On October 31, 2024 Merck (NYSE: MRK), known as MSD outside the United States and Canada, reported financial results for the third quarter of 2024 (Press release, Merck & Co, OCT 31, 2024, View Source [SID1234647605]).

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"Our third-quarter results were strong, as we continue to make progress heading into 2025 and beyond," said Robert M. Davis, chairman and chief executive officer, Merck. "Our pipeline is advancing and expanding, demonstrating our success in creating a sustainable innovation engine, and positioning Merck with a more diversified portfolio to drive growth. I continue to remain confident in the strength of our business and our ability to execute, and I want to thank our colleagues across the globe for their focus and commitment as we work to create lasting value for patients, shareholders and all our stakeholders."

Financial Summary

$ in millions, except EPS amounts

Third Quarter

2024

2023

Change

Change Ex-
Exchange

Sales

$16,657

$15,962

4%

7%

GAAP net income1

3,157

4,745

-33%

-30%

Non-GAAP net income that excludes certain items1,2*

3,985

5,427

-27%

-23%

GAAP EPS

1.24

1.86

-33%

-30%

Non-GAAP EPS that excludes certain items2*

1.57

2.13

-26%

-23%

*Refer to table on page 7.

In the third quarter of 2024, total worldwide sales were $16.7 billion, an increase of 4% compared with the third quarter of 2023; excluding the impact of foreign exchange, growth was 7%. Sales growth in the third quarter of 2024 was primarily due to increased usage of KEYTRUDA globally, contributions from new launches, including WINREVAIR and CAPVAXIVE, and strong growth in Merck’s Animal Health business. Revenue growth in the third quarter of 2024 was partially offset by lower sales of JANUVIA and JANUMET, lower combined sales of GARDASIL/GARDASIL 9 and lower sales of LAGEVRIO. Third-quarter GARDASIL/GARDASIL 9 sales declined year-over-year due to reduced demand in China; outside of China, the company achieved double-digit sales growth for GARDASIL/GARDASIL 9 in almost every major region globally.

For the third quarter of 2024, Generally Accepted Accounting Principles (GAAP) earnings per share (EPS) assuming dilution was $1.24 and non-GAAP EPS was $1.57. The declines in GAAP and Non-GAAP EPS in the third quarter of 2024 versus the prior year were largely due to a net charge of $0.79 per share in the aggregate for the acquisition of Eyebiotech Limited (EyeBio) and a related development milestone, the acquisition of CN201 (now known as MK-1045) from Curon Biopharmaceutical (Curon), as well as a payment received from Daiichi Sankyo related to the expansion of the existing development and commercialization agreement. There were no significant business development transaction charges in the third quarter of 2023.

Non-GAAP EPS in both periods excludes acquisition- and divestiture-related costs, costs related to restructuring programs, as well as income and losses from investments in equity securities.

Year-to-date results can be found in the attached tables.

Third-Quarter Sales Performance

The following table reflects sales of the company’s top products and significant performance drivers.

Third Quarter

$ in millions

2024

2023

Change

Change
Ex-
Exchange

Commentary

Total Sales

$16,657

$15,962

4%

7%

Approximately 2 percentage points of the negative impact of foreign exchange was due to devaluation of Argentine peso, which was largely offset by inflation-related price increases, consistent with practice in that market.

Pharmaceutical

14,943

14,263

5%

8%

Increase driven by growth in oncology and cardiovascular, partially offset by declines in diabetes, vaccines and virology.

KEYTRUDA

7,429

6,338

17%

21%

Growth driven by increased global uptake in earlier-stage indications, including triple-negative breast cancer (TNBC), renal cell carcinoma (RCC) and non-small cell lung cancer (NSCLC), as well as continued strong global demand from metastatic indications. Approximately 3 percentage points of the negative impact of foreign exchange was due to devaluation of Argentine peso, which was largely offset by inflation-related price increases.

GARDASIL/GARDASIL 9

2,306

2,585

-11%

-10%

Decline primarily due to lower demand in China compared with prior year, partially offset by higher sales in the U.S., driven by public-sector buying patterns, higher pricing and demand, as well as higher demand in most international regions.

PROQUAD, M-M-R II and VARIVAX

703

713

-1%

-1%

Decline primarily due to timing of shipments and lower tenders in Latin America, largely offset by higher demand in certain international markets.

JANUVIA/JANUMET

482

835

-42%

-38%

Decline primarily due to lower pricing in the U.S., as well as ongoing generic competition in many international markets.

BRIDION

420

424

-1%

0%

Relatively flat compared with prior year due to generic competition in certain international markets, particularly in Europe and Japan, largely offset by higher demand and pricing in the U.S.

LAGEVRIO

383

640

-40%

-36%

Decline primarily due to lower demand in Japan, partially offset by uptake from commercial launch in the U.S.

Lynparza*

337

299

13%

13%

Growth primarily due to higher global demand.

Lenvima*

251

260

-3%

-4%

Decline primarily due to timing of shipments in China in the prior year, partially offset by higher demand in the U.S.

VAXNEUVANCE

239

214

12%

13%

Growth largely driven by continued uptake from launches in Europe and Japan, partially offset by lower demand in the U.S. due to competition.

PREVYMIS

208

157

32%

36%

Growth primarily due to higher global demand, particularly in the U.S.

ROTATEQ

193

156

24%

25%

Growth primarily due to public-sector buying patterns in the U.S. and timing of shipments in China.

WINREVAIR

149

–

–

–

Represents continued uptake since launch in the U.S. in the second quarter.

WELIREG

139

54

156%

157%

Growth primarily driven by higher demand in the U.S., largely attributable to ongoing uptake of a new indication.

Animal Health

1,487

1,400

6%

11%

Growth primarily driven by higher demand and pricing for both Companion Animal and Livestock product portfolios, as well as sales related to July 2024 acquisition of Elanco aqua business. Approximately 2 percentage points of the negative impact of foreign exchange was due to devaluation of Argentine peso, which was largely offset by inflation-related price increases.

Livestock

886

874

1%

7%

Growth primarily driven by higher pricing and higher demand for poultry and swine products, as well as sales related to acquisition of Elanco aqua business.

Companion Animal

601

526

14%

17%

Growth primarily driven by uptake from new product launches, including the injectable formulation of BRAVECTO in certain international markets, as well as higher pricing across product portfolio. Sales of BRAVECTO were $266 million and $235 million in current and prior year quarters, respectively, which represented growth of 13%, or 16% excluding impact of foreign exchange.

Other Revenues**

227

299

-24%

-22%

Decline primarily due to lower payments received for out-licensing arrangements and lower royalty income.

*Alliance revenue for this product represents Merck’s share of profits, which are product sales net of cost of sales and commercialization costs.
**Other revenues are comprised primarily of revenues from third-party manufacturing arrangements and miscellaneous corporate revenues, including revenue-hedging activities.

Third-Quarter Expense, EPS and Related Information

The table below presents selected expense information.

$ in millions

GAAP

Acquisition-
and
Divestiture-
Related
Costs3

Restructuring
Costs

(Income)
Loss From
Investments
in Equity
Securities

Non-
GAAP2

Third Quarter 2024

Cost of sales

$4,080

$639

$192

$-

$3,249

Selling, general and administrative

2,731

43

31

–

2,657

Research and development

5,862

24

–

–

5,838

Restructuring costs

56

–

56

–

–

Other (income) expense, net

(162)

(27)

–

58

(193)

Third Quarter 2023

Cost of sales

$4,264

$552

$33

$-

$3,679

Selling, general and administrative

2,519

17

40

–

2,462

Research and development

3,307

10

–

–

3,297

Restructuring costs

126

–

126

–

–

Other (income) expense, net

126

(24)

–

17

133

GAAP Expense, EPS and Related Information

Gross margin was 75.5% for the third quarter of 2024 compared with 73.3% for the third quarter of 2023. The increase was primarily due to the favorable impact of product mix (including lower royalty rates related to KEYTRUDA and GARDASIL/GARDASIL 9), partially offset by higher restructuring costs (primarily reflecting asset impairment charges), as well as higher amortization of intangible assets.

Selling, general and administrative (SG&A) expenses were $2.7 billion in the third quarter of 2024, an increase of 8% compared with the third quarter of 2023. The increase was primarily due to higher administrative, promotional, selling, and acquisition-related costs, partially offset by the favorable impact of foreign exchange.

Research and development (R&D) expenses were $5.9 billion in the third quarter of 2024, an increase of 77% compared with the third quarter of 2023. The increase was primarily due to a charge of $1.35 billion for the acquisition of EyeBio and a $100 million charge for a related development milestone, as well as a charge of $750 million to acquire CN201 (MK-1045) from Curon. The increase in R&D expenses was also driven by higher compensation and benefit costs, as well as higher clinical development spending. The increase in R&D expenses was partially offset by the favorable impact of foreign exchange.

Other (income) expense, net, was $162 million of income in the third quarter of 2024 compared with $126 million of expense in the third quarter of 2023. The favorability was primarily due to a $170 million payment received from Daiichi Sankyo related to the expansion of the existing development and commercialization agreement, lower exchange losses and lower net interest expense.

The effective tax rate of 22.7% for the third quarter of 2024 includes a 7.2 percentage point combined unfavorable impact related to the EyeBio and Curon transactions.

GAAP EPS was $1.24 for the third quarter of 2024 compared with $1.86 for the third quarter of 2023. GAAP EPS in the third quarter of 2024 includes a net charge of $0.79 per share in the aggregate for the EyeBio, Curon and Daiichi Sankyo transactions. There were no significant business development transaction charges in the third quarter of 2023.

Non-GAAP Expense, EPS and Related Information

Non-GAAP gross margin was 80.5% for the third quarter of 2024 compared with 77.0% for the third quarter of 2023. The increase was primarily due to the favorable impact of product mix (including lower royalty rates related to KEYTRUDA and GARDASIL/GARDASIL 9).

Non-GAAP SG&A expenses were $2.7 billion in the third quarter of 2024, an increase of 8% compared with the third quarter of 2023. The increase was primarily due to higher administrative, promotional and selling costs, partially offset by the favorable impact of foreign exchange.

Non-GAAP R&D expenses were $5.8 billion in the third quarter of 2024, an increase of 77% compared with the third quarter of 2023. The increase was primarily due to a charge of $1.35 billion for the acquisition of EyeBio and a $100 million charge for a related development milestone, as well as a charge of $750 million to acquire CN201 (MK-1045) from Curon. The increase in R&D expenses was also driven by higher compensation and benefit costs, as well as higher clinical development spending. The increase in R&D expenses was partially offset by the favorable impact of foreign exchange.

Non-GAAP other (income) expense, net, was $193 million of income in the third quarter of 2024 compared with $133 million of expense in the third quarter of 2023. The favorability was primarily due to a $170 million payment received from Daiichi Sankyo related to the expansion of the existing development and commercialization agreement, lower exchange losses and lower net interest expense.

The non-GAAP effective tax rate of 21.9% for the third quarter of 2024 includes a 6.0 percentage point combined unfavorable impact related to the EyeBio and Curon transactions.

Non-GAAP EPS was $1.57 for the third quarter of 2024 compared with $2.13 for the third quarter of 2023. Non-GAAP EPS in the third quarter of 2024 includes a net charge of $0.79 per share in the aggregate for the EyeBio, Curon and Daiichi Sankyo transactions. There were no significant business development transaction charges in the third quarter of 2023.

A reconciliation of GAAP to non-GAAP net income and EPS is provided in the table that follows.

Third Quarter

$ in millions, except EPS amounts

2024

2023

EPS

GAAP EPS

$1.24

$1.86

Difference

0.33

0.27

Non-GAAP EPS that excludes items listed below2

$1.57

$2.13

Net Income

GAAP net income1

$3,157

$4,745

Difference

828

682

Non-GAAP net income that excludes items listed below1,2

$3,985

$5,427

Excluded Items:

Acquisition- and divestiture-related costs3

$679

$555

Restructuring costs

279

199

Loss from investments in equity securities

58

17

Decrease to net income

1,016

771

Estimated income tax (benefit) expense

(188)

(89)

Decrease to net income

$828

$682

Pipeline and Portfolio Highlights

In the third quarter, Merck continued to develop and augment its strong, diverse pipeline and achieve key regulatory and clinical milestones.

In cardiovascular disease, Merck continued to build on positive momentum in its U.S. launch of WINREVAIR. As of the end of September 2024, more than 3,700 patients have been prescribed WINREVAIR. The company also received the European Commission’s (EC) approval of WINREVAIR, in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of adult patients with PAH with World Health Organization (WHO) functional Class II to III. WINREVAIR is the first activin signaling inhibitor approved for the treatment of PAH in Europe. WINREVAIR has launched in Germany and Merck is working to obtain reimbursement for WINREVAIR in other countries in the EU, which should occur in most other major European markets in the second half of 2025.

In oncology, Merck continued to reinforce its leadership in women’s and earlier stages of cancers and demonstrate progress in its research pipeline. At the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024, three of the company’s data presentations were highlighted during Presidential Symposium sessions. These included overall survival (OS) data from the Phase 3 KEYNOTE-522 trial in high-risk, early-stage TNBC and from the Phase 3 KEYNOTE-A18 trial (also known as ENGOT-cx11/GOG-3047) in high-risk, locally advanced cervical cancer. In addition, new positive data on investigational candidates from Merck’s pipeline were presented, including for patritumab deruxtecan (HER3-DXd), an antibody-drug conjugate (ADC) being developed in collaboration with Daiichi Sankyo, and for sacituzumab tirumotecan (sac-TMT), an anti-TROP2 ADC being developed in collaboration with Kelun-Biotech.

The company also achieved several regulatory milestones, including new approvals for KEYTRUDA-based regimens in the U.S., Europe and Japan. In addition, Merck recently announced top-line results from the KEYNOTE-689 trial, which marks the first positive trial in two decades for patients with resected, locally advanced head and neck squamous cell carcinoma (LA-HNSCC).

In vaccines, the CDC’s Advisory Committee on Immunization Practices (ACIP) voted in October 2024 to recommend CAPVAXIVE for individuals 50 to 64 years of age. This decision expanded upon the initial unanimous recommendation in June 2024 for use of CAPVAXIVE in adults age 65 and older, among other cohorts.

At IDWeek 2024, Merck presented positive results from the Phase 2b/3 trial of clesrovimab (MK-1654), an investigational respiratory syncytial virus (RSV) preventative monoclonal antibody for infants. These results support the potential for clesrovimab to become the first and only single-dose immunization designed to protect infants with the same dose, regardless of weight, for the duration of their first RSV season (six months).

In immunology, long-term efficacy and safety data for tulisokibart (MK-7240), an investigational humanized monoclonal antibody directed to a novel target, tumor necrosis factor (TNF)-like cytokine 1A (TL1A), from the Phase 2 ARTEMIS-UC and APOLLO-CD studies in ulcerative colitis (UC) and Crohn’s disease (CD), were presented at the United European Gastroenterology (UEG) Week 2024 Congress. Both studies showed that, at week 50, maintenance of treatment efficacy was generally observed in 12-week induction responders. Phase 3 studies in UC and CD are ongoing.

In addition, Merck continued to expand and diversify its pipeline by securing strategic business development opportunities. Merck completed its acquisition of CN201 (MK-1045), a next-generation CD3xCD19 bispecific antibody with potential applications in B-cell malignancies and autoimmune diseases, from Curon. Merck also announced the expansion of the global development and commercialization agreement with Daiichi Sankyo to include MK-6070, an investigational delta-like ligand 3 (DLL3) targeting T-cell engager. The companies are planning to evaluate MK-6070 in combination with ifinatamab deruxtecan (I-DXd) in certain patients with small cell lung cancer (SCLC), as well as other potential combinations.

Notable recent news releases on Merck’s pipeline and portfolio are provided in the table that follows.

Oncology

FDA Approved KEYTRUDA Plus Pemetrexed and Platinum Chemotherapy as First-Line Treatment for Adult Patients With Unresectable Advanced or Metastatic Malignant Pleural Mesothelioma, Based on Results From Phase 3 KEYNOTE-483/CCTG IND.227 Trial

(Read Announcement)

EC Approved KEYTRUDA Plus Padcev as First-Line Treatment of Unresectable or Metastatic Urothelial Carcinoma in Adults, Based on Results From Phase 3 KEYNOTE-A39/EV-302 Trial

(Read Announcement)

KEYTRUDA Received 30th Approval From EC With Two New Indications in Gynecologic Cancers, Based on Results From Phase 3 KEYNOTE-868/NRG-GY018 and KEYNOTE-A18 Trials

(Read Announcement)

KEYTRUDA Received New Approvals in Japan for Certain Patients With NSCLC, Based on Results From Phase 3 KEYNOTE-671 Trial, and for Radically Unresectable Urothelial Carcinoma, Based on Results From Phase 3 KEYNOTE-A39/EV-302 and Phase 2 KEYNOTE-052 Trials

(Read Announcement)

KEYTRUDA Plus Chemotherapy Before Surgery and Continued as Single Agent After Surgery Reduced Risk of Death by More Than One-Third (34%) Versus Neoadjuvant Chemotherapy in High-Risk, Early-Stage TNBC, Based on Results From Phase 3 KEYNOTE-522

(Read Announcement)

KEYTRUDA Plus Chemoradiotherapy (CRT) Reduced Risk of Death by 33% Versus CRT Alone in Patients With Newly Diagnosed, High-Risk, Locally Advanced Cervical Cancer, Based on Results From Phase 3 KEYNOTE-A18/ENGOT-cx11/GOG-3047 Trial

(Read Announcement)

KEYTRUDA Ten-Year Data Demonstrated Sustained OS Benefit Versus Ipilimumab in Advanced Melanoma, Based on Results From Phase 3 KEYNOTE-006 Trial

(Read Announcement)

KEYTRUDA Plus Lenvima in Combination With Transarterial Chemoembolization (TACE) Significantly Improved Progression-Free Survival Compared to TACE Alone in Patients With Unresectable, Non-Metastatic Hepatocellular Carcinoma, Based on Results From Phase 3 LEAP-012 Trial

(Read Announcement)

KEYTRUDA Plus Trastuzumab and Chemotherapy Significantly Improved OS Versus Trastuzumab and Chemotherapy Alone in First-Line Treatment of Patients With HER2-Positive Advanced Gastric or GEJ Adenocarcinoma, Based on Results From Phase 3 KEYNOTE-811 Trial

(Read Announcement)

KEYTRUDA Met Primary Endpoint of Event-Free Survival as Perioperative Treatment Regimen in Patients With Resected, LA-HNSCC, Based on Results From Phase 3 KEYNOTE-689 Trial

(Read Announcement)

Patritumab Deruxtecan (HER3-DXd) Demonstrated Statistically Significant Improvement in Progression-Free Survival Versus Doublet Chemotherapy in Patients With Locally Advanced or Metastatic EGFR-Mutated NSCLC, Based on Results From Phase 3 HERTHENA-Lung02 Trial

(Read Announcement)

Ifinatamab Deruxtecan Continued to Demonstrate Promising Objective Response Rates in Patients With Extensive-Stage SCLC, Based on Results From Phase 2 IDeate-Lung01 Trial

(Read Announcement)

Merck and Moderna Initiated Phase 3 Trial Evaluating Adjuvant V940 (mRNA-4157) in Combination With KEYTRUDA After Neoadjuvant KEYTRUDA and Chemotherapy in Patients With Certain Types of NSCLC

(Read Announcement)

Merck Initiated Phase 3 Shorespan-007 Trial for Bomedemstat, an Investigational Candidate for the Treatment of Certain Patients With Essential Thrombocythemia

(Read Announcement)

Merck and Daiichi Sankyo Initiated Phase 3 IDeate-Lung02 Trial of Ifinatamab Deruxtecan in Patients With Relapsed SCLC

(Read Announcement)

Merck and Exelixis Signed Clinical Development Collaboration To Evaluate Investigational Zanzalintinib in Combination With KEYTRUDA in Head and Neck Cancer and in Combination With WELIREG in RCC

(Read Announcement)

Vaccines

Clesrovimab (MK-1654), an Investigational RSV Preventative Monoclonal Antibody, Significantly Reduced Incidence of RSV Disease and Hospitalization in Healthy Preterm and Full-Term Infants, Based on Results From Phase 2b/3 MK-1654-004 Trial

(Read Announcement)

CDC’s ACIP Recommended CAPVAXIVE for Pneumococcal Vaccination in Adults 50 Years of Age and Older

(Read Announcement)

CAPVAXIVE Demonstrated Positive Immune Responses in Adults With Increased Risk for Pneumococcal Disease, Based on Results From Phase 3 STRIDE-8 Trial

(Read Announcement)

Merck Announced Positive Top-line Results From Phase 3 Trial Evaluating Efficacy and Safety of GARDASIL 9 in Japanese Males

(Read Announcement)

Cardiovascular

EC Approved WINREVAIR in Combination With Other PAH Therapies for the Treatment of PAH in Adult Patients With Functional Class II-III, Based on Results From Phase 3 STELLAR Trial

(Read Announcement)

Immunology

Merck Presented New Long-Term Data for Tulisokibart (MK-7240), an Investigational Anti-TL1A Monoclonal Antibody, in Inflammatory Bowel Disease at UEG Week 2024

(Read Announcement)

Infectious Diseases

Merck and Gilead Announced Phase 2 Data Showing a Treatment Switch to an Investigational Oral Once-Weekly Combination Regimen of Islatravir and Lenacapavir (MK-8591D) Maintained Viral Suppression in Adults at Week 48

(Read Announcement)

Ophthalmology

Merck and EyeBio Initiated Phase 2b/3 Clinical Trial for MK-3000 for the Treatment of Diabetic Macular Edema

(Read Announcement)

Sustainability Highlights

Merck issued its 2023/2024 Impact Report, reaffirming its commitment to operating responsibly and enabling broad access to its products. The report noted how the company reached more than 550 million people around the world with its medicines and vaccines through commercial channels, clinical trials, voluntary licensing and product donations.

Full-Year 2024 Financial Outlook

The following table summarizes the company’s full-year financial outlook.

Full Year 2024

Updated

Prior

Sales*

$63.6 to $64.1 billion

$63.4 to $64.4 billion

Non-GAAP Gross margin2

Approximately 81%

Approximately 81%

Non-GAAP Operating expenses2**

$27.8 to $28.3 billion

$26.8 to $27.6 billion

Non-GAAP Other (income) expense, net2

Approximately $100 million expense

Approximately $350 million expense

Non-GAAP Effective tax rate2

16.0% to 17.0%

15.5% to 16.5%

Non-GAAP EPS2***

$7.72 to $7.77

$7.94 to $8.04

Share count (assuming dilution)

Approximately 2.54 billion

Approximately 2.54 billion

*The company does not have any non-GAAP adjustments to sales.

**Includes one-time R&D charges of $656 million for Harpoon Therapeutics, Inc. (Harpoon) acquisition, $1.45 billion for EyeBio acquisition and related development milestone payment, and $750 million for acquisition of CN201 (MK-1045) from Curon. Outlook does not assume any additional significant potential business development transactions.

***Includes net one-time charge of $1.05 per share in aggregate for the Harpoon, EyeBio and Curon transactions, and the cash payment received from Daiichi Sankyo.

Merck has not provided a reconciliation of forward-looking non-GAAP gross margin, non-GAAP operating expenses, non-GAAP other (income) expense, net, non-GAAP effective tax rate and non-GAAP EPS to the most directly comparable GAAP measures, given it cannot predict with reasonable certainty the amounts necessary for such a reconciliation, including intangible asset impairment charges, legal settlements, and gains and losses from investments in equity securities either owned directly or through ownership interests in investment funds, without unreasonable effort. These items are inherently difficult to forecast and could have a significant impact on the company’s future GAAP results.

Merck continues to experience strong growth, including from KEYTRUDA, new product launches and Animal Health. As a result, Merck is narrowing the range of its full-year sales outlook.

Merck now expects its full-year sales to be between $63.6 billion and $64.1 billion, including a negative impact of foreign exchange of approximately 3 percentage points, at mid-October 2024 exchange rates. Approximately 2 percentage points of the negative impact of foreign exchange is due to the devaluation of the Argentine peso, which is being largely offset by inflation-related price increases, consistent with practice in that market.

Merck now expects its full-year non-GAAP effective income tax rate to be between 16.0% and 17.0%, which includes an unfavorable impact related to the one-time charge associated with the acquisition of CN201 (MK-1045) from Curon.

Merck now expects its full-year non-GAAP EPS to be between $7.72 and $7.77. The outlook includes a negative impact of foreign exchange of approximately $0.30 per share. The negative impact of foreign exchange is primarily due to the devaluation of the Argentine peso, which is being largely offset by inflation-related price increases, consistent with practice in that market. This revised non-GAAP EPS range reflects a net charge of $0.24 per share for the following items not previously included in the outlook:

The acquisition of CN201 (MK-1045) from Curon.
Payment received from Daiichi Sankyo related to the expansion of the existing development and commercialization agreement.
Consistent with past practice, the financial outlook does not assume additional significant potential business development transactions.

Non-GAAP EPS excludes acquisition- and divestiture-related costs, costs related to restructuring programs, income and losses from investments in equity securities, as well as a tax benefit in 2024 due to a reduction in reserves for unrecognized income tax benefits, resulting from the expiration of the statute of limitations for assessments related to the 2019 federal tax return year.

Earnings Conference Call

Investors, journalists and the general public may access a live audio webcast of the earnings conference call on Thursday, October 31, at 9 a.m. ET via this weblink. A replay of the webcast, along with the sales and earnings news release, supplemental financial disclosures, and slides highlighting the results, will be available at www.merck.com.

All participants may join the call by dialing (800) 369-3351 (U.S. and Canada Toll-Free) or (517) 308-9448 and using the access code 9818590.

On October 31, 2024 Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical-stage T-cell reprogramming company advancing a pipeline of next-generation CAR T-cell therapies for patients with solid tumors or hematologic malignancies, reported that it has completed its acquisition of ImmPACT Bio USA Inc. ("ImmPACT"), a privately-owned clinical-stage cell therapy company (Press release, Lyell Immunopharma, OCT 31, 2024, View Source [SID1234647604]). The acquisition strengthens Lyell’s clinical-stage pipeline of CAR T-cell therapies and complements its suite of innovative technologies designed to generate longer-lasting, functional T cells to achieve more durable outcomes for patients. Lyell will accelerate the development of IMPT-314, a dual-targeting CD19/20 chimeric antigen receptor (CAR) T-cell product candidate for hematologic malignancies, including B-cell non-Hodgkin lymphoma. In connection with the acquisition, Sumant Ramachandra, M.D., Ph.D., MBA, the former Chief Executive Officer of ImmPACT Bio, has been appointed to the Lyell Board of Directors.

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"We’re excited to welcome ImmPACT to Lyell and look forward to working together to transform the treatment of cancer with next-generation cell therapies that offer patients improved outcomes," stated Lynn Seely, M.D., Lyell’s President and Chief Executive Officer. "We are focused on accelerating the development of IMPT-314 for patients with aggressive B-cell non-Hodgkin lymphoma and look forward to presenting initial data from the Phase 1-2 trial of IMPT-314 in patients treated in the 3rd line CAR-naïve setting at a major medical conference later this year."

"On behalf of my fellow directors, I am delighted to welcome Dr. Ramachandra to the Lyell Board," stated Rick Klausner, M.D., chair of Lyell’s Board of Directors. "Dr. Ramachandra’s experience and passion for developing innovative therapies for patients will help guide us as we integrate our two organizations and advance a pipeline of next-generation CAR T-cell therapies."

Dr. Ramachandra has served as the Chief Executive Officer of ImmPACT Bio USA, Inc. since November 2021. He also served as a member of the board of directors of ImmPACT from December 2021 to October 2024. Prior to joining ImmPACT, Dr. Ramachandra was most recently Chief Science, Technology and Medical Officer of Baxter International. In addition to these responsibilities, he was appointed President of Baxter Pharmaceuticals. Prior to Baxter, he worked at Pfizer, most recently as Senior Vice President, Head of Research & Development, Pfizer Essential Health. He served as Chief Scientific Officer at Hospira from 2008 to 2015 prior to Pfizer’s acquisition of Hospira in 2015. Before entering the industry in 2000, he was an intern and resident physician, medical services, at Massachusetts General Hospital, Harvard Medical School. Dr. Ramachandra completed his undergraduate degree in biochemistry, graduate degree (Ph.D.) in experimental pathology in the study of chronic lymphocytic leukemia and his medical degree (M.D.) at Rutgers University. In addition, he earned his M.B.A. at The Wharton School at the University of Pennsylvania.

As previously disclosed, following the closing of this acquisition, Lyell expects its cash balance will fund operations into 2027, through important clinical milestones for each pipeline program, including initiation of a pivotal trial for IMPT-314, which is expected to start in 2025.

On October 31, 2024 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that, following feedback from the U.S. Food and Drug Administration (FDA), the Company will be replacing TSS50, one of the co-primary endpoints in the Phase 3 SENTRY Trial (NCT04562389) with Abs-TSS (Press release, Karyopharm, OCT 31, 2024, View Source [SID1234647603]). Abs-TSS measures the average improvement in patient symptom scores over 24 weeks relative to the patient’s baseline symptom score.

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"There remains a tremendous unmet need in myelofibrosis, as less than half of patients achieve SVR35 with each of the approved JAK inhibitors and many patients eventually stop responding to these treatments," said Dr. Raajit Rampal, Director of the Center for Hematologic Malignancies and Director of the Myeloproliferative Neoplasms Program at Memorial Sloan Kettering Cancer Center. "The Phase 1 trial, which evaluates the combination of selinexor and ruxolitinib, shows an approximate doubling of SVR35 to nearly 80% compared to historical JAKi monotherapy, and meaningful improvements in Abs-TSS with an average 18.5 point improvement at week 24 compared to baseline. I believe these data are meaningful and impressive and provide a strong rationale for the Phase 3 SENTRY trial."

Data from the Company’s Phase 1 trial, evaluating the combination of selinexor 60mg plus ruxolitinib in JAKi naïve myelofibrosis patients, demonstrated that 79% of patients in the intent to treat population (n=14) achieved SVR35 and an average Abs-TSS improvement of 18.5 points in the efficacy evaluable population (n=9), at week 24 relative to baseline. Acknowledging the small sample size, these data are favorable to historical ruxolitinib monotherapy data which indicates that less than half of patients achieve SVR35 and an Abs-TSS improvement of 11 to 14 points1. The safety profile remains consistent and no new safety signals have been identified.

"Our confidence in the success of our Phase 3 SENTRY trial increases based on the change in the co-primary endpoint to Abs-TSS, the increased sample size and the data previously presented from our Phase 1 trial evaluating selinexor plus ruxolitinib in JAKi naïve myelofibrosis patients," said Reshma Rangwala, MD, PhD, Chief Medical Officer and Head of Research at Karyopharm. "Based upon strong enrollment, we remain on track to report top-line results in the second half of 2025."

"Improving symptomatic burden for patients with myelofibrosis is an important goal in therapy, directly linking to decreases in morbidity and likely mortality", said Dr. Ruben Mesa, President of Atrium Health Levine Cancer and Charles L. Spurr, MD Professor of Internal Medicine, Wake Forest University School of Medicine. "I am very encouraged by the benefits reported in Karyopharm’s Phase 1 trial of selinexor combined with standard of care ruxolitinib, especially regarding disease associated symptoms. Additionally, I am grateful that the ongoing Phase 3 trial will use Abs-TSS as a co-primary endpoint, which may better represent the cumulative benefit patients experience on symptom burden."

Abs-TSS is an accepted measure that has been used in other Phase 3 clinical trials in myelofibrosis to evaluate the benefit/risk of an add-on treatment, such as selinexor, to the current standard of care. The change to Abs-TSS is strongly supported by key leading investigators and patient advocacy organizations, which generally view improvement in Abs-TSS from baseline as a more accurate assessment of symptom improvement in head-to-head clinical trials, such as SENTRY.

"We are vocal advocates for evolving myelofibrosis clinical trial endpoints. Growing data that support a newer outcome measure like Abs-TSS that is also meaningful to patients is very encouraging," said Kapila Viges, Chief Executive Officer, MPN Research Foundation. "Efforts to develop effective treatments and combination therapies with patients’ goals for care in mind are important. For myelofibrosis patients and their families, options matter."

Company Conference Call Information

Karyopharm will host a conference call with management and Drs. Raajit Rampal and John Mascarenhas to discuss the Phase 3 SENTRY trial today, October 31, 2024, at 8:00 a.m. Eastern Time. To access the conference call, please dial (800) 836-8184 (local) or (646) 357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Karyopharm Therapeutics call. A live audio webcast, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source An archived webcast will be available on the Company’s website approximately two hours after the event.

References

1Phase 3 MANIFEST trial. Rampal R, et al. ASH (Free ASH Whitepaper) 2023. Oral 628; Phase 3 TRANSFORM-1 trial Pemmaraju N, et al. ASH (Free ASH Whitepaper) 2023 abstract 620.

About the Phase 3 SENTRY Trial

SENTRY (NCT04562389) is a pivotal, Phase 3 clinical trial evaluating a once-weekly dose of 60mg of selinexor in combination with twice-daily ruxolitinib versus placebo plus ruxolitinib in JAKi naïve patients with platelet counts >100 x 109/L. Karyopharm intends to enroll approximately 350 JAKi naïve patients with myelofibrosis in this Phase 3 trial; patients are randomized 2-to-1 to the selinexor arm. The co-primary endpoints will be spleen volume response rate ≥ 35% (SVR35) at week 24 and the change in absolute total symptom score (Abs-TSS) over 24 weeks relative to baseline.

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including but not limited to the European Union, the United Kingdom, China, South Korea, Canada, Israel and Taiwan. XPOVIO and NEXPOVIO is marketed by Karyopharm’s partners, Antengene, Menarini, Neopharm and FORUS in China, South Korea, Singapore, Australia, Hong Kong, Germany, Austria, Israel and Canada.

Please refer to the local Prescribing Information for full details.

Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including in endometrial cancer and myelofibrosis.

For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.

Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony‐stimulating factors.

Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.

Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.

Serious Infection: Monitor for infection and treat promptly.

Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.

Embryo‐Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.

Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3‐4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.

The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3‐4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions was infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.
Use In Specific Populations
Lactation: Advise not to breastfeed.

For additional product information, including full prescribing information, please visit www.XPOVIO.com.

To report SUSPECTED ADVERSE REACTIONS, contact Karyopharm Therapeutics Inc. at 1‐888‐209‐9326 or FDA at 1‐800‐FDA‐1088 or www.fda.gov/medwatch.

On October 31, 2024 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") reported its financial and operating results for the third quarter ended September 30, 2024, and provided an update on its recent corporate activities and outlook (Press release, Halozyme, OCT 31, 2024, View Source [SID1234647602]).

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"Our robust third quarter financial results highlight the strong execution and accelerating momentum we have across our business and exceeded expectations with total revenue growth of 34% and adjusted EBITDA growth of 60%. Based on the strong performance year-to-date, we have raised our 2024 guidance ranges and expect the advancement of our ENHANZE pipeline and new nominations from two global licensing agreements to support our future growth trajectory," said Dr. Helen Torley, president and chief executive officer of Halozyme. "In the quarter, the announcement of two highly anticipated partner approvals in the U.S. for Roche’s TECENTRIQ HYBREZA and OCREVUS ZUNOVO reinforces ENHANZE’s track record of 100% phase 3 study and subsequent regulatory success. The new nominations for ENHANZE from argenx, for a total of six targets, and ViiV Healthcare, for an additional undisclosed target, further demonstrate the value of our leading technology for rapid, large volume subcutaneous delivery."

Recent Partner Highlights:
•In October 2024, argenx initiated two studies evaluating VYVGART Hytrulo with ENHANZE, a Phase 3 study for adult patients with ocular myasthenia gravis ("oMG") and a Phase 2 study for kidney transplant recipients with antibody mediated rejection ("AMR").
•In October 2024, Janssen announced the European Commission approved DARZALEX SC for the treatment of patients newly diagnosed with multiple myeloma ("NDMM") who are eligible for autologous stem cell transplant ("ASCT") in combination with bortezomib, lenalidomide, and dexamethasone ("D-VRd").

•In September 2024, argenx expanded its global collaboration and license agreement nominating four additional targets that provides them exclusive access to our ENHANZE drug delivery technology for a total of six targets. Under the terms of the expanded exclusive agreement, we received upfront payments of $7.5 million per target nomination for a total of $30.0 million. argenx is obligated to make future milestone payments of up to $85.0 million per new nominated target, subject to achievements of specified development, regulatory and sales-based milestones. We are also entitled to receive royalties on net sales of commercialized products with our ENHANZE technology.
•In September 2024, ViiV expanded its global collaboration and license agreement providing ViiV the ability to exclusively access our ENHANZE drug delivery technology for one additional undisclosed target.
•In September 2024, Roche announced the U.S. Food and Drug Administration ("FDA") approved OCREVUS ZUNOVO with ENHANZE as a twice a year ten-minute subcutaneous ("SC") injection for the treatment of relapsing multiple sclerosis and primary progressive multiple sclerosis.
•In September 2024, Roche announced the FDA approved TECENTRIQ HYBREZA with ENHANZE for all approved adult indications of intravenous ("IV") TECENTRIQ and was made available to patients, resulting in a $12.0 million milestone payment.
•In September 2024, Janssen announced the submission of a supplemental Biologic License Application to the FDA for approval of a new indication of DARZALEX FASPRO in combination with D-VRd for the treatment of adult patients with NDMM for whom ASCT is deferred or who are ineligible for ASCT.
•In August 2024, the FDA designated Janssen’s Biologics License Application ("BLA") priority review status for amivantamab SC in combination with LAZCLUZE for currently approved or submitted indication of IV in certain patients with non-small cell lung cancer.
•In August 2024, Takeda submitted a New Drug Application in Japan seeking approval for TAK-771 with ENHANZE for treatment of chronic inflammatory demyelinating polyneuropathy/Multifocal Motor Neuropathy.
•In July 2024, Janssen announced the FDA approved DARZALEX FASPRO for an additional indication in NDMM patients who are eligible for ASCT in combination with D-VRd.
•In July 2024, argenx announced the National Medical Products Administration approved the BLA of efgartigimod SC for generalized myasthenia gravis in China.
•In July 2024, Acumen initiated a Phase 1 study of sabirnetug ("ACU193") co-formulated with ENHANZE for the treatment of early Alzheimer’s disease.

Third Quarter 2024 Financial Highlights:
•Revenue was $290.1 million, compared to $216.0 million in the third quarter of 2023. The 34% year-over-year increase was primarily driven by royalty revenue growth and an increase in milestone revenue. Revenue for the quarter included $155.1 million in royalties, an increase of 36% compared to $114.4 million in the third quarter of 2023, primarily attributable to increases in revenue of DARZALEX SC and Phesgo, and the prior year launch of VYVGART Hytrulo.
•Cost of sales was $49.4 million, compared to $54.8 million in the third quarter of 2023. The decrease was primarily due to lower device and bulk rHuPH20 sales.
•Amortization of intangibles expense was $17.8 million, compared to $20.3 million in the third quarter of 2023. The decrease was primarily due to an impairment charge of $2.5 million recognized in the prior year to fully impair the TLANDO product rights intangible asset.
•Research and development expense was $18.5 million, compared to $17.3 million in the third quarter of 2023. The increase was primarily due to increased compensation expense.

•Selling, general and administrative expense was $41.2 million, compared to $35.3 million in the third quarter of 2023. The increase was primarily due to increased compensation expense and consulting and professional service fees.
•Operating income was $163.2 million, compared to $88.3 million in the third quarter of 2023.
•Net Income was $137.0 million, compared to $81.8 million in the third quarter of 2023.
•EBITDA was $183.6 million, compared to $124.6 million in the third quarter of 2023. Adjusted EBITDA was $183.6 million, compared to $114.9 million in the third quarter of 2023.1
•GAAP diluted earnings per share was $1.05, compared to $0.61 in the third quarter of 2023. Non-GAAP diluted earnings per share was $1.27, compared to $0.75 in the third quarter of 2023.1
•Cash, cash equivalents and marketable securities were $666.3 million on September 30, 2024, compared to $336.0 million on December 31, 2023. The increase was primarily a result of cash generated from operations.

Financial Outlook for 2024
The Company is raising its financial guidance for 2024. For the full year 2024, the Company expects:

•Total revenue of $970 million to $1,020 million, representing growth of 17% to 23% over 2023 total revenue primarily driven by increases in royalty revenue, collaboration revenue and growth in product sales from XYOSTED. Revenue from royalties of $550 million to $565 million, representing growth of 23% to 26% over 2023.
•Adjusted EBITDA of $595 million to $625 million, representing growth of 40% to 47% over 2023.
•Non-GAAP diluted earnings per share of $4.00 to $4.20, representing growth of 44% to 52% over 2023. The Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

Table 1. 2024 Financial Guidance

Previous Guidance Range
New Guidance Range
Total Revenue $935 to $1,015 million
$970 to $1,020 million
Royalty Revenue $520 to $555 million
$550 to $565 million
Adjusted EBITDA $555 to $615 million
$595 to $625 million
Non-GAAP Diluted EPS $3.65 to $4.05
$4.00 to $4.20

Webcast and Conference Call
Halozyme will host its Quarterly Update Conference Call for the third quarter ended September 30, 2024 today, Thursday, October 31, 2024 at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed live with pre-registration via link: View Source The call will also be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit Halozyme.com.

On October 31, 2024 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion"), a clinical-stage TechBio company specializing in developing AI-Immunology powered vaccines, reported business update and provided third quarter 2024 financial results (Press release, Evaxion Biotech, OCT 31, 2024, View Source [SID1234647600]).

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Business highlights (since last quarterly update)

Since the second quarter 2024 business update, we have continued to execute strongly on our strategy and plans with several major milestones achieved. Key highlights include:

Significant expansion of the infectious disease vaccine development collaboration with MSD (tradename of Merck & co., Inc., Rahway, NJ, USA) in a transformative deal for Evaxion
Continuously increasing external interest and several ongoing partnerships discussions covering both our platform and pipeline
Strong progress in clinical and preclinical development with convincing phase 2 data presented for personalized cancer vaccine EVX-01 and preclinical Proof-of-Concept obtained for EVX-B2 mRNA Gonorrhea vaccine candidate
Launch of improved AI-Immunology platform for vaccine antigen prediction
Thomas Schmidt appointed as interim Chief Financial Officer
"We continued to make solid progress on our strategy execution in a busy third quarter and are very pleased to have achieved several important milestones across our company. The MSD agreement, which holds the potential to transform Evaxion over the coming years, and the groundbreaking EVX-01 phase 2 efficacy data, stand out among our many achievements. We continue to demonstrate our strong capabilities as a truly AI-based TechBio company and remain focused on advancing on-going partnerships as well as new partnership discussions, progressing the EVX-01 trial and carrying through preclinical studies as a basis for expanding our R&D pipeline," says Christian Kanstrup, CEO of Evaxion.

2024 Milestones

Milestones Target
EVX-B1 Conclusion of final MTA study with potential partner Q1 2024 ✓
AI-Immunology Launch of EDEN model version 5.0 Mid 2024
(ECCB, September) ✓
EVX-B2-mRNA EVX-B2-mRNA preclinical Proof-of-Concept obtained Q3 2024
(18th Vaccine Congress, September) ✓
EVX-01 Phase 2 one-year readout Q3 2024
(ESMO Congress, September) ✓
EVX-B3 Conclusion of target discovery and validation work in collaboration with MSD (tradename of Merck & Co., Inc., Rahway, NJ, USA)* H2 2024 (✓)
Precision ERV cancer vaccines Preclinical Proof-of-Concept obtained H2 2024
Funding Ambition for full year 2024 is to generate business development income or cash in equal to 2024 cash burn (excluding financing activities) of $14 million**
* MSD option and license agreement on EVX-B2 and EVX-B3 supersedes this milestone
** See update on the business development income ambition below

Research & Development update

We maintain a high activity level in Research & Development (R&D) from both a preclinical and clinical perspective. This work yielded outstanding results in the third quarter, first and foremost with the presentation of encouraging one-year data from the ongoing phase 2 trial with our lead asset EVX-01, an AI-Immunology designed personalized cancer vaccine, in patients with advanced melanoma (skin cancer).

The data demonstrates 69% Overall Response Rate, reduction in tumor target lesions in 15 out of 16 patients, an immunogenicity rate of 79%, and a positive correlation between our AI-Immunology platform predictions and immune responses induced by the individual neoantigens in the EVX-01 vaccine (p=0.00013). The observed immunogenicity rate means that 79% of EVX-01’s vaccine targets triggered a targeted immune response, which compares very favorably to what is seen with other approaches.

These clinical findings underscore the significant therapeutic potential of EVX-01 and are yet another validation of the AI-Immunology platform as a leading AI technology for fast and effective vaccine target discovery and design.

We were also successful in our preclinical research, obtaining Proof-of-Concept for novel mRNA Gonorrhea vaccine candidate EVX-B2. This was based on new data documenting that EVX-B2 mRNA triggers a targeted immune response that leads to the elimination of the gonorrhea bacteria. The same had earlier been shown for the protein-based version of EVX-B2, which is now part of our partnership with MSD. The mRNA data has been generated as part of our partnership with Afrigen Biologics.

Further to our pipeline, our R&D investments are also allocated to the continued improvement of our AI-Immunology platform. During the third quarter, we updated the platform with the launch of a new version of its EDEN AI prediction model. Among other improvements, the model can now predict toxin antigens, allowing for the development of improved bacterial vaccines. We expect this update to further solidify the strong interest seen in AI-Immunology from potential partners.

Business development income

Our strategy is based upon a multi-partner approach, making effective execution upon our business development plans crucial to our success. We were thrilled to sign the significantly expanded vaccine development collaboration with MSD during the third quarter. Further, we continue to see an increasing interest from potential partners and are excited by the current partnership opportunities both around existing pipeline assets as well as our AI-Immunology platform.

The agreement with MSD carries potential business development income of up to $10 million for 2025 on top of the $3.2 million upfront payment received in 2024. Based upon the current business development opportunities, we remain confident in our ability to execute upon our multi-partner strategy and bring in significant business development income.

Given that certain partnership discussions will be moving into 2025, we will – despite the strong interest – not be able to meet our 2024 ambition of generating business development income or cash in of $14 million. The discussions, having moved into 2025, will however support the generation of business development income for next year in addition to the potential up to $10 million from MSD.

Nasdaq dialogue

As communicated earlier, on May 7, 2024, we received a deficiency letter from Nasdaq Stock Market LLC ("Nasdaq") for failure to maintain stockholders’ equity of at least $2.5 million, following which we presented a plan to Nasdaq to regain compliance. Nasdaq provided us until November 4, 2024, to evidence compliance based upon the plan submitted.

We remain committed to ensuring compliance with the Nasdaq minimum stockholder’s equity requirement and maintain our Nasdaq listing. This is to be pursued through increasing shareholder’s equity via a combination of business development income and capital markets activities. However, current equity market environment, the geopolitical uncertainties and timing of business development activities have to date impacted timing for the full required increase in shareholder’s equity.

We do not expect to have regained compliance by November 4, 2024, and therefore expect Nasdaq to send us a delisting notification after such date. We then plan to appeal the delisting determination and request a hearing on the matter, following which a new 180-day extension could be granted based on our plan to regain compliance.

We are in constructive dialogue with Nasdaq around this process, though we will not receive any guarantee that another 180-day extension will be granted before the anticipated hearing.

Third quarter 2024 financial results

Cash position as of September 30, 2024, was $4.6 million, as compared to $5.6 million as of December 31, 2023. The cash position as of September 30, excludes the $3.2 million upfront from the MSD agreement which was received in October. The Company expects that its existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into March 2025.

Revenue of $3.0 million was recognized for the quarter ending September 30, 2024, as compared to nil for the quarter ending September 30, 2023. A minor proportion of this revenue derives from the existing EVX-B3 collaboration with MSD, while the majority relates to the newly signed option and license agreement with MSD.

Research and Development expenses were $2.6 million for the quarter ending September 30, 2024, as compared to $2.8 million for the quarter ending September 30, 2023. The decrease is primarily related to a reduced headcount.

General and Administrative expenses were $2.1 million for the quarter ending September 30, 2024, as compared to $2.9 million for the quarter ending September 30, 2023. The decrease was primarily due to a decrease in expenses to management remuneration following changes to executive management in 2023 and expenses related to this. In addition, various minor cost reductions related to overhead and professional fees are realized.

We generated a net loss of $1.9 million for the quarter ending September 30, 2024, or $(0.04) per basic and diluted share, as compared to a net loss of $5.7 million, or $(0.21) per basic and diluted share for the quarter ending September 30, 2023. The decreased loss was primarily driven by the recognized revenue and reduced general & administrative expenses.

Total equity amounts to $0.1 million as of September 30, 2024. Proceeds from the exercise of prefunded warrants amounted to $0.2 million for the quarter.

Evaxion Biotech A/S
Consolidated Statement of Financial Position Data (Unaudited)
(USD in thousands)

Sep 30,
2024 Dec 31,
2023
Cash and cash equivalents 4,576 5,583
Total assets 15,185 12,889
Total liabilities 15,111 17,618
Share capital 8,732 5,899
Other reserves 106,245 99,946
Accumulated deficit (114,903) (107,860)
Total equity before derivative warrant liability 74 (2,015)
Effect from derivative liabilities from investor warrants - (2,714)
Total equity 74 (4,729)
Total liabilities and equity 15,185 12,889

Based on the Company’s current cash position with an expected cash runway into March 2025, income from Business Development deals and/or further funding is required to mitigate the conclusion that there is significant doubt about the Company’s ability to continue as a going concern. Please refer to the Form 20-F, filed March 27, 2024, for additional background on the Company.

Evaxion Biotech A/S
Consolidated Statement of Comprehensive Loss Data (Unaudited)
(USD in thousands, except per share data)

Three Months Ended
September 30, Nine Months Ended
September 30,
2024 2023 2024 2023
Revenue 3,017 — 3,222 —
Research and development (2,614) (2,830) (8,202) (9,618)
General and administrative (2,134) (2,932) (5,728) (8,215)
Operating loss (1,731) (5,762) (10,708) (17,833)
Finance income 84 72 5,922 404
Finance expenses (384) (182) (2,665) (786)
Net loss before tax (2,031) (5,872) (7,451) (18,215)
Income tax benefit 96 194 513 613
Net loss for the period (1,935) (5,678) (6,938) (18,215)
Net loss attributable to shareholders of Evaxion Biotech A/S (1,935) (5,678) (6,938) (18,215)
Loss per share – basic and diluted (0.04) (0.21) (0.13) (0.66)
Number of shares used for calculation (basic and diluted) 55,255,329 27,659,878 51,905,948 26,754,440