On October 30, 2024 Cerus Corporation (Nasdaq: CERS) reported financial results for its third quarter and nine months ended September 30, 2024 (Press release, Cerus, OCT 30, 2024, View Source [SID1234647535]).

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

Three Months Ended

Nine Months Ended

September 30,

Change

September 30,

Change

2024

2023

$

%

2024

2023

$

%

Product Revenue

$

46,017

$

39,772

$

6,245

16

%

$

129,461

$

109,599

$

19,862

18

%

Government Contract Revenue

4,639

7,479

(2,840

)

-38

%

15,109

23,856

(8,747

)

-37

%

Total Revenue

$

50,656

$

47,251

$

3,405

7

%

$

144,570

$

133,455

$

11,115

8

%

Recent highlights include:

Partnered with U.S. Biomedical Advanced Research and Development Authority (BARDA) for new, six-year contract with value up to $248 million designed to support advancement of INTERCEPT Blood System for Red Blood Cells (RBCs) beyond U.S. Phase 3 studies through potential PMA licensure and into commercialization.
CE Mark review for INTERCEPT RBCs concluded without approval; assessing strategy for potential future regulatory submission in collaboration with Notified Body, TÜV-SÜD.
Further expanded U.S. manufacturing capacity for INTERCEPT Fibrinogen Complex (IFC) following receipt of three additional biologics license application (BLA) approvals by Cerus IFC production partners.
Multiple presentations across the INTERCEPT product portfolio at this month’s 2024 Association for Advancement of Blood & Biotherapies (AABB) Annual Meeting, including real-world case studies for IFC and clinical data from the U.S. Phase 3 ReCePI trial.
Submitted CE Mark dossier for LED illuminator in Europe to TÜV-SÜD.
Narrowed GAAP net loss attributable to Cerus Corporation to $2.9 million and generated positive non-GAAP adjusted EBITDA of $4.4 million for the third quarter.
Generated positive operating cash flows for the third straight quarter of 2024 bringing year-to-date positive operating cash flows to $6.4 million.
"Our performance, both commercially and financially, was very strong for the third quarter," stated William "Obi" Greenman, Cerus’ president and chief executive officer. "In addition to the organic growth in our North American INTERCEPT platelet business, we are pleased to have secured meaningful additional U.S. manufacturing capacity for IFC with the new BLA approvals, which will allow us to address the growing demand for the product. As a result of our strong performance during the first three quarters coupled with our expectations for future growth in our business segments, we are raising our full year product revenue guidance and increasing the lower end of our IFC guidance. Beyond the top-line, we have also delivered on our bottom-line and cashflow targets for Q3. We believe we are on track to hit our full-year 2024 goals as the year wraps up."

"With respect to our development pipeline, we are pleased to have submitted our LED illuminator regulatory filing in Europe in the third quarter," continued Greenman. "This much-anticipated new technology platform will enable operational improvements alongside the dependability that our customers have grown to expect from the INTERCEPT System. As we reported earlier this month, our RBC program remains a key focus for us in both the U.S., with the new BARDA contract accelerating a number of initiatives, as well as in the EU, where we anticipate an enhanced regulatory submission that we expect will incorporate the ReCePI Phase 3 clinical data."

Revenue

Product revenue during the third quarter of 2024 was $46.0 million, compared to $39.8 million during the prior year period. This year-over-year increase of 16% was driven primarily by growth in the Company’s platelets business, particularly in North America. Third-quarter product revenue included sales of IFC, which were $2.3 million, up from $1.7 million during the prior year period.

Third-quarter 2024 government contract revenue was $4.6 million, compared to $7.5 million during the prior year period. The Company’s government contract revenue was comprised of funding associated with research and development (R&D) activities related to the INTERCEPT Blood System for RBCs as well as efforts related to the development of next-generation pathogen reduction technology to treat whole blood and development of a lyophilized IFC. Reported government contract revenue during the third quarter of 2024 decreased versus the prior year period, primarily due to completion of the U.S. Phase 3 ReCePI clinical trial for INTERCEPT RBCs.

Product Gross Profit & Margin

Product gross profit for the third quarter of 2024 was $26.2 million, increasing by 20% over the prior year period. Product gross margin for the third quarter of 2024 improved to 56.9% compared to 54.9% for the prior year period. Absent any unanticipated factor, Cerus expects product gross margin levels to remain relatively consistent for the fourth quarter.

Operating Expenses

Total operating expenses for the third quarter of 2024 were $31.8 million, compared to $34.5 million for the same period of the prior year, reflecting a decrease of 8%. This decline resulted from decreases in R&D, partially offset by increases in selling, general and administrative (SG&A) expenses.

R&D expenses for the third quarter of 2024 were $14.0 million, compared to $16.8 million for the prior year period. The primary drivers for the decrease in R&D expenses were the completion of the Company’s ReCePI Phase 3 clinical trial during the first quarter of 2024 and the full impact of the previous year’s restructuring.

SG&A expenses for the third quarter of 2024 were $17.8 million, compared to $16.2 million for the prior year period. The primary driver for the increase in SG&A expenses was non-cash stock-based compensation. The Company continues to expect SG&A expenses will be relatively consistent for the balance of the year, driving increased leverage at these levels.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the third quarter of 2024 was $2.9 million, or $0.02 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $7.3 million, or $0.04 per basic and diluted share, for the third quarter of 2023. Net loss attributable to Cerus Corporation for the first nine months of 2024 was $18.4 million, compared to a net loss attributable to Cerus Corporation of $36.2 million for the first nine months of 2023.

Non-GAAP Adjusted EBITDA

Non-GAAP adjusted EBITDA for the third quarter of 2024 was a positive $4.4 million, compared to a loss of $1.0 million for the third quarter of 2023. Non-GAAP adjusted EBITDA was a positive $2.5 million for the first nine months of 2024, compared to a loss of $15.5 million for the first nine months of 2023. The Company continues to focus on achieving non-GAAP adjusted EBITDA breakeven for the full-year 2024. For additional information, please see definitions and the reconciliation of this non-GAAP measure to net loss attributable to Cerus Corporation accompanying this release.

Balance Sheet & Cash Flows

At September 30, 2024, the Company had cash and cash equivalents and short-term investments of $75.6 million, compared to $71.2 million at June 30, 2024, and $65.9 million at December 31, 2023.

As of September 30, 2024, the Company had $65.0 million outstanding on its term loan and $18.5 million drawn on its revolving credit facility. The Company’s revolving line of credit allows for an additional $16.5 million, which is dependent on eligible assets supporting the borrowing base.

For the third quarter of 2024, the Company generated positive cash flows of $4.1 million from operations compared to cash used in operations of $10.5 million during the prior year period. These improvements were in line with the Company’s expectations and will continue to be a focus area going forward.

Raising 2024 Product Revenue Guidance

The Company is raising its full-year 2024 product revenue guidance to a range of $177 million to $179 million from the prior range of $175 million to $178 million. Alongside this raise in guidance, the Company is raising the bottom end of its full-year 2024 IFC revenue guidance to a new range of $9 million to $10 million from $8 million to $10 million.

Quarterly Conference Call

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

A replay will be available on Cerus’ website approximately three hours after the call through November 20, 2024.

On October 30, 2024 Biogen Inc. (NASDAQ: BIIB) reported third quarter 2024 financial results (Press release, Biogen, OCT 30, 2024, View Source [SID1234647534]). Commenting on the quarter, President and Chief Executive Officer Christopher A. Viehbacher said:

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"In the third quarter Biogen made continued progress toward our goal of returning to sustainable growth. We continue to see momentum with ongoing product launches and we are increasingly excited about the potential of our pipeline. This quarter we had several significant positive developments in key areas of our late-stage pipeline which we believe underscore the potential value for both patients and shareholders. Importantly, the positive results for dapirolizumab pegol, as well as recent presentations of felzartamab data in IgAN, bolster our efforts to develop an industry-leading pipeline in immunology, where we are building upon data insights and increasing our capabilities to prepare for potential future launches."

Financial Highlights
Q3 ’24 Q3 ’23 △
r (CC*)
Total Revenue (in millions) $2,466 $2,530 (3)% (3)%
GAAP diluted EPS $2.66 $(0.47) 666% N/A
Non-GAAP diluted EPS $4.08 $4.36 (6)% N/A

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period.
N/A = not applicable.
* Percentage changes in revenue growth at constant currency (CC) are presented excluding the impact of changes in foreign currency exchange rates and hedging gains or losses. Foreign currency revenue values are converted into U.S. Dollars using the exchange rates from the end of the previous calendar year.

A reconciliation of GAAP to Non-GAAP financial measures can be found in Table 4 at the end of this news release.
Revenue Summary
(in millions) Q3 ’24 Q3 ’23 △
r (CC*)
Multiple sclerosis (MS) product revenue(1)
$1,054 $1,159 (9)% (9)%
Rare disease revenue(2)
$495 $450 10% 10%
Biosimilars revenue $197 $194 1% —%
Other product revenue(3)
$24 $2 NMF NMF
Total product revenue $1,769 $1,805 (2)% (2)%
Revenue from anti-CD20 therapeutic programs $446 $421 6% 6%
Contract manufacturing, royalty and other revenue $250 $304 (18)% (19)%
Total revenue $2,466 $2,530 (3)% (3)%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period. Numbers may not foot or recalculate due to rounding.
NMF = no meaningful figure.
(1) Multiple sclerosis includes TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI and FAMPYRA.
(2) Rare disease includes SPINRAZA, SKYCLARYS and QALSODY.
(3) Other includes ADUHELM, FUMADERM and ZURZUVAE.
•Third quarter 2024 ZURZUVAE revenue was approximately $22 million.
Expense Summary
(in millions) Q3 ’24 Q3 ’23 △
GAAP cost of sales*
$639 $660 3%
% of Total Revenue 26% 26%
Non-GAAP cost of sales*
$593 $660 10%
% of Total Revenue 24% 26%
GAAP R&D expense $543 $736 26%
Non-GAAP R&D expense $491 $539 9%
GAAP SG&A expense $588 $788 25%
Non-GAAP SG&A expense $556 $553 (1)%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period
* Excluding amortization and impairment of acquired intangible assets

•The decrease in third quarter 2024 GAAP and Non-GAAP cost of sales as a percentage of total revenue was driven primarily by product mix, particularly the year-over-year increase in revenue from new
2

product launches and decrease in contract manufacturing revenue, as well as lower idle capacity charges.

•In the third quarter 2024 as compared to the third quarter of 2023, the decrease in GAAP R&D of approximately $194 million was primarily driven by approximately $197 million of equity-based compensation expense recognized in 2023 related to the Reata Pharmaceuticals, Inc. (Reata) acquisition, cost-reduction measures realized in 2024 in connection with the Company’s R&D prioritization and Fit for Growth initiatives, as well as higher spend on clinical trials and close out costs incurred during 2023, partially offset by approximately $43 million of equity-based compensation expense recognized in 2024 related to the Human Immunology Biosciences, Inc. (HI-Bio) acquisition.

•In the third quarter 2024 as compared to the third quarter of 2023, the decrease in Non-GAAP R&D of approximately $48 million was primarily due to cost-reduction measures realized in 2024 in connection with the Company’s R&D prioritization and Fit for Growth initiatives, as well as higher spend on clinical trials and close out costs incurred during 2023.

•In the third quarter 2024 as compared to the third quarter of 2023, the decrease in GAAP SG&A expense of approximately $200 million was primarily due to approximately $196 million of equity-based compensation expense recognized in 2023 related to the acquisition of Reata and cost-reduction measures realized in 2024 in connection with the Company’s Fit for Growth initiative.

•In the third quarter 2024 as compared to the third quarter of 2023, the increase in Non-GAAP SG&A expense of approximately $3 million was primarily due to increased commercialization spend related to new product launches, partially offset by savings achieved from the Company’s Fit for Growth initiative.
Other Financial Highlights

•Third quarter 2024 GAAP and Non-GAAP collaboration profit sharing was a net expense of approximately $69 million, which includes approximately $60 million related to Biogen’s collaboration with Samsung Bioepis, and approximately $9 million related to Biogen’s collaboration with Sage Therapeutics, Inc. (Sage) and the commercialization of ZURZUVAE in the U.S.

•Third quarter 2024 GAAP other expense was approximately $15 million, primarily driven by net interest expense, partially offset by net realized and unrealized gains on strategic equity investments of approximately $39 million. Third quarter 2024 Non-GAAP other expense was approximately $54 million, primarily driven by net interest expense.

•Third quarter 2024 GAAP and Non-GAAP effective tax rates were 13.9% and 13.8%, respectively. Third quarter 2023 GAAP and Non-GAAP effective tax rates were 51.6% and 14.7%, respectively.
Financial Position

•Third quarter 2024 net cash flow from operations was approximately $936 million. Capital expenditures were approximately $35 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was approximately $901 million.

•As of September 30, 2024, Biogen had cash, cash equivalents, and marketable securities totaling approximately $1.7 billion and approximately $6.3 billion in total debt, resulting in net debt of approximately $4.6 billion.

•No shares of the Company’s common stock were repurchased in the third quarter of 2024. As of September 30, 2024, there was approximately $2.1 billion remaining under the share repurchase program authorized in October 2020.

•For the third quarter of 2024 the Company’s weighted average diluted shares were approximately 146 million.

Full Year 2024 Financial Guidance

For the full year 2024, Biogen now expects a Non-GAAP diluted EPS guidance range as follows:
Prior FY 2024 Guidance Updated FY 2024 Guidance
Non-GAAP diluted EPS
$15.75 to $16.25
Reflecting growth of ~9% at the mid-point*
$16.10 to $16.60
Reflecting growth of ~11% at the mid-point*

*Versus reported full year 2023

Biogen continues to expect total revenue to decline by a low-single digit percentage, with core pharmaceutical revenue, defined as product revenue plus Biogen’s 50% share of net LEQEMBI product revenue and cost of sales, including royalties, to be relatively flat for 2024 compared to 2023 as further declines in multiple sclerosis product revenue are expected to be offset by increases in revenue from new product launches.

Biogen continues to expect an improvement in the cost of sales as a percentage of total revenue for 2024 compared to 2023 driven by product mix and significantly lower idle capacity charges.

For 2024 compared to 2023, Biogen expects operating income to grow at a high-teen percentage with mid-single digit percentage point operating margin improvement. This is expected to be driven by improved cost of sales as a percentage of revenue, as well as lower operating expenses as a result of the Company’s Fit for Growth and R&D prioritization initiatives.

This financial guidance does not include any impact from potential acquisitions or business development transactions or pending and future litigation or any impact of potential tax or healthcare reform, as all are hard to predict.

This guidance also assumes that foreign exchange rates as of October 25, 2024, will remain in effect for the remainder of the year, net of hedging activities. Other modeling considerations will be provided on the conference call and webcast.

Biogen may incur charges, realize gains or losses, or experience other events or circumstances in 2024 that could cause any of these assumptions to change and/or actual results to vary from this financial guidance.

Biogen does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the Company is unable to predict with reasonable certainty the financial impact of items such as the transaction, integration, and certain other costs related to acquisitions or large business development transactions; unusual gains and losses; potential future asset impairments; gains and losses from equity security investments; and the ultimate outcome of pending or future significant litigation without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. For the same reasons, the Company is unable to address the significance of the unavailable information, which could be material to future results.

Key Recent Events

•In October, Biogen and Sage decided they will not pursue further development for zuranolone as a treatment for major depressive disorder.

Conference Call and Webcast

The Company’s earnings conference call for the third quarter will be broadcast via the internet at 8:30 a.m. ET on October 30, 2024 and will be accessible through the Investors section of Biogen’s website, www.biogen.com. Supplemental information in the form of a slide presentation is also accessible at the same location on the internet and will be subsequently available on the website for at least 90 days.

On October 30, 2024 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, Arvinas, OCT 30, 2024, View Source [SID1234647533]).

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"We maintained strong momentum across our portfolio in the third quarter and remain on track to report topline data from VERITAC-2, our Phase 3 clinical trial in metastatic breast cancer, in the fourth quarter of 2024 or the first quarter of 2025," said John Houston, Ph.D., Chairperson, Chief Executive Officer and President at Arvinas. "In partnership with our colleagues at Pfizer, we are excited by the possibility of changing the treatment paradigm for ER+/HER2- breast cancer and look forward to sharing results from VERITAC-2 in the coming months."

"Our path to reaching our goal of becoming a multi-product, commercial-stage organization with a robust pipeline across several indications is clearly defined, and our novel PROTAC platform technology has the potential to enable opportunities across multiple therapeutic areas," continued Dr. Houston. "We look forward to completing the multiple ascending dose portion of our Phase 1 clinical trial with ARV-102, our first PROTAC degrader with the potential to treat neurodegenerative diseases, and sharing data in 2025. We are also encouraged by the preclinical profile of ARV-393, our BCL6 PROTAC degrader currently being evaluated in a first-in-human Phase 1 clinical trial in patients with B-cell lymphomas. We look forward to sharing more about these programs in the coming months."

3Q 2024 Business Highlights and Recent Developments

Vepdegestrant

Continued enrollment globally in multiple clinical studies of vepdegestrant in ER+/HER2- metastatic breast cancer.
VERITAC-2: Phase 3 monotherapy clinical trial in patients with metastatic breast cancer (ClinicalTrials.gov Identifier: NCT05654623)
TACTIVE-U: Phase 1b/2 combination umbrella trial evaluating combinations of vepdegestrant with abemaciclib, ribociclib, or samuraciclib (ClinicalTrials.gov Identifiers: NCT05548127, NCT05573555, and NCT06125522).
TACTIVE-K: Phase 1/2 clinical trial with vepdegestrant plus Pfizer’s novel CDK4 inhibitor atirmociclib (ClinicalTrials.gov Identifier: NCT06206837)
ARV-102: Oral PROTAC LRRK2 degrader

In October, presented preclinical data at the 2024 Michael J. Fox Foundation Parkinson’s Disease Conference further supporting the potential of PROTAC-induced leucine-rich repeat kinase 2 (LRRK2) degradation as a potential treatment for patients with neurodegenerative diseases. New findings presented included data demonstrating:
Orally delivered ARV-102 crosses the blood-brain barriers and degrades LRRK2 in the cerebrospinal fluid (CSF) of non-human primates (NHPs).
Degradation of LRRK2 by ARV-102 induces changes in pathway (lysosomal and inflammation) biomarkers in the CSF of NHPs, which has not previously been demonstrated by kinase inhibitors of LRRK2.
In murine tauopathy models, oral PROTAC LRRK2 degrader treatment led to ~50% pathologic tau reduction.
Initiated the multiple ascending dose portion of the ongoing Phase 1 clinical trial in healthy volunteers.
ARV-393: Oral PROTAC BCL6 degrader

Continued recruiting patients in the first-in-human Phase 1 clinical trial in patients with B-cell lymphomas.
Anticipated Upcoming Milestones and Expectations

Vepdegestrant

As part of Arvinas global collaboration with Pfizer, the companies plan to:

Complete enrollment (4Q24) and announce topline data (4Q24/1Q25) for the VERITAC-2 Phase 3 monotherapy clinical trial in patients with metastatic breast cancer.
Present initial safety and pharmacokinetic data from the TACTIVE-U sub-study (NCT05548127) of abemaciclib in combination with vepdegestrant at the San Antonio Breast Cancer Symposium (SABCS) in December 2024.
Present data from the Phase 1 healthy volunteer pharmacokinetic trial of vepdegestrant in combination with midazolam to assess potential for drug-drug interaction at SABCS in December 2024 (NCT06256510).
Continue enrollment of the ongoing Phase 1b/2 combination umbrella trial evaluating combinations of vepdegestrant with abemaciclib, ribociclib, or samuraciclib (TACTIVE-U; ClinicalTrials.gov Identifiers: NCT05548127, NCT05573555, and NCT06125522).
Evaluate data from the study lead-in of the VERITAC-3 Phase 3 trial (NCT05909397) in patients with ER+/HER2- locally advanced or metastatic breast cancer (2H24).
Continue enrollment and evaluate preliminary data from the ongoing clinical trial with vepdegestrant plus Pfizer’s novel CDK4 inhibitor atirmociclib (TACTIVE-K).
Start Phase 3 combination trials in the first- and second-line settings (anticipated in 2025; pending emerging data and regulatory feedback).
First-line setting with vepdegestrant plus atirmociclib or palbociclib.
Second-line setting with vepdegestrant plus palbociclib and/or another CDK4/6 inhibitor.
ARV-102: Oral PROTAC LRRK2 degrader

Complete enrollment in the ongoing multiple ascending dose portion of a Phase 1 trial evaluating ARV-102 in healthy volunteers
Present data from the Phase 1 trial in 2025.
ARV-393: Oral PROTAC BCL6 degrader

Continue recruiting patients in the first-in-human Phase 1 clinical trial evaluating ARV-393 in patients with B-cell lymphomas.
Novel PROTAC KRAS G12D degrader

File an Investigational New Drug (IND) application in 2025.
Financial Guidance
Based on its current operating plan, Arvinas believes its cash, cash equivalents, and marketable securities as of September 30, 2024, is sufficient to fund planned operating expenses and capital expenditure requirements into 2027.

Third Quarter Financial Results

Cash, Cash Equivalents, and Marketable Securities Position: As of September 30, 2024, cash, cash equivalents and marketable securities were $1,121.6 million as compared with cash, cash equivalents, restricted cash and marketable securities of $1,266.5 million as of December 31, 2023. The decrease in cash, cash equivalents and marketable securities of $144.9 million for the nine months ended September 30, 2024 was primarily related to cash used in operations of $158.1 million (net of $150.0 million received from the Novartis agreements), inclusive of a one-time cash termination fee in the amount of $41.5 million related to the termination of our laboratory and office space lease with 101 College Street LLC in August 2024 and the purchase of lab equipment and leasehold improvements of $1.5 million, partially offset by proceeds from the exercise of stock options of $7.7 million and unrealized gains on marketable securities of $7.2 million.

Research and Development Expenses: Research and development expenses were $86.9 million for the quarter ended September 30, 2024, as compared with $85.9 million for the quarter ended September 30, 2023. The increase in research and development expenses of $1.0 million for the quarter was primarily due to an increase in compensation and related personnel expenses of $2.8 million, which are not allocated by program, partially offset by a decrease in external expenses of $2.2 million. External expenses include program-specific expenses, which decreased by $1.1 million, primarily driven by decreases in our ARV-766 and ARV-110 programs of $3.8 million and $1.6 million, respectively, partially offset by increases in our ARV-102 and ARV-393 programs of $2.5 million and $1.4 million, respectively, and our non-program specific expenses, which decreased by $1.1 million.

General and Administrative Expenses: General and administrative expenses were $75.8 million for the quarter ended September 30, 2024, as compared with $22.6 million for the quarter ended September 30, 2023. The increase in general and administrative expenses of $53.2 million for the quarter was primarily due to a loss on the termination of our laboratory and office space lease with 101 College Street LLC of $43.4 million as well as increases in personnel and infrastructure related costs of $5.0 million, professional fees of $3.4 million and in developing our commercial operations of $1.2 million.

Revenue: Revenue was $102.4 million for the quarter ended September 30, 2024 as compared with $34.6 million for the quarter ended September 30, 2023. Revenue for the quarter is related to the license agreement with Novartis, the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer, the collaboration and license agreement with Bayer and the collaboration and license agreement with Pfizer. The increase in revenue of $67.8 million was primarily due to revenue from the Novartis license agreement of $76.7 million, offset by a decrease in revenue from the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer of $7.6 million related to timing differences in clinical trials and program expenses and a decrease in revenue from Bayer of $1.1 million related to the termination of the Bayer Collaboration Agreement in August 2024.

Investor Call & Webcast Details

Arvinas will host a conference call and webcast today, October 30, 2024, at 8:00 a.m. ET to review its third quarter 2024 financial results and discuss recent corporate updates. Participants are invited to listen by going to the Events and Presentation section under the Investors page on the Arvinas website at www.arvinas.com. A replay of the webcast will be available on the Arvinas website following the completion of the event and will be archived for up to 30 days.

About Vepdegestrant
Vepdegestrant is an investigational, orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with ER positive (ER+)/human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) breast cancer. Vepdegestrant is being developed as a potential monotherapy and as part of combination therapy across multiple treatment settings for ER+/HER2- metastatic breast cancer.

In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will share worldwide development costs, commercialization expenses, and profits.

The U.S. Food and Drug Administration (FDA) has granted vepdegestrant Fast Track designation as a monotherapy in the treatment of adults with ER+/HER2- locally advanced or metastatic breast cancer previously treated with endocrine-based therapy.

On October 30, 2024 Amgen (NASDAQ:AMGN) reported financial results for the third quarter of 2024 (Press release, Amgen, OCT 30, 2024, View Source [SID1234647532]).

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"Strong growth in sales and earnings this quarter reflects the momentum we’re building throughout our business. We continue to invest heavily in our rapidly advancing pipeline, with a focus on delivering innovative therapies across our core therapeutic areas," said Robert A. Bradway, chairman and chief executive officer.

Key results include:

For the third quarter, total revenues increased 23% to $8.5 billion in comparison to the third quarter of 2023.
Product sales grew 24%, driven by 29% volume growth, partially offset by 2% lower net selling price. Excluding sales from our Horizon Therapeutics (Horizon) acquisition, product sales grew 8%, driven by volume growth of 12%.
Ten products delivered at least double-digit sales growth in the third quarter, including Repatha (evolocumab), TEZSPIRE (tezepelumab-ekko), BLINCYTO (blinatumomab), EVENITY (romosozumab-aqqg), and TAVNEOS (avacopan).
Our performance included $1.2 billion of sales from our rare disease products, driven by several first-in-class, early-in-lifecycle medicines, including TEPEZZA (teprotumumab-trbw), KRYSTEXXA (pegloticase), UPLIZNA (inebilizumab-cdon), and TAVNEOS (avacopan).
GAAP earnings per share (EPS) increased 62% from $3.22 to $5.22, driven by mark-to-market gains on our BeiGene, Ltd. (BeiGene) equity investment and higher revenues, partially offset by higher operating expenses, including amortization expense from Horizon-acquired assets and incremental expenses from Horizon.
GAAP operating income remained relatively unchanged at $2.0 billion, and GAAP operating margin decreased 5.8 percentage points to 25.1%.
Non-GAAP EPS increased 13% from $4.96 to $5.58, driven by higher revenues, partially offset by higher operating expenses, including incremental expenses from Horizon, and interest expense.
Non-GAAP operating income increased from $3.4 billion to $4.0 billion, and non-GAAP operating margin decreased 2.4 percentage points to 49.6%.
The Company generated $3.3 billion of free cash flow in the third quarter of 2024 versus $2.5 billion in the third quarter of 2023, driven by business performance and timing of working capital items, partially offset by lower interest income.
References in this release to "non-GAAP" measures, measures presented "on a non-GAAP basis" and "free cash flow" (computed by subtracting capital expenditures from operating cash flow) refer to non-GAAP financial measures. Adjustments to the most directly comparable GAAP financial measures and other items are presented on the attached reconciliations. Refer to Non-GAAP Financial Measures below for further discussion.

Product Sales Performance

General Medicine

Repatha (evolocumab) sales increased 40% year-over-year to $567 million in the third quarter, driven by 41% volume growth and 8% favorable changes to estimated sales deductions, partially offset by 10% lower net selling price.
EVENITY (romosozumab-aqqg) sales increased 30% year-over-year to $399 million in the third quarter, driven by volume growth.
Prolia (denosumab) sales increased 6% year-over-year to $1.0 billion in the third quarter, driven by 9% volume growth, partially offset by lower inventory levels.
Oncology

BLINCYTO (blinatumomab) sales increased 49% year-over-year to $327 million in the third quarter, primarily driven by volume growth.
Vectibix (panitumumab) sales increased 12% year-over-year to $282 million in the third quarter, primarily driven by volume growth.
KYPROLIS (carfilzomib) sales increased 8% year-over-year to $378 million in the third quarter, primarily driven by volume growth outside the U.S.
LUMAKRAS/LUMYKRAS (sotorasib) sales increased 88% year-over-year to $98 million in the third quarter, driven by volume growth and favorable changes to estimated sales deductions.
XGEVA (denosumab) sales increased 4% year-over-year to $541 million in the third quarter, driven by higher net selling price.
Nplate (romiplostim) sales increased 9% year-over-year to $456 million in the third quarter. U.S. government orders were $128 million in Q3’24 compared to $142 million in Q3’23. Excluding these U.S. government orders, Nplate sales grew 18% year-over-year in the third quarter, driven by 14% volume growth and higher net selling price.
IMDELLTRA (tarlatamab-dlle) generated $36 million of sales in the third quarter. Sales increased 200% quarter-over-quarter, driven by volume growth. IMDELLTRA is the first and only FDA-approved bispecific T-cell engager (BiTE) therapy for the treatment of extensive-stage small cell lung cancer (ES-SCLC).
MVASI (bevacizumab-awwb) sales decreased 8% year-over-year to $195 million in the third quarter. Going forward, we expect continued sales erosion driven by competition.
Inflammation

TEZSPIRE (tezepelumab-ekko) sales increased 67% year-over-year to $269 million in the third quarter, driven by volume growth.
Otezla (apremilast) sales decreased 1% year-over-year to $564 million in the third quarter, primarily driven by 7% lower net selling price, partially offset by 5% volume growth.
Enbrel (etanercept) sales decreased 20% year-over-year to $825 million in the third quarter, primarily driven by 13% unfavorable changes to estimated sales deductions and 12% lower net selling price. Going forward, we expect continued declining net selling price and relatively flat volumes.
AMJEVITA/AMGEVITA (adalimumab) sales increased 9% year-over-year to $166 million in the third quarter.
Rare Disease

Except for TAVNEOS, the products listed below were added through the acquisition of Horizon on Oct. 6, 2023.

TEPEZZA (teprotumumab-trbw) generated $488 million of sales in the third quarter. TEPEZZA is the first and only FDA-approved treatment for thyroid eye disease (TED).
KRYSTEXXA (pegloticase) generated $310 million of sales in the third quarter. KRYSTEXXA is the first and only FDA-approved treatment for chronic refractory gout.
UPLIZNA (inebilizumab-cdon) generated $106 million of sales in the third quarter. UPLIZNA is used to treat adults with neuromyelitis optica spectrum disorder.
TAVNEOS (avacopan) generated $80 million of sales in the third quarter. Sales increased 116% year-over-year, primarily driven by volume growth. TAVNEOS is a first-in-class treatment for severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis (ANCA-associated vasculitis).
Ultra-Rare products, which consist of RAVICTI (glycerol phenylbutyrate), PROCYSBI (cysteamine bitartrate), ACTIMMUNE (interferon gamma-1b), QUINSAIR (levofloxacin) and BUPHENYL (sodium phenylbutyrate), generated $188 million of sales in the third quarter.
Established Products

Our established products, which consist of EPOGEN (epoetin alfa), Aranesp (darbepoetin alfa), Parsabiv (etelcalcetide) and Neulasta (pegfilgrastim), generated $550 million of sales. Sales decreased 7% year-over-year for the third quarter, driven by volume declines. In the aggregate, we expect the year-over-year volume declines for this portfolio of products to continue.
Product Sales Detail by Product and Geographic Region

$Millions, except percentages

Q3 ’24

Q3 ’23

YOY Δ

U.S.

ROW

TOTAL

TOTAL

TOTAL

Repatha

$ 281

$ 286

$ 567

$ 406

40 %

EVENITY

289

110

399

307

30 %

Prolia

683

362

1,045

986

6 %

BLINCYTO

237

90

327

220

49 %

Vectibix

132

150

282

252

12 %

KYPROLIS

238

140

378

349

8 %

LUMAKRAS/LUMYKRAS

53

45

98

52

88 %

XGEVA

373

168

541

519

4 %

Nplate

345

111

456

419

9 %

IMDELLTRA

36

—

36

—

N/A

MVASI

136

59

195

213

(8 %)

TEZSPIRE

269

—

269

161

67 %

Otezla

460

104

564

567

(1 %)

Enbrel

817

8

825

1,035

(20 %)

AMJEVITA/AMGEVITA

28

138

166

152

9 %

TEPEZZA(1)

482

6

488

—

N/A

KRYSTEXXA(1)

310

—

310

—

N/A

UPLIZNA(1)

74

32

106

—

N/A

TAVNEOS

74

6

80

37

*

Ultra-Rare products(1)

180

8

188

—

N/A

EPOGEN

33

—

33

50

(34 %)

Aranesp

105

232

337

323

4 %

Parsabiv

32

38

70

95

(26 %)

Neulasta

84

26

110

124

(11 %)

Other products(2)

228

53

281

281

— %

Total product sales

$ 5,979

$ 2,172

$ 8,151

$ 6,548

24 %

N/A = not applicable

*Change in excess of 100%

(1) Horizon-acquired products, and the Ultra-Rare products consist of RAVICTI, PROCYSBI, ACTIMMUNE, QUINSAIR and BUPHENYL

(2) Consists of (i) Aimovig, RIABNI, KANJINTI, AVSOLA, NEUPOGEN, IMLYGIC, BEKEMV, Corlanor, WEZLANA/WEZENLA and Sensipar/Mimpara, where Biosimilars total $148 million in Q3 ’24 and $104 million in Q3 ’23; and (ii) Horizon-acquired products including RAYOS and PENNSAID

Operating Expense, Operating Margin and Tax Rate Analysis

On a GAAP basis for the third quarter:

Total Operating Expenses increased 32% year-over-year. Cost of Sales as a percentage of product sales increased 13.0 percentage points driven by higher amortization expense from Horizon acquisition-related assets and, to a lesser extent, higher royalties and profit share. Research & Development (R&D) expenses increased 34% driven by higher spend in later-stage clinical programs, marketed product support, and research and early pipeline, including Horizon-acquired programs. Selling, General & Administrative (SG&A) expenses increased 20% primarily driven by commercial expenses related to Horizon-acquired products. Other operating expenses consisted primarily of an impairment charge associated with an IPR&D asset and changes in the fair values of contingent consideration liabilities, both related to our Teneobio, Inc. acquisition from 2021.
Operating Margin as a percentage of product sales decreased 5.8 percentage points year-over-year to 25.1%.
Tax Rate decreased 2.4 percentage points year-over-year primarily due to the change in earnings mix as a result of the inclusion of the Horizon business, partially offset by quarter-to-date 2024 unrealized gains on our strategic equity investments, primarily BeiGene.
On a non-GAAP basis for the third quarter:

Total Operating Expenses increased 27% year-over-year. Cost of Sales as a percentage of product sales increased 0.4 percentage points driven by higher royalties and profit share, partially offset by lower manufacturing costs and changes in sales mix. R&D expenses increased 35% driven by higher spend in later-stage clinical programs, marketed product support, and research and early pipeline, including Horizon-acquired programs. SG&A expenses increased 21%, primarily driven by commercial expenses related to Horizon-acquired products.
Operating Margin as a percentage of product sales decreased 2.4 percentage points year-over-year to 49.6%.
Tax Rate decreased 2.7 percentage points year-over-year primarily due to the change in earnings mix as a result of the inclusion of the Horizon business.

$Millions, except percentages

GAAP

Non-GAAP

Q3 ’24

Q3 ’23

YOY Δ

Q3 ’24

Q3 ’23

YOY Δ

Cost of Sales

$ 3,310

$ 1,806

83 %

$ 1,454

$ 1,137

28 %

% of product sales

40.6 %

27.6 %

13.0 pts

17.8 %

17.4 %

0.4 pts

Research & Development

$ 1,450

$ 1,079

34 %

$ 1,440

$ 1,070

35 %

% of product sales

17.8 %

16.5 %

1.3 pts

17.7 %

16.3 %

1.4 pts

Selling, General & Administrative

$ 1,625

$ 1,353

20 %

$ 1,565

$ 1,293

21 %

% of product sales

19.9 %

20.7 %

(0.8) pts

19.2 %

19.7 %

(0.5) pts

Other

$ 71

$ 644

(89 %)

$ —

$ —

N/A

Total Operating Expenses

$ 6,456

$ 4,882

32 %

$ 4,459

$ 3,500

27 %

Operating Margin

operating income as % of product sales

25.1 %

30.9 %

(5.8) pts

49.6 %

52.0 %

(2.4) pts

Tax Rate

8.7 %

11.1 %

(2.4) pts

13.4 %

16.1 %

(2.7) pts

pts: percentage points

N/A = not applicable

Cash Flow and Balance Sheet

The Company generated $3.3 billion of free cash flow in the third quarter of 2024 versus $2.5 billion in the third quarter of 2023, driven by business performance and timing of working capital items, partially offset by lower interest income.
The Company’s third quarter 2024 dividend of $2.25 per share was declared on August 2, 2024, and was paid on September 6, 2024, to all stockholders of record as of August 16, 2024, representing a 6% increase from this same period in 2023.
During the third quarter, the Company reduced principal debt outstanding by $2.5 billion. Year-to-date, the Company has reduced principal debt outstanding by $4.5 billion.
Cash and investments totaled $9.0 billion and debt outstanding totaled $60.4 billion as of September 30, 2024.

$Billions, except shares

Q3 ’24

Q3 ’23

YOYΔ

Operating Cash Flow

$ 3.6

$ 2.8

$ 0.8

Capital Expenditures

$ 0.3

$ 0.2

$ 0.0

Free Cash Flow

$ 3.3

$ 2.5

$ 0.8

Dividends Paid

$ 1.2

$ 1.1

$ 0.1

Share Repurchases

$ 0.0

$ —

$ 0.0

Average Diluted Shares (millions)

542

538

4

Note: Numbers may not add due to rounding

$Billions

9/30/24

12/31/23

YTD Δ

Cash and Investments

$ 9.0

$ 10.9

$ (1.9)

Debt Outstanding

$ 60.4

$ 64.6

$ (4.2)

Note: Numbers may not add due to rounding

2024 Guidance

For the full year 2024, the Company now expects:

Total revenues in the range of $33.0 billion to $33.8 billion.
On a GAAP basis, EPS in the range of $8.71 to $9.56, and a tax rate in the range of 9.0% to 10.5%.
On a non-GAAP basis, EPS in the range of $19.20 to $20.00, and a tax rate in the range of 14.0% to 15.0%.
Capital expenditures to be approximately $1.3 billion.
Share repurchases not to exceed $500 million.
Third Quarter Product and Pipeline Update
The Company provided the following updates on selected product and pipeline programs:

General Medicine

MariTide (maridebart cafraglutide, AMG 133)

MariTide is a multispecific molecule that inhibits the gastric inhibitory polypeptide receptor (GIPR) and activates the glucagon like peptide 1 (GLP-1) receptor.
A Phase 2 study of MariTide is ongoing in adults who are living with overweight or obesity, with or without Type 2 diabetes mellitus. Topline data are anticipated in late 2024.
Planning for a broad Phase 3 program across multiple indications remains on track.
A Phase 2 study investigating MariTide was initiated for the treatment of Type 2 diabetes in patients with and without obesity.
AMG 513

A Phase 1 study of AMG 513 was initiated and is enrolling patients living with obesity.
Olpasiran (AMG 890)

Olpasiran is a potentially best-in-class small interfering ribonucleic acid (siRNA) molecule that reduces lipoprotein(a) (Lp(a)) synthesis in the liver.
The Ocean(a)-Outcomes trial, a Phase 3 cardiovascular outcomes study, is ongoing in patients with atherosclerotic cardiovascular disease and elevated Lp(a).
Repatha

EVOLVE-MI, a Phase 4 study of Repatha administered within 10 days of an acute myocardial infarction to reduce the risk of cardiovascular (CV) events, is ongoing.
VESALIUS-CV, a Phase 3 CV outcomes study of Repatha, is ongoing in patients at high CV risk without prior myocardial infarction or stroke.
In September data were presented from:
a sub-analysis of the FOURIER trial demonstrating that patients with obesity (BMI >35) are at an increased risk of CV events. Repatha treatment of patients with obesity resulted in a reduction in the composite endpoint of CV death, myocardial infarction, stroke, hospitalization for unstable angina or coronary revascularization at 3 years [HR 0.71 (0.59-0.86), and Absolute Risk Reduction = 5.65%]
the SLICE-CEA study, the first randomized trial of Repatha in patients with asymptomatic severe high-risk carotid artery stenosis. The study demonstrated that 6 months of Repatha treatment led to significant reductions in lipid rich necrotic core (LRNC) volume (p=0.017) and its components, intraplaque hemorrhage (IPH) (p=0.037) and lipid volume (p=0.023) in the carotid vessels, as assessed by MRI.
Oncology

IMDELLTRA

IMDELLTRA is a first-in-class delta-like ligand 3 (DLL3) targeting BiTE (bispecific T-cell engager) molecule.
The Company is advancing a comprehensive global clinical development program across extensive-stage and limited-stage small cell lung cancer (SCLC):
DeLLphi-304, a Phase 3 study comparing IMDELLTRA with standard of care chemotherapy in second-line extensive-stage small cell lung cancer (ES-SCLC), is ongoing.
DeLLphi-305, a Phase 3 study comparing IMDELLTRA and durvalumab with durvalumab alone, is enrolling patients with first-line ES-SCLC.
DeLLphi-306, a Phase 3 study comparing IMDELLTRA with placebo following concurrent chemoradiation therapy, is enrolling patients with limited-stage SCLC.
DeLLphi-308, a Phase 1b study evaluating subcutaneous tarlatamab, was initiated in patients with second line or later ES-SCLC.
DeLLphi-303, a Phase 1b study of IMDELLTRA in combination with a programmed cell death protein ligand-1 (PD-L1) inhibitor +/- carboplatin and etoposide, is ongoing in patients with first-line ES-SCLC.
DeLLphi-302, a Phase 1b study of IMDELLTRA in combination with AMG 404 in patients with second-line or later SCLC, is complete. AMG 404 is an anti-programmed cell death protein 1 (PD1) monoclonal antibody.
DeLLpro-300, a Phase 1b study of IMDELLTRA in patients with previously treated de novo or treatment-emergent neuroendocrine prostate cancer, is complete.
In September, data were presented from:
DeLLphi-301, a Phase 2 study of IMDELLTRA where extended follow-up data demonstrated sustained anti-cancer activity and a manageable safety profile in patients with ES-SCLC previously treated with platinum-based chemotherapy. Among 100 patients treated with the 10 mg dose level, the median duration of response was 9.7 months (95% CI, 6.9‒NE) and the median overall survival was 15.2 months. No new safety concerns were identified.
DeLLphi-303, a Phase 1b study of IMDELLTRA combined with a PD-L1 inhibitor as first-line ES-SCLC maintenance therapy. With a median follow-up of 10.0 months (range 1.4 – 20.4), IMDELLTRA in combination with a PD-L1 inhibitor, demonstrated a manageable safety profile with durable disease control [median duration of disease control: 9.3 months (95% CI: 5.6–not estimable)] and notable survival outcomes [median PFS 5.6 months (95% CI: 3.6–9.0), 9-month Kaplan-Meier estimate for OS of 88.9%].
BLINCYTO

Golden Gate, a Phase 3 study of BLINCYTO alternating with low-intensity chemotherapy, continues to enroll older adult patients with newly diagnosed Philadelphia chromosome (Ph)-negative B-cell precursor acute lymphoblastic leukemia (B-ALL).
A Phase 1/2 study of subcutaneous blinatumomab has completed enrollment in the dose-expansion and optimization phase in adult patients with relapsed or refractory Ph-negative B-ALL. The Company is planning to advance blinatumomab subcutaneous administration to a potentially registration-enabling Phase 2 portion of this study with initiation in H2 2025.
Xaluritamig (AMG 509)

Xaluritamig is a first-in-class bispecific T-cell engager targeting six-transmembrane epithelial antigen of prostate 1 (STEAP1).
The Company will initiate a Phase 3 study in post-taxane metastatic castrate resistant prostate cancer (mCRPC) in fourth quarter of 2024.
A Phase 1 monotherapy dose-expansion study of xaluritamig is ongoing in patients with mCRPC and has completed enrollment of patients in a reduced monitoring after treatment administration cohort. A fully outpatient treatment cohort continues to enroll patients to further improve administration convenience.
A Phase 1 combination of xaluritamig with enzalutamide or abiraterone continues to enroll patients with mCRPC in dose-escalation and dose-expansion respectively.
A Phase 1b study evaluating neoadjuvant xaluritamig therapy prior to radical prostatectomy was initiated in patients with newly diagnosed localized intermediate or high–risk prostate cancer.
A Phase 1b study was initiated and is now enrolling patients to evaluate xaluritamig in patients with high-risk nonmetastatic hormone-sensitive prostate cancer after definitive therapy.
In September results were presented from:
a Phase 1 dose exploration cohort evaluating xaluritamig monotherapy in patients with mCRPC where with a median follow-up of 27.9 months, the median OS was 17.7 months across all cohorts. An encouraging PSA90 rate (45.1%) was also observed in high-dose cohorts, and presence of PSA90 response was associated with survival (p = 0.0044), potentially serving as an early indicator for benefit in these patients.
a Phase 1 dose-expansion cohort evaluating xaluritamig monotherapy using multiple dosing regimens in patients with mCRPC demonstrated that the 1.5 mg Q2W target dosing regimen is the most favorable efficacy and safety profile and will be the recommended Phase 3 dose and schedule.
AMG 193

AMG 193 is a first-in-class small molecule methylthioadenosine (MTA)-cooperative protein arginine methyltransferase 5 (PRMT5) inhibitor.
A Phase 1/1b/2 study of AMG 193 continues to enroll patients with advanced methylthioadenosine phosphorylase (MTAP)-null solid tumors in the dose-expansion portion of the study.
A Phase 1b study of AMG 193 alone or in combination with other therapies is enrolling patients with advanced MTAP-null thoracic malignancies.
A Phase 1b study of AMG 193 in combination with other therapies in advanced MTAP-null gastrointestinal, biliary tract, and pancreatic cancers is enrolling patients.
A Phase 1/2 study of AMG 193 in combination with IDE397, an investigational methionine adenosyltransferase 2A (MAT2A) inhibitor, continues to enroll patients with advanced MTAP-null solid tumors.
A Phase 2 study evaluating the efficacy, safety, tolerability, and pharmacokinetics of AMG 193 was initiated in patients with MTAP-null previously treated advanced non-small cell lung cancer (NSCLC).
In September, data were presented from a Phase 1 dose-escalation and initial dose-expansion study of AMG 193 in patients with MTAP-null solid tumors. The data demonstrated monotherapy activity and an acceptable safety profile. These data open opportunities to explore both monotherapy and combination therapy development strategies.
Bemarituzumab

Bemarituzumab is a first-in-class fibroblast growth factor receptor 2b (FGFR2b) targeting monoclonal antibody.
FORTITUDE-101, a Phase 3 study of bemarituzumab plus chemotherapy, is ongoing in patients with first-line gastric cancer.
FORTITUDE-102, a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab in patients with first-line gastric cancer, has completed enrollment of the Phase 3 portion of the study.
FORTITUDE-103, a Phase 1b/2 study of bemarituzumab plus oral chemotherapy regimens with or without nivolumab, continues to enroll patients in first-line gastric cancer.
FORTITUDE-301, a Phase 1b/2 basket study of bemarituzumab monotherapy, is ongoing in patients with solid tumors with FGFR2b overexpression.
Nplate

The primary analysis of a Phase 3 study of Nplate as supportive care in chemotherapy-induced thrombocytopenia in gastrointestinal malignancies is complete. The Company continues to follow patients through a planned final analysis in H1 2025. Data presentation at a medical congress is anticipated in mid-2025.
LUMAKRAS/LUMYKRAS

CodeBreaK 202, a Phase 3 study of LUMAKRAS plus chemotherapy vs. pembrolizumab plus chemotherapy, is enrolling patients with first-line KRAS G12C–mutated and programmed PD-L1 negative advanced NSCLC.
Regulatory review by the European Medicines Agency (EMA) of the CodeBreaK 200 Phase 3 study of adults with previously treated locally advanced or metastatic KRAS G12C–mutated NSCLC along with data from the Phase 2 dose-comparison substudy is ongoing.
The U.S Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) date for the Phase 3 CodeBreaK 300 study of LUMAKRAS plus Vectibix vs. investigator’s choice of therapy in KRAS G12C–mutated metastatic colorectal cancer (CRC) from October 17, 2024, to January 17, 2025, to allow time for review of recently submitted supplemental data.
CodeBreaK 301, a Phase 3 study of LUMAKRAS in combination with Vectibix and FOLFIRI, is enrolling patients with first-line KRAS G12C–mutated CRC.
Inflammation

TEZSPIRE

The Company is planning to initiate Phase 3 studies in patients with moderate to very severe chronic obstructive pulmonary disease (COPD) and a BEC ≥ 150 cells/µl or greater. Study initiation is anticipated in H1 2025.
A Phase 3 study of TEZSPIRE is ongoing in patients with chronic rhinosinusitis with nasal polyps. Data readout is anticipated in H2 2024.
A Phase 3 study of TEZSPIRE continues to enroll patients with eosinophilic esophagitis.
In severe asthma, the WAYFINDER Phase 3b study and the PASSAGE Phase 4 real-world effectiveness study are fully enrolled. The SUNRISE Phase 3 study continues to enroll patients.
Rocatinlimab (AMG 451/KHK4083)

Rocatinlimab is a first-in-class T-cell rebalancing monoclonal antibody targeting the OX40 receptor.
The eight study ROCKET Phase 3 program continues to enroll patients with moderate-to-severe atopic dermatitis (AD). To date, over 3,200 patients have been enrolled in the ROCKET program, with six studies having completed enrollment.
In September, the Company reported results from the Phase 3 HORIZON study (part of the ROCKET program), evaluating rocatinlimab monotherapy vs. placebo in adults with moderate-to-severe AD. The study met its co-primary endpoints and all key secondary endpoints, achieving statistical significance versus placebo at week 24.
32.8% of patients in the rocatinlimab group achieved ≥ 75% reduction from baseline in Eczema Area and Severity Index score (EASI-75), compared to 13.7% placebo treated (19.1% difference, p<0.001).
19.3% of patients in the rocatinlimab group achieved a Validated Investigator’s Global Assessment for Atopic Dermatitis (vIGA-ADTM) score of 0 (clear) or 1 (almost clear) with a ≥ 2-point reduction from baseline, compared to 6.6% in placebo (12.8% difference, p<0.001).
In the U.S., a more stringent scoring method was used (revised Investigator’s Global Assessment, or rIGA) where 16.4% of patients in the rocatinlimab group achieved a score of 0 (clear) or 1 (almost clear) with a ≥ 2-point reduction from baseline, compared to 4.9% in placebo (11.5% difference, p<0.001).
Safety findings were consistent with those seen in the Phase 2b AD study.
Additional key data readouts from the ROCKET Phase 3 program are expected in late 2024 through H2 2025:
ROCKET SHUTTLE is a 24-week study evaluating rocatinlimab in combination with topical corticosteroids and/or topical calcineurin inhibitors in adult patients with moderate-to-severe AD. Data readout is anticipated in late 2024 to early 2025.
ROCKET IGNITE is a 24-week study evaluating rocatinlimab monotherapy in adult patients with moderate-to-severe AD. Data readout is anticipated in late 2024 to early 2025.
ROCKET ASCEND is a study evaluating rocatinlimab maintenance therapy in adult and adolescent patients with moderate-to-severe AD. Data readout is anticipated in H2 2025.
ROCKET ASTRO is a 52-week study evaluating rocatinlimab in adolescent patients with moderate-to-severe AD. Data readout is anticipated in H2 2025.
A Phase 2 study of rocatinlimab is enrolling patients with moderate-to-severe asthma.
A Phase 3 study of rocatinlimab is enrolling patients with prurigo nodularis.
Otezla

In September data were presented from:
a Phase 3 study of Japanese patients with Palmoplantar Pustulosis (PPP) demonstrating maintained or increased efficacy (compared with the week 16 primary analysis) and no new safety signals with 52 weeks of Otezla treatment.
the DISCREET moderate-to-severe genital psoriasis study demonstrating that improvements in disease severity, symptoms, and quality of life with Otezla treatment were maintained at week 32. No new safety signals were observed.
the FOREMOST study of patients with oligoarticular psoriatic arthritis, where Otezla demonstrated sustained benefits in skin, joint, fatigue, and pain symptoms through 48-weeks.
Blinatumomab

Blinatumomab is a bispecific T-cell engager (BiTE) molecule targeting CD19.
A Phase 2 study of blinatumomab was initiated in patients with autoimmune disease with an initial focus on systemic lupus erythematosus (SLE) with nephritis.
Inebilizumab

Inebilizumab is a monoclonal antibody targeting CD19.
A Phase 2 study of inebilizumab was initiated in patients with autoimmune disease with an initial focus on SLE with nephritis.
Efavaleukin alfa (AMG 592)

Efavaleukin alfa is an interleukin 2 (IL 2) mutein Fc fusion protein.
A Phase 2b study of efavaleukin alfa in patients with ulcerative colitis was terminated due to the study meeting a prespecified futility threshold, and not related to safety concerns.
Ordesekimab (AMG 714/PRV-015)

Ordesekimab is a monoclonal antibody that binds interleukin-15.
A Phase 2b study of ordesekimab is ongoing in patients with nonresponsive celiac disease.
AMG 104 (AZD8630)

AMG 104 is an inhaled anti-thymic stromal lymphopoietin (TSLP) fragment antigen-binding (Fab).
A Phase 2 study was initiated in patients with asthma.
Rare Disease

TAVNEOS

A Phase 3, open-label study of TAVNEOS in combination with Rituximab or a cyclophosphamide-containing regimen, is enrolling patients from 6 years to < 18 years of age with active ANCA-associated vasculitis (Granulomatosis with Polyangiitis (GPA) / Microscopic Polyangiitis (MPA)).
TEPEZZA

In September, TEPEZZA was approved for the treatment of active Thyroid Eye Disease (TED) by Japan’s Ministry of Health, Labour and Welfare (MHLW). Regulatory review in multiple additional geographies continues.
A Phase 3 study of TEPEZZA in Japan continues to enroll patients with chronic or low clinical activity score TED.
A Phase 3 study evaluating the subcutaneous route of administration of teprotumumab is enrolling patients with TED.
KRYSTEXXA

Data from the AGILE study evaluating the safety, tolerability, and efficacy of KRYSTEXXA administered with a shorter infusion duration in patients with uncontrolled gout receiving methotrexate as co-administration will be presented at the American College of Rheumatology Convergence (ACR) in November.
UPLIZNA

The Company recently presented results of the Phase 3 MINT clinical study evaluating the efficacy and safety of UPLIZNA for the treatment of generalized myasthenia gravis (gMG).
UPLIZNA demonstrated a clinically meaningful and statistically significant Myasthenia Gravis Activities of Daily Living (MG-ADL) score improvement after two doses compared to placebo at Week 26: -4.2 overall improvement, -1.9 placebo adjusted (p < 0.0001, primary endpoint).
In the acetylcholine receptor autoantibody-positive (AChR+) population, UPLIZNA demonstrated a clinically meaningful and statistically significant MG-ADL score improvement compared to placebo at Week 26: -4.2 overall improvement, -1.8 placebo adjusted (p = 0.0015, secondary endpoint).
In the muscle-specific kinase autoantibody-positive (MuSK+) population, UPLIZNA demonstrated a clinically meaningful and statistically significant MG-ADL score improvement compared to placebo at Week 26: -3.9 overall improvement, -2.2 placebo adjusted (p = 0.0297, secondary endpoint).
UPLIZNA demonstrated a statistically significant Quantitative Myasthenia Gravis (QMG) score improvement after two doses compared to placebo at week 26: -4.8 overall improvement, -2.5 placebo adjusted (p = 0.0002, secondary endpoint).
In the AChR+ population, UPLIZNA demonstrated a clinically meaningful and statistically significant QMG score improvement compared to placebo at week 26: -4.4 overall improvement, -2.5 placebo adjusted (p = 0.0011, secondary endpoint).
In the MuSK+ population, the mean change from baseline in QMG score at Week 26 showed a trend favoring UPLIZNA but was not statistically significant (-5.2 for UPLIZNA and -3.0 for placebo, difference -2.3, p=0.1326).
Patients who entered the study taking corticosteroids were tapered down starting at Week 4 to prednisone 5 mg per day by Week 24.
The overall safety results during the placebo-controlled period of the study were consistent with the known safety profile of UPLIZNA.
52-week data from the AChR+ cohort and from AChR+ and MuSK+ patients in the open-label period of the study will be presented at a future date.
Planning for regulatory submissions for gMG is underway.
In August, the FDA granted Breakthrough Therapy Designation for UPLIZNA in the treatment of Immunoglobulin G4-related diseases (IgG4-RD) based upon data from the Phase 3 MITIGATE study. Regulatory filing activities for IgG4-RD are underway.
MITIGATE results will be presented at ACR in November.
Dazodalibep

Dazodalibep is a fusion protein that inhibits CD40L.
Two Phase 3 studies of dazodalibep in Sjögren’s disease are enrolling patients. The first study is in patients with moderate-to-severe systemic disease activity, and the second study is in patients with moderate-to-severe symptomatic burden and low systemic disease activity.
Daxdilimab

Daxdilimab is a fully human monoclonal antibody targeting immunoglobulin-like transcript 7 (ILT7).
A Phase 2 study of daxdilimab is ongoing in patients with moderate-to-severe active primary discoid lupus erythematosus refractory to standard of care.
A Phase 2 study of daxdilimab is ongoing in patients with dermatomyositis and antisynthetase inflammatory myositis.
Fipaxalparant

Fipaxalparant is a lysophosphatidic acid receptor 1 (LPAR1) antagonist.
A Phase 2 study of fipaxalparant in patients with idiopathic pulmonary fibrosis is complete. The study did not meet any of the primary or secondary endpoints. Development of fipaxalparant in this indication will be discontinued.
A Phase 2 study of fipaxalparant has completed enrollment of patients with diffuse cutaneous systemic sclerosis.
Biosimilars

In August, the FDA approved PAVBLUTM (aflibercept-ayyh) (ABP 938), a biosimilar candidate to EYLEA (aflibercept), for the treatment of retinal conditions, including neovascular age-related macular degeneration (wet AMD), macular edema following retinal vein occlusion, diabetic macular edema, and diabetic retinopathy.
A randomized, double-blind pharmacokinetic similarity study of ABP 206 compared with OPDIVO (nivolumab) is enrolling patients with resected stage III or stage IV melanoma in the adjuvant setting.
A randomized, double-blind comparative clinical study of ABP 206 compared with OPDIVO is enrolling patients with treatment-naïve unresectable or metastatic melanoma.
A randomized, double-blind combined pharmacokinetic/comparative clinical study of ABP 234 compared to KEYTRUDA (pembrolizumab) is enrolling patients with advanced or metastatic non-squamous non-small cell lung cancer.
TEZSPIRE is being developed in collaboration with AstraZeneca.
AMG 104 is being developed in collaboration with AstraZeneca.
Rocatinlimab, formerly AMG 451/KHK4083, is being developed in collaboration with Kyowa Kirin.
Ordesekimab, formerly AMG 714 and also known as PRV-015, is being developed in collaboration with Provention Bio, a Sanofi Company. For the purposes of the collaboration, Provention Bio conducts a clinical trial and leads certain development and regulatory activities for the program.
Xaluritamig, formerly AMG 509, is being developed pursuant to a research collaboration with Xencor, Inc.
IDE397 is an investigational MAT2A inhibitor from IDEAYA Biosciences.
EYLEA is a registered trademark of Regeneron Pharmaceuticals, Inc.
OPDIVO is a registered trademark of Bristol-Myers Squibb Company.
KEYTRUDA is a registered trademark of Merck & Co., Inc.

On October 30, 2024 Alterome Therapeutics, Inc., a clinical stage biopharmaceutical company pioneering the development of next generation, small molecule targeted therapies for the treatment of cancer, reported the dosing of the first patient in AKTive-001, a Phase 1/1b trial of ALTA2618 in adults with AKT1 E17K-mutant advanced solid tumors (Press release, Alterome Therapeutics, OCT 30, 2024, View Source [SID1234647531]).

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ALTA2618 is the first mutant-selective allosteric inhibitor of AKT1 E17K and is designed to avoid on-target toxicities associated with inhibition of AKT family kinases. AKT1 E17K is a clinically validated oncogene that drives ~5% of breast cancer, particularly hormone positive disease, and 1-2% of all cancer, including endometrial (~3%) and prostate (~2%) cancers.

"Dosing the first patient in the AKTive-001 trial marks a significant milestone for Alterome as we advance the first program from our mutation-selective and isoform-selective discovery pipeline to the clinic," said Eric Murphy, Ph.D., co-founder and CEO of Alterome Therapeutics. "ALTA2618 was designed to selectively target the AKT1 E17K mutation by pioneering a novel covalent approach to capture the lysine mutation. From concept to clinic, ALTA2618 illustrates the relentless execution of our team and dedication to developing novel mutation-selective therapies that will improve outcomes for patients with cancer."

AKTive-001 is a Phase 1/1b, open-label, dose escalation and multiple expansion cohort study evaluating ALTA2618 as an oral monotherapy. The study will evaluate the safety, tolerability, pharmacokinetics, and preliminary clinical activity of ALTA2618 in patients aged 18 years or above with AKT1 E17K-mutated advanced unresectable or metastatic solid tumors (NCT06533059). Additional clinical sites across Europe, Asia and Australia will initiate enrollment over the next year.