On May 2, 2024 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS) (GLOBAL NEWSWIRE), a clinical-stage biopharmaceutical company, reported that it has entered into a securities purchase agreement with new and existing investors to raise approximately $30.6 million dollars in aggregate gross proceeds through the sale of shares of its common stock, par value $0.0001 per share (the "Common Stock") and pre-funded warrants to purchase Common Stock in lieu thereof (the "Pre-Funded Warrants"), and accompanying common warrants to purchase Common Stock (or Pre-Funded warrants in lieu thereof) (the "Common Warrants," and together with the Common Stock and Pre-Funded Warrants, the "Securities"), excluding the proceeds, if any, from the exercise of the Pre-Funded Warrants and the Common Warrants and before deducting offering expenses (Press release, Corvus Pharmaceuticals, MAY 2, 2024, View Source [SID1234642583]).

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The offering includes participation from health-care dedicated investors including Point72, Samlyn Capital, Armistice Capital, OrbiMed, Puissance Capital and Altamont Pharmaceutical Holdings, and other existing investors including Richard Miller, the Company’s chief executive officer.

The purchase and sale will be completed via a registered direct offering of 13,512,699 shares of Common Stock and accompanying Common Warrants to purchase 13,078,509 shares of Common Stock (or Pre-Funded Warrants in lieu thereof) at a combined offering price of $1.7312 per Share, and Pre-Funded Warrants to purchase 4,144,085 shares of Common Stock and accompanying Common Warrants to purchase 4,010,927 shares of Common Stock (or Pre-Funded Warrants in lieu thereof) at a combined offering price of $1.7311 per share underlying each Pre-Funded Warrant and Common Warrant, which equals the offering price per share of the Common Stock and Common Warrant less the $0.0001 exercise price per share of the Pre-Funded Warrants. The Pre-Funded Warrants have an exercise price of $0.0001 per share of Common Stock and are exercisable at any time after the date of issuance, subject to certain ownership limitations. The Common Warrants have an exercise price of $3.50 per share of Common Stock (or $3.4999 per Pre-Funded Warrant in lieu thereof) and are exercisable at any time after the date of issuance, subject to certain ownership limitations, and expire on June 30, 2025.

All of the Securities are being offered by Corvus. The offering is expected to close on or about May 6, 2024, subject to the satisfaction of customary closing conditions. Since the offering of the Securities was made without an underwriter or a placement agent, the Company will not be paying any underwriting discounts or placement agent fees in connection with the offering.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

The Securities are being offered by Corvus Pharmaceuticals pursuant to a registration statement on Form S-3 (File No. 333-270921) previously filed and declared effective by the Securities and Exchange Commission ("SEC"). A final prospectus supplement and accompanying base prospectus relating to and describing the terms of the proposed offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov.

On May 2, 2024 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers, reported financial results for the first quarter ended March 31, 2024, and provided a business update (Press release, Cardiff Oncology, MAY 2, 2024, View Source [SID1234642581]).

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"During the start of 2024, we presented several important new data sets supporting our first-line RAS-mutated mCRC strategy and the broader opportunity for onvansertib," said Mark Erlander, Ph.D., Chief Executive Officer of Cardiff Oncology. "The data from the ONSEMBLE trial replicated, in a second independent and randomized dataset, the bev naïve signal from our earlier Phase 1b/2 KRAS-mutated mCRC trial. And the Phase 1b data published in the peer-reviewed journal Clinical Cancer Research, and the additional data we presented in one of our five posters at AACR (Free AACR Whitepaper), further substantiated our lead program in RAS-mutated mCRC. The additional AACR (Free AACR Whitepaper) posters also point toward new indications for onvansertib in RAS wild-type mCRC, small cell lung cancer and ovarian cancer. Looking ahead, we believe that our upcoming data readout from our first-line trial in RAS-mutated mCRC has the potential to serve as a key value inflection point for our company and revolutionize the treatment of RAS-mutated mCRC, an area with no new treatments approved in over two decades."
Upcoming expected milestones

•The Company has updated its expectation of the timing for an initial readout from the first-line RAS-mutated metastatic colorectal cancer (mCRC) randomized CRDF-004 trial to 2H 2024, based on the enrollment trends observed from active clinical trial sites
Company highlights for the quarter ended March 31, 2024 and subsequent weeks include:
•Presented updated data at AACR (Free AACR Whitepaper) Annual Meeting 2024 that supports ongoing first-line RAS-mutated mCRC clinical study.
◦In a poster titled "A Phase 1b/2 Clinical Study of Onvansertib in Combination with FOLFIRI/Bev Revealed a New Role of PLK1 in regulating the Hypoxia Pathway in KRAS-mutated mCRC," updated clinical and preclinical data further supported the ongoing CRDF-004 Phase 2 trial of onvansertib + chemo/bev for the first-line treatment of RAS-mutated mCRC patients, who by definition are bev naïve.
◦In vitro, onvansertib inhibited activation of the hypoxia pathway through the regulation of transcription factor HIF1α and its downstream targets.

◦Bev naïve patients treated with onvansertib + FOLFIRI/bev demonstrated a significantly greater overall response rate [odds ratio=13.64, p<0.001] and longer PFS [hazard ratio=0.21, p=0.003] compared to bev exposed patients.
•Announced first patient dosed for lead program in randomized first-line Phase 2 trial, CRDF-004, for patients with RAS-mutated mCRC.
◦The trial, whose clinical execution is being conducted by our partner, Pfizer Ignite, is designed to confirm the dose of onvansertib for a subsequent registrational trial, and generate safety and efficacy data for onvansertib when added to standard of care (SoC) vs. SoC alone.
◦Interim topline results from CRDF-004 are expected in 2H 2024. Contingent upon the results, Cardiff Oncology will initiate a Phase 3, randomized trial, CRDF-005, with registrational intent.
•Provided a clinical update on Phase 2 randomized second-line ONSEMBLE trial in mCRC.
◦New clinical data from second-line randomized ONSEMBLE trial, that closed for enrollment in 2023, provide further evidence of onvansertib’s role in improving the efficacy of SoC (FOLFIRI+bev) therapy in bev naïve patients.
◦In the trial (n = 21), patients who were bev naïve and who received onvansertib in combination with SoC demonstrated an objective response rate (ORR) of 50% (2 of 4). No clinical responses were observed in bev naïve patients who received SoC alone (n = 3), or patients who were previously exposed to bev (n = 14).
◦The ONSEMBLE data serves as a second independent, randomized, prospective data set providing evidence of the strong efficacy signal in bev naïve patients.
•Announced the publication of data from Phase 1b study in second line KRAS-mutated mCRC in the peer-reviewed journal Clinical Cancer Research.
◦Findings from the Phase 1b portion of company’s Phase 1b/2 study in second-line KRAS-mutated mCRC highlight the safety and promising efficacy of onvansertib in combination with SoC.
◦These peer-reviewed published data are part of the Phase 1b/2 data announced in August 2023 and informed the shift to investigation in first-line RAS-mutated mCRC (CRDF-004).
•Presented three posters at AACR (Free AACR Whitepaper) Annual Meeting 2024 in therapeutic areas outside of RAS-mutated mCRC
◦In a poster titled "The PLK1 Inhibitor Onvansertib is Active as Monotherapy and in Combination with Cetuximab in RAS Wild-type mCRC Patient-derived Xenografts," single agent onvansertib successfully induced tumor stasis or regression in 70% (14/20) of the RAS wild-type mCRC patient-derived xenograft (PDX) models tested which included both models sensitive to cetuximab (5/7, 71%) and resistant to cetuximab (9/13, 69%). Additionally, onvansertib in combination with cetuximab induced tumor stasis or regression in 90% (18/20) of the models tested. The antitumor activity of the combination was superior compared to monotherapy with either agent in both cetuximab sensitive and resistant models.

◦In a poster titled "The PLK1 Inhibitor, Onvansertib, Synergizes with Paclitaxel in Small Cell Lung Cancer (SCLC)," onvansertib in combination with paclitaxel was well-tolerated and demonstrated superior efficacy over monotherapies in cisplatin sensitive and resistant SCLC PDX models. These preclinical findings in SCLC and previous data generated in breast cancer suggest that onvansertib in combination with paclitaxel has the potential to become a highly promising combination strategy across multiple cancer indications.

◦In a poster titled "In vivo anti-tumor activity of onvansertib, a PLK1 inhibitor, combined with gemcitabine or carboplatin in platinum-resistant ovarian carcinoma patient-derived xenograft models," onvansertib was synergistic in vitro in both combinations in an ovarian cancer cell line. Both combinations demonstrated antitumor activity in vivo in platinum-resistant ovarian cancer PDX models and was well tolerated. These data support the potential of onvansertib to improve SoC treatments for platinum-resistant ovarian cancer patients.

First Quarter 2024 Financial Results
Liquidity, cash burn, and cash runway
As of March 31, 2024, Cardiff Oncology had approximately $67.2 million in cash, cash equivalents, and short-term investments.
Net cash used in operating activities for the first quarter of 2024 was approximately $7.7 million, a decrease of approximately $1.0 million from $8.7 million for the same period in 2023.
Based on its current expectations and projections, the Company believes its current cash resources are sufficient to fund its operations into Q3 of 2025.
Operating results
Total operating expenses were approximately $11.1 million for the three months ended March 31, 2024, a decrease of $1.0 million from $12.1 million for the same period in 2023. The decrease in operating expenses was primarily due to a reduction in chemistry, manufacturing and controls costs compared to the prior period.
Conference Call and Webcast
Cardiff Oncology will host a corresponding conference call and live webcast at 4:30 p.m. ET/1:30 p.m. PT on May 2, 2024. Individuals interested in listening to the live conference call may do so by using the webcast link in the "Investors" section of the company’s website at www.cardiffoncology.com. A webcast replay will be available in the investor relations section on the company’s website following the completion of the call.

On May 2, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the first quarter ended March 31, 2024, and provided an update on the Company’s operations (Press release, BridgeBio, MAY 2, 2024, View Source [SID1234642580]).

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"In addition to the important scientific work we have done to advance our patient mission this quarter, we have also taken a first step towards streamlining our firm and thereby unlocking value for patients and investors alike," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "We believe the launch of BBOT better aligns investors with an opportunity that was being hidden in our pipeline, and our partnership with these new investors enables us to prosecute these oncology programs more quickly and with higher fidelity. In tandem with our progress in corporate simplification and balance sheet strengthening, we continue to prepare for what we believe will be a world class launch this year in ATTR-CM and we continue to enroll our achondroplasia, ADH1 and LGMD2I/R9 clinical trials, all of which are expected to complete enrollment this year."

BridgeBio’s late stage programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):

The EMA accepted the Company’s MAA for acoramidis for review, with an expected approval in 2025. This is in addition to the Prescription Drug User Fee Act (PDUFA) date of November 29, 2024 set by the U.S. FDA for the Company’s NDA for acoramidis for the treatment of ATTR-CM.

In March 2024, the Company entered into a licensing agreement granting Bayer exclusive rights to commercialize acoramidis for ATTR-CM in Europe; in exchange, BridgeBio is entitled to receive among other consideration:

Tiered royalties beginning in the low-thirties percent, and

Up to $310 million in upfront and near-term milestone payments, as well as additional sales milestones.

The Company presented cardiac magnetic resonance imaging evidence from an exploratory substudy where treatment with acoramidis was associated with possible cardiac structural and functional improvement compared with placebo, with potential cardiac amyloid regression. This evidence is consistent with clinical improvement observed in the Company’s ATTRibute-CM Phase 3 study of acoramidis in patients with ATTR-CM.

Additional detailed results of the Company’s ATTRibute-CM study are planned for presentation at 2024 medical meetings, including four abstracts accepted for presentation at the European Society of Cardiology Heart Failure conference in May 2024, and eleven abstracts accepted for presentation at the International Symposium on Amyloidosis in May 2024.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:

The PROPEL 3 global Phase 3 registrational study of infigratinib in achondroplasia continues to enroll; study completion continues to be anticipated for 2025.

The Company anticipates initiating its clinical program for hypochondroplasia in 2024, as part of its commitment to exploring the potential of infigratinib on the wider medical and functional impacts of achondroplasia, hypochondroplasia and other skeletal dysplasias.

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):

The Company believes there is potential to pursue Accelerated Approval for BBP-418 based on recent interactions with the FDA on the use of glycosylated αDG levels as a surrogate endpoint.

FORTIFY, the global Phase 3 registrational trial of BBP-418 in LGMD2I/R9, continues to enroll in the U.S., Europe and Australia. Full enrollment of the interim analysis population is expected in 2024.

Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):

CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, continues to enroll; the Company anticipates sharing topline data from CALIBRATE in 2025.
Recent Corporate Updates:

The Company announced the launch of BridgeBio Oncology Therapeutics (BBOT) with a $200 million private financing to accelerate the development of its novel precision oncology pipeline.
The $200 million financing was led by Cormorant Asset Management and co-led by Omega Funds, with participation from affiliates of Deerfield Management, GV (Google Ventures), EcoR1 Capital, Wellington Management, Enavate Sciences, Surveyor Capital (a Citadel company), Aisling Capital, Casdin Capital, and Longwood Fund.
BBOT will be led by Eli Wallace, Ph.D. as CEO and Pedro Beltran, Ph.D. as CSO; the Board of Directors for BBOT will be chaired by Frank McCormick, Ph.D., David A. Wood Distinguished Professor of Tumor Biology and Cancer Research at UCSF and advisor to the National Cancer Institute’s RAS Initiative at Frederick National Laboratory for Cancer Research. Raymond Kelleher, M.D., Ph.D., Managing Director Cormorant Asset Management, Michelle Doig, Partner, Omega Funds, and Neil Kumar, Ph.D., CEO of BridgeBio will serve as directors.
First Quarter 2024 Financial Results:

Cash, Cash Equivalents, Marketable Securities and Short-term Restricted Cash

Cash, cash equivalents, marketable securities and short-term restricted cash, totaled $519.8 million as of March 31, 2024, compared to $392.6 million of cash, cash equivalents and short-term restricted cash as of December 31, 2023. The $127.2 million net increase in cash, cash equivalents, marketable securities and short-term restricted cash was primarily attributable to net proceeds received from the term loan under the credit facility with Blue Owl of $437.7 million, net proceeds received of $315.3 million from various equity financings, proceeds from the sale of investments in equity securities of $63.2 million, and special cash dividends received from investments in equity securities of $25.7 million. These were primarily offset by refinancing of the Company’s previous senior secured credit and inclusive of prepayment fees and exit-related costs in aggregate of $473.4 million, net cash used in operating activities of $219.5 million and purchases of equity securities of $20.3 million during the three months ended March 31, 2024.

Revenue

Revenue for the three months ended March 31, 2024, were $211.1 million, as compared to $1.8 million for the same period in the prior year. The $209.3 million net increase for the three months ended March 31, 2024, compared to the same period in the prior year, was primarily due to the recognition of non-refundable upfront payments under the Bayer and the Kyowa Kirin exclusive license agreements. The remaining license and services revenue was recognized in connection with the Navire-BMS License Agreement, which was entered into in May 2022 and for which BMS provided notice of termination in March 2024 with a termination effective in June 2024.

Operating Costs and Expenses

Operating costs and expenses for the three months ended March 31, 2024 were $210.8 million compared to $128.0 million for the same period in the prior year.

The overall increase of $82.8 million in operating costs and expenses for the three months ended March 31, 2024, compared to the same period in the prior year, was primarily due to an increase of $48.1 million in research and development and other expenses (R&D) to advance the Company’s pipeline of development programs, and an increase of $34.7 million in selling, general and administrative (SG&A) expenses mainly to support commercialization readiness efforts. Operating costs and expenses for the three months ended March 31, 2024, include $22.5 million of nonrecurring deal-related costs for transactions that were closed during the period.

Restructuring, impairment and related charges for the three months ended March 31, 2024 amounted to $3.4 million. These charges primarily consisted of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs. Restructuring, impairment and related charges for the same period in the prior year was $3.4 million. These charges primarily consisted of winding down, exit costs, and severance and employee-related costs.

Stock-based compensation expenses included in operating costs and expenses for the three months ended March 31, 2024 were $28.9 million, of which $12.8 million is included in R&D expenses, and $16.1 million is included in SG&A expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $23.5 million, of which $11.8 million is included in R&D expenses, and $11.7 million is included in SG&A expenses.

"With our recent equity financing activities, our licensing deal with Bayer for European commercial rights to acoramidis, our royalty funding agreement for $500 million upon FDA approval of acoramidis, and now the private financing of BBOT to advance our oncology pipeline into the clinic, we are in a strong financial position to launch acoramidis in the U.S. at the end of this year and deliver three Phase 3 readouts in 2025," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio.

On May 2, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported the completion of a $200M private financing of its former subsidiary, TheRas, Inc. d/b/a BridgeBio Oncology Therapeutics (BBOT), to accelerate the development of its oncology portfolio (Press release, BridgeBio, MAY 2, 2024, View Source [SID1234642579]). The oversubscribed financing was led by Cormorant Asset Management and co-led by Omega Funds with participation from affiliates of Deerfield Management, GV (Google Ventures), EcoR1 Capital, Wellington Management, Enavate Sciences, Surveyor Capital (a Citadel company), Aisling Capital, Casdin Capital, and Longwood Fund.

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BBOT will be advancing three initial programs:

BBO-8520, a direct inhibitor of KRASG12C that binds to both the ON and OFF states of the protein; BBOT is currently enrolling patients in the ONKORAS-101 trial for patients with KRASG12C mutant non-small cell lung cancer
BBO-10203, a PI3Kα:RAS breaker that blocks the specific interaction between RAS and PI3Ka to inhibit PI3Kα / AKT effector signaling in tumors while bypassing glucose metabolic signaling to avoid hyperglycemia; BBOT expects to file an Investigational New Drug application (IND) for BBO-10203 in Q2 2024 and, subject to clearance of the IND, will begin enrolling patients later this year
BBO-11818, a pan-KRAS inhibitor that targets both the ON and OFF states of KRASG12X for which BBOT expects to file an IND in early 2025
In addition to these assets, BBOT will continue to undertake a robust discovery-stage research program focused on targeting additional oncogenic drivers within the RAS and PI3K pathways.

BridgeBio Oncology Therapeutics will be led by Eli Wallace, PhD, as CEO and Pedro Beltran, PhD, as CSO. "We are excited to progress our portfolio of novel small molecules to treat patients suffering from RAS pathway malignancies. In conjunction with BridgeBio and an amazing suite of investors, we look forward to seeing all three of our programs progress into the clinic over the next 12 months," said Dr. Wallace.

The Board of Directors of BBOT will be chaired by Frank McCormick, PhD, David A. Wood Distinguished Professor of Tumor Biology and Cancer Research at UCSF and advisor to the National Cancer Institute’s RAS Initiative at Frederick National Laboratory for Cancer Research. Raymond Kelleher, MD, PhD, Managing Director, Cormorant Asset Management, Michelle Doig, Partner, Omega Funds, and Neil Kumar, PhD, CEO of BridgeBio, will serve as directors.

"I’m thrilled to partner with BridgeBio and this stellar investor syndicate to advance BBOT’s unique portfolio of precision oncology assets targeting RAS-driven cancers," said lead investor Raymond Kelleher, MD, PhD, of Cormorant Asset Management. "BBOT’s innovative chemical biology approaches to inhibit a wide spectrum of KRAS mutations in both the "on" and "off" states, as well as bypass mechanisms exploiting PI3K signaling, promise to transform the treatment of this important class of cancers."

"We are very excited about BBOT’s pipeline of potential first-in-class assets and to join a team of world class experts in RAS-driven malignancies, who we believe are positioned to have significant positive impact on cancer patients and build stakeholder value," added Michelle Doig from Omega Funds.

On May 2, 2024 Blueprint Medicines Corporation (Nasdaq: BPMC) reported its financial results, provided a business update for the first quarter ended March 31, 2024, and provided updated financial guidance (Press release, Blueprint Medicines, MAY 2, 2024, View Source [SID1234642578]).

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"We delivered another very strong quarter in our launch of AYVAKIT in indolent systemic mastocytosis (ISM) and have entered 2024 in a position of strength with great momentum across all aspects of our business. The first few quarters of a launch are critical in defining the sales trajectory for a product, and our revenue to-date positions us squarely on the path to achieve more than $2 billion in peak sales for AYVAKIT in systemic mastocytosis," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "Importantly, our growing revenue also enables us to invest in additional, compelling opportunities across our pipeline, where we can drive longer-term growth. This includes advancing our portfolio focused in allergy and inflammation as we move BLU-808 into the clinic in the second half of this year. I am proud of our team’s strong operational execution against our core value drivers, which enables Blueprint to realize the harmony between our mission of bringing new, innovative medicines to patients and building a robust and thriving business that will create substantial value for our shareholders."

First Quarter 2024 Highlights and Recent Progress

Mast cell disorders

·

Achieved AYVAKIT net product revenues of $92.5 million for first quarter of 2024, representing more than 135 percent growth year-over-year.

·

Presented long-term data from the PIONEER trial of AYVAKIT in ISM, demonstrating durable symptom impact and a well-tolerated safety profile, supporting long-term treatment and consistent with real-world experience observed in the commercial setting. Read the presentation here.

·

Presented preclinical data for BLU-808, a highly selective and potent investigational oral wild-type KIT inhibitor with best-in-class potential, for chronic urticaria and other mast cell disorders. BLU-808 treatment led to dose-dependent inhibition and depletion of mast cells in multiple in vivo studies, and also improved lung function in an ovalbumin-induced asthma model. Based on these data, Blueprint is on track to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for BLU-808 in the second quarter of 2024 to enable the initiation of a Phase 1 study in healthy volunteers. Read the presentation here.

·

Highlighted Blueprint’s strategy to leverage the company’s proven expertise in developing mast cell-targeted therapies to address large medical needs in allergy and inflammation. The company plans to host additional educational webcasts highlighting the evolving science around Blueprint’s portfolio strategy in the future. Watch the replay here.

HR+/HER2- breast cancer

·

Advanced the development of BLU-222, an oral, potent, and selective CDK2 inhibitor in combination with ribociclib and fulvestrant in patients with HR+/HER2- breast cancer, with plans to present the first positive combination safety data with signal of early clinical activity for a CDK2 inhibitor in combination with an approved CDK4/6 inhibitor at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

Corporate

·

Announced that Rigel Pharmaceuticals, Inc. is purchasing the U.S. rights to research, develop, manufacture, and commercialize GAVRETO (pralsetinib), which allows continuity of patient access to GAVRETO in the U.S.

·

Published third annual Sustainability Report, highlighting Blueprint’s 2023 progress on environmental, social, and governance (ESG) initiatives and strategy and reflecting the results of a materiality assessment to focus our strategy on the most important ESG topics for the company and shareholders. Read the report here.

2024 Financial Guidance

Blueprint Medicines now anticipates approximately $390 million to $410 million in global AYVAKIT net product revenues for all approved indications in 2024, an increase from the previous range of $360 million to $390 million. This updated guidance reflects continued execution in the global launch for ISM, as well as a stronger than anticipated first quarter. The company continues to expect that full-year operating expenses and cash burn will decline in 2024 as compared to 2023, and that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will enable the company to maintain a durable capital position to achieve a self-sustainable financial profile.

Key Upcoming Milestones

The company plans to achieve the following remaining milestones in the first half of 2024:

Mast cell disorders

·

Submit an IND to FDA for BLU-808 in the second quarter of 2024 to enable the initiation of a Phase 1 study in healthy volunteers.

HR+/HER2- breast cancer

·

Present data for BLU-222 in combination with ribociclib and fulvestrant for HR+/HER2- breast cancer at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.

·

Continue ongoing strategic business development discussions.

First Quarter 2024 Results

·

Revenues: Revenues were $96.1 million for the first quarter of 2024, including $92.5 million of net product revenues from sales of AYVAKIT/AYVAKYT and $3.6 million in collaboration revenues. Blueprint Medicines recorded revenues of $63.3 million in the first quarter of 2023, including $39.1 million of net product revenues from sales of AYVAKIT/AYVAKIT and $24.2 million in collaboration revenues.

·

Cost of Sales: Cost of sales was $3.2 million for the first quarter of 2024, as compared to $3.2 million for the first quarter of 2023. The relatively flat cost of sales was primarily attributed to the increased sales of lower cost dosages of AYVAKIT/AYVAKIT.

·

R&D Expenses: Research and development expenses were $88.2 million for the first quarter of 2024, as compared to $112.1 million for the first quarter of 2023. This decrease was primarily due to operational efficiency across our portfolio as we execute across our top priority programs and the timing of manufacturing of clinical trial materials. Research and development expenses included $10.9 million in stock-based compensation expenses for the first quarter of 2024.

·

SG&A Expenses: Selling, general and administrative expenses were $83.6 million for the first quarter of 2024, as compared to $71.0 million for the first quarter of 2023. This increase was primarily due to an increase in activities related to the commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $13.4 million in stock-based compensation expenses for the first quarter of 2024.

·

Net Income (Loss): Net income was $89.1 million for the first quarter of 2024, as compared to a net loss of $129.6 million for the first quarter of 2023. The net income was primarily driven by a one-time non-cash debt extinguishment gain of $173.7 million recorded in connection with the Royalty Pharma termination agreement in the first quarter of 2024.

·

Cash Position: As of March 31, 2024, cash, cash equivalents and investments were $735.6 million, as compared to $767.2 million as of December 31, 2023. 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 first quarter 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 186292. 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:

·

Citizens JMP Life Sciences Conference on Monday, May 13, 2024 at 10:00 am ET.

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Goldman Sachs 45th Annual Global Healthcare Conference on Monday, June 10, 2024 at 10:40 am ET.

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