On February 13, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported its financial results for the quarter and full year ended December 31, 2024 and shared financial guidance for 2025 (Press release, Ultragenyx Pharmaceutical, FEB 13, 2025, View Source [SID1234650260]).

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"We have created a next-generation rare disease company on a pathway to profitability with meaningful revenue growth from multiple global products and a series of potential new drug approvals," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "The major regulatory and clinical catalysts ahead of us this year are the pending PDUFA decision for our gene therapy to treat Sanfilippo syndrome, submission of our second gene therapy biologics license application in Glycogen Storage Disease Type Ia, readout of our pivotal Phase 3 results in osteogenesis imperfecta, and completion of enrollment in our Phase 3 trial in Angelman syndrome."

Fourth Quarter and Full Year 2024 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended December 31, Year Ended December 31,
2024 2023 2024 2023
Crysvita
Product sales $ 22,415 $ 18,379 $ 134,709 $ 75,697
Revenue in Profit-Share Territory 85,534 70,124 248,966 231,574
Royalty revenue in European Territory 7,473 5,612 25,849 20,783
Total Crysvita Revenue 115,422 94,115 409,524 328,054
Dojolvi 31,103 23,286 88,194 70,633
Evkeeza 10,374 2,102 32,162 3,642
Mepsevii 7,978 7,889 30,350 30,441
Other — — — 1,479
Total revenues $ 164,877 $ 127,392 $ 560,230 $ 434,249

Total Revenues
Ultragenyx reported $165 million in total revenue for the fourth quarter of 2024, which represents 29% growth compared to the same period in 2023. Fourth quarter 2024 Crysvita revenue was $115 million, which represents 23% growth compared to the same period in 2023. This includes product sales of $22 million from Latin America and Turkey, which represents 22% growth compared to the same period in 2023. Dojolvi revenue in the fourth quarter 2024 was $31 million, which represents 34% growth compared to the same period in 2023. Evkeeza revenue in the fourth quarter 2024 was $10 million.

Total revenue for the year ended December 31, 2024 was $560 million, which represents 29% growth compared to the prior year. Full year 2024 Crysvita revenue was $410 million, which represents 25% growth compared to the prior year. This includes product sales of $135 million from Latin America and Turkey, which represents 78% growth compared to the prior year. Dojolvi revenue in 2024 was $88 million, which represents 25% growth compared to the prior year. Evkeeza revenue in 2024 was $32 million, as demand continues to build following launches in the company’s territories outside of the United States.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended December 31, Year Ended December 31,
2024 2023 2024 2023
Total revenues $ 164,877 $ 127,392 $ 560,230 $ 434,249
Operating expenses:
Cost of sales 16,894 12,051 76,728 45,209
Research and development 187,766 160,557 697,865 648,449
Selling, general and administrative 82,495 76,833 321,610 309,799
Total operating expenses 287,155 249,441 1,096,203 1,003,457
Net loss $ (133,385 ) $ (123,190 ) $ (569,183 ) $ (606,639 )
Net loss per share, basic and diluted $ (1.39 ) $ (1.52 ) $ (6.29 ) $ (8.25 )

Operating Expenses
Total operating expenses for the fourth quarter of 2024 were $287 million, including non-cash stock-based compensation of $40 million. Total operating expenses for the year ended December 31, 2024 were $1,096 million, including $158 million of non-cash stock-based compensation.

Net Loss
For the fourth quarter of 2024, Ultragenyx reported net loss of $133 million, or $1.39 per share basic and diluted, compared with a net loss for the fourth quarter of 2023 of $123 million, or $1.52 per share basic and diluted. For the year ended December 31, 2024, Ultragenyx reported net loss of $569 million, or $6.29 per share basic and diluted, compared with a net loss the prior year of $607 million, or $8.25 per share, basic and diluted.

Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $745 million as of December 31, 2024. Net cash used in operations for the year ended December 31, 2024 was $414 million.

2025 Financial Guidance
Revenues are expected to grow approximately 14-20% compared to 2024. The company will continue to prioritize expense management, focusing its investments on the execution of multiple upcoming commercial launches and advancing multiple Phase 3 programs. Together, this is expected to lead to a reduction in 2025 net cash used in operations compared to 2024.

For the full year 2025:

Total revenue to be in the range of $640 million to $670 million
Crysvita revenue to be in the range of $460 million to $480 million
Dojolvi revenue to be in the range of $90 million to $100 million
Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Phase 3 Orbit study progressing to second interim analysis (IA2) expected in mid-2025

Patients continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical trials, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing towards the second interim analysis in mid-2025 or a final analysis in the fourth quarter 2025. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2. Patients in the Cosmic study also are continuing to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim and final analyses.

GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study enrolling; expect enrollment completion in second half of 2025

Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to enroll approximately 120 children ages four to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Participants will be randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a 10% allocation of alpha) will be the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Enrollment in the Phase 3 Aspire study is expected to complete in the second half of 2025.

The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in 2025.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Biologics license application (BLA) submitted; expect Prescription Drug User Fee Act (PDUFA) decision on the application and potential launch in second half of 2025

In December 2024, Ultragenyx submitted a BLA to the U.S. Food and Drug Administration for UX111 supported by the available data, including from the ongoing pivotal Transpher A study. New clinical data were recently presented at WORLDSymposium 2025, that demonstrated treatment with UX111 led to a statistically significant improvement in the Bayley-III raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with MPS IIIA compared to natural history data from untreated patients. These clinical endpoints were correlated with substantial and sustained reduction in levels of heparan sulfate in cerebrospinal fluid. A PDUFA decision and potential launch are expected in the second half of 2025.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA filing expected in mid-2025

Ultragenyx previously announced positive topline results from the Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48.

After the 48-week primary efficacy analysis period, crossover patients (previously treated with placebo) were eligible to receive DTX401. These patients were able to titrate cornstarch much more rapidly once they were confirmed to have been treated and had timely direct access to their glucose levels. Patients from the original DTX401 treatment arm who have reached 78 weeks also continued to reduce their daily cornstarch intake, while maintaining glycemic control. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety concerns identified as of the data cut-off.

These results have been discussed with regulatory authorities in a pre-BLA meeting and will be included as part of a BLA submission in mid-2025.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Enrollment in Phase 3 study complete

Enrollment in the Phase 3 Enh3ance study has been completed with a total of 37 patients. The pivotal study enrolled participants 12 years of age and older, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by change in 24-hour ammonia levels and removal of ammonia-scavenger medications and protein-restricted diet. Based on the recently amended protocol, the change in 24-hour ammonia levels will be measured through Week 36 after which the study would unblind and patients will be followed for a total of up to 64 weeks to determine the complete responders able to remove safely both ammonia-scavenger medications and protein-restricted diet control.

UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; expect Cohort 4 enrollment completion in second half of 2025

In Stage 1 of the Phase 1/2/3 Cyprus2+ study, 15 patients across three sequential dose cohorts were enrolled and demonstrated clinical activity as well as improvements in copper metabolism. Multiple responders completely tapered off their standard-of-care treatment with responses seen in all three dose cohorts.

The company expects to enroll a fourth cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. Enrollment in Cohort 4 is expected to complete in the second half of 2025.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, February 13, 2025, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and full year 2024 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for three months.

On February 13, 2025 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer and obesity drugs, reported the receipt of newly issued patents in the United States and New Zealand, and updated investors on the extent of its global intellectual property portfolio (Press release, Bio-Path Holdings, FEB 13, 2025, View Source [SID1234650244]).

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Bio-Path received Notice of Allowance from the United States Patent and Trademark Office for U.S. Patent No. 17/339,366 titled, "P-ethoxy nucleic acids for STAT3 inhibition." The New Zealand Intellectual Property Office has granted Patent No. 741793 titled, "P-ethoxy nucleic acids for liposomal formulation." These new patents build on earlier patents granted that protect the platform technology for DNAbilize, the Company’s novel RNAi nanoparticle drugs.

"We are proud of our growing global intellectual property portfolio as it safeguards our technology, serves as a deterrent to would-be competitors and creates value around our core competencies," said Peter Nielsen, Chief Executive Officer of Bio-Path. "These efforts are designed to protect our investments and advance our clinical programs to bring new medicines to patients suffering with obesity and cancer. Importantly, our newly issued U.S. patent provides additional intellectual property protection around our STAT3 program, where we intend to advance into non-small cell lung cancer."

Bio-Path continues to expand its intellectual property portfolio by filing patent applications that are applicable to its technology and business strategy. Bio-Path’s patent portfolio currently includes seven issued patents in the U.S. and 61 issued patents in foreign jurisdictions, providing protection in 26 countries. The Company has three additional pending patent applications in the U.S. and five additional allowed patent applications in foreign jurisdictions.

On February 13, 2025 Oncotelic Therapeutics, Inc (OTCQB:OTLC). Oncotelic Therapeutics, a leader in RNA-based therapeutics, reported that Vuong Trieu, Ph.D., Chairman & CEO, will be delivering a featured presentation at the SWCR 2025 Conference on March 24th (Press release, Oncotelic, FEB 13, 2025, View Source [SID1234650261]). His talk will highlight the central role of Transforming Growth Factor Beta 2 (TGFB2) in the convergence of disease pathways spanning cancer, lupus, and aging, as well as its potential as a therapeutic target in immuno-oncology.

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Dr. Trieu’s presentation, titled "Transforming Growth Factor Beta 2 in Aging, Cancer, Lupus, and Immuno-Oncology: A Convergence of Disease Pathways and Therapeutic Potential," will delve into the immunosuppressive mechanisms of TGFB2 and its profound impact on solid tumors, autoimmune disorders, and age-related diseases. His research underscores how high TGFB2 expression fosters tumor-associated macrophage infiltration, disrupts antigen presentation, and drives resistance to immunotherapy-ultimately contributing to poor patient outcomes.

A key focus of the presentation will be the clinical development of OT-101, an antisense therapeutic targeting TGFB2, which is currently in Phase 3 clinical trials (STOP-PC study) for pancreatic ductal adenocarcinoma (PDAC). The talk will explore how TGFB2 inhibition-alone or in combination with immune checkpoint inhibitors or interferon-based strategies-may reshape the tumor microenvironment, boost immune response, and enhance therapeutic efficacy.

Dr. Vuong Trieu’s Vision for TGFB2 as a Therapeutic Target

"TGFB2 has emerged as a master regulator of immune suppression, not just in cancer but across multiple chronic diseases," said Dr. Trieu. "Our work on OT-101 aims to neutralize TGFB2-driven resistance, unlocking the full potential of immunotherapy and improving outcomes for patients with pancreatic cancer, gliomas, and beyond."

On February 13, 2025 (the "Closing Date"), Sarepta Therapeutics, Inc. (the "Company") and Sarepta Therapeutics Investments, Inc., a wholly owned subsidiary of the Company (together with the Company, the "Obligors") reported to have entered into a credit agreement (the "Credit Agreement") with JPMorgan Chase Bank, N.A., as administrative agent (in such capacity, the "Administrative Agent") and as collateral agent (in such capacity, the "Collateral Agent") and the lenders party thereto (Filing, 8-K, Sarepta Therapeutics, FEB 13, 2025, View Source [SID1234650287]). The Credit Agreement provides for a five-year, $600 million senior secured revolving credit facility (the "Revolving Credit Facility").

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Interest rates under the Revolving Credit Facility are variable and equal to the Secured Overnight Financing Rate plus a credit spread adjustment of 0.10% per annum ("Adjusted SOFR"), plus a margin of 1.125% to 1.75% per annum based on the Company’s total gross leverage ratio, or, at the Company’s option, at a base reference rate equal to the highest of (a) the federal funds rate plus 0.50%, (b) the rate of interest last quoted by the Administrative Agent as its "base rate" and (c) the one-month Adjusted SOFR rate plus 1.00%, plus a margin of 0.125% to 0.75% per annum based on the Company’s total gross leverage ratio.

The Company will pay customary agency fees and a commitment fee based on the daily unused portion of the Revolving Credit Facility at a rate of 0.20% to 0.35% per annum based on the Borrower’s total gross leverage ratio. The Revolving Credit Facility is not subject to amortization and will mature on the fifth anniversary of the Closing Date.

On the Closing Date, each of the Obligors and the Collateral Agent entered into a pledge and security agreement, pursuant to which the Obligors granted a security interest in substantially all of their respective assets, in each case, subject to customary exceptions and exclusions.

The Credit Agreement contains customary representations and warranties, affirmative covenants, negative covenants and events of default. The Credit Agreement also contains financial covenants that are tested on the last day of each of the Company’s fiscal quarters. These financial covenants include a (x) maximum secured net leverage ratio of 3.5:1.0, subject to a 4.0:1.0 covenant holiday following certain permitted acquisitions or permitted collaborations, and (y) minimum consolidated interest coverage ratio of 2.5:1.0.

The foregoing description of the Credit Agreement does not purport to be complete and is subject to, and qualified in its entirety by reference to, the full text of the Credit Agreement, attached hereto as Exhibit 10.1 and incorporated herein by reference.

On February 13, 2025 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2024 and provided financial guidance (Press release, Blueprint Medicines, FEB 13, 2025, View Source [SID1234650245]).

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"We are entering 2025 in the strongest position we have ever been in as a company with a focus on driving growth and innovation through operational excellence. AYVAKIT is a medicine that is changing patients’ lives, and the SM market is larger and growing faster than we originally expected. Our 2025 guidance of $680 to $710 million represents robust growth, putting AYVAKIT firmly on track to achieve $2 billion in revenue by 2030. And with the larger potential SM franchise peak opportunity of $4 billion, we have a plan to drive innovation in the treatment of SM with elenestinib as we move beyond symptom control to disease modification in the HARBOR registration study, building a durable SM franchise that we anticipate will grow throughout the next decade," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Our next potential blockbuster opportunity, BLU-808, is also coming into focus with the healthy volunteer data reported last month. BLU-808 demonstrated a best-in-class profile for an oral wild-type KIT inhibitor enabling our differentiated approach to modulating mast cell activity across thousands of patients with mast-cell-mediated allergic and inflammatory diseases. BLU-808 offers us the opportunity to find the right balance of efficacy and tolerability to realize true pipeline-in-a-medicine potential. Altogether, our portfolio represents a unique set of assets, targeting fundamental mast cell biology, that will drive durable and diversified growth over the next decade with significant operating leverage."

Fourth Quarter 2024 Highlights and Recent Progress

Mast cell disorders

● Achieved AYVAKIT net product revenues of $479.0 million, including $421.8 million in the US and $57.1 million ex-US, representing 135 percent growth year-over-year. In the fourth quarter of 2024, AYVAKIT achieved $144.1 million, including $124.1 million in the US and $20.0 million ex-US.
● Blueprint estimates the peak revenue opportunity for the company’s SM franchise is $4 billion, with $2 billion in annual revenues expected to be achieved by AYVAKIT by 2030.
● Presented AYVAKIT data at the 2024 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, showing a significant survival benefit in treatment-naïve patients with advanced SM, and sustained improvement in bone density for advanced SM patients with low bone mass at baseline. View the presentations here.
● Reported in January 2025 results from the Phase 1 healthy volunteer study of BLU-808, a wild-type KIT inhibitor, showing a differentiated profile that enables the evaluation of tunable dosing strategies. BLU-808 was well-tolerated at all doses tested, showed consistent pharmacokinetics supporting once daily oral dosing, and achieved dose-dependent reductions in tryptase exceeding 80 percent. These positive data support initiation of multiple proof-of-concept studies planned in 2025.

Corporate

● Presented the company’s 2025 corporate outlook and strategy to drive continued growth at the 43rd Annual J.P. Morgan Healthcare Conference. View the press release here.
● Following closing of the IDRx acquisition by GSK, Blueprint anticipates approximately $80 million in gross proceeds from an equity stake in IDRx.
● Announced the appointment of Sherwin Sattarzadeh to Chief Business Officer, overseeing business development, portfolio leadership and project management, and alliance management. His previous roles over a decade of service at Blueprint include Head of Regulatory Affairs, Chief of Staff to the Chief Executive Officer, and Senior Vice President of Strategic Operations. He succeeds Helen Ho, Ph.D. who is departing the company to pursue another career opportunity.

2025 Financial Guidance

Blueprint reported it anticipates approximately $680 million to $710 million in global AYVAKIT net product revenues for all approved indications in 2025. The midpoint of this range represents 45 percent year-over-year revenue growth. In 2024, Blueprint reduced cash burn by more than 50 percent and expects further reduction in 2025 as the company continues to invest in advancing prioritized programs, balancing investing in innovation with financial discipline. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will maintain a durable capital position to enable the company to achieve a self-sustainable financial profile.

Key Upcoming Milestones

The company plans to achieve the following milestones in the first half of 2025:

● Present 14 abstracts at the 2025 American Academy of Allergy, Asthma and Immunology (AAAAI) / World Allergy Organization (WAO) Joint Congress, including:
o Three-year safety and efficacy data from the PIONEER trial of AYVAKIT.
o Bone density improvements shown by AYVAKIT in the PIONEER trial.
o Phase 1 healthy volunteer data for BLU-808, a highly potent and selective oral inhibitor of wild-type KIT, consistent with top-line results reported in January 2025.
● Initiate two proof-of-concept studies of BLU-808 in patients with chronic spontaneous urticaria/chronic inducible urticaria, and allergic rhinitis/allergic conjunctivitis.

Fourth Quarter and Year End 2024 Results

● Revenues: Revenues were $146.4 million for the fourth quarter of 2024, including $144.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.2 million in collaboration revenues. Revenues for the year ended December 31, 2024 were $508.8 million, including $479.0 million of net product revenues from sales of AYVAKIT/AYVAKYT, and $29.9 million in collaboration and license revenues. Blueprint Medicines recorded $72.0 million and $249.4 million in revenues in the fourth quarter and year ended December 31, 2023, respectively.
● Cost of Sales: Cost of sales was $7.4 million for the fourth quarter of 2024 and $20.2 million for the year ended December 31, 2024, as compared to $0.3 million and $8.5 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to higher sales to our collaboration and other partners and an increase in product sales volume.
● R&D Expenses: Research and development expenses were $83.7 million for the fourth quarter of 2024 and $341.4 million for the year ended December 31, 2024, as compared to $97.5 million and $427.7 million for the fourth quarter and year ended December 31, 2023, respectively. This decrease was primarily due to operational efficiency across our portfolio as we executed across our top priority programs, and the timing of manufacturing of clinical trial materials. Research and development expenses also included $11.7 million in stock-based compensation expenses for the fourth quarter of 2024 and $47.5 million in stock-based compensation for the year ended December 31, 2024.
● SG&A Expenses: Selling, general and administrative expenses were $96.5 million for the fourth quarter of 2024 and $359.3 million for the year ended December 31, 2024, as compared to $79.3 million and $295.1 million for the fourth quarter and year ended December 31, 2023, respectively. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $16.6 million in stock-based compensation expenses for the fourth quarter of 2024 and $61.5 million in stock-based compensation for the year ended December 31, 2024.
● Net Loss: Net loss was $(50.0) million for the fourth quarter of 2024 and $(67.1) million for the year ended December 31, 2024, or a diluted net loss per share of $(0.79) and diluted net loss per share of $(1.07), respectively, as compared to a net loss of $(110.9) million for the fourth quarter of 2023 and a net loss of $(507.0) million for the year ended December 31, 2023, or a diluted net loss per share of $(1.82) and a diluted net loss per share of $(8.37), respectively.
● Cash Position: As of December 31, 2024, cash, cash equivalents and investments were $863.9 million, as compared to $767.2 million as of December 31, 2023.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss fourth quarter and full year 2024 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international), and referring to conference ID 349846. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences

Blueprint Medicines will participate in two upcoming investor conferences:

● 45th Annual Cowen Health Care Conference on Monday, March 3, 2025 at 1:10 p.m. ET.
● H.C. Wainwright 3rd Annual Autoimmune & Inflammatory Disease Virtual Conference on Thursday, March 27, 2025 at 12:00 p.m. ET.

Live webcasts of the presentations will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source Replays of the webcasts will be archived on the Blueprint Medicines website for 30 days following the presentations.