On October 15, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Galinpepimut-S (GPS), an immunotherapeutic targeting Wilms Tumor-1 (WT1), for the treatment of pediatric acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, OCT 15, 2024, View Source [SID1234647206]).

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"GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Receiving RPDD from the FDA is another acknowledgment of the critical need for new treatment options for AML and our results in adult patients. In our Phase 2 trial in adult patients which included patients as young as 25, clinical benefits were significantly higher in younger patients, which was expected based on the mechanism of action of GPS that is mediated via the immune system that is generally better preserved in younger patients, and even more so in children. With both of our development candidates, GPS and SLS009, now granted RPDD for AML, this recognition further reinforces our commitment to delivering potential new therapies to children affected by this challenging condition."

AML prognosis with currently available treatments in the refractory and/or relapsed pediatric patient population remains poor. In a representative study, the 5-year overall survival (OS) rate in relapsed pediatric AML was 33% for all patients, and in patients whose remission lasted less than 12 months only 15.7%. In patients who did not achieve complete remission after one course of chemotherapy, 5-year overall survival was 0%. About 50% of children with pediatric AML relapse. Generally, the only therapy considered curative in relapsed and refractory patients is a bone marrow transplant and the primary goal of chemotherapy is to achieve remission so that pediatric patients can be transplanted.

In adult AML patients in first complete remission, GPS showed a median OS of 67.6 months across all ages with a favorable safety profile in an earlier Phase 2 study and induced T-lymphocytes response in both cytotoxic CD8+ cells and memory and helper CD4-+ cells with its innovative heteroclitic technology. In that study, outcomes were even better in younger patients in whom neither the median disease-free survival (DFS) nor OS was reached, i.e. among younger patients more than half of the patients were alive and leukemia-free for more than 5 years after treatment commenced.

Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for GPS for the treatment of pediatric AML is approved by the FDA, SELLAS will be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million.

On October 15, 2024 PharmaMar (MSE: PHM) and its partner Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported positive top-line results from the Phase 3 clinical trial evaluating Zepzelca (lurbinectedin) in combination with the PD-L1 inhibitor atezolizumab (Tecentriq) compared to atezolizumab alone when administered as a maintenance treatment for adults with extensive-stage Small Cell Lung Cancer (ES-SCLC) following induction therapy with carboplatin, etoposide and atezolizumab (Press release, PharmaMar, OCT 15, 2024, View Source [SID1234647205]). The combination of lurbinectedin and atezolizumab demonstrated a statistically significant improvement in the primary endpoints of overall survival (OS) and progression-free survival (PFS), as assessed by an independent review facility (IRF), compared to treatment with atezolizumab alone.

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"Each year, approximately 63,000 to 72,000 new cases of Small Cell Lung Cancer (SCLC) are reported in Europe. A majority of these patients are diagnosed with extensive stage disease, which is aggressive and often difficult to treat, with poor prognosis,[i],[ii],[iii]" said Luis Paz-Ares, M.D., Ph.D., head of medical oncology at the Hospital Universitario 12 de Octubre in Madrid, Spain, and IMforte trial principal investigator. "These trial results demonstrate the efficacy of lurbinectedin, in combination with standard-of-care atezolizumab for patients in first-line maintenance treatment, a much-needed advancement for patients with extensive disease."

"The results of the Phase 3 IMforte trial are highly encouraging and showed a statistically significant benefit for the lurbinectedin and atezolizumab combination for extensive-stage small cell lung cancer patients receiving this treatment in the first-line maintenance setting. These results demonstrate the potential of this regimen to delay disease progression and extend survival for patients with this aggressive disease," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "We are pleased with these clinically meaningful results and plan to submit an sNDA in the first half of 2025 to support this combination in the first-line maintenance setting. We thank the investigators and patients who are involved in this trial, along with our partners at Roche."

"Lurbinectedin monotherapy is currently the standard of care in 2L SCLC. In Europe, it is only approved in Switzerland and early access and compassionate use programs have already allowed some European patients to benefit from lurbinectedin," said Javier Jiménez, Chief Medical Officer of PharmaMar.

The combination was generally well-tolerated. The preliminary safety data in the ongoing trial is consistent with the known safety profiles of lurbinectedin and atezolizumab with no new safety signals observed in the combination arm.

Jazz and Roche plan to submit these data for presentation at a future medical meeting.

PharmaMar will submit a marketing authorisation application (MAA) to the EMA in the first half of 2025 to request regulatory approval in the European Union (EU). Lurbinectedin is available for use in 16 territories around the world.

On October 15, 2024 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage radiopharma company focused on developing innovative treatments for cancer patients, reported the filing of a provisional patent covering new therapeutic radiopharmaceuticals based on a family of linkers used to connect radioisotopes with targeting agents, including Monopar’s uPAR targeting antibody MNPR-101 (Press release, Monopar Therapeutics, OCT 15, 2024, View Source [SID1234647204]).

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Highlights of the patent filing include:

Composition of Matter: Claims cover a family of linkers, as well as Monopar’s uPAR targeting agents linked with these along with therapeutic radioisotopes
Stability and Biodistribution: These proprietary new linkers have been created to enhance the stability and biodistribution of Monopar’s array of therapeutic radiopharmaceuticals
Versatility: The newly developed linker family works with a wide range of isotopes and targeting molecules, including small molecules/peptides and antibodies
"This provisional patent could enable us to use these linkers to create new proprietary radiopharmaceuticals going after well-established, high-value cancer targets that we are interested in," commented Andrew Cittadine, Monopar’s Chief Operating Officer. "We also believe that these linkers may be of great interest to others in the industry, opening the door to potential licensing and development collaborations."

"These novel linkers and compositions of matter exemplify Monopar’s passion and commitment to being an innovator in the radiopharma space," said Chandler Robinson, MD, Monopar’s Chief Executive Officer.

On October 15, 2024 Johnson & Johnson (NYSE: JNJ) reported results for third-quarter 2024. "Johnson & Johnson’s strong results in the third quarter reflect the unique breadth of our business and commitment to delivering the next wave of healthcare innovation," said Joaquin Duato, Chairman and Chief Executive Officer (Press release, Johnson & Johnson, OCT 15, 2024, View Source [SID1234647203]). "During the quarter, we advanced our pipeline with regulatory approvals for TREMFYA and RYBREVANT, submitted an IDE for our general surgery robotic system, OTTAVA, and launched VELYS Spine and Shockwave E8 IVL Catheter, further strengthening our confidence in our near-and long-term growth targets."
Unless otherwise noted, the financial results and earnings guidance included below reflect the continuing operations of Johnson & Johnson.

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Overall financial results
Q3
($ in Millions, except EPS) 2024 2023 % Change
Reported Sales
$22,471
$21,351
5.2%
Net Earnings
$2,694

$4,309
(37.5)%
EPS (diluted)
$1.11

$1.69
(34.3)%
Q3
Non-GAAP* ($ in Millions, except EPS) 2024 2023 % Change
Operational Sales1,2
6.3%
Adjusted Operational Sales1,3
5.4%
Adjusted Operational Sales ex. COVID-19 Vaccine1,3

5.6%
Adjusted Net Earnings1,4
$5,876
$6,777
(13.3)%
Adjusted EPS (diluted)1,4
$2.42
$2.66
(9.0)%
Free Cash Flow (YTD)6,7
~$14,000
$11,974

1Non-GAAP financial measure; refer to reconciliations of non-GAAP financial measures included in accompanying schedules
2Excludes the impact of translational currency
3Excludes the net impact of acquisitions and divestitures and translational currency
4Excludes intangible amortization expense and special items
5Excludes COVID-19 Vaccine
6Non-GAAP measure; defined as cash flow from operating activities, less additions to property, plant and equipment. Cash flow from operations, the most directly comparable GAAP financial measure, will be included in subsequent SEC filings.
7Q3 YTD 2024 is estimated as of October 15, 2024. Q3 YTD 2023 includes approximately 8 months contribution from the Consumer Health segment.
Note: values may have been rounded

Regional sales results
Q3 % Change
($ in Millions) 2024 2023 Reported
Operational1,2
Currency
Adjusted
Operational1,3
U.S.
$12,909
$11,996
7.6%
7.6
–
6.5
International
9,562
9,355
2.2
4.6
(2.4)
4.0
Worldwide
$22,471
$21,351
5.2%
6.3
(1.1)
5.4

1Non-GAAP financial measure; refer to reconciliations of non-GAAP financial measures included in accompanying schedules
2Excludes the impact of translational currency
3Excludes the net impact of acquisitions and divestitures and translational currency
Note: values may have been rounded

Segment sales results
Q3 % Change
($ in Millions) 2024 2023 Reported
Operational1,2
Currency
Adjusted
Operational1,3
Innovative Medicine
$14,580
$13,893
4.9%
6.3
(1.4)
6.4
MedTech
7,891
7,458
5.8
6.4
(0.6)
3.7
Worldwide
$22,471
$21,351
5.2%
6.3
(1.1)
5.4

1Non-GAAP financial measure; refer to reconciliations of non-GAAP financial measures included in accompanying schedules
2Excludes the impact of translational currency
3Excludes the net impact of acquisitions and divestitures and translational currency
Note: values may have been rounded

Third Quarter 2024 segment commentary:
Operational sales* reflected below excludes the impact of translational currency.
Innovative Medicine
Innovative Medicine worldwide operational sales grew 6.3%. Growth was driven by DARZALEX (daratumumab), ERLEADA (apalutamide), Other Oncology, and CARVYKTI (ciltacabtagene autoleucel) in Oncology, TREMFYA (guselkumab) in Immunology, SPRAVATO (esketamine) in Neuroscience, and OPSUMIT (macitentan) in Pulmonary Hypertension. Growth was partially offset by STELARA (ustekinumab) and SIMPONI/SIMPONI ARIA (golimumab) in Immunology.

MedTech
MedTech worldwide operational sales grew 6.4%*, with net acquisitions and divestitures positively impacting growth by 2.7%. Operational sales growth was driven primarily by electrophysiology products and Abiomed in Cardiovascular, previously referred to as Interventional Solutions, contact lenses in Vision and wound closure products in General Surgery. Growth was partially offset by endocutter products in Advanced Surgery.

Full-year 2024 guidance:
Johnson & Johnson does not provide GAAP financial measures on a forward-looking basis because the company is unable to predict with reasonable certainty the ultimate outcome of legal proceedings, unusual gains and losses, acquisition-related expenses, and purchase accounting fair value adjustments without unreasonable effort. These items are uncertain, depend on various factors, and could be material to Johnson & Johnson’s results computed in accordance with GAAP.
Johnson & Johnson is updating its 2024 guidance, including adjusted operational EPS guidance, to reflect improved performance and the impact from the recent acquisition of V-Wave.

Non-GAAP* 2024
July 2024 Adjusted Operational EPS1,2
$10.05
Improved performance outlook $0.10
October 2024 Adjusted Operational EPS1,2 pre-M&A
$10.15
M&A impact (V-Wave) ($0.24)
October 2024 Adjusted Operational EPS1,2
$9.91

1Non-GAAP financial measure; excludes the impact of translational currency
2Non-GAAP financial measure; excludes intangible amortization expense and special items
Note: Adjusted operational EPS figures reflect midpoint of issued guidance

($ in Billions, except EPS) October 2024 July 2024
Adjusted Operational Sales1,2,5
Change vs. Prior Year / Mid-point
5.7% – 6.2% / 6.0% 5.5% – 6.0% / 5.8%
Operational Sales2,5/ Mid-point
Change vs. Prior Year / Mid-point
$89.4B – $89.8B / $89.6B
6.3% – 6.8% / 6.6%
$89.2B – $89.6B / $89.4B
6.1% – 6.6% / 6.4%
Estimated Reported Sales3,5/ Mid-point
Change vs. Prior Year / Mid-point
$88.4B – $88.8B / $88.6B
5.1% – 5.6% / 5.4%
$88.0B – $88.4B / $88.2B
4.7% – 5.2% / 5.0%
Adjusted Operational EPS (Diluted)2,4/ Mid-point
Change vs. Prior Year / Mid-point
$9.86 – $9.96 / $9.91
(0.6)% – 0.4% / (0.1)%
$10.00 – $10.10 / $10.05
0.8% – 1.8% / 1.3%
Adjusted EPS (Diluted)3,4 / Mid-point
Change vs. Prior Year / Mid-point
$9.88 – $9.98 / $9.93
(0.4)% – 0.6% / 0.1%
$9.97 – $10.07 / $10.02
0.5% – 1.5% / 1.0%

1Non-GAAP financial measure; excludes the net impact of acquisitions and divestitures
2Non-GAAP financial measure; excludes the impact of translational currency
3Calculated using Euro Average Rate: October 2024 = $1.09 and July 2024 = $1.08 (Illustrative purposes only)
4Non-GAAP financial measure; excludes intangible amortization expense and special items
5Excludes COVID-19 Vaccine
Note: percentages may have been rounded
Other modeling considerations will be provided on the webcast.

Notable announcements in the quarter:
The information contained in this section should be read together with Johnson & Johnson’s other disclosures filed with the Securities and Exchange Commission, including its Current Reports on Form 8-K, Quarterly Reports on Form 10-Q and Annual Reports on Form 10-K. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. The reader is also encouraged to review all other news releases and information available in the Investor Relations section of the company’s website at News Releases, as well as Innovative Medicine News Center, MedTech News & Events, and www.factsabouttalc.com.
Regulatory
Johnson & Johnson submits application to the European Medicines Agency for DARZALEX (daratumumab) SC-based quadruplet regimen for newly diagnosed multiple myeloma patients1
Press Release
Johnson & Johnson files for U.S. FDA approval of DARZALEX FASPRO-based quadruplet regimen for newly diagnosed multiple myeloma patients for whom transplant is not planned
Press Release
DARZALEX (daratumumab)-based quadruplet regimen receives positive CHMP opinion for transplant-eligible patients with newly diagnosed multiple myeloma
Press Release
RYBREVANT (amivantamab-vmjw) plus standard of care approved in the U.S. as first and only targeted regimen to cut risk of disease progression by more than half in second-line EGFR-mutated advanced lung cancer
Press Release
Johnson & Johnson seeks first EU approval of nipocalimab to treat a broad population of patients living with antibody-positive generalised myasthenia gravis
Press Release
TREMFYA (guselkumab) receives U.S. FDA approval for adults with moderately to severely active ulcerative colitis, strengthening Johnson & Johnson’s leadership in inflammatory bowel disease
Press Release

Johnson & Johnson seeks first approval of nipocalimab to treat broadest population living with antibody positive generalized myasthenia gravis
Press Release
European Commission approves RYBREVANT (amivantamab) in combination with chemotherapy for the treatment of adult patients with advanced EGFR-mutated non-small cell lung cancer after failure of prior therapy
Press Release
European Commission approves BALVERSA (erdafitinib) for adult patients with unresectable or metastatic urothelial carcinoma
Press Release
RYBREVANT (amivantamab-vmjw) plus LAZCLUZE (lazertinib) approved in the U.S. as a first-line chemotherapy-free treatment for patients with EGFR-mutated advanced lung cancer
Press Release
DARZALEX FASPRO (daratumumab and hyaluronidase-fihj)-based quadruplet regimen approved in the U.S. for patients with newly diagnosed multiple myeloma who are transplant-eligible
Press Release
Johnson & Johnson seeks U.S. FDA approval of SPRAVATO (esketamine) as the first and only monotherapy for adults with treatment-resistant depression
Press Release
Data Releases
TREMFYA (guselkumab) demonstrates impressive results across biologic-naïve and biologic-refractory patients in Crohn’s disease and ulcerative colitis1
Press Release
CARVYKTI is the first and only cell therapy to significantly extend overall survival versus standard therapies for patients with multiple myeloma as early as second line
Press Release
DARZALEX (daratumumab)-based maintenance regimens show clinically meaningful deep and durable responses in transplant-eligible patients with newly diagnosed multiple myeloma
Press Release
Novel combination of TALVEY (talquetamab-tgvs) and TECVAYLI (teclistamab-cqyv) suggests high response rates and durable responses in triple-class refractory patients with relapsed or refractory multiple myeloma, including those with extramedullary disease
Press Release
TALVEY (talquetamab-tgvs) and DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) based combination shows deep and durable responses in patients with relapsed or refractory multiple myeloma
Press Release
Johnson & Johnson is transforming solid tumor cancer outcomes with new data at the 2024 World Conference on Lung Cancer and European Society for Medical Oncology Congress
Press Release
Groundbreaking nipocalimab study of pregnant individuals at high risk for early onset severe hemolytic disease of the fetus and newborn published in The New England Journal of Medicine
Press Release
Product Launch
Johnson & Johnson MedTech Launches VOLT Plating System1
Press Release
Johnson & Johnson Rolls Out New TECNIS Odyssey Next-Generation Intraocular Lens Offering Cataract Patients Precise Vision at Every Distance in Any Lighting
Press Release
Shockwave Medical Expands U.S. Peripheral IVL Portfolio with Enhanced Catheter
Press Release
DePuy Synthes Launches its First Active Spine Robotics and Navigation Platform

Other
Johnson & Johnson Completes Acquisition of V-Wave1
Press Release
Johnson & Johnson Announces that its Subsidiary, Red River Talc LLC, has Filed a Voluntary Prepackaged Chapter 11 Case to Resolve All Current and Future Ovarian Cancer Talc Claims
Press Release
Johnson & Johnson’s Executive Vice President, Chief Human Resources Officer Peter Fasolo to Retire; Kristen Mulholland Named Chief Human Resources Officer, Effective October 1, 2024
Press Release

1Subsequent to the quarter

Webcast information:
Johnson & Johnson will conduct a conference call with investors to discuss this earnings release today at 8:30 a.m., Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Johnson & Johnson website. A replay and podcast will be available approximately two hours after the live webcast in the Investor Relations section of the company’s website at events-and-presentations.

On October 15, 2024 Immuneering Corporation (Nasdaq: IMRX), a clinical-stage oncology company seeking to develop and commercialize universal-RAS/RAF medicines for broad populations of cancer patients, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to IMM-1-104 in the treatment of pancreatic cancer (Press release, Immuneering, OCT 15, 2024, View Source [SID1234647202]). IMM-1-104 is currently being evaluated in a Phase 2a clinical trial in patients with advanced solid tumors, including pancreatic cancer, in which positive initial response data was recently reported for first line pancreatic cancer patients treated in combination with modified gemcitabine/nab-paclitaxel.

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"The FDA’s granting of orphan drug designation for IMM-1-104 underscores the urgent need for new therapies that meaningfully improve outcomes for pancreatic cancer patients and represents an important milestone in the development of our lead asset," said Ben Zeskind, Ph.D., Co-Founder and CEO of Immuneering. "I believe our recently announced positive initial Phase 2a data, from our arm investigating IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel in pancreatic cancer, speaks to IMM-1-104’s potential to improve upon the current standard of care in this indication. Importantly, in the same trial we are also studying IMM-1-104 in combination with modified FOLFIRINOX, as well as in monotherapy for pancreatic cancer. We look forward to providing initial data from at least one additional arm of the Phase 2a portion of our Phase 1/2a trial before the end of the year."

FDA orphan drug designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation may qualify sponsors for incentives, including tax credits for qualified clinical trials, exemptions from certain FDA fees and additional time for post-approval marketing exclusivity. Earlier this year, Immuneering was granted FDA Fast Track designation for IMM-1-104 for the treatment of both first and second-line pancreatic cancer.

About IMM-1-104

IMM-1-104 aims to achieve universal-RAS activity that selectively impacts cancer cells to a greater extent than healthy cells, through Deep Cyclic Inhibition of the MAPK pathway with once-daily dosing. IMM-1-104 is currently being evaluated in a Phase 1/2a study in patients with advanced solid tumors harboring RAS mutations (NCT05585320).