On May 4, 2023 Cardinal Health (NYSE: CAH) reported third quarter fiscal year 2023 revenues of $50.5 billion, an increase of 13% from the third quarter of fiscal year 2022 (Press release, Cardinal Health, MAY 4, 2023, View Source [SID1234631020]). Third quarter GAAP operating earnings were $572 million and GAAP diluted earnings per share (EPS) were $1.34. Third quarter non-GAAP operating earnings increased 11% to $606 million due to a significant increase in Pharmaceutical segment profit, partially offset by a decline in Medical segment profit. Non-GAAP diluted EPS increased 20% to $1.74, reflecting the improvement in non-GAAP operating earnings, a lower share count and lower interest expense, partially offset by a higher non-GAAP effective tax rate.

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"Our third quarter results were led by continued momentum and growth in the Pharmaceutical segment," said Jason Hollar, CEO of Cardinal Health. "With the strong overall performance in the quarter, we are pleased to raise our full year non-GAAP EPS guidance by $0.35 at the midpoint. In Medical, we continue to see improvement in underlying performance and remain confident in our Medical Improvement Plan initiatives. Across the enterprise, we continue to operate with urgency to drive our businesses forward and create value for our shareholders."

Q3 FY23 summary

Q3 FY23

Q3 FY22

Y/Y

Revenue

$50.5 billion

$44.8 billion

13 %

Operating earnings/(loss)

$572 million

$(97) million

N.M.

Non-GAAP operating earnings

$606 million

$545 million

11 %

Net earnings/(loss) attributable to Cardinal Health, Inc.

$345 million

$(1,391) million

N.M.

Non-GAAP net earnings attributable to Cardinal Health, Inc.

$447 million

$402 million

11 %

Effective Tax Rate2

36.3 %

(916.5) %

Non-GAAP Effective Tax Rate

22.4 %

20.1 %

Diluted EPS attributable to Cardinal Health, Inc.

$1.34

$(5.05)

N.M.

Non-GAAP diluted EPS attributable to Cardinal Health, Inc.

$1.74

$1.45

20 %

Segment results

Pharmaceutical segment

Q3 FY23

Q3 FY22

Y/Y

Revenue

$ 46.8 billion

$ 41.0 billion

14 %

Segment profit

$ 600 million

$ 487 million

23 %

Third-quarter revenue for the Pharmaceutical segment increased 14% to $46.8 billion, driven by brand and specialty pharmaceutical sales growth from existing customers.

Pharmaceutical segment profit increased 23% to $600 million in the third quarter, driven by positive generics program performance and a higher contribution from brand and specialty products.

Medical segment

Q3 FY23

Q3 FY22

Y/Y

Revenue

$ 3.7 billion

$ 3.9 billion

(5) %

Segment profit

$ 20 million

$ 59 million

(66) %

Third-quarter revenue for the Medical segment decreased 5% to $3.7 billion, driven by lower Products and Distribution sales, primarily due to PPE volumes and pricing.

Medical segment profit decreased 66% to $20 million in the third quarter, primarily due to lower Products and Distribution volumes and unfavorable sales mix. Additionally, these results reflect both net unfavorable non-recurring adjustments, including simplification actions, and an improvement in PPE margins.

Fiscal year 2023 outlook1

The company raised and narrowed its fiscal year 2023 guidance range for non-GAAP diluted earnings per share attributable to Cardinal Health, Inc. to $5.60 to $5.80, from $5.20 to $5.50.

This guidance includes an update to fiscal year 2023 Pharmaceutical segment profit outlook to 10.5% to 12% growth, from 4% to 6.5% growth and Medical segment profit outlook to a decline of approximately 50%, from flat to a decline of 20%.

Additionally, the company now expects interest and other in the range of $95 to $105 million, a non-GAAP effective tax rate of 22% to 23%, diluted weighted average shares outstanding of 262 to 263 million, capital expenditures of ~$450 million and adjusted free cash flow of $2.0 to $2.3 billion.

The company does not provide forward-looking guidance on a GAAP basis as certain financial information, the probable significance of which cannot be determined, is not available and cannot be reasonably estimated. See "Use of Non-GAAP Measures" following the attached schedules for additional explanation.

Investor Day

The company plans to host an Investor Day at 9:00 a.m. Eastern Standard Time on June 8 in New York City to detail its growth strategies and provide updates on its long-term outlook, capital allocation framework and the ongoing business and portfolio review. The event will be live-webcast and archived on Cardinal Health’s Investor Relations website.

Business Review Committee and Cooperation Agreement update

Business Review Committee and Cooperation Agreement update Cardinal Health’s management and Board of Directors, with support from the Business Review Committee, continue to work through the comprehensive review of the company’s strategy, portfolio, capital allocation framework, and operations. Given the importance of the work in maximizing Cardinal Health’s potential for the benefit of all stakeholders, the Board has extended the term of the Business Review Committee for an additional year through July 15, 2024. In connection with this extension, Cardinal Health has also extended the term of the company’s Cooperation Agreement with Elliott Investment Management L.P. ("Elliott") until the later of July 15, 2024 or until Elliott’s representative ceases to serve on, or resigns from, the company’s Board of Directors.

Recent highlights

Cardinal Health initiated and completed a $250 million dollar accelerated share repurchase program in the third quarter, resulting in a total of $1.5 billion year-to-date share repurchases in fiscal year 2023.
Cardinal Health announced the opening of two new distribution centers in Central Ohio. Both facilities support the company’s Medical segment, focusing on its U.S. Medical Products and Distribution and at-Home Solutions businesses.
Cardinal Health announced its collaboration with Signify Health to offer in-home clinical and medication management services through its Outcomes business.
Cardinal Health was selected by Autolus to provide core distribution capabilities required for U.S. commercialization of CAR T-cell therapies.
Cardinal Health recently introduced the Kangaroo OMNI Enteral Feeding platform, which is designed to accurately deliver thick formula and meet enteral feeding needs, from the hospital to home and infancy to end of life. OMNI is expected to launch in the United States and Canada in early fiscal year 2024.
Webcast

Cardinal Health will host a webcast today at 8:30 a.m. Eastern Standard Time to discuss third-quarter results. To access the webcast and corresponding slide presentation, go to the Investor Relations page at ir.cardinalhealth.com. No access code is required.

Presentation slides and a webcast replay will be available on the Investor Relations page for 12 months.

On May 4, 2023 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition, a well-validated oncology drug target, to develop novel therapies across a range of cancers, reported financial results for the first-quarter ended March 31, 2023, and provided a business update (Press release, Cardiff Oncology, MAY 4, 2023, View Source [SID1234631019]).

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"We are delighted to have dosed the first patient in our Phase 2 ONSEMBLE trial. This randomized trial will evaluate the efficacy of onvansertib combined with standard of care in patients with KRAS/NRAS-mutated mCRC, who historically have limited treatment options," said Mark Erlander, PhD, Chief Executive Officer of Cardiff Oncology. "In addition, we are excited to have appointed Fairooz Kabbinavar, MD, FACP, as our new Chief Medical Officer, who brings deep expertise in the treatment of patients with CRC as well as expertise in using bevacizumab in the clinical setting. We believe Dr. Kabbinavar adds significant value to our clinical development program for onvansertib. We also formally introduced the full membership of our Scientific Advisory Board, a group of highly esteemed oncology experts who are providing a diverse range of insights that strengthen our clinical programs."

Upcoming potential milestones

•Metastatic pancreatic ductal adenocarcinoma (mPDAC) data readout from Phase 2 trial expected in mid-2023
•Small cell lung cancer (SCLC) data readout from investigator-initiated (with UPMC) Phase 2 trial expected in mid-2023
•Triple negative breast cancer (TNBC) data readout from investigator-initiated (with Dana-Farber Cancer Institute) Phase 1b/2 trial expected in the fourth-quarter of 2023 or first-quarter of 2024
•mCRC randomized data readout from Phase 2 ONSEMBLE trial expected in the second-half of 2024

Company Highlights for the quarter ended March 31, 2023

•Announced the Appointment of Fairooz Kabbinavar, MD, FACP, as Chief Medical Officer. Dr. Kabbinavar oversees the clinical development program for the Company’s investigational drug onvansertib and reports directly to Chief Executive Officer, Mark Erlander, PhD.
•Formally introduced the full membership of its Scientific Advisory Board (SAB). The SAB is comprised of a distinguished group of academic and industry experts who bring depth and breadth of knowledge in oncology drug development, clinical trial design, translational science and clinical research. In most cases, these individuals have been advising the Company for several years and will now collectively serve a critical role as we advance the clinical development of

onvansertib. The Company anticipates that over time the membership of the SAB may expand to add additional expertise in new indications or stages of clinical development.
•Announced First Patient Dosed in ONSEMBLE Phase 2 Randomized Trial of Onvansertib in Patients with Metastatic Colorectal Cancer. The Phase 2 ONSEMBLE trial includes patients with mCRC who have a documented KRAS or NRAS mutation and have previously received one prior chemotherapy regimen with or without bevacizumab in the first line metastatic setting. Patients are being randomized to onvansertib plus FOLFIRI/bevacizumab versus FOLFIRI/bevacizumab (standard of care).

First-Quarter 2023 Financial Results

Liquidity, cash burn, and cash runway

As of March 31, 2023, Cardiff Oncology had approximately $97.0 million in cash, cash equivalents, and short-term investments.

Net cash used in operating activities for the first quarter of 2023 was approximately $8.7 million, a decrease of approximately $1.5 million from $10.2 million for the same period in 2022.

Based on its current expectations and projections, the Company believes its current cash resources are sufficient to fund its operations into 2025.

Operating results

Total operating expenses were approximately $12.1 million for the three months ended March 31 2023, an increase of $1.0 million from $11.1 million for the same period in 2022. The increase in operating expenses was primarily due to higher costs associated with clinical programs and outside service costs related to the development of the company’s lead drug candidate, salaries and staff costs primarily due to increased headcount.

On May 4, 2023 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, reported financial results for the first quarter ended March 31, 2023, as well as recent business highlights (Press release, C4 Therapeutics, MAY 4, 2023, View Source [SID1234631018]).

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"Thus far in 2023, we have executed against key milestones, including progressing three orally bioavailable degrader programs through the clinic and presented new preclinical data from our most recent clinical program, CFT1946, at AACR (Free AACR Whitepaper)," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "This preclinical data highlights the capabilities of our TORPEDO platform to develop catalytically efficient and orally bioavailable degraders and we look forward to continuing to advance both MonoDAC and BiDAC clinical programs to bring new therapeutic options to patients with difficult-to-treat diseases."

FIRST QUARTER 2023 AND RECENT HIGHLIGHTS

CFT7455: CFT7455 is an oral degrader of IKZF1/3 for the treatment of multiple myeloma (MM) and non-Hodgkin’s lymphomas (NHL).

Progressed the Phase 1/2 Clinical Trial: The dose escalation portion of the CFT7455 Phase 1/2 clinical trial continues in MM and NHL. The three arms of the trial are evaluating CFT7455 as a monotherapy for MM, in combination with dexamethasone for MM and as a monotherapy for NHL.
CFT8634: CFT8634 is an oral degrader of BRD9 for the treatment of synovial sarcoma and SMARCB1-null solid tumors.

Encouraging Initial Pharmacokinetic (PK) and Pharmacodynamic (PD) Data: In January 2023, shared PK and PD data from the initial escalation cohorts of the ongoing CFT8634 Phase 1/2 clinical trial demonstrating dose proportional exposure, strong oral bioavailability and deep BRD9 degradation.
Progressed the Phase 1/2 Clinical Trial: The dose escalation portion of the CFT8634 Phase 1/2 clinical trial continues in synovial sarcoma and SMARCB1-null solid tumors.
CFT1946: CFT1946 is an oral degrader targeting BRAF V600 mutations for the treatment of solid tumors including non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and melanoma.

Dosed First Patient in Phase 1/2 Clinical Trial: The CFT1946 Phase 1/2 clinical trial was initiated in January. Five trial sites are now open and enrolling patients with BRAF V600 solid tumors, including NSCLC, CRC and melanoma.
Presented New Preclinical Data at AACR (Free AACR Whitepaper): In April 2023, C4T presented new preclinical data on the discovery and characterization of CFT1946 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2023 Annual Meeting. The preclinical data demonstrated that CFT1946 is a potent and mutant-selective BiDAC degrader of BRAF V600 and is superior to inhibitors in in vitro and in vivo models with BRAF V600E driven disease and in the escape mutant BRAF V600E/NRAS-Q61K-driven model. The data further demonstrate C4T’s medicinal chemistry abilities to access catalytically efficient and orally bioavailable degraders by combining C4T’s cereblon toolkit with rational ligand and linker modifications.
KEY UPCOMING MILESTONES

CFT7455: Present Phase 1 dose escalation data from the Phase 1/2 clinical trial of Arm B1, evaluating CFT7455 as a monotherapy in MM, in the second half of 2023.
CFT8634: Present Phase 1 dose escalation data from the Phase 1/2 clinical trial for synovial sarcoma and SMARCB1-null solid tumors in the second half of 2023.
CFT1946: Present a Trial in Progress poster at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 3, 2023 at 8:00 AM CST, titled "A Phase 1/2 Study of CFT1946, A Novel Bifunctional Degradation Activating Compound, or BiDAC Degrader, of Mutant BRAF V600 as Monotherapy and in Combination with Trametinib, in Mutant BRAF V600 Solid Tumors."
CFT8919: Submit an Investigational New Drug (IND) application for CFT8919 for the treatment of NSCLC in the first half of 2023.
UPCOMING INVESTOR EVENTS

June 7, 2023: Management will participate in the Jefferies Global Healthcare Conference in New York.
FIRST QUARTER 2023 FINANCIAL RESULTS

Revenue: Total revenue for the first quarter of 2023 was $3.8 million, compared to $7.7 million for the first quarter of 2022. Total revenue reflects revenue recognized under collaboration agreements with Roche, Biogen and Calico.

Research and Development (R&D) Expense: R&D expense for the first quarter of 2023 was $29.0 million, compared to $26.2 million for the first quarter of 2022. The increase in R&D expense was primarily attributable to increased clinical expenses from the ongoing CFT7455, CFT8634 and CFT1946 Phase 1/2 clinical trials.

General and Administrative (G&A) Expense: G&A expense for the first quarter of 2023 was $10.9 million, compared to $12.8 million for the first quarter of 2022. The decrease in G&A expense was primarily attributable to decreased professional fees and stock compensation expense compared to 2022.

Net Loss and Net Loss per Share: Net loss for the first quarter of 2023 was $34.8 million, compared to $31.6 million for the first quarter of 2022. Net loss per share for the first quarter of 2023 was $0.71 compared to $0.65 for the first quarter of 2022.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of March 31, 2023, were $305.0 million, compared to $337.1 million as of December 31, 2022. The decrease in cash was primarily driven by expenditures to fund operations. C4T expects that its cash, cash equivalents and marketable securities as of March 31, 2023, along with cost savings, will be sufficient to fund planned operating expenses and capital expenditures into 2025.

About CFT7455

CFT7455 is an orally bioavailable MonoDAC degrader designed to be highly potent and selective against its intended targets of Ikaros (IKZF1) and Aiolos (IKZF3). CFT7455 binds with high affinity to the E3 ligase adapter protein, cereblon, to target and degrade IKZF1/3 for the treatment of multiple myeloma and non-Hodgkin’s lymphomas, including peripheral T cell lymphoma and mantle cell lymphoma. In early clinical data, CFT7455 demonstrated deep and durable degradation of IKZF1/3. C4T is enrolling patients in its ongoing Phase 1/2 clinical trial of CFT7455. More information about this trial may be accessed at www.clinicaltrials.gov (identifier: NCT04756726).

About CFT8634

CFT8634 is an orally bioavailable BiDAC degrader designed to be potent and selective against BRD9. BRD9 was previously considered an undruggable target due to the inability of bromodomain inhibitors to effectively treat cancers dependent on BRD9. Unlike BRD9 inhibition, BRD9 degradation has been shown to be efficacious in pre-clinical models of synovial sarcoma. C4T is enrolling patients in its ongoing Phase 1/2 clinical trial of CFT8634 for the treatment of synovial sarcoma and SMARCB1-null solid tumors. More information about this trial may be accessed at www.clinicaltrials.gov (identifier: NCT05355753).

About CFT1946

CFT1946 is an orally bioavailable BiDAC degrader designed to be potent and selective against BRAF V600 mutant targets. In preclinical studies, CFT1946 is active in vivo and in vitro in models with BRAF V600E driven disease and in models resistant to BRAF inhibitors. C4T is advancing CFT1946 to the clinic to study treatment for BRAF V600 mutant solid tumors including non-small cell lung cancer, colorectal cancer, and melanoma. C4T is enrolling patients in its ongoing Phase 1/2 clinical trial of CFT1946. More information about this trial may be accessed at www.clinicaltrials.gov (identifier: NCT05668585).

On May 4, 2023 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, 2023 and provided an update on the Company’s operations (Press release, BridgeBio, MAY 4, 2023, View Source [SID1234631017]).

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"We were excited to announce the results from the 5th cohort of our Phase 2 trial of infigratinib for achondroplasia – an oral agent with a well-tolerated safety profile and best-in-class efficacy to date," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "We thank the children and physicians who have partnered with us on this study and are eager to take the next steps for this program together. Infigratinib pairs with our ADH1 program, which reads out its Phase 3 next year, to form a high-quality endocrine franchise that we are continuing to build out. With this dataset for achondroplasia in hand, we look forward to July and the next big readout from our pipeline, the 30-month data from our ATTR-CM Phase 3 trial of acoramidis that recently completed last patient last visit. We thank the ATTR-CM patient and physician communities who made this trial possible, and we look forward to sharing the results with you."

BridgeBio’s key programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM):
The Phase 3 ATTRibute-CM study has completed last patient last visit.
The Company expects to announce topline registrational data for the month 30 primary endpoint, a hierarchical composite including all-cause mortality and cardiovascular-related hospitalizations, in late July, 2023.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia:
In March 2023, the Company reported data from the fifth dosing cohort of the Phase 2 dose-escalation trial PROPEL 2, demonstrating a mean change from baseline in annualized height velocity (AHV) of +3.03 cm/year for the ten children that have had six-month visits.
80% of the ten children with six-month visits in the fifth cohort were responders, as defined by an increase from baseline AHV of at least 25%.
As a result of treatment, the median absolute AHV reached 7.6 cm/yr, which is beyond the 99th percentile of growth for children living with achondroplasia.
Combined with the previously reported Cohort 4 change from baseline in AHV value of +1.52 cm/yr, the Cohort 5 data demonstrate a strong dose response for infigratinib.
To date, the study has shown a well-tolerated safety profile, with no study drug related treatment emergent adverse events (TEAEs) in Cohort 5. No serious adverse events (SAEs) or discontinuations due to AEs were reported in any cohort.
The Company has begun enrolling children in the run-in for a registrational Phase 3 trial.
The Company believes infigratinib, if approved, has the potential to capture a significant share of the market based on blinded market research.
BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2I (LGMD2I):
The Company announced development of a validated novel bioassay that directly measures glycosylated αDG, which is central to LGMD2I disease and enables monitoring of responses to disease-modifying therapies in LGMD2I patients.
The Company also reported positive 15-month results from the ongoing Phase 2 clinical trial in October 2022, showing a doubling of glycosylated αDG in LGMD2I patients treated with BBP-418; a sustained decrease of ≥70% in creatine kinase (CK), a marker of muscle breakdown; and improvements in ambulatory and clinical function measures after 15 months of treatment.
The Company intends to initiate a global Phase 3 registrational trial of BBP-418 for LGMD2I in mid-2023, and has engaged with regulatory authorities to align on a trial design.
BBP-418 has a potentially-addressable population of 7,000 patients in the United States and European Union.
There are currently no disease-modifying treatments available for LGMD2I.
Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1):
The pivotal Phase 3 CALIBRATE trial of encaleret for ADH1 continues to proceed.
Population genetics analyses estimate approximately 25,000 carriers of gain-of-function variants of the CaSR, the underlying cause of ADH1, in the United States and European Union.
The Company anticipates sharing topline data from CALIBRATE in the first half of 2024.
If approved, encaleret could be the first therapy specifically indicated for the treatment of ADH1.
BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH):
The Phase 1/2 gene therapy trial of BBP-631 for CAH continued to progress; as of February 1, 2023, BBP-631 has been generally well-tolerated in four patients treated at the first two dose levels.
The Company plans to provide a data update by the end of 2023.
CAH is one of the most prevalent genetic diseases potentially addressable with adeno-associated virus (AAV) gene therapy, with more than 75,000 cases estimated in the United States and European Union.
RAS cancer portfolio:
BridgeBio is continuing to develop the three main programs of its RAS franchise:
BBO-8520, an investigational, next-generation small molecule direct KRASG12C(ON) inhibitor candidate that is designed to directly bind and inhibit KRASG12C in both its ON (GTP-bound) and OFF (GDP-bound) conformations, which remains on track to file an IND and enter the clinic in the second half of 2023.
A PI3Kα:RAS breaker program, investigational small molecules that are designed to block Ras-driven PI3Kα activation with a novel and potentially broad mechanism of action to target not only PI3Kα mutant tumors and RAS mutant tumors, but potentially other tumors driven by RTK activation of RAS signaling. The Company remains on track to select a development candidate in 2023, with IND filing to follow in 2024.
The Company’s pan-KRAS program, which targets multiple KRAS mutants including KRASG12D and KRASG12V, which are present in a large percentage of colorectal, pancreatic, and non-small cell lung cancer tumors. Development candidate selection for this program is planned for late 2023 or early 2024.
Corporate Update:

Follow-on offering:
The Company closed an underwritten public offering of shares of its common stock with gross proceeds of approximately $150 million.
First Quarter 2023 Financial Results:
Cash, Cash Equivalents, Marketable Securities and Restricted Cash (Current)

Cash, cash equivalents, marketable securities and restricted cash (current), totaled $467.0 million as of March 31, 2023, compared to $466.2 million as of December 31, 2022. The net increase of $0.8 million in cash, cash equivalents, marketable securities and restricted cash (current) is primarily attributable to the net proceeds received of $143.0 million from the follow-on public offering, and proceeds from common stock issuance under ESPP and stock option exercises of $2.0 million, partially offset by net cash used in operating activities of $144.3 million.

Operating Costs and Expenses

Operating costs and expenses decreased by $47.4 million to $128.0 million for the three months ended March 31, 2023, compared to $175.4 million for the same period in the prior year. The overall decrease in operating costs and expenses for the first quarter of 2023 compared to the comparative period was mainly due to decreases in research, development and other (R&D) expenses of $15.5 million resulting from the Company’s reprioritization of its R&D programs; selling, general and administrative expenses of $12.6 million resulting from its company-wide streamlining of costs; and restructuring, impairment and related charges of $19.3 million since the majority of the restructuring initiatives were initiated in the first quarter of 2022. The effects of the Company’s restructuring initiative that was started in the first quarter of 2022 are continuing to be realized due to the Company’s reductions in its operating costs and expenses. Restructuring, impairment and related charges for the three months ended March 31, 2023 of $3.4 million, were primarily comprised of winding down, exit and other related costs. Restructuring, impairment and related charges for the same period in prior year were $22.7 million, were primarily related to impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs. The Company continues to evaluate restructuring alternatives to drive operational changes in business processes, efficiencies, and cost savings.

Stock-based compensation expenses included in operating costs and expenses for the first quarter of 2023 were $23.5 million, of which $11.8 million is included in research, development and other (R&D) expenses, $11.7 million is included in selling, general and administrative expenses, and nil is included in restructuring, impairment and related charges. Stock-based compensation expenses included in operating costs and expenses for the first quarter of 2022 were $24.3 million, of which $8.6 million is included in research, development and other (R&D) expenses, $14.6 million is included in selling, general and administrative expenses, and $1.2 million is included in restructuring, impairment and related charges.

"Strengthening our balance sheet through our recent $150 million follow-on offering extends our runway into the second half of 2024, and puts us in a strong position to take advantage of our optionality as we head into this summer’s ATTR-CM readout," said Brian Stephenson, Ph.D., CFA, Chief Financial Officer of BridgeBio. "We will continue to explore ways to extend our runway further through considering potential partnerships and royalty transactions."

On May 4, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the first quarter ended March 31, 2023 (Press release, Blueprint Medicines, MAY 4, 2023, View Source [SID1234631016]).

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"Our first quarter was marked by executional progress across the multiple growth opportunities that Blueprint Medicines has to expand our impact and transform outcomes for thousands of patients worldwide. We drove strong AYVAKIT revenue growth, presented seminal data for AYVAKIT in indolent systemic mastocytosis, and worked to progress our broad pipeline by rapidly resolving the partial clinical hold for BLU-222 and generating early clinical data across our portfolio," said Kate Haviland, Chief Executive Officer at Blueprint Medicines. "Blueprint Medicines is ready to further solidify our leadership in systemic mastocytosis and scale the impact of AYVAKIT by addressing the medical needs of a substantially larger group of patients with our anticipated FDA label expansion for indolent systemic mastocytosis in a few weeks."

First Quarter 2023 Highlights and Recent Progress

Systemic mastocytosis (SM)

· Presented full registrational PIONEER trial data for AYVAKIT in patients with indolent systemic mastocytosis (ISM) at the 2023 American Academy of Asthma, Allergy, and Immunology (AAAAI) Annual Conference. These data included a statistically significant and clinically meaningful improvement in total symptom score that deepened over time, with improvements shown across all individual symptoms and in quality-of-life measures. AYVAKIT showed a favorable safety profile compared to placebo, supporting potential for chronic treatment. Read the press release here.
· U.S. Food and Drug Administration (FDA) target action date for AYVAKIT supplemental new drug application for ISM is May 22, 2023.

EGFR-driven non-small cell lung cancer (NSCLC)

· Announced plans to present results from the ongoing dose escalation of the Phase 1/2 CONCERTO trial of BLU-451 in EGFR exon 20 insertion-positive NSCLC, showing early safety and clinical activity, at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
· Announced plans to present updated results from the dose escalation of the Phase 1/2 SYMPHONY trial showing safety and tolerability of BLU-945 both as a monotherapy and in combination with osimertinib in late-line EGFR-driven NSCLC at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.
· Presented real-world data showing that NSCLC patients with an EGFR L858R mutation have worse outcomes compared to patients with an EGFR exon 19 deletion mutation when treated with first-line osimertinib, and new preclinical data showing the combination of BLU-945 and osimertinib enhanced tumor regression and survival compared to osimertinib alone in a treatment-naïve EGFR L858R-mutant cell-derived model at the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Read the poster presentation here.
· Today announced acceptance of an investigational new drug (IND) application to the FDA for BLU-525.

CDK2-vulnerable breast and other cancers

· Announced the FDA lifted the partial clinical hold on patient enrollment in the VELA trial of BLU-222. Patients already enrolled in the trial have continued on the study and trial sites are working to resume patient enrollment.
· Announced plans to present results from the ongoing dose escalation of the Phase 1/2 VELA trial of BLU-222 in breast cancer and other cancers vulnerable to CDK2 inhibition, showing evidence of monotherapy safety and pathway modulation, at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.

Key Upcoming Milestones

The company plans to achieve the following milestones by mid-2023:

· Obtain FDA approval and initiate launch of AYVAKIT in ISM in mid-2023.
· Present initial CONCERTO trial dose escalation data in EGFR exon 20 NSCLC at ASCO (Free ASCO Whitepaper) 2023.
· Present initial VELA trial dose escalation data at ASCO (Free ASCO Whitepaper) 2023.
· Nominate a development candidate targeting wild-type KIT for chronic urticaria by mid-2023.

First Quarter 2023 Results

· Revenues: Revenues were $63.3 million for the first quarter of 2023, including $39.1 million of net product revenues from sales of AYVAKIT/AYVAKYT and $24.2 million in collaboration revenues. Blueprint Medicines recorded revenues of $62.7 million in the first quarter of 2022, including $23.8 million of net product revenues from sales of AYVAKIT/AYVAKIT and $38.9 million in collaboration revenues.
· Cost of Sales: Cost of sales was $3.2 million for the first quarter of 2023, as compared to $5.1 million for the first quarter of 2022.
· R&D Expenses: Research and development expenses were $112.1 million for the first quarter of 2023, as compared to $103.1 million for the first quarter of 2022. This increase was primarily due to increased compensation related costs driven by increased headcount, along with increased costs related to early discovery efforts. Research and development expenses included $10.1 million in stock-based compensation expenses for the first quarter of 2023.
· SG&A Expenses: Selling, general and administrative expenses were $71.0 million for the first quarter of 2023, as compared to $57.1 million for the first quarter of 2022. This increase was primarily due to increased compensation related costs driven by increased headcount, along with increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $13.1 million in stock-based compensation expenses for the first quarter of 2023.
· Net Loss: Net loss was $129.6 million for the first quarter of 2023, or a net loss per share of $2.15, as compared to a net loss of $106.0 million for the first quarter of 2022, or a net loss per share of $1.79.
· Cash Position: As of March 31, 2023, cash, cash equivalents and investments were $961.3 million, as compared to $1,078.5 million as of December 31, 2022.

2023 Financial Guidance

Blueprint Medicines has updated its financial guidance and now anticipates approximately $135 million to $145 million in AYVAKIT net product revenues for advanced SM and GIST in 2023, and $40 million to $50 million in collaboration revenues from existing collaborations in 2023. This guidance excludes revenue from the anticipated AYVAKIT indication expansion in ISM in mid-2023. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve 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 first quarter 2023 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 929-526-1599 (international), and referring to conference ID 668091. 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:

· Goldman Sachs 44th Annual Global Healthcare Conference on Wednesday, June 14, 2023 at 12:20 pm ET.

A live webcast of each presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcasts will be archived on Blueprint Medicines’ website for 30 days following each presentation.