On October 31, 2024 Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, reported that Jeffrey Stein, Ph.D., President and Chief Executive Officer, will participate in the following November investor conferences (Press release, Cidara Therapeutics, OCT 31, 2024, View Source [SID1234647599]).

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Details are as follows:

Event: Guggenheim Securities Healthcare Innovation Conference
Date: Monday, November 11, 2024
Time: 1:30 PM ET
Format: Fire side chat
Webcast: View Source

A replay of the presentation will be available in the Investors section on the Company’s website at www.cidara.com. The replay of the presentation will be available for 90 days.

Event: Jefferies London Healthcare Conference
Date: Tuesday, November 19 – Thursday, November 21, 2024

Cidara will participate in one-on-one investor meetings during these events. Investors interested in meeting with Cidara at the conferences should contact the appropriate conference directly.

On October 31, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported financial results for the third quarter ended September 30, 2024, as well as recent business highlights (Press release, C4 Therapeutics, OCT 31, 2024, View Source [SID1234647584]).

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"2024 has been a successful year for C4T marked by strong execution across our entire portfolio, which has continued to position us as a leader in targeted protein degradation science. We recently shared initial clinical data on CFT1946 demonstrating a well-tolerated safety profile and evidence of monotherapy anti-tumor activity in BRAF inhibitor exposed patients, and we delivered our second development candidate to Biogen," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "We look forward to continuing this momentum with cemsidomide Phase 1 data in multiple myeloma and in non-Hodgkin’s lymphoma being presented at ASH (Free ASH Whitepaper), and multiple CFT1946 data readouts expected in 2025. Our continued focus on discovery research, clinical development and collaborations sets us up for an exciting future and for the potential to improve patients’ lives."

THIRD 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. Dose escalation continues as the maximum tolerated dose has not yet been reached. For the combination with dexamethasone MM arm, patients are now enrolling at dose level 6 (100 µg once daily (QD)) and enrollment in the dose level 5 (75 µg QD) expansion cohort is complete. The monotherapy NHL arm continues to progress and dose level 5 (100 µg QD) is the highest dose level evaluated to date.
CFT1946: CFT1946 is an oral degrader targeting BRAF V600 mutations for the potential treatment of solid tumors including colorectal cancer (CRC), melanoma and non-small cell lung cancer (NSCLC).
•Presented New Preclinical Data at the 7th Annual Targeted Protein Degradation (TPD) Summit: In October 2024, C4T shared new in vivo data for CFT1946 describing Kpu,u (range: 0.34 – 0.88), experimentally determined using independent methods in two different species. These results demonstrate CFT1946’s ability to cross the blood brain barrier and highlight the potential for activity in primary and metastatic central nervous system (CNS) disease.
•Presented Monotherapy Clinical Data Demonstrating Proof of Mechanism and Early Evidence of Proof of Concept from the Ongoing CFT1946 Phase 1 Trial at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024: CFT1946 demonstrated a well-tolerated safety profile and evidence of monotherapy anti-tumor activity, which supports further clinical development as monotherapy and in combination with MEK and EGFR targeted therapies.
•Advanced the Phase 1/2 Clinical Trial. The CFT1946 Phase 1/2 trial for BRAF V600 mutant solid tumors continues to enroll patients across multiple arms of the trial:
◦CFT1946 Monotherapy Dose Escalation in BRAF V600 Mutant Solid Tumors: Patients continue to enroll in the 640 mg twice daily (BID) pharmacodynamic (PD) backfill cohort.
◦CFT1946 Monotherapy Melanoma Expansion Cohorts: Enrollment at the 320 mg BID dose level is complete and patients are enrolling at the 640 mg BID dose level.
◦CFT1946 with Cetuximab Phase 1b Dose Escalation for CRC: The 160 mg BID dose level has been declared safe and patients are enrolling at the 320 mg BID dose level.
RESEARCH UPDATES:
•Presented at the 7th Annual TPD Summit Highlighting C4T’s TORPEDO Platform: In October 2024, at the 7th Annual TPD Summit, C4T presented kinetics-based pharmacokinetic (PK) and PD models, demonstrating the company’s ability to predict clinical PD responses across target classes.
COLLABORATIONS:
•Delivered Second Development Candidate to Biogen. In September 2024, C4T earned an additional $8 million milestone payment after Biogen accepted delivery of a second development candidate in an undisclosed indication. Biogen is responsible for all future clinical development and commercialization for this program. This marks the final development candidate under this strategic collaboration.
CORPORATE UPDATES:
•In October 2024, C4T appointed Paige Mahaney, Ph.D., as the company’s chief scientific officer, succeeding Stew Fisher, Ph.D., who is retiring to pursue personal interests. Dr. Mahaney brings over 25 years of pharmaceutical executive leadership with multidisciplinary expertise in discovery research and development along with successfully building clinical portfolios across a wide range of disease indications and treatment modalities.
•In September 2024, C4T appointed Stephen Fawell, Ph.D., to the Board of Directors. Dr. Fawell brings decades of experience leading discovery and development strategies for global pharmaceutical companies.
KEY UPCOMING MILESTONES
Cemsidomide:

•Present updated dose escalation and expansion cohort data from the 50 µg M/W/F, 37.5 µg QD, 62.5 µg QD, and 75 µg QD dose levels in approximately 40 patients from the ongoing Phase 1/2 clinical trial with dexamethasone in R/R MM at the ASH (Free ASH Whitepaper) Annual Meeting in December 2024.
•Present dose escalation data from the 25 µg M/W/F, 50 µg M/W/F, 37.5 µg QD, 62.5 µg QD, and 100 µg QD dose levels in approximately 25 patients from the ongoing monotherapy Phase 1/2 clinical trial in R/R NHL at the ASH (Free ASH Whitepaper) Annual Meeting in December 2024.
•Complete Phase 1 dose exploration in R/R MM and R/R NHL by year-end.
CFT1946:
•Initiate the Phase 1b dose escalation cohort evaluating CFT1946 in combination with trametinib in melanoma by year-end.
•Present data from multiple anticipated readouts in 2025, including:
◦Full monotherapy CFT1946 dose escalation cohorts in BRAF V600 mutant solid tumors.
◦Expansion cohorts evaluating CFT1946 as a monotherapy in melanoma.
◦Phase 1b dose escalation cohort evaluating CFT1946 in combination with cetuximab in CRC.
UPCOMING INVESTOR EVENTS:
•November 20, 2024, at 8 am GT: Management will participate in a fireside chat at the Jefferies London Healthcare conference taking place in London, UK.
•December 8, 2024: Management will host an investor call to discuss MM and NHL data from the ongoing Phase 1/2 trial of cemsidomide.
THIRD QUARTER 2024 FINANCIAL RESULTS
Revenue: Total revenue for the third quarter of 2024 was $15.4 million, compared to $11.1 million for the third quarter of 2023. The increase in revenue was primarily due to recognition of an $8.0 million milestone from Biogen as well as $2.9 million of revenue recognized under our license and supply agreement with Betta, partially offset by reduced revenue from our agreement with Roche due to the completion of research activities in the third quarter of 2023 for a nominated target. Total revenue for the third quarter of 2024 reflects revenue recognized under our collaborations with Biogen, Betta, Merck, Merck KGaA, Darmstadt, Germany (MKDG) and Roche, and total revenue recognized in the third quarter of 2023 reflects revenue recognized under collaboration agreements with Biogen and Roche.
Research and Development (R&D) Expense: R&D expense for the third quarter of 2024 was $31.8 million, compared to $28.3 million for the third quarter of 2023. The increase in R&D expense was primarily due to increased clinical trial expense as cemsidomide and CFT1946 continue to advance, partially offset by lower personnel costs.
General and Administrative (G&A) Expense: G&A expense was $11.8 million for the third quarter of 2024, compared to $10.5 million for the third quarter of 2023. The increase in G&A expense was primarily attributable to higher personnel expense related to stock-based compensation.
Net Loss and Net Loss per Share: Net loss for the third quarter of 2024 was $24.7 million, compared to $27.0 million for the third quarter of 2023. Net loss per share for the third quarter of 2024 was $0.35 compared to $0.55 for the third quarter of 2023.
Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of September 30, 2024 were $284.4 million, compared to $295.7 million as of June 30, 2024, and $281.7 million as of December 31, 2023. The reduction in cash, cash equivalents and marketable securities during the third quarter was primarily the result of cash used in operating activities, partially offset by $10.3 million in net proceeds raised through our at-the-market (ATM) facility. C4T expects that its cash, cash equivalents and marketable securities as of September 30, 2024 will be sufficient to fund planned operating expenses and capital expenditures into 2027.

On October 31, 2024 Bristol Myers Squibb (NYSE: BMY) reported results for the third quarter of 2024 (Press release, Bristol-Myers Squibb, OCT 31, 2024, View Source [SID1234647583]).

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"We made important strides in the third quarter with the landmark U.S. approval of Cobenfy in schizophrenia, continued sales momentum, strong cash flow generation and key pipeline achievements," said Christopher Boerner, Ph.D., board chair and chief executive officer, Bristol Myers Squibb. "We’re focused on closing out the year with strong execution as we deliver on our Growth Portfolio, prioritize high-growth opportunities and continue delivering transformational results for patients."

Third Quarter

$ in millions, except per share amounts

2024

2023

Change

Change Excl.
F/X**

Total Revenues

$11,892

$10,966

8%

10%

Earnings Per Share — GAAP*

0.60

0.93

(35)%

N/A

Earnings Per Share — Non-GAAP* **

1.80

2.00

(10)%

N/A

Acquired IPRD Charge and Licensing Income Net Impact on Earnings Per Share

(0.09

)

(0.03

)

N/A

N/A

*GAAP and Non-GAAP earnings per share include the net impact of Acquired IPRD charges and licensing income.

**See "Use of Non-GAAP Financial Information".

THIRD QUARTER RESULTS
All comparisons are made versus the same period in 2023 unless otherwise stated.

Bristol Myers Squibb posted third quarter revenues of $11.9 billion, an increase of 8%, or 10% when adjusted for foreign exchange impacts, primarily driven by the Growth Portfolio and Eliquis, partially offset by generic erosion of Sprycel due to the loss of exclusivity.
U.S. revenues increased 9% to $8.2 billion, and International revenues increased 7% to $3.7 billion, primarily due to the Growth Portfolio and higher demand for Eliquis, partially offset by generic erosion of Sprycel due to the loss of exclusivity. The negative impact from foreign exchange on International revenues was 4%.
On a GAAP basis, gross margin decreased from 77.1% to 75.1%, and on a non-GAAP basis decreased from 77.3% to 76.0%, primarily due to product mix.
On a GAAP and non-GAAP basis, marketing, selling and administrative expenses remained relatively flat at $2.0 billion.
On a GAAP basis, research and development expenses increased 6%, and 8% on a non-GAAP basis, to $2.4 billion, primarily due to recent acquisitions.
On a GAAP and non-GAAP basis, Acquired IPRD increased to $262 million from $80 million. On a GAAP and non-GAAP basis, licensing income was $25 million compared to $12 million.
On a GAAP basis, amortization of acquired intangible assets increased 7% to $2.4 billion, primarily due to the RayzeBio acquisition in 2024 and approval of Augtyro in the fourth quarter of 2023.
On a GAAP basis, the effective tax rate increased from 9.5% to 27.5%, and on a non-GAAP basis increased from 11.6% to 18.5%, primarily due to jurisdictional earnings mix and adjustments in 2023 to reflect IRS income tax guidance issued in 2023 regarding deductibility of certain non-U.S. research and development expenses.
On a GAAP basis, the company reported net income attributable to Bristol Myers Squibb of $1.2 billion, or $0.60 per share, during the third quarter of 2024 compared to $1.9 billion, or $0.93 per share, for the same period a year ago. The company reported non-GAAP net earnings attributable to Bristol Myers Squibb of $3.7 billion, or $1.80 per share, during the third quarter of 2024 compared to $4.1 billion, or $2.00 per share, for the same period a year ago. In addition to the items above, GAAP and non-GAAP earnings per share were impacted by higher interest expense.

THIRD QUARTER PRODUCT REVENUE HIGHLIGHTS

($ amounts in millions)

Quarter Ended September
30, 2024

% Change from Quarter
Ended September 30,
2023

% Change from
Quarter Ended
September 30,
2023 Ex-F/X**

U.S.

Int’l (c)

WW (d)

U.S.

Int’l (c)

WW (d)

Int’l (c)

WW (d)

Growth Portfolio

Opdivo

$

1,366

$

994

$

2,360

2%

7%

4%

16%

7%

Orencia

706

230

936

—%

6%

1%

13%

3%

Yervoy

399

243

642

11%

10%

11%

17%

13%

Reblozyl

358

89

447

79%

85%

80%

90%

81%

Opdualag

216

17

233

33%

>200%

40%

>200%

40%

Abecma

77

47

124

12%

96%

33%

100%

34%

Zeposia

105

42

147

11%

50%

20%

46%

19%

Breyanzi

173

51

224

125%

>200%

143%

>200%

143%

Camzyos

135

21

156

101%

>200%

129%

>200%

129%

Sotyktu

51

15

66

(18)%

>200%

—%

>200%

—%

Augtyro

10

—

10

N/A

N/A

N/A

N/A

N/A

Krazati

32

2

34

N/A

N/A

N/A

N/A

N/A

Other Growth Products (a)

172

261

433

15%

61%

39%

64%

41%

Total Growth Portfolio

3,800

2,012

5,812

15%

22%

18%

29%

20%

Legacy Portfolio

Eliquis

2,045

957

3,002

15%

3%

11%

2%

11%

Revlimid

1,212

200

1,412

—%

(9)%

(1)%

(6)%

(1)%

Pomalyst/Imnovid

697

201

898

15%

(24)%

3%

(24)%

3%

Sprycel

225

65

290

(44)%

(45)%

(44)%

(42)%

(43)%

Abraxane

151

102

253

(15)%

24%

(3)%

37%

1%

Other Legacy Products (b)

102

123

225

17%

(18)%

(5)%

(19)%

(5)%

Total Legacy Portfolio

4,432

1,648

6,080

4%

(7)%

1%

(6)%

1%

Total Revenues

$

8,232

$

3,660

$

11,892

9%

7%

8%

11%

10%

**

See "Use of Non-GAAP Financial Information".

(a)

Includes Nulojix, Onureg, Inrebic, Empliciti and royalty revenue.

(b)

Includes other mature brands.

(c)

Beginning in 2024, Puerto Rico revenues are included in International revenues. Prior period amounts have been reclassified to conform to the current presentation.

(d)

Worldwide (WW) includes U.S. and International (Int’l).

THIRD QUARTER PRODUCT REVENUE HIGHLIGHTS
Growth Portfolio
Growth Portfolio worldwide revenues increased to $5.8 billion compared to $4.9 billion in the prior year period, representing growth of 18% on a reported basis or 20% when adjusted for foreign exchange impacts. Growth Portfolio revenues were primarily driven by higher demand for Reblozyl, Breyanzi, Camzyos and Opdualag.

Legacy Portfolio
Revenues for the Legacy Portfolio in the third quarter were $6.1 billion compared to $6.0 billion in the prior year period, representing growth of 1% on a reported basis and when adjusted for foreign exchange impacts. Legacy Portfolio revenues were primarily driven by higher demand for Eliquis , partially offset by a decline in demand for Sprycel due to generic erosion.

PRODUCT AND PIPELINE UPDATE
Bristol Myers Squibb recently achieved several important clinical and regulatory milestones, including the U.S. approval of Cobenfy and the disclosure of long-term cardiovascular and oncology data that underscore the strength of the company’s science.

With Cobenfy, the company is re-establishing its presence in neuroscience and introducing the first new pharmacological approach to treat schizophrenia in decades.

Today, the company is providing an update on data from two Phase 3 oncology trials, CheckMate -8HW and CheckMate -901. Please see the table below for more information.

Neuroscience

Category

Asset

Milestone

Regulatory

Cobenfy TM
(xanomeline and
trospium
chloride)

The U.S. Food and Drug Administration (FDA) approved Cobenfy, previously referred to as KarXT, for the treatment of schizophrenia in adults, with a mechanism of action distinct from current therapies. The approval is based on data from the EMERGENT clinical program, which includes three placebo-controlled efficacy and safety trials and two open-label trials evaluating the long-term safety and tolerability of Cobenfy for up to one year.

Cardiovascular

Category

Asset

Milestone

Clinical &
Research

Camzyos
(mavacamten)

Long-term follow-up results from the EXPLORER-LTE cohort of the MAVA-Long-Term Extension study evaluating Camzyos in adult patients with New York Heart Association (NYHA) class II-III symptomatic obstructive hypertrophic cardiomyopathy demonstrated that patients experienced consistent and sustained improvements in echocardiographic measures and biomarkers after up to 3.5 years of continuous treatment.

Patients experienced an improvement in symptoms and functional capacity as measured by NYHA class and patient-reported outcomes. The safety profile of Camzyos for up to 3.5 years remained consistent with the established safety profile and no new safety signals were identified.

Oncology

Category

Asset

Milestone

Regulatory

Opdivo
(nivolumab)

The FDA approved Opdivo for the treatment of adult patients with resectable (tumors ≥ 4cm or node positive) non-small cell lung cancer (NSCLC) and no known epidermal growth factor receptor mutations or anaplastic lymphoma kinase rearrangements, for neoadjuvant treatment, in combination with platinum-doublet chemotherapy, followed by single-agent Opdivo as adjuvant treatment after surgery. The approval is based on results from the Phase 3 randomized CheckMate -77T trial.

Opdivo +
Yervoy
(ipilimumab)

The FDA accepted the supplemental Biologics License Application for Opdivo plus Yervoy as a potential first-line treatment for adult patients with unresectable hepatocellular carcinoma. The acceptance is based on results from the Phase 3 CheckMate -9DW trial. The FDA assigned a Prescription Drug User Fee Act goal date of April 21, 2025.

Clinical &
Research

Opdivo

The Phase 3 CheckMate -8HW trial evaluating Opdivo plus Yervoy compared to Opdivo monotherapy across all lines of therapy as a treatment for patients with microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer met the dual primary endpoint of progression-free survival (PFS) as assessed by Blinded Independent Central Review at a pre-specified interim analysis. Previously, Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful improvement in PFS compared to chemotherapy.

Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful improvement in PFS compared to Opdivo monotherapy across all lines of therapy. The study is ongoing to assess various secondary endpoints, including overall survival (OS). The safety profile for the combination of Opdivo plus Yervoy remained consistent with previously reported data, with no new safety signals identified.

Opdivo

The Phase 3 CheckMate -901 trial evaluating Opdivo plus Yervoy versus standard-of-care non-cisplatin-based chemotherapy in patients with unresectable or metastatic urothelial carcinoma (UC) who are ineligible for cisplatin-based chemotherapy did not meet its primary endpoint of OS. The safety profile for Opdivo and Yervoy was consistent with previously reported data, with no new safety signals identified.

Opdivo has previously shown clinical benefit across various stages of UC. These results do not impact those data or approved indications.

nivolumab +
relatlimab high
dose

The company announced plans to initiate a Phase 3 trial evaluating the fixed-dose combination of nivolumab and high-dose relatlimab plus chemotherapy as a first-line treatment for stage IV or recurrent non-squamous NSCLC with tumor cell PD-L1 expression of 1 to 49%. The decision was supported by findings from the Phase 2 RELATIVITY-104 trial.

Opdivo +
Yervoy

10-year follow-up data from the Phase 3 CheckMate -067 trial showed continued durable improvement in survival with first-line Opdivo plus Yervoy therapy and Opdivo monotherapy, versus Yervoy alone, in patients with previously untreated advanced or metastatic melanoma. With a minimum follow up of 10 years, median OS was 71.9 months with Opdivo plus Yervoy, the longest reported median OS in a Phase 3 advanced melanoma trial.

Hematology

Category

Asset

Milestone

Regulatory

Breyanzi
(lisocabtagene
maraleucel)

The European Medicines Agency (EMA) validated the Type II variation application to expand the indication for Breyanzi to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. The application is supported by data from the Phase 2 TRANSCEND FL study. Validation of the application confirms the submission is complete and begins the EMA’s centralized review process.

In addition, Japan’s Ministry of Health, Labour and Welfare approved the supplemental New Drug Application for Breyanzi for the treatment of relapsed or refractory FL after one prior line of systemic therapy in patients with high-risk FL and after two or more lines of systemic therapy.

Immunology

Category

Asset

Milestone

Clinical &
Research

Zeposia
(ozanimod)

Data from the Phase 3 DAYBREAK trial demonstrated that decreased rates of brain volume loss were sustained in the open-label extension (OLE) for patients treated with Zeposia for relapsing forms of multiple sclerosis.

A separate DAYBREAK OLE safety analysis demonstrated declining or stable incidence rates of treatment-emergent adverse events, with relatively low rates of infections, serious infections and opportunistic infections over more than eight years of treatment with Zeposia .

Financial Guidance
Bristol Myers Squibb is raising its 2024 line-item guidance as noted below.

2024 Line-Item Guidance

Non-GAAP 2

July
(Prior)

October
(Updated)

Total Revenues

Upper end of low single-
digit range

~5% increase

Total Revenues
(excl. F/X)

Upper end of low single-
digit range

~6% increase

Gross Margin %

Between ~74% and ~75%

Between ~74.5% and ~75%

Operating Expenses 1

Low single-digit increase

~4% to ~5% increase

Other income/(expense)

~($50M)

~$125M

Effective tax rate

~66%

~60%

Diluted EPS

$0.60 – $0.90

$0.75 – $0.95

1 Operating Expenses = MS&A and R&D, excluding Acquired IPRD and Amortization of acquired intangible assets.

2 See "Use of Non-GAAP Financial Information."

The 2024 financial guidance excludes the impact of any potential future strategic acquisitions, divestitures, specified items that have not yet been identified and quantified, and the impact of future Acquired IPRD charges. To the extent we have quantified the impact of significant R&D charges or other income resulting from upfront or contingent milestone payments in connection with asset acquisitions or licensing of third-party intellectual property rights, we may update this information from time to time on our website www.bms.com, in the "Investors" section. Non-GAAP guidance assumes current exchange rates. The financial guidance is subject to risks and uncertainties applicable to all forward-looking statements as described elsewhere in this press release.

A reconciliation of forward-looking non-GAAP measures, including non-GAAP EPS, to the most directly comparable GAAP measures is not provided because comparable GAAP measures for such measures are not reasonably accessible or reliable due to the inherent difficulty in forecasting and quantifying measures that would be necessary for such reconciliation. Namely, we are not, without unreasonable effort, able to reliably predict the impact of accelerated depreciation and impairment charges, legal and other settlements, gains and losses from equity investments and other adjustments. In addition, the company believes such a reconciliation would imply a degree of precision and certainty that could be confusing to investors. These items are uncertain, depend on various factors and may have a material impact on our future GAAP results. See "Cautionary Statement Regarding Forward-Looking Statements" and "Use of Non-GAAP Financial Information."

Environmental, Social & Governance (ESG)
As a leading biopharmaceutical company, Bristol Myers Squibb’s passion for making an impact extends beyond the discovery, development and delivery of innovative medicines that help patients prevail over serious diseases. To learn more about our priorities and goals, please visit our latest ESG report.

Conference Call Information
Bristol Myers Squibb will host a conference call today, Thursday, October 31, 2024, at 8:00 a.m. ET, during which company executives will review quarterly financial results and address inquiries from investors and analysts. Investors and the general public are invited to listen to a live webcast of the call at View Source." target="_blank" title="View Source." rel="nofollow">View Source

Investors and the public can register for the live conference call here. Those unable to register can access the live conference call by dialing in the U.S. toll-free 1-833-816-1116 or international +1 412-317-0705. Materials related to the call will be available at View Source prior to the start of the conference call.

A replay of the webcast will be available at View Source approximately three hours after the conference call concludes. A replay of the conference call will be available beginning at 11:30 a.m. ET on October 31, 2024, through 11:30 a.m. ET on November 14, 2024, by dialing in the U.S. toll free 1-877-344-7529 or international +1 412-317-0088, confirmation code: 9624003.

On October 31, 2024 Bicycle Therapeutics plc (NASDAQ: BCYC), a pharmaceutical company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported recent business progress and financial results for the third quarter ended September 30, 2024 (Press release, Bicycle Therapeutics, OCT 31, 2024, View Source [SID1234647582]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"In the third quarter, we continued to make significant advancements across our business and pipeline. At ESMO (Free ESMO Whitepaper), we reported updated data for our clinical-stage molecules, further supporting their promising monotherapy response rates and differentiated safety profiles. Additionally, last week we shared exciting first human imaging data for our first radiopharmaceuticals molecule, validating the potential of MT1-MMP as a novel cancer target and providing important information on the ability of our Bicycle molecules to deliver radioisotopes to the tumor," said Kevin Lee, Ph.D., CEO of Bicycle Therapeutics. "Altogether, we believe these data demonstrate the power and broad capabilities of our Bicycle technology platform, and we look forward to providing additional updates later this year."

Third Quarter 2024 and Recent Events

· Announced first human imaging data for a Bicycle Radionuclide Conjugate (BRC) targeting MT1-MMP and outlined strategy for leadership in next-generation radiopharmaceuticals.

o Data presented at the European Association of Nuclear Medicine 2024 Congress validate the potential of MT1-MMP as a novel target in the treatment of cancer, demonstrate the translatability of BRC preclinical data and highlight the potential of Bicycle molecules for targeted radionuclide therapy.

§ In an oral presentation, the German Cancer Consortium shared results of fluorine-18-labelled FDG-PET/CT imaging and gallium-68-labelled BRC MT1-MMP PET/CT imaging in a 65-year-old male diagnosed with advanced pulmonary adenocarcinoma, the most common type of non-small cell lung cancer, in the lung and lymph nodes confirmed by endobronchial ultrasound biopsy. Both scans revealed multiple lymph node metastases and bone metastases in the sternum. MT1-MMP imaging demonstrated tracer uptake in the primary tumor in the lung and lymph node and bone metastases, consistent with FDG imaging. Additionally, the MT1-MMP BRC tracer showed renal excretion, with all other organs showing only a negligible tracer uptake. Clear imaging contrast was also observed at early time points.

§ In an e-poster presented by Bicycle Therapeutics, preclinical data demonstrate the suitability of Bicycle molecules to deliver indium to tumors in vivo due to their favorable properties including specific tumor uptake, rapid tumor penetration and rapid renal elimination. Additionally, imaging showed how the biodistribution profile of BRCs can be optimized to maintain high tumor uptake and retention while significantly reducing kidney levels. These data build on the body of preclinical data that the company has published in this area demonstrating the use of Bicycle molecules to effectively deliver various radioisotopes, such as lutetium and lead, to tumors.

o Bicycle Therapeutics’ strategy in radiopharmaceuticals focuses on pursuing novel targets with first-in-class potential and selecting the isotope that best aligns with the target biology and indication. In line with this strategy, the company selected EphA2, a novel tumor antigen that is widely expressed in many cancers, as its second BRC target and signed a letter of intent with leading isotope technology company Eckert & Ziegler to put in place an agreement to supply a range of radioisotopes and develop and manufacture BRC molecules.

· Presented updated clinical results across oncology pipeline at the European Society for Medical Oncology Congress 2024.

o Zelenectide pevedotin (formerly BT8009) is a Bicycle Toxin Conjugate (BTC) molecule targeting Nectin-4, a well-validated tumor antigen. Updated results from the ongoing Phase 1/2 Duravelo-1 trial evaluating 5 mg/m2 weekly of zelenectide pevedotin monotherapy in patients with metastatic urothelial cancer (mUC) who had not previously been treated with enfortumab vedotin showed a 45% overall response rate (ORR) in efficacy-evaluable patients (n=38), with a median duration of response of 11.1 months among patients with confirmed responses (n=14). Zelenectide pevedotin continued to demonstrate an emerging differentiated safety profile, particularly around adverse events of interest such as peripheral neuropathy, skin reactions and eye disorders.

The global Phase 2/3 Duravelo-2 registrational trial of zelenectide pevedotin in patients with mUC is currently enrolling. Additional data updates for zelenectide pevedotin monotherapy in other tumor types and in combination with pembrolizumab in first-line mUC are planned by year end.

o BT5528 is a BTC molecule targeting tumor antigen EphA2, which has historically been difficult to target using other drug conjugate approaches. Updated results from the ongoing Phase 1/2 trial evaluating 6.5 mg/m2 every two weeks and 5 mg/m2 weekly of BT5528 monotherapy in patients with advanced solid tumors showed an emerging differentiated safety profile and antitumor activity, including a 45% ORR in mUC patients enrolled in the dose expansion cohort (n=11) receiving 6.5 mg/m2 every two weeks.

Bicycle Therapeutics is currently assessing BT5528 at 6.5 mg/m2 every two weeks in combination with nivolumab, with results expected in 2025.

o An analysis of BTC clinical data showed that treatment-related peripheral neuropathy (TRPN) in patients receiving either zelenectide pevedotin or BT5528 occurred at low rates and were primarily low grade. In 223 patients from ongoing Phase 1/2 studies, TRPN occurred in 28% of zelenectide pevedotin-treated patients and in 19% of BT5528-treated patients, nearly all of which were low grade (Grade 1-2). One Grade 3 event (neuralgia) was reported in a patient treated with zelenectide pevedotin following prior therapy with enfortumab vedotin, while no Grade 3-4 events were observed for BT5528.

These data showing low rates of TRPN at primarily low severity with BTC molecules support the hypothesis that the antibody-drug construct may be a primary driver of peripheral neuropathy rather than monomethyl auristatin E (MMAE) toxicity as was previously believed.

o BT7480 is a Nectin-4 targeted CD137 agonist designed to overcome immune agonist toxicities and activate the immune system in Nectin-4 expressing tumors. Initial data from the Phase 1/2 dose escalation trial evaluating BT7480 in patients with advanced solid tumors showed an emerging differentiated safety and tolerability profile, with low rates of severe adverse events among 39 patients assigned to receive one of 10 different doses (0.002-3.5 mg/kg weekly). Preliminary biomarker analyses support BT7480 dual targeting of CD137 and Nectin-4 as demonstrated by enhanced immune cell activation, aligned with the proposed mechanism of action of BT7480.

As the maximum tolerated dose for BT7480 has not yet been reached, Bicycle Therapeutics is continuing dose exploration in combination studies, starting with nivolumab.

· Promoted Zafar Qadir to Chief Legal Officer and General Counsel. Since joining Bicycle Therapeutics in April 2020, Mr. Qadir has managed critical responsibilities to support the company’s growth by leading the legal, compliance and intellectual property functions. Over the course of his career, Mr. Qadir has more than a decade of corporate, legal, intellectual property, regulatory and compliance experience and has played a pivotal role in Bicycle Therapeutics’ key partnerships and transactions.

Third Quarter 2024 Financial Results

· Cash and cash equivalents were $890.9 million as of September 30, 2024, compared to $526.4 million as of December 31, 2023. The increase in cash and cash equivalents is primarily due to net proceeds from our PIPE financing in May 2024 and share option exercises, offset by the repayment of our debt facility with Hercules Capital, Inc. in July 2024 and cash used in operating activities.

· Research and Development (R&D) expenses were $48.3 million for the three months ended September 30, 2024, compared to $39.9 million for the three months ended September 30, 2023. The increase in expense of $8.4 million was primarily due to increased clinical program expenses for zelenectide pevedotin development and increased personnel-related expenses, including incremental share-based compensation of $1.1 million, offset by decreased clinical program expenses for Bicycle Tumor-Targeted Immune Cell Agonist molecule development, lower discovery, platform and other expenses, and higher U.K. R&D tax credits period over period.

· General and administrative expenses were $18.3 million for the three months ended September 30, 2024, compared to $16.3 million for the three months ended September 30, 2023. The increase of $2.0 million was primarily due to increased personnel-related expenses, including incremental share-based compensation expense of $0.7 million.

· Net loss was $50.8 million, or $(0.74) basic and diluted net loss per share, for the three months ended September 30, 2024, compared to net loss of $49.9 million, or $(1.26) basic and diluted net loss per share, for three months ended September 30, 2023.

On October 31, 2024 argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, reported its third quarter 2024 financial results and provided a business update (Press release, argenx, OCT 31, 2024, View Source [SID1234647581]).

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"We delivered significant patient impact with VYVGART over the quarter, expanding our gMG footprint and delivering innovation to CIDP patients three months into launch," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "We continued to advance our goal of reaching more gMG patients earlier in their treatment journey, supported by VYVGART’s strong safety and efficacy profile, and real world data showing the ability to meaningfully reduce steroid use. Expanding upon our leadership in gMG, we are now paving the future in CIDP. The strength of our data, combined with execution across the team to reach key stakeholders, contributed to the initial success of our CIDP launch, with more than 300 patients on therapy at the end of the third quarter. There remains significant opportunity ahead as we work towards achieving our Vision 2030, with innovation implemented across our pipeline to deliver transformative outcomes to more patients."

Advancing ‘Vision 2030’ in Third Quarter 2024

Vision 2030 is the next phase in argenx’s long-term commitment to transform the treatment of autoimmune diseases by strengthening its leadership in FcRn biology, investing in its continuous pipeline of differentiated antibody candidates, and scaling in a disciplined way to ensure innovation remains core to the argenx mission. As a part of this vision, argenx plans to reach at least 50,000 patients globally, advance the pipeline to achieve 10 labeled indications, and bring five new molecules into Phase 3 by 2030.

Reaching 50,000 Patients Globally by 2030

VYVGART (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting FcRn and is now approved for both intravenous use and subcutaneous injection (SC) (efgartigimod alfa and hyaluronidase-qvfc) in three indications, including generalized myasthenia gravis (gMG) globally, primary immune thrombocytopenia (ITP) in Japan (IV-only), and chronic inflammatory demyelinating polyneuropathy (CIDP) in the U.S. (SC-only).
•Generated global net product revenues (inclusive of both VYVGART and VYVGART SC) of $573 million in the third quarter of 2024
•Multiple VYVGART regulatory submissions completed or underway for gMG, including:
◦Swissmedic approved VYVGART for the treatment of gMG in Switzerland
◦Regulatory decisions on approval expected in Australia and Saudi Arabia in 2024, and South Korea in 2025
•Multiple VYVGART SC regulatory submissions under review or planned for CIDP, including:
◦Regulatory submissions completed in Japan, Europe, and China with decisions on approval expected in 2025
◦Regulatory submission to be completed in Canada by end of 2024
•VYVGART now reimbursed in 11 countries in Europe, with new agreements in place in France Luxembourg, and Belgium

•FDA review of VYVGART SC pre-filled syringe (PFS) for gMG and CIDP ongoing with Prescription Drug User Fee Act (PDUFA) target action date of April 10, 2025

Advancing Pipeline to Achieve 10 Labeled Indications by 2030

argenx continues to demonstrate breadth and depth within its immunology pipeline, advancing multiple pipeline-in-a-product candidates. argenx is solidifying its leadership in FcRn biology, with efgartigimod currently in development in 15 indications. argenx is also advancing its first-in-class C2 inhibitor, empasiprubart, which is being evaluated in multifocal motor neuropathy (MMN), delayed graft function (DGF), dermatomyositis (DM), and CIDP. In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).
•Registrational studies ongoing of efgartigimod in thyroid eye disease (TED)
•Registrational studies ongoing to support label-expansion into broader MG, including ADAPT SERON in seronegative gMG and ADAPT OCULUS in ocular MG
•Registrational study in primary Sjögren’s disease (SjD) on track to start by end of 2024
•Confirmatory study of efgartigimod in primary ITP to start by end of 2024 to enable registration in U.S.
•Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and DM) expected by end of 2024
•Update on BALLAD study development plan evaluating efgartigimod in bullous pemphigoid (BP) expected by end of 2024
•Decision made to discontinue development of efgartigimod in membranous nephropathy (MN); proof-of concept study ongoing with efgartigimod in lupus nephritis (LN)
•Proof-of-concept study ongoing with efgartigimod in antibody mediated rejection (AMR), with systemic sclerosis (SSc) to start by end of 2024
•Registrational study of empasiprubart in MMN to start by end of 2024
•Additional proof-of-concept studies of empasiprubart ongoing, including VARVARA study in DGF and EMPACIFIC study in DM
•Registrational study of empasiprubart in CIDP to start in 2025
•Ongoing Phase 1b/2a studies of ARGX-119 to assess early signal in patients with CMS and ALS

Investing in Immunology Innovation Program to Support Five New Molecules in Phase 3 by 2030

argenx continues to invest in its Immunology Innovation Program (IIP) to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213, targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121, a first-in-class molecule targeting IgA; ARGX-109, targeting IL-6, which plays an important role in inflammation, and ARGX-220, a first-in-class sweeping antibody for which the target has not yet been disclosed.
•Phase 1 studies of ARGX-213 and ARGX-121 expected to start in second half of 2025
•Investigational new drug (IND) applications for ARGX-220 and ARGX-109 on track to be filed by end of 2025

THIRD QUARTER 2024 FINANCIAL RESULTS
argenx SE
UNAUDITED CONDENSED CONSOLIDATED INTERIM STATEMENTS OF PROFIT OR LOSS
Three Months Ended Nine Months Ended
September 30 September 30
(in thousands of $ except for shares and EPS) 2024 2023 2024 2023
Product net sales $ 572,997 $ 329,097 $ 1,448,915 $ 816,432
Collaboration revenue 239 692 2,905 3,047
Other operating income 15,642 10,050 38,999 31,275
Total operating income $ 588,878 $ 339,839 $ 1,490,819 $ 850,754
Cost of sales $ (59,072) $ (35,999) $ (154,633) $ (78,358)
Research and development expenses (235,940) (191,755) (686,195) (553,119)
Selling, general and administrative expenses (277,698) (191,930) (769,392) (503,079)
Loss from investment in a joint venture (1,981) (743) (5,294) (2,623)
Total operating expenses $ (574,691) $ (420,427) $ (1,615,514) $ (1,137,179)
Operating profit/(loss) $ 14,187 $ (80,588) $ (124,695) $ (286,425)
Financial income $ 40,586 $ 30,049 $ 118,414 $ 67,078
Financial expense (676) (231) (1,760) (626)
Exchange gains/(losses) 33,927 (32,509) 6,712 (23,345)
Profit/(loss) for the period before taxes $ 88,024 $ (83,279) $ (1,329) $ (243,318)
Income tax (expense)/benefit $ 3,386 $ 10,637 $ 60,208 $ 47,437
Profit/(loss) for the period $ 91,410 $ (72,642) $ 58,879 $ (195,881)
Profit/(loss) for the period attributable to:
Owners of the parent $ 91,410 $ (72,642) $ 58,879 $ (195,881)
Weighted average number of shares outstanding 60,087,498 58,128,233 59,633,179 56,512,254
Basic profit/(loss) per share (in $) 1.52 (1.25) 0.99 (3.47)
Diluted profit/(loss) per share (in $) 1.39 (1.25) 0.91 (3.47)
Net increase in cash, cash equivalents and current financial assets compared to year-end 2023 and 2022 $ 194,523 $ 993,035
Cash and cash equivalents and current financial assets at the end of the period $ 3,374,367 $ 3,185,583

DETAILS OF THE FINANCIAL RESULTS
Total operating income for the third quarter and year-to-date in 2024 was $589 million and $1,491 million, respectively, compared to $340 million and $851 million for the same periods in 2023, and mainly consists of:
•Product net sales of VYVGART for the three months ended and nine months ended September 30, 2024, were $573 million and $1,449 million, compared to $329 million and $816 million for the same periods in 2023.

•Other operating income for the third quarter and year-to-date in 2024 was $16 million and $39 million, respectively, compared to $10 million, and $31 million for the same periods in 2023. The other operating income for the three and nine months ended September 30, 2024 primarily relates to research and development tax incentives and payroll tax rebates.
Total operating expenses for the third quarter and year-to-date in 2024 were $575 million and $1,616 million, respectively, compared to $420 million and $1,137 million for the same periods in 2023, and mainly consists of:
•Cost of sales for the third quarter and year-to-date in 2024 was $59 million and $155 million, respectively, compared to $36 million and $78 million for the same periods in 2023. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART Hytrulo.

•Research and development expenses for the third quarter and year-to-date in 2024 were $236 million and $686 million, respectively, compared to $192 million and $553 million for the same periods in 2023. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates.

•Selling, general and administrative expenses for the third quarter and year-to-date in 2024 were $278 million and $769 million, respectively, compared to $192 million and $503 million for the same periods in 2023. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to global commercialization of VYVGART and VYVGART Hytrulo, and personnel expenses.
Financial income for the third quarter and year-to-date in 2024 was $41 million and $118 million, respectively, compared to $30 million and $67 million for the same periods in 2023. The increase in financial income is mainly due to an increase in interest income on current financial assets and cash and cash equivalents.
Exchange gains for the third quarter and year-to-date in 2024 were $34 million and $7 million respectively, respectively, compared to $(33) million and $(23) million of exchange losses for the same periods in 2023. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets position in Euro.
Income tax for the third quarter and year-to-date in 2024 was $3 million and $60 million of tax benefit, respectively, compared to $11 million and $47 million of tax benefit for the same periods in 2023. Tax benefit for the nine months ended September 30, 2024 consists of $29 million of income tax expense and $89 million of deferred tax income, compared to $24 million of income tax expense and $71 million of deferred tax income for the comparable prior period.
Net income for the three and nine month periods ended September 30, 2024, was $91 million and $59 million, respectively, compared to a net loss of $(73) million and $(196) million over the prior year periods. On a per weighted average share basis, the earnings per share was $0.99 for the nine months ended September 30, 2024 and a net loss per share of $(3.47) for the nine months ended September 30, 2023.

Cash, cash equivalents and current financial assets totaled $3.4 billion as of September 30, 2024, compared to $3.2 billion as of December 31, 2023. The increases in cash and cash equivalents and current financial assets over the period was from financing activities due to the exercise of stock options which is offset by net cash flows used in operating and investing activities.

FINANCIAL GUIDANCE

With the increase in cash, cash equivalents and current financial assets in the quarter and year-to-date, the previously issued cash guidance no longer applies. The financial guidance on the combined selling, general and administrative expenses and research and development expenses remains unchanged at approximately $2 billion.

EXPECTED 2024 FINANCIAL CALENDAR

•February 27, 2025: Full-year 2024 financial results and 4Q 2024 business update

CONFERENCE CALL DETAILS

The third quarter 2024 financial results and business update will be discussed during a conference call and webcast presentation today at 1:30 pm CET/8:30 am ET. A webcast of the live call and replay may be accessed on the Investors section of the argenx website at argenx.com/investors.