which we may raise through a combination of the sale of equity, debt financings, or other sources, including potential collaborations. To the extent that we raise additional capital through the future sale of equity or debt, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing common stockholders. If we raise additional funds through collaboration arrangements in the future, we may have to relinquish valuable rights to our technologies, future revenue streams or drug candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market drug candidates that we would otherwise prefer to develop and market ourselves.
Previous wording
In May 2023, we entered into a sales agreement with Cowen and Company, LLC, or TD Cowen, under which we could, from time to time, issue and sell shares of our common stock having aggregate sales proceeds of up to $200.0 million, in a series of one or more at-the-market equity offerings, or the 2023 ATM Program. TD Cowen is not required to sell any specific share amounts but acts as the Company’s sales agent, using commercially reasonable efforts consistent with its normal trading and sales practices. Pursuant to the sales agreement, shares will be sold under the shelf registration statement on Form S-3ASR (Registration No. 333-277424), which became automatically effective upon filing on February 27, 2024. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. For the three and six months ended June 30, 2024, we did not sell any shares of common stock under the 2023 ATM Program. As of July 25, 2024, we had $157.9 million available under the 2023 ATM Program.
Current wording
In May 2023, we entered into a sales agreement with Cowen and Company, LLC, or TD Cowen, under which we could, from time to time, issue and sell shares of our common stock having aggregate sales proceeds of up to $200.0 million, in a series of one or more at-the-market equity offerings, or the 2023 ATM Program. TD Cowen is not required to sell any specific share amounts but acts as the Company’s sales agent, using commercially reasonable efforts consistent with its normal trading and sales practices. Pursuant to the sales agreement, shares will be sold under the shelf registration statement on Form S-3ASR (Registration No. 333-277424), which became automatically effective upon filing on February 27, 2024. Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary. For the three and nine months ended September 30, 2024, we did not sell any shares of common stock under the 2023 ATM Program. As of September 30, 2024, we had $157.9 million available under the 2023 ATM Program.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
Net Cash Provided by Investing Activities