Cerus Corporation Announces Second Quarter 2024 Financial Results and Increases Full-Year 2024 Product Revenue Guidance Range

On August 1, 2024 Cerus Corporation (Nasdaq: CERS) reported financial results for its second quarter and six months ended June 30, 2024 (Press release, Cerus, AUG 1, 2024, View Source [SID1234645254]).

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Total revenue for the three and six months ended June 30, 2024 was comprised of ( in thousands, except %) :

Three Months Ended

Six Months Ended

June 30,

Change

June 30,

Change

2024

2023

$

%

2024

2023

$

%

Product Revenue

$

45,079

$

38,853

$

6,226

16

%

$

83,444

$

69,827

$

13,617

20

%

Government Contract Revenue

5,440

8,875

(3,435

)

-39

%

10,470

16,377

(5,907

)

-36

%

Total Revenue

$

50,519

$

47,728

$

2,791

6

%

$

93,914

$

86,204

$

7,710

9

%

Recent highlights include:

Advanced efforts to expand the U.S. manufacturing capacity for INTERCEPT Fibrinogen Complex (IFC).
Achievement of 100% adoption of INTERCEPT platelets by Canadian Blood Services, resulting in the pathogen inactivation of the majority of platelets in Canada.
Narrowed GAAP net loss attributable to Cerus Corporation to $5.8 million and generated non-GAAP adjusted EBITDA of $0.8 million for the second quarter.
Generated positive operating cash flows for the second straight quarter of 2024 bringing year-to-date positive operating cash flows to $2.4 million.
Cash and cash equivalents and short-term investments were $71.2 million at June 30, 2024.
The Company is increasing its full-year 2024 annual product revenue guidance range from $172-175 million to $175-178 million which includes $8-10 million for IFC.
The Company continues to expect future improvements to GAAP net loss attributable to Cerus Corporation for the full-year 2024 and remains committed to adjusted EBITDA breakeven for the full-year 2024.
"We are very pleased to post another strong quarter to continue our growth trajectory in 2024. North American sales in our platelet business led the way, including both organic U.S. sales growth and 100% adoption by Canadian Blood Services. Given our expectations for continued momentum in the business, we are raising our full-year 2024 product revenue guidance today," stated William "Obi" Greenman, Cerus’ president and chief executive officer. "Our IFC business is accelerating with respect to the increasing recognition of its pivotal early role in the care of bleeding patients and its improved operational ease of use for hospital transfusion services. Two recent publications spotlighting the hospital experience with IFC document these benefits well, and there are expanding numbers of hospital case studies being presented at upcoming conferences, including the AABB Annual Meeting in the fall."

"We are also extremely pleased with our execution to date on key financial measures that we discussed at the beginning of the year, namely adjusted EBITDA and operating cash flows," continued Greenman. "We will continue our efforts to improve on these metrics during the back half of 2024, which is expected to help further strengthen our financial position."

Revenue

Product revenue during the second quarter of 2024 was $45.1 million, compared to $38.9 million during the prior year period. This year-over-year increase of 16% was driven primarily by growth in our platelets business, particularly in North America. Second-quarter product revenue included sales of IFC, which were $2.0 million, up from $1.4 million during the prior year period.

Second-quarter 2024 government contract revenue was $5.4 million, compared to $8.9 million during the prior year period. Our government contract revenue was comprised of funding associated with research and development (R&D) activities related to the INTERCEPT Blood System for Red Blood Cells (RBCs) as well as efforts related to the development of next-generation pathogen reduction technology to treat whole blood and development of a lyophilized IFC. Reported government contract revenue during the second quarter of 2024 decreased versus the prior year period, primarily due to completion of the U.S. Phase 3 ReCePI clinical trial for INTERCEPT RBCs.

Product Gross Profit & Margin

Product gross profit for the second quarter of 2024 was $24.7 million, increasing by 16% over the prior year period. Product gross margin for the second quarter of 2024 was relatively stable year over year at 54.7% compared to 54.9% for the second quarter of 2023. Absent any unanticipated factor, we expect product gross margin levels will remain relatively consistent with second-quarter 2024 levels for the duration of 2024.

Operating Expenses

Total operating expenses for the second quarter of 2024 were $33.9 million, compared to $41.9 million for the same period of the prior year, reflecting a year-over-year decrease of 19%. This decline resulted from year-over-year decreases in both R&D and selling, general and administrative (SG&A) expenses, driven by the restructuring implemented in the second quarter of 2023, as well as completion of key initiatives by the Company.

R&D expenses for the second quarter of 2024 were $15.0 million, compared to $19.2 million for the second quarter of 2023. The primary drivers for the decrease in R&D expenses were the restructuring implemented in the second quarter of last year and the completion of the Company’s ReCePI trial in the first quarter of 2024.

SG&A expenses narrowed for the second quarter of 2024 and totaled $19.0 million, compared to $20.5 million for the second quarter of 2023. The primary driver for the decrease in SG&A expenses was again the restructuring implemented in the second quarter of last year.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the second quarter of 2024 was $5.8 million, or $0.03 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $13.3 million, or $0.07 per basic and diluted share, for the second quarter of 2023. Net loss attributable to Cerus Corporation for the first half of 2024 was $15.5 million, compared to a net loss attributable to Cerus Corporation of $28.9 million for the first half of 2023.

Non-GAAP Adjusted EBITDA

Non-GAAP adjusted EBITDA for the second quarter of 2024 was $0.8 million, compared to a loss of $4.7 million for the second quarter of 2023. Non-GAAP adjusted EBITDA narrowed to a loss of $1.9 million for the first half of 2024, compared to a loss of $14.5 million for the first half of 2023. The Company continues to focus on achieving non-GAAP adjusted EBITDA breakeven for the full-year 2024 period as a whole. For additional information, please see definitions and the reconciliation of this non-GAAP measure to net loss attributable to Cerus Corporation accompanying this release.

Balance Sheet & Cash Flows

At June 30, 2024, the Company had cash and cash equivalents and short-term investments of $71.2 million, compared to $65.9 at December 31, 2023.

As of June 30, 2024, the Company had $65.0 million outstanding on its term loan and $18.8 million drawn on its revolving credit facility which represents a payback of $1.2 million for the quarter. The Company’s revolving line of credit allows for an additional $16.2 million.

For the second quarter of 2024, the Company generated positive cash flows of $0.4 million from operations compared to cash used in operations of $7.6 million during the prior year period. These improvements were in line with the Company’s expectations and will continue to be a focus area going forward.

Increasing 2024 Product Revenue Guidance

Given the strong performance in the first half and continued growing conviction around IFC demand, the Company expects full-year 2024 product revenue will be in the range of $175 million to $178 million, representing increased growth expectations of approximately 12-14% over full-year 2023 results. Previously, the Company’s 2024 product revenue guidance range was $172 million to $175 million. The Company is reiterating full-year 2024 IFC revenue guidance of $8 million to $10 million.

Quarterly Conference Call

The Company will host a conference call at 4:30 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at View Source

A replay will be available on Cerus’ website approximately three hours after the call through August 22, 2024.

FDA Grants Orphan Drug Designation to Cellectis’ CLLS52 (alemtuzumab) For ALL Treatment

On August 1, 2024 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as part of the lymphodepletion regimen associated with UCART22, evaluated in the BALLI-01 clinical trial in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) (Press release, Cellectis, AUG 1, 2024, View Source [SID1234645253]).

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"We are excited that the FDA granted CLLS52 (alemtuzumab) ODD designation status. The importance of adding alemtuzumab to the lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity", said Mark Frattini, M.D., Ph.D. Chief Medical Officer at Cellectis.

Cellectis is the inventor of the combination of CD52 knockout UCART cells with a lymphodepleting regimen containing an anti-CD52 antibody such as alemtuzumab. The CD52 knockout aims to render the UCART product candidates resistant to alemtuzumab as part of the lymphodepleting regimen. Cellectis’ UCART22 product candidate has the CD52 gene inactivated by TALEN gene editing technology.

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent.

C4 Therapeutics Reports Second Quarter 2024 Financial Results and Recent Business Highlights

On August 1, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported financial results for the second quarter ended June 30, 2024, as well as recent business highlights (Press release, C4 Therapeutics, AUG 1, 2024, View Source [SID1234645252]).

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"Our strong execution during the first half of the year has built momentum across our clinical, discovery and partnered programs, each of which has the potential to advance targeted protein degradation science and transform patients’ lives," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "Our two lead programs continue to advance in the clinic, and we are on track to share multiple Phase 1 datasets during the second half of the year. At the upcoming ESMO (Free ESMO Whitepaper) Congress, we will present initial CFT1946 Phase 1 data, marking the first clinical presentation for a BRAF V600X degrader, which has the potential to address many of the liabilities that occur with inhibitors. Additionally, we expect to share updated cemsidomide Phase 1 data in relapsed refractory multiple myeloma and relapsed refractory non-Hodgkin’s lymphoma in the fourth quarter."

SECOND QUARTER 2024 AND RECENT ACHIEVEMENTS

Cemsidomide: Cemsidomide is an oral degrader of IKZF1/3 for the potential treatment of relapsed/refractory (R/R) multiple myeloma (MM) and R/R non-Hodgkin’s lymphoma (NHL).

Advanced the Phase 1/2 Clinical Trial. The cemsidomide Phase 1/2 trial in combination with dexamethasone for R/R MM and as a monotherapy for R/R NHL continues to enroll patients. For the combination with dexamethasone MM arm, dose level 4 (75 µg QD) has been declared safe and additional patients are enrolling in this expansion cohort. Dose escalation continues as the maximum tolerated dose has not yet been reached. For the monotherapy NHL arm, patients are enrolling at dose level 5 (100 µg QD).
CFT1946: CFT1946 is an oral degrader targeting BRAF V600X mutations for the potential treatment of solid tumors including colorectal cancer (CRC), melanoma and non-small cell lung cancer (NSCLC).

Advanced the Phase 1/2 Clinical Trial. The CFT1946 Phase 1/2 trial for BRAF V600X mutant solid tumors continues to enroll patients. Enrollment is complete at dose level 5 (640 mg BID), with patients currently in the dose limiting toxicity evaluation period for this dose level. Simultaneously, patients continue to be evaluated for pharmacokinetic, pharmacodynamic and anti-tumor activity at the 160 mg BID and 320 mg BID dose levels. Additionally, patients are now enrolling in a monotherapy exploratory expansion cohort for BRAF inhibitor refractory melanoma at the 320 mg BID dose level.

The Phase 1b portion of the trial evaluating CFT1946 in combination with cetuximab for CRC has also been opened and patients are enrolling at the 160 mg BID dose level.
Preliminary CFT1946 Monotherapy Data Accepted as a Mini Oral Presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024. Monotherapy data from the ongoing CFT1946 Phase 1 trial will be presented on Saturday, September 14, 2024 from 2:45 – 2:50 CEST at the ESMO (Free ESMO Whitepaper) Congress 2024 in Barcelona, Spain.
CORPORATE UPDATES:

In June 2024, Ron Cooper was appointed as chairman of C4T’s Board of Directors as a part of C4T’s commitment to strategically transform the Board to lead the company into its next phase of growth. Mr. Cooper brings decades of deep biopharmaceutical executive leadership across discovery, development and commercialization.
KEY UPCOMING MILESTONES

Cemsidomide:

Present updated data from at least three dose levels from the dose escalation and expansion cohorts of the ongoing Phase 1/2 clinical trial in R/R MM in Q4 2024.
Present data from at least four dose levels from the dose escalation portion of the ongoing Phase 1/2 clinical trial in R/R NHL in Q4 2024.
Complete Phase 1 dose exploration in R/R MM and R/R NHL by year-end 2024.
CFT1946:

Present data from at least five dose levels from the ongoing Phase 1 monotherapy dose escalation trial in BRAF V600X solid tumors as a mini oral presentation on Saturday, September 14, 2024 from 2:45 – 2:50 CEST at the ESMO (Free ESMO Whitepaper) Congress 2024 in Barcelona, Spain.
UPCOMING INVESTOR EVENTS:

September 5, 2024 at 9:30 AM ET: Management will participate in a fireside chat at the Wells Fargo Healthcare Conference taking place in Boston, MA.
September 16, 2024: Management will host a webcast to discuss the CFT1946 data presented at the ESMO (Free ESMO Whitepaper) Congress 2024.
September 17 – 19, 2024: Management will participate in the Cantor Global Healthcare Conference taking place in New York, NY.
SECOND QUARTER 2024 FINANCIAL RESULTS

Revenue: Total revenue for the second quarter of 2024 was $12.0 million, compared to $2.7 million for the second quarter of 2023. The increase in revenue was primarily due to the receipt of an $8.0 million milestone payment from Biogen after the company accepted delivery of a development candidate. Total revenue for the second quarter of 2024 reflects revenue recognized under our collaborations with Merck KGaA, Darmstadt, Germany (MKDG), Merck, Roche and Biogen, and total revenue recognized in the second quarter of 2023 reflects revenue recognized under collaboration agreements with Roche and Biogen.

Research and Development (R&D) Expense: R&D expense for the second quarter of 2024 was $23.8 million, compared to $29.9 million for the second quarter of 2023. The reduction in R&D expense was primarily due to the prioritization of our internal discovery efforts and stopping clinical development for CFT8634, partially offset by increased clinical trial expense as cemsidomide and CFT1946 continue to advance.

General and Administrative (G&A) Expense: G&A expense was $9.7 million for the second quarter of 2024, compared to $10.3 million for the second quarter of 2023. The decrease in G&A expense was primarily attributable to a reduction in external consulting spend.

Net Loss and Net Loss per Share: Net loss for the second quarter of 2024 was $17.7 million, compared to $35.9 million for the second quarter of 2023. Net loss per share for the second quarter of 2024 was $0.26 compared to $0.73 for the second quarter of 2023.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of June 30, 2024 were $295.7 million, compared to $299.2 million as of March 31, 2024, and $281.7 million as of December 31, 2023. The reduction in cash, cash equivalents and marketable securities during the second quarter was primarily the result of operating expenses offset by receipt of the upfront payment from our collaborator MKDG and a milestone payment from Biogen. C4T expects that its cash, cash equivalents and marketable securities as of June 30, 2024 will be sufficient to fund planned operating expenses and capital expenditures into 2027.

BridgeBio Pharma Reports Second Quarter 2024 Financial Results and Business Update

On August 1, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases, reported its financial results for the second quarter ended June 30, 2024, and provided an update on the Company’s operations (Press release, BridgeBio, AUG 1, 2024, View Source [SID1234645250]).

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"Our team has continued its preparation for the commercial launch of acoramidis while also executing against our goal of fully enrolling our three Phase 3 clinical programs by the end of 2024. We are well positioned to launch acoramidis and achieve three readouts in 2025. Our differentiated capability to develop multiple candidates for genetic-based diseases provides a unique opportunity to create significant value for patients and shareholders," said Dr. Neil Kumar, CEO and Founder of BridgeBio.

Pipeline overview:

Program Status Next expected milestone
Acoramidis for ATTR-CM NDA filed with FDA November 29, 2024 PDUFA date
Encaleret for ADH1 Enrolling CALIBRATE, Phase 3 study Enrollment completion in 2024
BBP-418 for LGMD2I/R9 Enrolling FORTIFY, Phase 3 study Enrollment completion in 2024
Low-dose infigratinib for achondroplasia Enrolling PROPEL 3, Phase 3 study Enrollment completion in 2024
Low-dose infigratinib for hypochondroplasia Enrolling observational run-in for ACCEL 2, Phase 2 study Enrollment completion date to be announced
BBP-631 for congenital adrenal hyperplasia (CAH) Dose finding / analysis in Phase 2 study Program update in August 2024
BBP-812 for Canavan disease Enrolling at high dose in Phase 1/2 study Key regulatory interactions

Program updates:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
During the 2024 International Symposium of Amyloidosis (ISA), five new analyses were disclosed through oral presentations and posters, discussing the following:
Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular hospitalization (CVH) in ATTR-CM participants through Month 30.
Acoramidis treatment resulted in a significant improvement in the composite endpoint of ACM and CVH in ATTR-CM participants, with benefit evident as early as Month 3.
In ATTRibute-CM, participants with at least one CVH had a significantly higher risk of mortality, highlighting the need for ATTR-CM treatments that reduce the risk of CVH.
BridgeBio also shared the rationale and design of ACT-EARLY, the acoramidis ATTR amyloidosis prevention trial, which it expects to initiate later this year.
At this year’s European Society of Cardiology Heart Failure (ESC-HF) Congress 2024, BridgeBio shared four positive analyses, which included the following data:
In a pre-specified Cochran-Mantel-Haenszel sensitivity analysis applied to the entire ITT population of the study (N=632), acoramidis significantly reduced ACM (p=0.04), with no safety signals of potential clinical concern.
Among ATTRibute-CM participants enrolled with Stage 4 chronic kidney disease (CKD) (N=21), acoramidis treatment was associated with proportionally fewer deaths compared with placebo, with no safety signals of potential clinical concern.
At Month 30 of the ATTRibute-CM study, acoramidis treatment resulted in a statistically significant and clinically important reduction in the progressive decline in health-related quality of life as assessed by the EuroQoL Health Outcomes Assessment tool, EQ-5D-5L.
Acoramidis treatment also reduced the decline in health status and quality of life as shown by statistically significant and clinically meaningful benefits in the Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score and supported by numerical and consistent benefits in individual KCCQ domains.
In ATTRibute-CM, acoramidis significantly improved NT-proBNP indices that can be a signal of ATTR-CM disease progression and be predictive of subsequent mortality risk.
During the 2024 American College of Cardiology (ACC), BridgeBio presented cardiac magnetic resonance (CMR) imaging evidence consistent with clinical improvement observed in the ATTRibute-CM. The data demonstrate that targeting near-complete TTR stabilization with acoramidis may enable cardiac remodeling and functional recovery in patients with ATTR-CM.
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.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9):
BridgeBio has surpassed its interim analysis enrollment target for its Phase 3 FORTIFY study of BBP-418 in individuals living with LGMD2I/R9, with top-line results from the interim analysis expected in 2025.
Recent Type C interactions with U.S. FDA focused on the validated glycosylated alpha-dystroglycan (αDG) bioassay and our interim analysis plans reinforce BridgeBio’s belief that there is potential to pursue Accelerated Approval for BBP-418.
Rare Pediatric Disease Designation for BBP-418 highlights that LGMD2I/R9 is a rare disease with serious manifestations, which primarily impacts children. If BBP-418 is approved, BridgeBio may qualify for a Priority Review Voucher, which can be applied to another therapy in the Company’s pipeline for a shorter timeline during the review process of a NDA or can be sold to another company looking to receive priority review for one of its applications.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
BridgeBio shared data from Month 12 and 18 for Cohort 5 of PROPEL 2 (0.25 mg/kg/day), its Phase 2 trial in achondroplasia with the oral treatment, infigratinib, with results including:
A statistically significant and sustained increase in AHV, with a mean change from baseline of +2.51cm/yr at Month 12, and +2.50 cm/yr at Month 18 (p=0.0015).
At Month 18, there was a statistically significant improvement in body proportionality (p-value of 0.001). The mean upper to lower body segment ratio was 1.88 at Month 18, as compared to 2.02 at baseline.
Infigratinib continues to be well-tolerated as a single daily oral therapy with no adverse events (AEs) assessed as treatment-related in any participant in Cohort 5.
Infigratinib for achondroplasia was granted Fast Track Designation and Rare Pediatric Drug Designation by the U.S. FDA. If infigratinib is approved, BridgeBio may qualify for a Priority Review Voucher.
In May 2024, the first participant consented to be part of ACCEL, the observational run-in study for infigratinib in children living with hypochondroplasia.
Second 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 $587.2 million as of June 30, 2024, compared to $392.6 million of cash, cash equivalents and short-term restricted cash as of December 31, 2023. The $194.6 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 $434.0 million, net proceeds received from various equity financings of $314.8 million, 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 term loan, inclusive of prepayment fees and exit-related costs in aggregate of $473.4 million, net cash used in operating activities of $144.8 million, purchases of equity securities of $20.3 million, and repurchase of shares to satisfy tax withholdings of $4.7 million during the six months ended June 30, 2024.

Revenue

Revenue for the three and six months ended June 30, 2024 were $2.2 million and $213.3 million, respectively, as compared to $1.6 million and $3.5 million for the same periods in the prior year.

The increase of $0.6 million in revenue for the three months ended June 30, 2024, compared to the same period in the prior year, was primarily due to the recognition of services revenue under the exclusive license and collaboration agreements with Bayer and Kyowa Kirin. Revenue for the three months ended June 30, 2023 primarily consists of the recognition of services revenue under the Navire-BMS License Agreement, which terminated effective June 2024.

The increase of $209.8 million in revenue for the six months ended June 30, 2024, compared to the same period in the prior year, was primarily due to $202.9 million from recognition of non-refundable upfront payments and service revenue under the Bayer and the Kyowa Kirin exclusive license and collaboration agreements.

Operating Costs and Expenses

Operating costs and expenses for the three and six months ended June 30, 2024 were $177.7 million and $388.5 million, respectively, compared to $147.7 million and $275.7 million for the same periods in the prior year.

The overall increase of $30.0 million in operating costs and expenses for the three months ended June 30, 2024, compared to the same period in the prior year, was primarily due to an increase of $23.4 million in selling, general and administrative (SG&A) expenses mainly to support commercialization readiness efforts, an increase of $7.2 million in research and development and other expenses (R&D) to advance the Company’s pipeline of research and development programs, offset by a decrease of $0.6 million in restructuring, impairment and related charges.

The overall increase of $112.8 million in operating costs and expenses for the six months ended June 30, 2024, compared to the same period in the prior year, was primarily due to an increase of $58.1 million in SG&A expenses mainly to support commercialization readiness efforts, and an increase of $55.3 million in R&D expenses to advance the Company’s pipeline of research and development programs, offset by a decrease of $0.6 million in restructuring, impairment and related charges. Operating costs and expenses for the six months ended June 30, 2024, include $22.5 million of nonrecurring deal-related costs for transactions that were closed during the three months ended March 31, 2024.

Restructuring, impairment and related charges for the three and six months ended June 30, 2024 amounted to $2.9 million and $6.3 million, respectively. 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 periods in the prior year was $3.5 million and $6.9 million, respectively. 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 June 30, 2024 were $21.5 million, of which $4.9 million is included in R&D expenses, $16.5 million is included in SG&A expenses, and $0.1 million is included in Restructuring expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $27.2 million, of which $13.2 million is included in R&D expenses, and $14.0 million is included in SG&A expenses.

Stock-based compensation expenses included in operating costs and expenses for the six months ended June 30, 2024 were $50.3 million, of which $17.7 million is included in R&D expenses, $32.5 million is included in SG&A expenses, and $0.1 million is included in Restructuring expenses. Stock-based compensation expenses included in operating costs and expenses for the same period in the prior year were $50.7 million, of which $25.0 million is included in R&D expenses, and $25.7 million is included in SG&A expenses.

Total Other Income (Expense), net
Total other income (expense), net for the three and six months ended June 30, 2024 were $100.0 million and $63.5 million, respectively, compared to ($14.6) million and ($31.2) million for the same periods in the prior year.

The increase in total other income (expense), net of $114.6 million for the three months ended June 30, 2024, compared to the same period in the prior year, was primarily due to the Company’s gain on deconsolidation of a subsidiary of $126.3 million. This was partially offset by a net loss from an equity method investment of $7.9 million and an increase in interest expense of $2.3 million.

The increase in total other income (expense), net of $94.7 million for the six months ended June 30, 2024, compared to the same period in the prior year, was primarily due to the Company’s gain on deconsolidation of a subsidiary of $126.3 million and an increase in other income (expense), net of $8.1 million mainly from income or mark to market fair value adjustments from the Company’s investments in equity securities. These were partially offset by a loss on extinguishment of debt of $26.6 million, a net loss from an equity method investment of $7.9 million and an increase in interest expense of $5.7 million.

Net Loss Attributable to Common Stockholders of BridgeBio and Net Loss per Share

For the three months and six months ended June 30, 2024, the Company recorded a net loss attributable to common stockholders of BridgeBio of $73.5 million and $108.7 million, respectively, compared to $157.9 million and $298.1 million, respectively for the three months and six months ended June 30, 2023.

For the three months and six months ended June 30, 2024, the Company reported a net loss per share of $0.39 and $0.59, respectively compared to $0.98 and $1.90, respectively for the three months and six months ended June 30, 2023.

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Operations
(in thousands, except shares and per share amounts)

Three Months Ended June 30, Six Months Ended June 30,
2024 2023 2024 2023
(Unaudited) (Unaudited)
Revenue $ 2,168 $ 1,641 $ 213,288 $ 3,467
Operating costs and expenses:
Research, development and other expenses 115,293 108,087 256,863 201,599
Selling, general and administrative 59,523 36,122 125,330 67,230
Restructuring, impairment and related charges 2,891 3,531 6,291 6,900
Total operating costs and expenses 177,707 147,740 388,484 275,729
Loss from operations (175,539 ) (146,099 ) (175,196 ) (272,262 )
Other income (expense), net:
Interest income 5,195 4,514 9,270 8,667
Interest expense (22,937 ) (20,594 ) (46,408 ) (40,715 )
Gain on deconsolidation of a subsidiary 126,294 — 126,294 —
Loss on extinguishment of debt — — (26,590 ) —
Net loss from equity method investment (7,925 ) — (7,925 ) —
Other income (expense), net (632 ) 1,476 8,851 875
Total other income (expense), net 99,995 (14,604 ) 63,492 (31,173 )
Net loss (75,544 ) (160,703 ) (111,704 ) (303,435 )
Net loss attributable to redeemable convertible noncontrolling interests and noncontrolling interests 2,088 2,804 3,032 5,380
Net loss attributable to common stockholders of BridgeBio $ (73,456 ) $ (157,899 ) $ (108,672 ) $ (298,055 )
Net loss per share, basic and diluted $ (0.39 ) $ (0.98 ) $ (0.59 ) $ (1.90 )
Weighted-average shares used in computing net loss per share, basic and diluted 187,586,680 160,535,435 183,145,995 156,645,838

Three Months Ended June 30, Six Months Ended June 30,
Stock-based Compensation 2024 2023 2024 2023
(Unaudited) (Unaudited)
Research, development and other expenses $ 4,937 $ 13,229 $ 17,716 $ 25,008
Selling, general and administrative 16,471 13,947 32,542 25,645
Restructuring, impairment and related charges 43 — 43 —
Total stock-based compensation $ 21,451 $ 27,176 $ 50,301 $ 50,653

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Balance Sheets
(In thousands)

June 30, December 31,

2024 2023
Assets (Unaudited) (1)
Cash, cash equivalents and marketable securities $ 447,771 $ 375,935
Investments in equity securities — 58,949
Receivables from licensing and collaboration agreements 660 1,751
Short-term restricted cash 139,409 16,653
Prepaid expenses and other current assets 28,396 24,305
Investment in nonconsolidated entity 117,006 -
Property and equipment, net 9,840 11,816
Operating lease right-of-use assets 7,267 8,027
Intangible assets, net 25,123 26,319
Other assets 18,903 22,625
Total assets $ 794,375 $ 546,380
Liabilities, Redeemable Convertible Noncontrolling Interests and Stockholders’ Deficit
Accounts payable $ 18,126 $ 10,655
Accrued and other liabilities 96,517 122,965
Operating lease liabilities 11,682 13,109
Deferred revenue 32,059 9,823
2029 Notes, net 737,882 736,905
2027 Notes, net 544,270 543,379
Term loan, net 435,447 446,445
Other long-term liabilities 485 5,634
Redeemable convertible noncontrolling interests (223 ) 478
Total BridgeBio stockholders’ deficit (1,092,925 ) (1,354,257 )
Noncontrolling interests 11,055 11,244
Total liabilities, redeemable convertible noncontrolling interests and stockholders’ deficit $ 794,375 $ 546,380

(1 ) The condensed consolidated financial statements as of and for the year ended December 31, 2023 are derived from the audited consolidated financial statements as of that date.

BRIDGEBIO PHARMA, INC.
Condensed Consolidated Statements of Cash Flows
(Unaudited)
(In thousands)

Six Months Ended June 30,
2024 2023
Operating activities:
Net loss $ (111,704 ) $ (303,435 )
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation 38,511 49,085
Loss on extinguishment of debt 26,590 —
Accretion of debt 3,683 4,580
Depreciation and amortization 3,170 3,270
Noncash lease expense 2,093 2,024
Accrual of payment-in-kind interest on term loan — 6,742
Net loss from equity method investment 7,925 —
Loss (gain) on deconsolidation of subsidiaries (126,294 ) 1,241
Gain from investment in equity securities, net (8,136 ) (2,399 )
Fair value adjustment of warrants — (222 )
Other noncash adjustments (1,911 ) (328 )
Changes in operating assets and liabilities:
Receivables from licensing and collaboration agreements 1,091 8,466
Prepaid expenses and other current assets (6,506 ) 1,057
Other assets 942 32
Accounts payable 8,858 (4,098 )
Accrued compensation and benefits (8,378 ) (11,071 )
Accrued research and development liabilities 7,067 (11,322 )
Operating lease liabilities (2,981 ) (2,443 )
Deferred revenue 22,236 (3,184 )
Accrued professional and other liabilities (1,090 ) 4,330
Net cash used in operating activities (144,834 ) (257,675 )
Investing activities:
Purchases of marketable securities (93,811 ) (19,754 )
Maturities of marketable securities 55,000 41,550
Purchases of investments in equity securities (20,271 ) (71,504 )
Proceeds from sales of investments in equity securities 63,229 67,068
Proceeds from special cash dividends received from investments in equity securities 25,682 —
Payment for an intangible asset (3,190 ) —
Purchases of property and equipment (749 ) (440 )
Decrease in cash and cash equivalents resulting from deconsolidation of subsidiaries (98 ) (503 )
Net cash provided by investing activities 25,792 16,417
Financing activities:
Proceeds from term loan under Financing Agreement 450,000 —
Issuance costs and discounts associated with term loan under Financing Agreement (15,986 ) —
Repayment of term loan under Loan and Security Agreement (473,417 ) —
Proceeds from issuance of common stock through public offerings, net 314,759 144,049
Proceeds from BridgeBio common stock issuances under ESPP 2,364 1,809
Proceeds from stock option exercises, net of repurchases 778 312
Repurchase of RSU shares to satisfy tax withholding (4,679 ) (1,715 )
Other financing activities — 4,563
Net cash provided by financing activities 273,819 149,018
Net increase (decrease) in cash, cash equivalents and restricted cash 154,777 (92,240 )
Cash, cash equivalents and restricted cash at beginning of period 394,732 416,884
Cash, cash equivalents and restricted cash at end of period $ 549,509 $ 324,644

Six Months Ended June 30,
2024 2023
Supplemental Disclosure of Cash Flow Information:
Cash paid for interest $ 49,046 $ 28,738
Supplemental Disclosures of Noncash Investing and Financing Information:
Unpaid public offering issuance costs $ 18 $ —
Deferred and unpaid issuance costs recorded to "Accrued and other liabilities" $ 74 $ —
Unpaid property and equipment $ 70 $ 131
Transfers to noncontrolling interests $ (1,929 ) $ (5,940 )
Reconciliation of Cash, Cash Equivalents and Restricted Cash:
Cash and cash equivalents $ 407,958 $ 302,438
Restricted cash 139,409 19,930
Restricted cash — Included in "Other assets" 2,142 2,276
Total cash, cash equivalents and restricted cash at end of period shown in the
condensed consolidated statements of cash flows $ 549,509 $ 324,644

Blueprint Medicines Reports Second Quarter 2024 Results and Raises AYVAKIT®/AYVAKYT® (avapritinib) Full Year Revenue Guidance

On August 1, 2024 Blueprint Medicines Corporation (Nasdaq: BPMC) reported financial results, provided a business update for the second quarter ended June 30, 2024, and provided updated financial guidance (Press release, Blueprint Medicines, AUG 1, 2024, View Source [SID1234645249]).

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"This quarter marks a milestone, as we celebrate one full year since the U.S. approval of AYVAKIT for indolent systemic mastocytosis. We have delivered yet another very strong quarter of revenue as we continue to build this new rare disease market, and we are well on path to achieve more than $2 billion in potential peak sales. In addition, we have invested in our next pillars of growth, building on AYVAKIT as the cornerstone of a mast cell disease franchise. Our wild-type KIT inhibitor BLU-808 has entered a Phase 1 healthy volunteer study and we expect to initiate the registration-enabling HARBOR Part 2 study of our next-generation KIT D816V inhibitor elenestinib in indolent systemic mastocytosis later this year," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "With a significant and growing revenue base from AYVAKIT, a next wave of therapies in our pipeline that we believe can address even larger scale patient opportunities, and a financial profile anchored in sustainable topline revenue growth that enables us the flexibility to invest in the next wave of innovation, we are building Blueprint Medicines for long term value."

Second Quarter 2024 Highlights and Recent Progress

Mast cell disorders

· Achieved AYVAKIT net product revenues of $114.1 million for second quarter of 2024, representing more than 185 percent growth year-over-year.

· Achieved clearance of an Investigational New Drug application for BLU-808 by the U.S. Food and Drug Administration and initiated the healthy volunteer study. BLU-808 is a highly selective and potent investigational oral wild-type KIT inhibitor with best-in-class potential, for chronic urticaria and other mast cell disorders.

· Presented multiple datasets highlighting the long-term safety and durable clinical outcomes of AYVAKIT/AYVAKYT (avapritinib) across the spectrum of systemic mastocytosis (SM) at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) and European Hematology Association (EHA) (Free EHA Whitepaper) conferences. Read the presentations here.

Cell cycle inhibition

· Presented data from the Phase 1 VELA study of BLU-222, an oral, potent and selective CDK2 inhibitor, at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting. This is the first positive combination safety data with signals of early clinical activity for a CDK2 inhibitor in combination with an approved CDK4/6 inhibitor, ribociclib, and fulvestrant, in patients with HR+/HER2- breast cancer. Read the presentation here.

2024 Financial Guidance

Blueprint Medicines now anticipates approximately $435 million to $450 million in global AYVAKIT net product revenues for all approved indications in 2024, an increase from the previous range of $390 million to $410 million. 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 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 second half of 2024:

Mast cell disorders

· Initiate registration-enabling Part 2 of the HARBOR trial in indolent systemic mastocytosis (ISM).

Cell cycle inhibition

· Continue strategic business development discussions.

· Complete Phase 1 combination dose escalation for BLU-222 by end of year to inform registration plans.

Second Quarter 2024 Results

· Revenues: Revenues were $138.2 million for the second quarter of 2024, including $114.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $24.0 million in collaboration, license and other revenues. Revenues were $57.6 million in the second quarter of 2023, including $39.9 million of net product revenues from sales of AYVAKIT/AYVAKYT and $17.7 million in collaboration, license and other revenues.

· Cost of Sales: Cost of sales was $7.6 million for the second quarter of 2024, as compared to $2.3 million for the second quarter of 2023. The increase was primarily due to the sale of GAVRETO (pralsetinib) product to Rigel.

· R&D Expenses: Research and development expenses were $84.3 million for the second quarter of 2024, as compared to $110.1 million for the second 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 $12.3 million in stock-based compensation expenses for the second quarter of 2024.

· SG&A Expenses: Selling, general and administrative expenses were $89.3 million for the second quarter of 2024, as compared to $71.9 million for the second quarter of 2023. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $15.7 million in stock-based compensation expenses for the second quarter of 2024.

· Net Loss: Net loss was $50.0 million for the second quarter of 2024, as compared to a net loss of $132.8 million for the second quarter of 2023.

· Cash Position: As of June 30, 2024, cash, cash equivalents and investments were $868.5 million, as compared to $767.2 million as of December 31, 2023. This increase was primarily due to taking an additional draw under our 2022 debt facility with Sixth Street Partners. Blueprint Medicine’s cash and investments provide a durable capital position which, together with anticipated product revenues, the company believes will enable it to reach a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss second 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 299779. 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.