AMGEN REPORTS THIRD QUARTER FINANCIAL RESULTS

On October 31, 2023 Amgen (NASDAQ:AMGN) reported financial results for the third quarter of 2023 (Press release, Amgen, OCT 31, 2023, View Source [SID1234636513]).

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Key results include:

Total revenues increased 4% to $6.9 billion in comparison to the third quarter of 2022, resulting from a 5% increase in product sales. Product sales growth was driven by 11% volume growth, partially offset by 3% lower net selling price and 3% unfavorable changes to estimated sales deductions.
Volume growth of 11% included double-digit volume growth from BLINCYTO (blinatumomab), EVENITY (romosozumab-aqqg), Repatha (evolocumab) and Nplate (romiplostim).
U.S. volume grew 11% and ex-U.S. volume grew 12%, including 27% volume growth in the Asia Pacific region.
GAAP earnings per share (EPS) decreased 19% from $3.98 to $3.22, driven by a net impairment charge in Q3 2023 of approximately $650 million following a decision to discontinue development of AMG 340, partially offset by increased revenues.
GAAP operating income decreased from $2.7 billion to $2.0 billion, and GAAP operating margin decreased 11.7 percentage points to 30.9%.
Non-GAAP EPS increased 6% from $4.70 to $4.96, driven by increased revenues, partially offset by higher operating expenses.
Non-GAAP operating income increased from $3.3 billion to $3.4 billion, and non-GAAP operating margin decreased 0.5 percentage points to 52.0%.
The Company generated $2.5 billion of free cash flow for the third quarter of 2023 versus $2.8 billion in the third quarter of 2022.
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

Total product sales increased 5% for the third quarter of 2023 versus the third quarter of 2022. Unit volumes grew 11%, partially offset by 3% lower net selling price and 3% unfavorable changes to estimated sales deductions.

General Medicine

Repatha sales increased 31% year-over-year for the third quarter, driven by 44% volume growth, partially offset by lower net selling price. In the U.S., sales grew 29%, driven by 45% volume growth, partially offset by lower net selling price resulting from higher rebates to support and expand access for patients. Outside the U.S., sales grew 34%, driven by 43% volume growth, partially offset by lower net selling price. Repatha remains the global proprotein convertase subtilisin/kexin type 9 (PCSK9) segment leader, with over 2 million patients treated since launch.

Prolia (denosumab) sales increased 14% year-over-year for the third quarter, primarily driven by 7% volume growth and higher net selling price. We are on track to treat over 7 million patients with Prolia in 2023.

EVENITY sales increased 53% year-over-year to a record $307 million for the third quarter, driven by strong volume growth. U.S. volumes grew 41% year-over-year and volumes outside the U.S. grew 63%.

Aimovig (erenumab-aooe) sales decreased 12% year-over-year for the third quarter, driven by lower net selling price, partially offset by favorable changes to estimated sales deductions.
Inflammation

TEZSPIRE (tezepelumab-ekko) generated $161 million of sales in the third quarter. Quarter-over-quarter sales increased 21%, driven by 18% volume growth that benefited from the pre-filled, single-use pen, which was approved for self-administration by the U.S. Food and Drug Administration (FDA) in the first quarter. Healthcare providers are increasingly recognizing TEZSPIRE’s unique, differentiated profile and its broad potential to treat the 2.5 million patients worldwide with severe asthma who are uncontrolled, without any phenotypic or biomarker limitation.

TAVNEOS (avacopan) generated $37 million of sales in the third quarter. Quarter-over-quarter sales increased 23%, driven by volume growth. U.S. volumes grew 18% quarter-over-quarter. In the U.S., approximately 2,300 patients have now been treated with TAVNEOS.

Otezla (apremilast) sales decreased 10% year-over-year for the third quarter, driven by lower net selling price, unfavorable changes to estimated sales deductions and lower inventory levels, partially offset by 1% volume growth. In the U.S., net selling price declined, driven by higher rebates to support and expand access for commercial and Medicare Part D patients. Otezla demand in the quarter continued to be impacted by free drug programs for newly launched competition. For the remainder of 2023, we expect demand to be affected by these free drug programs.

We expect future growth for Otezla to be driven by its established efficacy and safety profile, strong payer coverage with limited prior authorization requirements and ease of administration. Otezla remains the only approved oral systemic therapy with a broad indication and is well-positioned to help the 1.5 million U.S. patients with mild-to-moderate psoriasis who cannot be optimally addressed by a topical and can benefit from a systemic treatment like Otezla.

Enbrel (etanercept) sales decreased 6% year-over-year for the third quarter, primarily driven by an 8% decline from unfavorable changes to estimated sales deductions, resulting from a $47 million favorable adjustment in the third quarter of 2022 compared to a $37 million unfavorable adjustment in this quarter. Year-over-year volume increased 1% in the third quarter, driven by an increase in new patients starting treatment as a result of improved payer coverage. For the remainder of 2023, we expect this improved coverage will lead to growth in new patients and declining net selling price.

AMJEVITA/AMGEVITA (adalimumab) sales increased 30% year-over-year for the third quarter, driven by 53% volume growth, partially offset by lower net selling price. Ex-U.S. sales increased 10% year-over-year, driven by 22% volume growth, partially offset by lower net selling price. U.S. sales increased 21% quarter-over-quarter, driven by 41% volume growth, partially offset by lower inventory levels.
Hematology-Oncology

BLINCYTO sales increased 55% year-over-year to a record $220 million for the third quarter, driven by 56% volume growth, supported by broad prescribing for patients with B-cell precursor acute lymphoblastic leukemia.

Vectibix (panitumumab) sales increased 2% year-over-year for the third quarter to a record $252 million, driven by higher net selling price and 4% volume growth, partially offset by unfavorable foreign exchange impact.

KYPROLIS (carfilzomib) sales increased 10% year-over-year for the third quarter, primarily driven by 8% volume growth.

LUMAKRAS/LUMYKRAS (sotorasib) generated $52 million of sales for the third quarter. Year-over-year sales decreased 31% for the third quarter, primarily driven by unfavorable changes to estimated sales deductions related to ongoing reimbursement negotiations in France.

XGEVA (denosumab) sales increased 5% year-over-year for the third quarter, driven by higher net selling price.

Nplate sales increased 45% year-over-year for the third quarter, driven by 43% volume growth resulting from a $142 million order from the U.S. government.

MVASI (bevacizumab-awwb) sales increased 2% year-over-year for the third quarter, driven by 17% volume growth and favorable changes to estimated sales deductions, partially offset by lower net selling price. The published third quarter Average Selling Price (ASP) for MVASI in the U.S. declined 19% year-over-year and 1% quarter-over-quarter. Going forward, we expect continued net selling price erosion driven by increased competition.

KANJINTI (trastuzumab-anns) sales decreased 72% year-over-year for the third quarter, driven by lower net selling price, unfavorable changes to estimated sales deductions and volume declines.
Established Products

Total sales of our established products, which include EPOGEN (epoetin alfa), Aranesp (darbepoetin alfa), Parsabiv (etelcalcetide) and Neulasta (pegfilgrastim), decreased 30% year-over-year for the third quarter, driven by lower net selling price and volume declines. In the aggregate, we expect the year-over-year net selling price and volume erosion for this portfolio of products to continue.
Product Sales Detail by Product and Geographic Region

$Millions, except percentages


Q3 ’23


Q3 ’22


YOY Δ


US


ROW


TOTAL


TOTAL


TOTAL

Repatha


$ 183


$ 223


$ 406


$ 309


31 %

Prolia


673


313


986


862


14 %

EVENITY


214


93


307


201


53 %

Aimovig


88


6


94


107


(12 %)

TEZSPIRE


161



161


55


*

TAVNEOS


32


5


37



NM

Otezla


462


105


567


627


(10 %)

Enbrel


1,026


9


1,035


1,106


(6 %)

AMJEVITA/AMGEVITA


23


129


152


117


30 %

BLINCYTO


147


73


220


142


55 %

Vectibix


116


136


252


247


2 %

KYPROLIS


231


118


349


318


10 %

LUMAKRAS/LUMYKRAS


48


4


52


75


(31 %)

XGEVA


374


145


519


495


5 %

Nplate


322


97


419


288


45 %

MVASI


140


73


213


209


2 %

KANJINTI


7


13


20


72


(72 %)

EPOGEN


50



50


136


(63 %)

Aranesp


107


216


323


358


(10 %)

Parsabiv


59


36


95


100


(5 %)

Neulasta


92


32


124


247


(50 %)

Other products**


136


31


167


166


1 %

Total product sales


$ 4,691


$ 1,857


$ 6,548


$ 6,237


5 %


*Change in excess of 100%


**Consists of AVSOLA, RIABNI, Corlanor, NEUPOGEN, IMLYGIC, Sensipar/Mimpara and BEKEMV, as well as sales in prior periods of our divested Bergamo and GENSENTA subsidiaries.

NM = not meaningful


Operating Expense, Operating Margin and Tax Rate Analysis

On a GAAP basis:

Total Operating Expenses increased 22%. Cost of Sales margin increased 2.1 percentage points, primarily driven by higher profit share, higher amortization expense from acquisition-related assets and changes in product mix. Research & Development (R&D) expenses decreased 3%, as lower spend in research and early pipeline was partially offset by higher spend in later-stage clinical programs and marketed products. Selling, General & Administrative (SG&A) expenses increased 5%, primarily driven by higher general and administrative expenses, including higher acquisition-related expenses. Other operating expenses consisted of a net impairment charge for AMG 340.
Operating Margin as a percentage of product sales decreased 11.7 percentage points to 30.9%.
Tax Rate increased 0.7 percentage points, primarily driven by the 2022 Puerto Rico tax law change that replaced the excise tax with an income tax beginning in 2023, partially offset by net favorable items and earnings mix, including the tax benefit from the net impairment charge for AMG 340.
On a non-GAAP basis:

Total Operating Expenses increased 4%. Cost of Sales margin increased 1.3 percentage points, primarily driven by higher profit share and changes in product mix. R&D expenses decreased 2%, as lower spend in research and early pipeline was partially offset by higher spend in later-stage clinical programs and marketed products. SG&A expenses increased 1%.
Operating Margin as a percentage of product sales decreased 0.5 percentage points in the third quarter to 52.0%.
Tax Rate increased 3.2 percentage points, primarily due to the 2022 Puerto Rico tax law change that replaced the excise tax with an income tax beginning in 2023 and earnings mix.

Cash Flow and Balance Sheet

The Company generated $2.5 billion of free cash flow in the third quarter of 2023 versus $2.8 billion in the third quarter of 2022.
The Company’s third quarter 2023 dividend of $2.13 per share was declared on August 1, 2023, and was paid on September 8, 2023, to all stockholders of record as of August 18, 2023, representing a 10% increase from 2022.
Cash and investments totaled $34.7 billion and debt outstanding totaled $60.5 billion as of September 30, 2023.

2023 Guidance

For the full year 2023, the Company now expects:

Total revenues in the range of $28.0 billion to $28.4 billion.
On a GAAP basis, EPS in the range of $11.23 to $12.73, and a tax rate in the range of 14.0% to 15.5%.
On a non-GAAP basis, EPS in the range of $18.20 to $18.80, and a tax rate in the range of 16.5% to 17.0%.
Capital expenditures to be approximately $950 million.
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:

Oncology
Tarlatamab (AMG 757)

In October, data were presented from the global Phase 2 DeLLphi-301 study and simultaneously published in the New England Journal of Medicine, evaluating tarlatamab, a first-in-class investigational delta-like ligand 3 (DLL3) targeting BiTE (bispecific T-cell engager) molecule, in patients with advanced-stage small cell lung cancer (SCLC) who had failed two or more prior lines of treatment. With a median follow-up of 10.6 months, an intention-to-treat analysis that included 100 patients at the selected 10 mg dose for tarlatamab demonstrated an objective response rate (ORR; primary endpoint) of 40% (97.5% confidence interval: 29-52). There were no new safety signals observed compared to the Phase 1 study.
Data from the DeLLphi-301 study are being submitted to the U.S. Food and Drug Administration (FDA) which recently granted Breakthrough Therapy Designation to tarlatamab for the treatment of adult patients with extensive-stage SCLC with disease progression on or after platinum-based chemotherapy.
DeLLphi-304, a Phase 3 study comparing tarlatamab with standard of care chemotherapy in second-line SCLC, is enrolling patients.
Two additional Phase 3 studies of tarlatamab in earlier lines of SCLC are planned.
DeLLphi-300, a Phase 1 study of tarlatamab in relapsed/refractory SCLC, continues to enroll patients.
DeLLphi-302, a Phase 1b study of tarlatamab in combination with AMG 404, an anti-programmed cell death protein 1 (PD1) monoclonal antibody, in second-line or later SCLC, is ongoing.
DeLLphi-303, a Phase 1b study of tarlatamab in combination with standard of care in first-line SCLC, continues to enroll patients.
DeLLpro-300, a Phase 1b study of tarlatamab in de novo or treatment-emergent neuroendocrine prostate cancer, has completed enrollment.
BLINCYTO

In September, based upon the E1910 study, the FDA granted Breakthrough Therapy Designation to BLINCYTO for the treatment of adult and pediatric patients with CD19-positive, Philadelphia chromosome-negative (Ph-), B-cell precursor acute lymphoblastic leukemia (B-ALL) during the consolidation phase of multiphase therapy. Global regulatory authority submissions are planned in late 2023 to early 2024 for the Phase 3 E1910 study conducted by the National Cancer Institute, the Eastern Cooperative Oncology Group and the American College of Radiology Imaging Network (ECOG ACRIN) Cancer Research Group.
In October, National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology1 (NCCN Guidelines) for Pediatric Acute Lymphoblastic Leukemia were updated to broaden the recommendation for BLINCYTO as consolidation to include both early and late first relapse patients with bone marrow relapse with or without extramedullary involvement and adding the COG1331 regimen to the list of recommended treatment regimens.
Golden Gate, a Phase 3 study of BLINCYTO alternating with low-intensity chemotherapy in older adults with newly diagnosed Ph- B-ALL, continues to enroll patients.
A Phase 1/2 study of subcutaneous BLINCYTO in adults with relapsed or refractory Ph- B-ALL continues to enroll patients.
Xaluritamig (AMG 509)

In October, initial data from a Phase 1b study were presented and simultaneously published in Cancer Discovery on xaluritamig, a first-in-class bispecific molecule targeting six-transmembrane epithelial antigen of prostate 1 (STEAP1) demonstrating encouraging anti-tumor activity in heavily pretreated patients with metastatic castrate-resistant prostate cancer (mCRPC). Efficacy was greater at higher doses (doses ≥0.75 mg target dose) where PSA50 was 59% (n=44) and RECIST objective response rate was 41% (n=37). The safety profile was clinically manageable, with CRS that was generally low grade and primarily in cycle 1.
A Phase 1b monotherapy and combination dose-escalation and expansion study of xaluritamig in mCRPC continues to enroll patients.
Two additional Phase 1 studies of xaluritamig to evaluate preliminary efficacy and safety in patients with early prostate cancer are planned.
AMG 193

Initial results from a Phase 1/1b study of AMG 193, a first-in-class small molecule methylthioadenosine (MTA)-cooperative protein arginine methyltransferase 5 (PRMT5) inhibitor, demonstrate promising monotherapy activity across six methylthioadenosine phosphorylase (MTAP)-null tumor types. Dose-limiting adverse events and treatment discontinuations were typically due to gastrointestinal events and were manageable and reversible.
This Phase 1/1b/2 study of AMG 193 continues to enroll patients with advanced MTAP-null solid tumors.
A Phase 1/2 study of AMG 193 in combination with IDE397, an investigational methionine adenosyltransferase 2A (MAT2A) inhibitor, is enrolling patients.
LUMAKRAS/LUMYKRAS

In October, data were presented and simultaneously published in the New England Journal of Medicine from the global Phase 3 CodeBreaK 300 trial evaluating two doses of LUMAKRAS (960 mg or 240 mg) in combination with Vectibix. Both doses demonstrated a statistically significant superiority in PFS over the investigator’s choice of standard therapy in patients with chemorefractory KRAS G12C-mutated metastatic colorectal cancer (CRC). No new safety signals were observed. Discussions continue with regulatory agencies on a potential approval pathway for this indication.
In September, LUMAKRAS was included in the colon cancer and the rectal cancer NCCN Guidelines1 (category 2A) and is recommended for treatment of previously treated metastatic colorectal or rectal cancer with KRAS G12C-mutated tumors in combination with cetuximab or panitumumab.
The Company is planning to initiate a Phase 3 study of LUMAKRAS in combination with Vectibix and FOLFIRI in first-line KRAS G12C-mutated CRC.
In September, data were presented from an arm of the CodeBreaK 101 clinical trial, a Phase 1b study evaluating LUMAKRAS with carboplatin and pemetrexed in adult patients with KRAS G12C-mutated advanced NSCLC. LUMAKRAS treatment resulted in encouraging objective response rates and disease control rates in first-line and second-line patients.
A global, randomized Phase 3 study of LUMAKRAS plus chemotherapy vs. pembrolizumab plus chemotherapy in first-line KRAS G12C-mutated and programmed cell death protein ligand-1 (PD-L1) negative advanced NSCLC was initiated.
Regulatory review by the FDA and the European Medicines Agency (EMA) of the CodeBreaK 200 Phase 3 trial of previously treated patients with KRAS G12C-mutated advanced NSCLC, along with data from the Phase 2 dose comparison substudy, is ongoing.
In November, data2 comparing sotorasib 960 mg versus 240 mg in adults with pretreated KRAS G12C-mutated advanced NSCLC will be presented at a European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Plenary session.
Bemarituzumab

FORTITUDE-101, a Phase 3 study of bemarituzumab, a first-in-class fibroblast growth factor receptor 2b (FGFR2b) targeting monoclonal antibody, plus chemotherapy in first-line gastric cancer, continues to enroll patients.
FORTITUDE-102, a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab in first-line gastric cancer, continues to enroll patients in the Phase 3 portion of the study.
FORTITUDE-103, a Phase 1b study of bemarituzumab plus oral chemotherapy regimens with or without nivolumab in first-line gastric cancer, continues to enroll patients.
FORTITUDE-301, a Phase 1b/2 basket study of bemarituzumab monotherapy in solid tumors with FGFR2b overexpression, is ongoing.
FORTITUDE-201, a Phase 1b study of bemarituzumab monotherapy and in combination with standard of care therapy, in squamous NSCLC with FGFR2b overexpression, will be discontinued.
AMG 340

A Phase 1 dose-escalation study of AMG 340, a lower T-cell affinity BiTE molecule targeting prostate-specific membrane antigen (PSMA), in mCRPC will be discontinued.
General Medicine
Maridebart cafraglutide (AMG 133)

A Phase 2 study of maridebart cafraglutide, a multispecific molecule that inhibits the gastric inhibitory polypeptide receptor (GIPR) and activates the glucagon like peptide 1 (GLP-1) receptor, in overweight or obese adults with or without type 2 diabetes mellitus has completed enrollment with topline data anticipated in late 2024.
AMG 786

A Phase 1 study of AMG 786, a small molecule obesity program, is ongoing with initial data readout anticipated in H1 2024. This molecule has a different target than AMG 133 and is not an incretin-based therapy.
Olpasiran (AMG 890)

A Phase 3 cardiovascular outcomes study of olpasiran, a potentially best-in-class small interfering ribonucleic acid (siRNA) molecule that reduces lipoprotein(a) (Lp(a)) synthesis in the liver, in participants with atherosclerotic cardiovascular disease and elevated Lp(a) continues to enroll patients.
In August, data were presented from the final analysis of the Phase 2 OCEAN(a)-DOSE study. The results from the off-treatment extension period (at least 24 weeks since the last dose) show that patients previously dosed every 12 weeks for up to 36 weeks with ≥ 75 mg of olpasiran sustained a ~ 40-50% placebo-adjusted percent reduction in Lp(a) nearly a year after the last dose. No new safety concerns were identified during the off-treatment extension period.
Repatha

New data from the FOURIER Open Label Extension (OLE) study reinforcing the safety and efficacy of Repatha, including results from the evaluation of long-term neurocognitive safety in atherosclerotic cardiovascular disease patients treated with Repatha, will be presented at the American Heart Association Scientific Sessions in November.
EVOLVE-MI, a Phase 4 study of Repatha administered shortly after an acute myocardial infarction and designed to reduce the risk of cardiovascular events in hospitalized acute coronary syndrome patients, continues to enroll patients.
A Phase 3 cardiovascular outcomes study (VESALIUS-CV) in patients at high cardiovascular risk without prior myocardial infarction or stroke is ongoing.
Inflammation
TEZSPIRE

In severe asthma, the WAYFINDER Phase 3b study is fully enrolled. The PASSAGE Phase 4 real-world effectiveness study and the SUNRISE Phase 3 study continue to enroll patients.
A Phase 3 study of TEZSPIRE in chronic rhinosinusitis with nasal polyps is fully enrolled.
A Phase 3 study of TEZSPIRE in eosinophilic esophagitis continues to enroll patients.
A Phase 2 study of TEZSPIRE in chronic obstructive pulmonary disease is fully enrolled. Data readout is anticipated in H1 2024.
A Phase 2b study of TEZSPIRE in chronic spontaneous urticaria did not meet the primary endpoint comparing tezepelumab with placebo in the overall and anti-IgE-naïve populations at week 16. By the end of the study at week 32 (18 weeks after last dose), a sustained treatment effect was observed in both tezepelumab dose groups in anti-IgE naïve patients. The Company and its partner AstraZeneca are determining next steps for this indication and plan to publish full results in the future.
Rocatinlimab (AMG 451/KHK4083)

In October, three posters of post-hoc analysis data from the Phase 2b study of rocatinlimab, a first-in-class anti-OX40 monoclonal antibody, in patients with moderate to severe atopic dermatitis were presented. These included patient reported outcomes data demonstrating the benefit of rocatinlimab.
The ROCKET Phase 3 program, composed of seven studies evaluating rocatinlimab in moderate to severe atopic dermatitis, continues to enroll adult and adolescent patients. To date, over 1,500 patients have been randomized into the ROCKET program.
A Phase 2 study of rocatinlimab in moderate to severe uncontrolled asthma is planned.
Otezla

In October, results were presented from the DISCREET Phase 3 study which demonstrated that Otezla treated patients across subgroups experienced greater improvement in genital psoriasis and genital itch at week 16 compared to placebo, with women achieving numerically greater responses.
A Phase 3 study evaluating the efficacy and safety of Otezla in Japanese patients with palmoplantar pustulosis successfully met the primary and secondary endpoints.
TAVNEOS

Data will be presented from the ADVOCATE study at the American College of Rheumatology and American Society of Nephrology Scientific sessions in November on the efficacy and safety of TAVNEOS in key sub-groups including patients 65 years old and older and patients with renal involvement.
Efavaleukin alfa (AMG 592)

A Phase 2b study of efavaleukin alfa, an interleukin 2 (IL 2) mutein Fc fusion protein, in ulcerative colitis continues to enroll patients.
Ordesekimab (AMG 714/PRV-015)

A Phase 2b study of AMG 714, a monoclonal antibody that binds interleukin-15, in nonresponsive celiac disease continues to enroll patients.
Biosimilars

The clinical comparative study portion of a randomized, double-blind pivotal study evaluating pharmacokinetic (PK) similarity of ABP 206 compared with OPDIVO (nivolumab) in resected stage III or stage IV melanoma patients in the adjuvant setting was initiated.
The FDA accepted the Biologics License Application for ABP 938, an investigational biosimilar to EYLEA (aflibercept).
1National Comprehensive Cancer Network (NCCN) makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.
2 Previously provided in the publicly available ODAC briefing book.
TEZSPIRE 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.
OPDIVO is a registered trademark of Bristol-Myers Squibb Company.
EYLEA is a registered trademark of Regeneron Pharmaceuticals, Inc.

Alligator Bioscience Announces Capital Markets Day 2023

On October 31, 2023 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that it will hold a Capital Markets Day on December 1, 2023 (Press release, Alligator Bioscience, OCT 31, 2023, View Source [SID1234636512]). The event will be hosted by members of Alligator’s executive management team who will provide an overview of the Company’s strategy, its prorietary and partnered assets, and its technology platforms.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Capital Markets Day Details

Date/Time: Friday, December 1, 2023 at 2:00 PM CET / 8:00 AM EST
Location: 7A Posthuset, Vasagatan 28, Stockholm, Sweden
Format: In-person event to be streamed live
To attend the event in person, contact IR & Communications Manager, Greta Eklund, at [email protected]. Please note that the number of seats is limited.

The event will be live-streamed to Alligator’s Youtube channel, accessible through this link.

Capital Markets Day Agenda

Overview of Alligator Bioscience: Chief Executive Officer Søren Bregenholt will present an overview of the Company’s strategic direction and its prorietary and partnered assets
Lead Asset Mitazalimab: Chief Medical Officer Dr. Sumeet Ambarkhane will provide an update on the clinical data generated so far by mitazalimab along with a broader view of the Company’s CD40 program
Key Opinon Leader: Dr. Gregory Beatty, Associate Professor of Medicine at the University of Pennsylvania, will explain the current treatment landscape for pancreatic cancer and the potential of mitazalimab to change the treatment paradigm

Alligator’s Technology Platforms: Chief Technology Officer Laura von Schantz will discuss Alligator’s proprietary technology platforms Neo-X-Prime and Ruby, including the pivotal role Ruby is playing in Alligator’s research collaboration and license agreement with Orion Corporation

ATOR-4066: Chief Scientific Officer Peter Ellmark will look at the progress Alligator is making with ATOR-4066, a third generation bispecific antibody targeting CD40 and CEACAM5, developed by the Company’s Neo-X-Prime platform

4-1BB: Chief Medical Officer Dr. Sumeet Ambarkhane will provide the latest updates on ATOR-1017, Alligator’s 4-1BB monoclonal antibody with best-in-class potential, and ALG.APV-527, a bispecific antibody with a tumor-directed 4-1BB agonistic effect being co-developed with Aptevo Therapeutics

Live question and answer sessions will be held after each presentation and CEO Søren Bregenholt will provide concluding remarks and present Alligator’s strategic outlook at the end of the event.

Akoya Biosciences to Showcase New Spatial Biology 2.0 Solutions and Spatial Phenotyping Data at SITC 2023

On October 31, 2023 Akoya Biosciences Inc., (Nasdaq: AKYA) The Spatial Biology Company, reported that it will be showcasing its latest Spatial Biology 2.0 solutions and ultrahigh-plex (100+ protein biomarkers) spatial phenotyping data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 38th Annual Meeting, being held in San Diego, California, November 1-5, 2023 (Press release, Akoya Biosciences, OCT 31, 2023, View Source [SID1234636511]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

At booth #719, Akoya Biosciences will highlight its new PhenoCycler-Fusion 2.0 and PhenoImager HT 2.0 platforms, which offer significant advancements in speed and scale for spatial biology research.

PhenoCycler-Fusion 2.0: Features multi-slide automation, enabling researchers to generate ultrahigh-plex spatial phenotyping data for larger and more complex samples at unprecedented speed.
PhenoImager HT 2.0: Features onboard spectral unmixing, which accelerates biomarker development and validation workflows.
"We continue to evolve our spatial biology platforms to enable even greater speed and scale, which is essential for our customers as they discover and validate new biomarkers," said Brian McKelligon, CEO of Akoya Biosciences. "We’re excited to be at SITC (Free SITC Whitepaper) where we will highlight these innovations and share an extensive array of new spatial phenotyping data. Many members of our global network of CRO service providers will also be at SITC (Free SITC Whitepaper). These organizations are a critical resource for biopharmaceutical companies as the industry continues to integrate spatial biology into their discovery and translational workflows."

Ultrahigh-plex Spatial Phenotyping Reveals Markers Associated with Response to Immunotherapy

Among the posters being presented at SITC (Free SITC Whitepaper) is one describing the spatial tumor microenvironment landscapes of cutaneous squamous cell carcinoma (cSCC) tissues from patients over the course of immunotherapy treatment. An ultrahigh-plex antibody panel encompassing cell lineages, activation states, immune checkpoints, structural and metabolic markers was deployed on the PhenoCycler-Fusion platform for analysis of the tissue microenvironment. Cellular phenotypes, spatial neighborhoods and functional states driving tumor pathogenesis and response to immunotherapy were identified. The poster will be presented by Arutha Kulasinghe, Ph.D., leader of the Clinical-oMx Group at the Frazer Institute of the University of Queensland, Australia, and founding scientific director of the Queensland Spatial Biology Centre.

Dr. Kulasinghe, his colleagues, and Akoya collaborators recently published a peer-reviewed article describing another ultrahigh-plex spatial biology study that revealed a high degree of intra-tumoral heterogeneity and distinct immune microenvironments in a tumor tissue section from a head and neck squamous cell carcinoma (HNSCC) patient with a partial response to treatment with an immune checkpoint inhibitor.

These studies reinforce the value of single-cell ultrahigh-plex spatial phenotyping as a powerful tool for defining spatial tissue signatures associated with therapy response and resistance.

Expanding Global Network of Contract Research Organizations (CROs) Providing Spatial Phenotyping Solutions

Fourteen of Akoya’s eighteen qualified CRO service providers will also be exhibiting at SITC (Free SITC Whitepaper). Akoya’s CRO network continues to grow rapidly, reflecting the demand for spatial phenotyping solutions across the biopharmaceutical industry. Offering biomarker testing services, these CROs enable drug developers and academic research institutions to accelerate the discovery and development of new immuno-therapies.

"We have seen great success leveraging Akoya’s ultrahigh-plex spatial biology technology to deliver better insights into the tumor microenvironment for our biopharma clients," said Christiaan Neelemen, President, Research & Clinical Services, Discovery Life Sciences, a member of Akoya’s CRO network providing end-to-end spatial immunophenotyping services to help define mechanisms of action, disease indications, and patient stratification. "With Akoya’s reliable multiplex technology, we can perform faster and higher quality analysis on a single tissue slide, helping us better understand complex disease mechanisms so our clients can develop more effective treatments."

"The demand for spatial phenotyping services and analysis continues to grow at an unprecedented pace," said Sunil Bodapati, Co-Founder and Chief Executive Officer of Enable Medicine, also a member of Akoya’s CRO network. "Akoya’s spatial biology platforms offer a combination of speed, throughput, and workflow efficiency. Our analysis platform, applied to high-plex datasets, allows us to rapidly deliver the spatial insights our clients need to advance their discovery and translational research and set the stage for the long-term success of their initiatives."

Details of the SITC (Free SITC Whitepaper) posters to be presented by Akoya and collaborators are as follows:

Title: The Mutational Landscape Defines the Proteome and Spatial Organization of Tumor, Stroma, and
Immune Cells in Ovarian Cancer
Session Date and Time: Friday, Nov. 3 from 9 a.m.–7 p.m.
Poster Number: 75

Title: Ultrahigh-plex Spatial Phenotyping of the Glioma Tumor Landscape in IDH-1wt and IDH-1R132H Patient Tissues
Session Date and Time: Friday, Nov. 3 from 9 a.m.–7 p.m.
Poster Number: 97

Title: Identifying a Role for Macrophages in Predicting Immunotherapy Responses of Non-Small Cell Lung Cancer (NSCLC)
Session Date and Time: Friday, Nov. 3 from 9 a.m.–7 p.m.
Poster Number: 105

Title: High Dimensional to High-throughput Spatial Phenotyping: An Integrated Pathway from Discovery to Translational and Clinical Studies
Session Date and Time: Friday, Nov. 3 from 9 a.m.–7 p.m.
Poster Number: 117

Title: Temporo-Spatial Monitoring of the Tumor Microenvironment in Cutaneous Squamous Cell
Carcinoma
Session Date and Time: Saturday, Nov. 4 from 9 a.m.–8:30 p.m.
Poster Number: 90

Title: Location, Location, Location: Spatial Analysis of Ultrahigh-Plex Immunofluorescence Panel in Head
and Neck Cancer
Session Date and Time: Saturday, Nov. 4 from 9 a.m.–8:30 p.m.
Poster Number: 124

Title: Successful Immune-Checkpoint Therapy Promotes Spatial Reorganization of the Lymphoid and
Myeloid Cellular Populations
Session Date and Time: Saturday, Nov. 4 from 9 a.m.–8:30 p.m.
Poster Number: 570

For more information about Akoya’s activities at SITC (Free SITC Whitepaper) 2023, please visit our website.

Adaptimmune Reports Positive Data with Lete-cel1 from an Interim Analysis of the Pivotal IGNYTE-ESO Trial for People with Synovial Sarcoma or MRCLS

On October 31, 2023 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported the outcome of a protocol-defined interim analysis of data from the pivotal IGNYTE-ESO trial with lete-cel for people with synovial sarcoma or myxoid/round cell liposarcoma (MRCLS) who received ≥ 2 prior lines of therapy (Press release, Adaptimmune, OCT 31, 2023, View Source [SID1234636508]).

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Additionally, Dr. Melissa Burgess of University of Pittsburgh Medical Center will present a poster summarizing results of Substudy 14 of the IGNYTE-ESO clinical trial exploring the feasibility, efficacy, and safety of lete-cel in the first-line setting for treatment-naïve patients with metastatic or unresectable synovial sarcoma or MRCLS on Thursday November 2nd at the Connective Tissue Oncology Society (CTOS) annual meeting taking place in Dublin, Ireland.

Elliot Norry, MD, Adaptimmune’s Chief Medical Officer: "The remarkable responses we are seeing in the interim analysis from IGNYTE-ESO provide us with a compelling opportunity to continue the clinical development of lete-cel for both synovial sarcoma and MRCLS. We’ll be able to leverage our learnings and capabilities from afami-cel as we consider next steps to further develop lete-cel."

Interim analysis data from the IGNYTE-ESO pivotal trial with lete-cel

Lete-cel, an engineered T-cell therapy targeted against NY-ESO-1, is being investigated for the treatment of synovial sarcoma or MRCLS in the pivotal IGNYTE-ESO (NCT03967223) trial in patients who received ≥ 2 prior lines of therapy.

The interim analysis for efficacy includes data from 45 people with synovial sarcoma or MRCLS who have received lete-cel in the IGNYTE-ESO trial and who had at least 6 months follow up. At the time of this analysis, 18/45 (40%) (99.6% CI: 20.3%, 62.3%) people with synovial sarcoma or MRCLS had RECISTv1.1 responses by independent review with two complete responses and 16 partial responses. The response rate was 9/23 (39%) for people with synovial sarcoma and 9/22 (41%) for people with MRCLS by independent review.

Duration of Response (DoR) is still being followed in 9/18 (50%) of responders at the time of the data cut-off. The median duration of response was 10.6 months (95% CI: 3.3, NE). The DoR ranges from 1.18+ to 16.6+ months and 12 out of 18 patients were censored for this analysis.

At the time of this data cut, 18/45 of people with synovial sarcoma or MRCLS had RECISTv1.1 responses with lete-cel, by independent review. The pre-defined success criteria for this planned interim analysis required at least 14 responders out of 45 patients and the primary endpoint for efficacy will require 16 responders out of 60 patients by independent review.

By investigator review, 15/45 (33%) of people with synovial sarcoma or MRCLS had RECISTv1.1 responses. The response rate was 7/23 (30%) for people with synovial sarcoma and 8/22 (36%) for people with MRCLS by investigator review.

The safety population included 73 people who had received lete-cel at the time of this interim analysis. Safety findings were consistent with the known profile of lete-cel from previous data. 65 (89%) patients had cytokine release syndrome (CRS), with the majority of CRS cases at grades 1 and 2, and 9 (12%) at grade 3 and no cases reported at Grade 4 or 5. 64 (88%) patients had hematopoietic cytopenias and the majority (86%) of events were Grade 3 or higher. Less than 5% of patients experienced immune effector cell-associated neurotoxicity (ICANS) and all events were grade 1. Overall, the safety profile of lete-cel was acceptable, including CRS and reversible hematologic toxicities.

The primary analysis will be conducted in the first half of 2024 when the 60th treated patient has 12 months of follow-up.

Data from Substudy 1 of the IGNYTE-ESO trial (CTOS 2023)

Substudy 1 was designed to explore the feasibility, efficacy, and safety of lete-cel in the first-line setting for treatment-naïve patients with metastatic or unresectable synovial sarcoma or MRCLS.

Of the five evaluable patients in the substudy, one exhibited a complete response, with an additional three partial responses, yielding an overall response rate of 80% (4/5) by investigator assessment. All five patients experienced cytokine release syndrome, all cases resolved; four were treated with tocilizumab. Overall, the substudy reveals encouraging efficacy in this small population of treatment-naïve patients in the advanced/metastatic setting with 80% ORR, with all responses ongoing at the time of this analysis.

Overview of IGNYTE-ESO trial design

IGNYTE-ESO is a Phase 2, open-label trial for people with advanced synovial sarcoma or MRCLS to evaluate the efficacy, safety, and tolerability of lete-cel. Lete-cel’s engineered TCR T-cells target NY-ESO-1+ tumors. NY-ESO-1 is highly expressed in synovial sarcoma and MRCLS in the context of HLA-A*02.

Key eligibility criteria include ECOG performance status of 0 or 1; HLA*02 positive with confirmed NY-ESO expression in ≥ 30% of tumor cells ≥ 2+ by immunohistochemistry; aged ≥ 10 years; and patients must have measurable disease according to RECIST v1.1 at the time of treatment. The IGNYTE-ESO master protocol include two substudies – Substudy 1 was designed to investigate lete-cel in previously untreated advanced (metastatic or unresectable) synovial sarcoma or MRCLS; and Substudy 2 lete-cel in advanced (metastatic or unresectable) synovial sarcoma or MRCLS post-anthracycline chemotherapy. Eligible patients received lete-cel doses between 1-15 × 10^9 transduced T-cells after receiving lymphodepleting chemotherapy.

Approximately 10 people were planned to be treated in Substudy 1, 5 patients were treated and enrollment was stopped. Approximately 60 people were planned to be treated in Substudy 2 and enrollment is complete.

About lete-cel 5

Lete-cel is an engineered TCR T-cell therapy against the solid tumor antigen NY-ESO-1.

About synovial sarcoma

There are approximately 50 types of soft tissue sarcomas which are categorized by tumors that appear in fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues.1 Synovial sarcoma accounts for approximately 5% to 10% of all soft tissue sarcomas (there are approximately 13,400 new soft tissue cases in the U.S. each year).2 One third of patients with synovial sarcoma will be diagnosed under the age of 30.2 The five-year survival rate for people with metastatic disease is just 20% and most people undergoing standard of care treatment for advanced disease experience recurrence and go through multiple lines of therapy, often exhausting all options.

About Myxoid/round cell liposarcoma (MRCLS)

Myxoid/round cell liposarcoma (MRCLS) is a type of soft tissue sarcoma that is predominantly found in the limbs. MRCLS accounts for approximately 5% to 10% of all soft tissue sarcomas.1 One-third of MRCLS cases will become metastatic with tumors spreading to unusual bone and soft tissue locations. MRCLS commonly presents at an age ranging from 35-55 years and has a poor prognosis because it recurs locally and tends to metastasize quickly and widely. The 5-year survival rate for metastatic MRCLS is only 5%.

OncoBone Ventures announces collaboration with metastasis specialist Mestastop Solutions

On October 31, 2023 OncoBone Ventures enters into collaboration with India-based Mestastop Solutions, a pioneer in the metastasis field (Press release, OncoBone Ventures, OCT 31, 2023, View Source [SID1234636482]). Mestastop has dissected the whole metastasis process in three patent protected platforms METAssay, METSCAN and METVivo that they can use to identify cancer patients who are at a higher risk of metastasis, as well as pertinent targets for drug discovery.

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

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Arnab and Debabani Roy Chowdhury, Founders and Directors of Mestastop state: "We were happy to partner with OncoBone Ventures, who are focused on developing novel therapies for cancer patients with bone metastases, for whom there are currently no effective therapies available. The collaboration includes developing novel in vitro models as a screening platform for evaluating efficacy of therapies on cancer bone metastases."

Tiina Kähkönen, Founder and CSO of OncoBone Ventures comments: ‘We have been truly fortunate to find a collaborator like Mestastop Solutions who shares our passion to develop therapies for cancer patients with metastatic disease. Together we have the required expertise to successfully develop a pioneering in vitro bone metastasis platform that will be of great importance for the development of novel therapies affecting bone metastases."

Jussi Halleen, Founder and CEO of OncoBone Ventures adds: "OncoBone Ventures aims to utilize the developed in vitro platform as a screening tool before testing efficacy of our pipeline assets in our current in vivo Bone Metastasis Technology Platform. This will lead to faster and more cost-effective development of efficient therapies for patients with bone metastases."

More information about Mestastop Solutions in View Source