On February 15, 2024 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, reported financial results for the fiscal quarter and fiscal year ended November 30, 2023, and provided a corporate update (Press release, Nurix Therapeutics, FEB 15, 2024, View Source [SID1234640146]).

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"Building on a very successful 2023, marked by impressive clinical data for both NX-5948 and NX-2127, Nurix has hit the ground running in 2024, with plans to accelerate enrollment in the NX-5948 leukemia and lymphoma program and enable development in inflammatory diseases," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "2023 was also a great year for our partnerships, generating significant non-dilutive funding and expanding our pipeline in both oncology and inflammation with our IRAK-4 degrader. We anticipate continued success with our partners Gilead, Sanofi, and Pfizer in the coming year."

Recent Business Highlights

•Nurix presented clinical data for Bruton’s tyrosine kinase (BTK) degrader NX-5948 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting: In December 2023, Nurix reported data from the dose escalation stage of the Phase 1 trial demonstrating dose-dependent pharmacokinetics (PK), resulting in rapid, robust, and sustained BTK degradation in all patients treated. NX-5948 was well-tolerated across all doses. Preliminary efficacy data demonstrated clinical benefit in six of seven patients with chronic lymphocytic leukemia (CLL). Durable responses were seen across indications in non-Hodgkin lymphoma (NHL) patients, with almost half the patients continuing to receive treatment as of the data cut-off date. Dose escalation in the NX-5948 trial continues across all indications and the study is actively enrolling patients in the United States, the United Kingdom, and the Netherlands.
•NX-5948 received U.S. FDA Fast Track designation: In January 2024, the FDA granted Fast Track designation for NX-5948 for the treatment of adult patients with relapsed or refractory CLL or small lymphocytic lymphoma after at least two lines of therapy, including a BTK inhibitor (BTKi) and a B-cell lymphoma 2 (BCL2) inhibitor. The FDA’s Fast Track designation is intended to facilitate and expedite the development and review of drug candidates to treat serious conditions and fulfill an unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.
•Nurix presented clinical data for NX-2127, a dual BTK and IKZF1/3 degrader, at the ASH (Free ASH Whitepaper) Annual Meeting: In December 2023, Nurix reported data from its Phase 1a dose escalation and Phase 1b dose expansion cohorts in CLL, mantle cell lymphoma (MCL) and diffuse large B-cell lymphoma (DLBCL). NX-2127 exhibited dose-dependent PK, leading to robust and sustained degradation of BTK and biologically relevant degradation of IKZF1 (Ikaros). Treatment with NX-2127 resulted in encouraging rapid and durable responses in the heavily pre-treated patient population including patients with BTK inhibitor resistance mutations. Durable complete responses were reported in two patients with MCL and DLBCL which remained ongoing for over one year. NX-2127 had a manageable safety profile that was consistent with previous reports for BTK-targeted and immunomodulatory therapies.

•High profile publications provide scientific basis for BTK scaffold function and degrader mechanism: In February 2024, Nurix announced the publication of a manuscript in the journal Science titled: "Kinase Impaired BTK Mutations Are Susceptible to Clinical Stage BTK and IKZF1/3 Degrader NX-2127" that elucidates a previously unappreciated oncogenic scaffold function of BTK responsible for clinical resistance to enzymatic inhibitors and shows that NX-2127 can overcome this resistance across a broad range of acquired mutations. A second manuscript was published contemporaneously in The Journal of Medicinal Chemistry entitled "Discovery and Preclinical Pharmacology of NX-2127, an Orally Bioavailable Degrader of Bruton’s Tyrosine Kinase with Immunomodulatory Activity for the Treatment of Patients with B Cell Malignancies," which details the discovery and optimization of NX-2127.
Upcoming Program Highlights*
•NX-5948: NX-5948 is an investigational, orally bioavailable, small molecule degrader of BTK. NX-5948 is currently being evaluated in a Phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies. In 2024, Nurix expects to define doses for Phase 1b cohort expansion in CLL and NHL and accelerate Phase 1 clinical trial enrollment to enable pivotal trials. Nurix plans to present additional clinical data with higher dose levels and longer treatment duration in mid-2024. In addition, Nurix expects to complete ongoing preclinical studies that can enable an investigational new drug (IND) application for NX-5948 in autoimmune indications. Additional information on the clinical trial can be accessed at www.clinicaltrials.gov (NCT05131022).

•NX-2127: NX-2127 is an orally bioavailable degrader of BTK with immunomodulatory activity for the treatment of patients with relapsed or refractory B-cell malignancies. Nurix is conducting a Phase 1a/b clinical trial of NX-2127, which includes three Phase 1b expansion cohorts in patients with DLBCL, MCL and CLL. Screening and enrollment of new study participants have been paused due to a partial clinical hold placed on the study by the FDA. Patients currently enrolled in the clinical study who are deriving clinical benefit may continue to receive treatment in accordance with the ongoing study protocol. In 2024, Nurix expects to resolve the partial clinical hold to enable the introduction of newly manufactured drug product into the ongoing Phase 1 clinical trial. Additional information on the clinical trial can be accessed at www.clinicaltrials.gov (NCT04830137).

•NX-1607: Nurix’s lead drug candidate from its targeted protein elevation portfolio, NX-1607, is an orally bioavailable inhibitor of the E3 ligase Casitas B-lineage lymphoma proto-oncogene B (CBL-B) for immuno-oncology indications including a range of solid tumor types and lymphoma. Nurix is evaluating NX-1607 in an ongoing, Phase 1 trial in monotherapy and in a combination cohort utilizing paclitaxel in adults in a range of oncology indications. In 2024, Nurix expects to present data from the Phase 1a dose-escalation portion of the trial of NX-1607 and to define dose(s) to enable Phase 1b cohort expansion. Additional information on the clinical trial can be accessed at www.clinicaltrials.gov (NCT05107674).
•NX-0479/GS-6791: GS-6791 (previously NX-0479) is a potent, selective, oral IRAK4 degrader. Degradation of IRAK4 by GS-6791 has potential applications in the treatment of rheumatoid arthritis and other inflammatory diseases. Nurix’s partner, Gilead, is responsible for conducting IND-enabling studies and advancing this program to clinical development.
•Selection of new drug candidate: Nurix expects to select a new targeted protein degrader development candidate in 2024.
•Continued pipeline advancement of strategic collaborations with Gilead, Sanofi and Pfizer: Nurix expects to continue to achieve substantial research collaboration milestones throughout the terms of its collaborations with Gilead, Sanofi and Pfizer.
*Expected timing of events throughout this press release is based on calendar year quarters.
Fiscal Fourth Quarter and Full Year 2023 Financial Results
Revenue for the three months and twelve months ended November 30, 2023, was $15.2 million and $77.0 million, respectively, compared with $6.8 million and $38.6 million for the three and twelve months ended November 30, 2022, respectively. The increase for the twelve-month period was primarily due to a higher percentage of completion of performance obligations and an increase in the value of milestones achieved in the current period. The increase was also due to the receipt of $20.0 million related to the license option exercise payment from Gilead. During the year ended November 30, 2023, Nurix achieved research milestones under its collaborations with Gilead and Sanofi totaling $12.5 million and $7.0 million, respectively.

Research and development expenses for the three months and twelve months ended November 30, 2023, were $49.7 million and $189.1 million, respectively, compared to $46.1 million and $184.5 million for the three and twelve months ended November 30, 2022, respectively. For the twelve-month period, there was an increase in compensation and related personnel costs and an increase in clinical costs as Nurix continued its clinical trial programs and ongoing patient enrollment, offset by a decrease in research related costs and in contract manufacturing.
General and administrative expenses for the three months and twelve months ended November 30, 2023, were $10.8 million and $42.9 million, respectively, compared to $9.4 million and $38.0 million for the three and twelve months ended November 30, 2022, respectively. The increase for the twelve-month period was primarily related to an increase in non-cash stock-based compensation expense and an increase in professional service costs related to the Pfizer collaboration agreement, offset by a decrease in outside consulting costs.
Net loss for the three months and twelve months ended November 30, 2023, was $42.0 million or ($0.77) per share and $143.9 million or ($2.65) per share, respectively, compared with $46.7 million or ($0.87) per share and $180.4 million or ($3.71) per share for the three and twelve months ended November 30, 2022, respectively.
Cash, cash equivalents and marketable securities was $295.3 million as of November 30, 2023, compared to $268.7 million as of August 31, 2023.

On February 15, 2024 Labcorp (NYSE: LH), a global leader of innovative and comprehensive laboratory services, reported results for the fourth quarter and year ending Dec. 31, 2023, and full year 2024 guidance (Press release, LabCorp, FEB 15, 2024, View Source [SID1234640145]).

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Labcorp Logo (PRNewsfoto/Labcorp)

"We made significant progress on our strategy in 2023 and delivered strong results," said Adam Schechter, chairman and CEO. "Our teams developed and brought important diagnostic testing advancements to market and supported our biopharma clients as they advanced their new drug pipelines and treatments. With the successful integration of several hospital partnerships including Ascension, and the spin of Fortrea, we enter 2024 with a strong foundation and momentum. We remain focused on advancing science, technology and innovation to fuel our growth and fulfill our mission to improve health and improve lives."

In the fourth quarter and throughout the year, Labcorp executed on its enterprise strategy. The company closed its previously announced transaction with Legacy Health to acquire select assets of Legacy’s outreach laboratory business. Labcorp now manages Legacy’s inpatient hospital laboratories, serving patients throughout Oregon and Southwest Washington state.

In November, Ovia Health by Labcorp announced that it will offer a first-of-its-kind Fertility and Family Building Benefit, allowing employers and health plans to offer customizable solutions to employees and health plan members to support their family-building needs. Additionally, the company also became the first lab to launch a new, FDA-cleared blood test for risk assessment and clinical management of severe preeclampsia.

Last month, the company announced a strategic collaboration with Hawthorne Effect, Inc. to create new service offerings that support decentralized clinical trials and aim to improve the patient experience, accessibility, and efficiency.

On January 12, 2024, the company announced a quarterly cash dividend of $0.72 per share of common stock, payable on March 13, 2024, to stockholders of record at the close of business on February 27, 2024.

Consolidated Results

Fourth Quarter Results

Revenue for the quarter was $3.03 billion, an increase of 3.5% from $2.93 billion in the fourth quarter of 2022. The increase was due to organic revenue of 1.5%, acquisitions, net of divestitures of 1.4%, and foreign currency translation of 0.7%. The 1.5% increase in organic revenue was driven by a 5.0% increase in the company’s organic Base Business, partially offset by a (3.5%) decrease in COVID-19 PCR and antibody testing (COVID-19 Testing). Compared to the Base Business last year, Base Business revenue grew 7.4%. Base Business includes Labcorp’s operations except for COVID-19 Testing.

Operating loss for the quarter was ($122.8) million, or (4.0)% of revenue, compared to operating income of $28.5 million, or 1.0%, in the fourth quarter of 2022. The company recorded impairment charges, amortization, restructuring charges, and special items, which together totaled $517.6 million in the quarter, compared to $384.0 million during the same period in 2022. Included in these numbers are goodwill and other asset impairment charges of $333.8 million compared to $260.5 million in the fourth quarter 2022, which are primarily related to the Early Development business. Adjusted operating income (excluding impairment charges, amortization, restructuring charges, and special items) for the quarter was $394.9 million, or 13.0% of revenue, compared to $412.6 million, or 14.1%, in the fourth quarter of 2022. The decrease in adjusted operating income was due to a reduction in COVID-19 Testing.

Net losses from continuing operations for the quarter were $(166.8) million compared to net earnings of $37.1 million in the fourth quarter of 2022. Diluted EPS from continuing operations were $(1.95) in the quarter compared to $0.42 during the same period in 2022. Adjusted EPS (excluding impairment charges, amortization, restructuring charges, and special items) were $3.30 in the quarter compared to $3.05 in the fourth quarter of 2022.

Operating cash flow from continuing operations for the quarter was $579.6 million compared to $607.2 million in the fourth quarter of 2022. The decrease in operating cash flow was due to lower COVID-19 Testing. Capital expenditures totaled $165.4 million compared to $99.1 million a year ago. The increase in capital expenditures was primarily timing related. As a result, free cash flow from continuing operations (operating cash flow less capital expenditures) was $414.2 million compared to $508.1 million in the fourth quarter of 2022.

At the end of the quarter, the company’s cash balance and total debt were $0.54 billion and $5.05 billion, respectively. During the quarter, the company invested $154.8 million on acquisitions, paid out $61.1 million in dividends, and settled its accelerated share repurchase program, receiving approximately 1.1 million additional shares. At the end of the quarter, the company had $530.4 million of share repurchase authorization remaining.

Full Year Results

Revenue was $12.16 billion, an increase of 2.5% from $11.86 billion in 2022. The increase was due to acquisitions, net of divestitures of 1.7%, organic revenue of 0.6%, and favorable foreign currency translation of 0.2%. The 0.6% increase in organic revenue was due to an 8.7% increase in the Company’s organic Base Business, partially offset by an (8.1%) decrease in COVID-19 Testing.

Operating income was $725.6 million, or 6.0% of revenue, compared to $1,436.5 million, or 12.1%, in 2022. The company recorded impairment charges, amortization, restructuring charges, and special items, which together totaled $989.3 million compared to $736.0 million during 2022. Adjusted operating income (excluding impairment charges, amortization, restructuring charges, and special items) was $1.71 billion, or 14.1% of revenue, compared to $2.17 billion, or 18.3%, in 2022. The decrease in adjusted operating income was due to lower COVID-19 Testing.

Net earnings from continuing operations were $380.4 million compared to $1,003.5 million in 2022. Diluted EPS were $4.33 compared to $10.94 in 2022. Adjusted EPS (excluding impairment charges, amortization, restructuring charges, and special items) were $13.56 compared to $16.66 in 2022.

Operating cash flow from continuing operations was $1.20 billion compared to $1.76 billion in 2022. The decrease in operating cash flow was primarily due to lower COVID-19 Testing and items related to the Fortrea spin-off. Capital expenditures totaled $453.6 million compared to $429.3 million in 2022. As a result, free cash flow from continuing operations (operating cash flow less capital expenditures) was $748.7 million compared to $1,335.5 million in 2022.

During the year the company repurchased $1.00 billion of stock representing approximately 4.8 million shares and invested $0.67 billion on acquisitions.

Fourth Quarter Segment Results

The following segment results exclude impairment charges, amortization, restructuring charges, special items, and unallocated corporate expenses.

Diagnostics Laboratories

Revenue for the quarter was $2.35 billion, an increase of 2.6% from $2.29 billion in the fourth quarter of 2022. The increase was primarily due to acquisitions of 1.8% and organic revenue of 0.8%. The 0.8% increase in organic revenue was due to a 5.3% increase in the Base Business, partially offset by a (4.5%) decrease in COVID-19 Testing. Total Base Business growth compared to the Base Business in the prior year was 7.6%.

Total volume (measured by requisitions) increased by 2.4% as organic volume increased by 0.3% and acquisition volume contributed 2.1%. Organic volume was impacted by a (2.6%) decrease in COVID-19 Testing, partially offset by a 3.0% increase in Base Business. Price/mix increased by 0.2% due to organic base business growth of 2.4%, partially offset by COVID-19 Testing of (1.9%), and acquisitions of (0.3%). Base Business volume increased 5.2% compared to the Base Business last year. Price/mix was up 2.4% in the Base Business compared to the Base Business last year.

Adjusted operating income for the quarter was $353.7 million, or 15.1% of revenue, compared to $387.0 million, or 16.9%, in the fourth quarter of 2022. The decrease in adjusted operating income was due to a reduction in COVID-19 Testing. The benefit of higher organic demand, acquisitions, and launchpad savings was partially offset by higher personnel costs. The decrease in adjusted operating income margin was due to the reduction in COVID-19 Testing and the mix impact from recently closed hospital partnerships.

Biopharma Laboratory Services

Revenue for the quarter was $694.8 million, an increase of 7.1% from $648.8 million in the fourth quarter of 2022. The increase was primarily due to organic revenue of 4.0%, and foreign currency translation of 3.1%.

Adjusted operating income for the quarter was $109.0 million, or 15.7% of revenue, compared to $95.2 million, or 14.7%, in the fourth quarter of 2022. Adjusted operating income and margin increased due to organic growth and LaunchPad savings, partially offset by higher personnel and stranded costs.

Net orders and net book-to-bill during the trailing twelve months were $2.89 billion and 1.04, respectively. Backlog at the end of the quarter was $8.25 billion, an increase of 4.9% compared to last year. The company expects approximately $2.47 billion of its backlog to convert into revenue in the next twelve months.

2024 Guidance

The following guidance assumes foreign exchange rates effective as of Dec. 31, 2023, for the full year. Enterprise level guidance includes the estimated impact from currently anticipated capital allocation, including acquisitions, share repurchases and dividends.

On February 15, 2024 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported that members of its senior management team will present at the Citi 2024 Virtual Oncology Leadership Summit on Wednesday, February 21, 2024, at 1:00 p.m. ET (Press release, CRISPR Therapeutics, FEB 15, 2024, View Source [SID1234640142]).

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A live webcast of the fireside chat will be available on the "Events & Presentations" page in the Investors section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 14 days following the presentation.

On February 15, 2024 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, reported its results for the quarter and year ended December 31, 2023 (Press release, Corcept Therapeutics, FEB 15, 2024, View Source [SID1234640140]).

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Financial Results

•Fourth quarter revenue of $135.4 million, a 31 percent increase over the same period in 2022
•Full year 2023 revenue of $482.4 million, a 20 percent increase over 2022
•Reiterated 2024 revenue guidance of $600 – $630 million
•Fourth quarter net income of $31.4 million, compared to $16.6 million in fourth quarter 2022
•2023 net income of $106.1 million, compared to $101.4 million in 2022
•Cash and investments of $425.4 million as of December 31, 2023
"The medical field is increasingly recognizing that Cushing’s syndrome is much more prevalent than was previously assumed. Our strong 2023 commercial results reflect that physicians are more regularly screening for hypercortisolism. The results demonstrate our ability to support these physicians as they manage this complex disease. We are confident these trends will continue and are reiterating our 2024 revenue guidance of $600 – $630 million," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer.
Corcept’s fourth quarter 2023 revenue was $135.4 million, compared to $103.1 million in the fourth quarter of 2022. Revenue for the full year was $482.4 million, compared to $401.9 million in 2022.
Diluted net income per common share was $0.28 in the fourth quarter of 2023, compared to $0.14 in the fourth quarter of 2022. For the full year, it was $0.94 compared to $0.87 in 2022.
Cash and investments were $425.4 million at December 31, 2023 compared to $436.6 million at December 31, 2022. In 2023, Corcept paid $154.5 million to purchase its common stock in connection with the April 2023 tender offer, the exercise of employee stock options and vesting of restricted stock grants.
Clinical Development
"Our clinical development programs are advancing rapidly and will reach important milestones this year. We are on-track to submit our NDA for relacorilant in Cushing’s syndrome and will report data from our trials in Cushing’s syndrome (GRACE, GRADIENT and CATALYST), ovarian cancer (ROSELLA) and ALS (DAZALS)," added Dr. Belanoff.
Cushing’s Syndrome
•GRACE – Phase 3 trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome – results from open label and randomized withdrawal phases expected in the second quarter
•Relacorilant New Drug Application (NDA) – NDA submission for Cushing’s syndrome expected in the second quarter
•GRADIENT – Phase 3 trial of relacorilant in patients with Cushing’s syndrome caused by adrenal adenomas – enrollment continues; results expected in the second half of this year
•CATALYST – Phase 4 trial examining the prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes; patients with hypercortisolism may enter a randomized, double-blind, placebo-controlled study of Korlym – enrollment continues; preliminary prevalence phase results: 24%

prevalence rate in the first 700 patients enrolled; final results from prevalence and treatment phases expected by year-end
"We are on-track to submit our relacorilant NDA in the second quarter. Relacorilant has demonstrated tremendous promise as a treatment for patients with Cushing’s syndrome and we are eager to make it more broadly available," said Bill Guyer, PharmD, Corcept’s Chief Development Officer.
"Our CATALYST trial is the largest study ever conducted to establish the prevalence of hypercortisolism in patients with difficult-to-control diabetes. We expect CATALYST’s findings to greatly stimulate physicians’ efforts to screen for hypercortisolism in patients with difficult-to-control diabetes and to treat them when hypercortisolism is found. Almost all of these patients currently go undiagnosed. We believe that CATALYST will be a landmark study and look forward to presenting the final results from the prevalence phase at the American Diabetes Association’s 84th Scientific Sessions in June," added Dr. Guyer.
Oncology
•ROSELLA – Pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with platinum-resistant ovarian cancer – enrollment continues; results expected by year-end
•Open-label, Phase 1b trial of relacorilant plus pembrolizumab in patients with adrenal cancer with cortisol excess – enrollment completed; results expected by mid-year
•Randomized, placebo-controlled, Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer in collaboration with the University of Chicago – enrollment continues
"If our ROSELLA trial replicates the results of our Phase 2 trial in patients with platinum-resistant ovarian cancer (results published in The Journal of Clinical Oncology, June 2023), it will constitute a major medical advance and could establish the combination of relacorilant and nab-paclitaxel as a new standard of care for women with this devastating disease. We expect data from ROSELLA by the end of this year," said Dr. Guyer.
Amyotrophic Lateral Sclerosis (ALS)
•DAZALS – Randomized, double-blind, placebo-controlled, Phase 2 trial of dazucorilant in patients with ALS – enrollment continues; results expected by year-end
"ALS is a lethal illness with an urgent need for better treatment. Dazucorilant showed great promise in animal models of ALS – improving motor performance and reducing neuroinflammation and muscular atrophy. Our DAZALS study is investigating dazucorilant’s potential to significantly improve the lives of patients with ALS. We expect data by the end of this year," said Dr. Guyer.
Non-alcoholic Steatohepatitis (NASH)
•MONARCH – Randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in patients with biopsy-confirmed NASH – enrollment continues
"Miricorilant has the potential to greatly benefit the millions of patients with NASH. Our Phase 1b study demonstrated that miricorilant effectively reduces liver fat, improves liver health and key metabolic and lipid measures and is well-tolerated. We look forward to building on these promising results in our MONARCH study," said Dr. Guyer.

Conference Call

We will hold a conference call on February 15, 2024, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number and a unique access PIN. Each access PIN will accommodate one caller.

On February 15, 2024 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2023 and provided financial guidance (Press release, Blueprint Medicines, FEB 15, 2024, View Source [SID1234640138]).

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"As we enter 2024, AVAYKIT continues to be the foundation of our thriving and growing commercial business. Our 2024 guidance of $360 to $390 million sets us up to nearly double AYVAKIT product revenue this year, driven by the launch in ISM as we focus on reaching more patients in both the U.S. and Europe. AYVAKIT’s compelling efficacy and safety profile, coupled with the chronic nature of ISM, make the cumulative effect of patients staying on therapy an important revenue driver this year, beyond and in addition to new patient starts. AYVAKIT is firmly on the path to becoming a multibillion-dollar product, providing Blueprint with durable revenue growth well into the next decade. Beyond AYVAKIT and ISM, we are balancing investment in our most compelling pipeline opportunities and maintaining financial discipline, strengthening our financial profile, and accelerating our path to profitability," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "We are particularly excited about our expanding portfolio targeting allergic-inflammatory diseases where mast cells play a central role, and we are looking forward to sharing more on these programs throughout this year."

Fourth Quarter 2023 Highlights and Recent Progress

Mast cell disorders

·

Achieved AYVAKIT net product revenues of $204.2 million and $71.0 million for the full year and the fourth quarter of 2023, respectively, representing 84 percent growth year-over-year.

·

Announced approval by the European Commission for AYVAKYT as the first and only treatment for indolent systemic mastocytosis (ISM). Read the press release here.

·

Presented data demonstrating the compelling benefit-risk profile of elenestinib in ISM from Part 1 of the HARBOR trial and analyses of real-world data highlighting the burden of and urgency to treat ISM at the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. Read the presentations here.

Breast cancer and other solid tumors

·

Advanced the development of BLU-222 in combination with ribociclib and fulvestrant in patients with HR+/HER2- breast cancer.

·

Announced the development candidate nomination of BLU-956, a next-generation CDK2 inhibitor.

Corporate

·

Presented a 2024 corporate overview and strategy at the J.P. Morgan 42nd Annual Healthcare Conference. Read the press release here.

·

Identified an alternate partner for GAVRETO (pralsetinib) in the U.S. and are working with all involved parties to enable continuity of access to GAVRETO in the U.S. The company plans to provide an update to coincide with the late February 2024 termination date of the existing collaboration agreement with Roche.

·

The company continues to expect that the wind-down of the Roche collaboration for GAVRETO and the discontinuation of global development and marketing in territories outside the U.S. and Greater China will result in significantly lower year-over-year operating expenses related to GAVRETO and will have no material impact to its overall operating expense plans in 2024.

2024 Financial Guidance

Blueprint Medicines reported it anticipates approximately $360 million to $390 million in global AYVAKIT net product revenues for all approved indications in 2024. The midpoint of this range represents more than 80 percent year-over-year revenue growth, the majority of which is expected to be driven by ISM. The company continues to expect that operating expenses and cash burn will further decline in 2024, and 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 2024:

Mast cell diseases

·

Present long-term safety and efficacy data from the PIONEER trial of AYVAKIT in ISM at the 2024 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting.

·

Present preclinical data for BLU-808, a highly selective and potent oral inhibitor of wild-type KIT, at the 2024 AAAAI Annual Meeting.

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Submit an investigational new drug application for BLU-808 in the second quarter of 2024.

Breast cancer and other solid tumors

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Continue ongoing strategic business development discussions.

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Present data for BLU-222 in combination with ribociclib and fulvestrant in patients with HR+/HER2- breast cancer in the first half of 2024.

Fourth Quarter and Year End 2023 Results

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Revenues: Revenues were $72.0 million for the fourth quarter of 2023, including $71.0 million of net product revenues from sales of AYVAKIT/AYVAKYT and $0.9 million in collaboration revenues. Revenues for the year ended December 31, 2023 were $249.4 million, including $204.2 million of net product revenues from sales of AYVAKIT/AYVAKYT, and $45.2 million in collaboration and license revenues. Blueprint Medicines recorded $38.8 million and $204.0 million in revenues in the fourth quarter and year ended December 31, 2022, respectively.

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Cost of Sales: Cost of sales was $0.3 million for the fourth quarter of 2023 and $8.5 million for the year ended December 31, 2023, as compared to $4.8 million and $17.8 million for the fourth quarter and year ended December 31, 2022, respectively. This decrease was mainly due to a decrease in inventory write-downs and the cost of collaboration-related sales.

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R&D Expenses: Research and development expenses were $97.5 million for the fourth quarter of 2023 and $427.7 million for the year ended December 31, 2023, as compared to $117.8 million and $477.4 million for the fourth quarter and year ended December 31, 2022, respectively. This decrease was primarily due to a focused approach towards optimizing operational efficiency across Blueprint Medicine’s portfolio as the company executes across its top priority programs and the timing of manufacturing of clinical trial materials. Research and development expenses also included $10.0 million in stock-based compensation expenses for the fourth quarter of 2023 and $41.5 million in stock-based compensation for the year ended December 31, 2023.

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SG&A Expenses: Selling, general and administrative expenses were $79.3 million for the fourth quarter of 2023 and $295.1 million for the year ended December 31, 2023, as compared to $64.0 million and $237.4

million for the fourth and year ended December 31, 2022, respectively. This increase was primarily due to an increase in compensation and personnel related costs driven by the company’s first quarter 2023 field force expansion to support the AYVAKIT launch in ISM and an increase in commercial and related activities primarily related to the commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $12.6 million in stock-based compensation expenses for the fourth quarter of 2023 and $51.1 million in stock-based compensation for the year ended December 31, 2023.

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Net Income (Loss): Net loss was $(110.9) million for the fourth quarter of 2023 and $(507.0) million for the year ended December 31, 2023, or a diluted net loss per share of $(1.82) and diluted net loss per share of $(8.37), respectively, as compared to a net loss of $(158.6) million for the fourth quarter of 2022 and a net loss of $(557.5) million for the year ended December 31, 2022, or a diluted net loss per share of $(2.65) and a diluted net loss per share of $(9.35), respectively.

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Cash Position: As of December 31, 2023, cash, cash equivalents and marketable securities were $767.2 million, as compared to $1,078.5 million as of December 31, 2022. Blueprint Medicine’s cash and investments provide a durable capital position which enables the company to reach a self-sustainable profile.

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 2023 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 071930. 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 one upcoming investor conference:

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Cowen 44th Annual Health Care Conference on Monday, March 4, 2024 at 9:10 am ET.

A live webcast of the presentation will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source A replay of the webcast will be archived on the Blueprint Medicines website for 30 days following the presentation.