Janssen Submits Supplemental Biologics License Application to U.S. FDA Seeking Approval of RYBREVANT® (amivantamab-vmjw) Plus Chemotherapy for the Treatment of Patients with EGFR-Mutated Non-Small Cell Lung Cancer Who Progressed on or after Osimertinib

On November 20, 2023 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking the approval of RYBREVANT (amivantamab-vmjw) in combination with chemotherapy (carboplatin and pemetrexed) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or L858R substitution after disease progression on or after osimertinib (Press release, Johnson & Johnson, NOV 20, 2023, View Source [SID1234637873]).

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"New treatment options are urgently needed in the post-osimertinib setting, where patients continue to face unacceptable survival rates," said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. "As we strive to transform the standard of care in patients with EGFR-mutated NSCLC, we are committed to working closely with the FDA during review of this submission for RYBREVANT in this expanded patient population."

This application is supported by data from the Phase 3 MARIPOSA-2 (NCT04988295) study evaluating the efficacy and safety of RYBREVANT and chemotherapy in patients with locally advanced or metastatic EGFR ex19del or L858R substitution NSCLC who had disease progression on or after treatment with osimertinib.1,2

Results from the MARIPOSA-2 study were recently presented in a Presidential Symposium at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) 2023 Congress (Abstract #LBA15) and simultaneously published in the Annals of Oncology.

About the MARIPOSA-2 Study
MARIPOSA-2 (NCT04988295), which enrolled 657 patients, is a randomized, open-label Phase 3 study evaluating the efficacy and safety of two combination regimens of RYBREVANT (with and without lazertinib) and chemotherapy. Patients with locally advanced or metastatic EGFR ex19del or L858R substitution NSCLC who had disease progression on or after treatment with osimertinib were randomized to treatment with RYBREVANT plus chemotherapy, RYBREVANT plus chemotherapy with lazertinib, or chemotherapy alone. The dual primary endpoint was used to compare the PFS (using RECIST v1.1 guidelines*) as assessed by blinded independent central review (BICR) for each experimental arm to chemotherapy alone. Secondary endpoints included objective response as assessed by BICR, OS, DOR, time to subsequent therapy, PFS2 and intracranial PFS.1

All study participants underwent serial brain imaging to allow for the robust assessment of intracranial endpoints and to assess the CNS activity of RYBREVANT plus chemotherapy with and without lazertinib. As brain metastases can lead to significant burden and poor outcomes for patients, this aspect of the study design provides critical information in an area of high unmet need. The study enrolled 657 patients with locally advanced or metastatic EGFR ex19del or L858R substitution NSCLC who had disease progression on or after treatment with osimertinib.2

About RYBREVANT
RYBREVANT (amivantamab-vmjw), a fully-human bispecific antibody targeting EGFR and MET with immune cell-directing activity, received accelerated approval by the U.S. Food and Drug Administration (FDA) in May 2021 for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.2 This indication is approved under accelerated approval based on ORR and DOR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. RYBREVANT is also approved in this indication in Europe, and other markets around the world. In August 2023, Janssen submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration for the expanded approval of RYBREVANT in combination with chemotherapy (carboplatin-pemetrexed) for the first-line treatment of patients with NSCLC with EGFR exon 20 insertion mutations. A marketing authorization application for this indication has also been submitted to the European Medicines Agency.

The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Non-Small Cell Lung Cancer† prefer next-generation sequencing-based strategies over polymerase chain reaction-based approaches for the detection of EGFR exon 20 insertion variants and include amivantamab-vmjw (RYBREVANT) as a subsequent therapy option with a Category 2A recommendation for patients that have progressed on or after platinum-based chemotherapy with or without immunotherapy and have EGFR exon 20 insertion mutation-positive advanced NSCLC.3 ‡§

In addition to the Phase 3 MARIPOSA-2 study, RYBREVANT is being studied in multiple clinical trials in NSCLC, including:

The Phase 3 MARIPOSA (NCT04487080) study assessing RYBREVANT in combination with lazertinib versus osimertinib and versus lazertinib alone in the first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR ex19del or L858R substitution mutations. Data for this randomized Phase 3 study presented at the ESMO (Free ESMO Whitepaper) 2023 Congress demonstrated statistically significant and clinically meaningful improvement in PFS in patients receiving RYBREVANT plus lazertinib versus osimertinib.4
The Phase 3 PAPILLON (NCT04538664) study assessing RYBREVANT in combination with carboplatin-pemetrexed versus carboplatin-pemetrexed in the first-line treatment of patients with advanced or metastatic NSCLC with EGFR exon 20 insertion mutations. Data for this randomized Phase 3 study presented at the ESMO (Free ESMO Whitepaper) 2023 Congress demonstrated statistically significant and clinically meaningful improvement in PFS in patients receiving RYBREVANT versus chemotherapy.5
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in participants with advanced NSCLC.6
The Phase 1/1b CHRYSALIS-2 (NCT04077463) study evaluating RYBREVANT in combination with lazertinib and lazertinib as a monotherapy in patients with advanced NSCLC with EGFR mutations.7
The Phase 1 PALOMA (NCT04606381) study assessing the feasibility of subcutaneous (SC) administration of amivantamab based on safety and pharmacokinetics and to determine a dose, dose regimen and formulation for amivantamab SC delivery.8
The Phase 2 PALOMA-2 (NCT05498428) study assessing subcutaneous amivantamab in participants with advanced or metastatic solid tumors including EGFR-mutated NSCLC.9
The Phase 3 PALOMA-3 (NCT05388669) study assessing lazertinib with subcutaneous amivantamab compared to intravenous amivantamab in participants with EGFR-mutated advanced or metastatic NSCLC.10
The Phase 1/2 METalmark (NCT05488314) study assessing RYBREVANT and capmatinib combination therapy in locally advanced or metastatic NSCLC.11
The Phase 1/2 PolyDamas (NCT05908734) study assessing RYBREVANT and cetrelimab combination therapy in locally advanced or metastatic NSCLC.12
The Phase 2 SKIPPirr study (NCT05663866) exploring how to decrease the incidence and/or severity of first-dose infusion-related reactions with RYBREVANT in combination with lazertinib in relapsed or refractory EGFR-mutated advanced or metastatic NSCLC.13
For more information, visit: View Source

About Lazertinib
Lazertinib is an oral, third-generation, brain-penetrant EGFR TKI that targets both the T790M mutation and activating EGFR mutations while sparing wild type-EGFR. An analysis of the efficacy and safety of lazertinib from the Phase 3 LASER301 study was published in The Journal of Clinical Oncology in 2023.14 In 2018, Janssen Biotech, Inc., entered into a license and collaboration agreement with Yuhan Corporation for the development of lazertinib.

About Non-Small Cell Lung Cancer
Worldwide, lung cancer is one of the most common cancers, with NSCLC making up 80 to 85 percent of all lung cancer cases.15,16 The main subtypes of NSCLC are adenocarcinoma, squamous cell carcinoma and large cell carcinoma.17 Among the most common driver mutations in NSCLC are alterations in EGFR, which is a receptor tyrosine kinase controlling cell growth and division.18 EGFR mutations are present in 10 to 15 percent of Western patients with NSCLC with adenocarcinoma histology and occur in 40 to 50 percent of Asian patients.17,18,19,20,21,22 EGFR ex19del or EGFR L858R substitutions are the most common EGFR mutations.23 The five-year survival rate for all people with advanced NSCLC and EGFR mutations treated with EGFR TKIs is less than 20 percent.24,25 Patients with EGFR ex19del or L858R mutations have a real-world five-year OS of 19 percent.26

RYBREVANT IMPORTANT SAFETY INFORMATION2

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions
RYBREVANT can cause infusion-related reactions (IRR); signs and symptoms of IRR include dyspnea, flushing, fever, chills, nausea, chest discomfort, hypotension, and vomiting.

Based on the safety population, IRR occurred in 66% of patients treated with RYBREVANT. Among patients receiving treatment on Week 1 Day 1, 65% experienced an IRR, while the incidence of IRR was 3.4% with the Day 2 infusion, 0.4% with the Week 2 infusion, and cumulatively 1.1% with subsequent infusions. Of the reported IRRs, 97% were Grade 1-2, 2.2% were Grade 3, and 0.4% were Grade 4. The median time to onset was 1 hour (range 0.1 to 18 hours) after start of infusion. The incidence of infusion modifications due to IRR was 62% and 1.3% of patients permanently discontinued RYBREVANT due to IRR.

Premedicate with antihistamines, antipyretics, and glucocorticoids and infuse RYBREVANT as recommended. Administer RYBREVANT via a peripheral line on Week 1 and Week 2. Monitor patients for any signs and symptoms of infusion reactions during RYBREVANT infusion in a setting where cardiopulmonary resuscitation medication and equipment are available. Interrupt infusion if IRR is suspected. Reduce the infusion rate or permanently discontinue RYBREVANT based on severity.

Interstitial Lung Disease/Pneumonitis
RYBREVANT can cause interstitial lung disease (ILD)/pneumonitis. Based on the safety population, ILD/pneumonitis occurred in 3.3% of patients treated with RYBREVANT, with 0.7% of patients experiencing Grade 3 ILD/pneumonitis. Three patients (1%) discontinued RYBREVANT due to ILD/pneumonitis. 

Monitor patients for new or worsening symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, fever). Immediately withhold RYBREVANT in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed.

Dermatologic Adverse Reactions
RYBREVANT can cause rash (including dermatitis acneiform), pruritus and dry skin. Based on the safety population, rash occurred in 74% of patients treated with RYBREVANT, including Grade 3 rash in 3.3% of patients. The median time to onset of rash was 14 days (range: 1 to 276 days). Rash leading to dose reduction occurred in 5% of patients, and RYBREVANT was permanently discontinued due to rash in 0.7% of patients.

Toxic epidermal necrolysis occurred in one patient (0.3%) treated with RYBREVANT.

Instruct patients to limit sun exposure during and for 2 months after treatment with RYBREVANT. Advise patients to wear protective clothing and use broad spectrum UVA/UVB sunscreen. Alcohol-free emollient cream is recommended for dry skin.

If skin reactions develop, start topical corticosteroids and topical and/or oral antibiotics. For Grade 3 reactions, add oral steroids and consider dermatologic consultation. Promptly refer patients presenting with severe rash, atypical appearance or distribution, or lack of improvement within 2 weeks to a dermatologist. Withhold, dose reduce or permanently discontinue RYBREVANT based on severity.

Ocular Toxicity
RYBREVANT can cause ocular toxicity including keratitis, dry eye symptoms, conjunctival redness, blurred vision, visual impairment, ocular itching, and uveitis. Based on the safety population, keratitis occurred in 0.7% and uveitis occurred in 0.3% of patients treated with RYBREVANT. All events were Grade 1-2. Promptly refer patients presenting with eye symptoms to an ophthalmologist. Withhold, dose reduce or permanently discontinue RYBREVANT based on severity.

Embryo-Fetal Toxicity
Based on its mechanism of action and findings from animal models, RYBREVANT can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential of the potential risk to the fetus. Advise female patients of reproductive potential to use effective contraception during treatment and for 3 months after the final dose of RYBREVANT.

Adverse Reactions
The most common adverse reactions (≥20%) were rash (84%), IRR (64%), paronychia (50%), musculoskeletal pain (47%), dyspnea (37%), nausea (36%), fatigue (33%), edema (27%), stomatitis (26%), cough (25%), constipation (23%), and vomiting (22%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased lymphocytes (8%), decreased albumin (8%), decreased phosphate (8%), decreased potassium (6%), increased alkaline phosphatase (4.8%), increased glucose (4%), increased gamma-glutamyl transferase (4%), and decreased sodium (4%).

Please read the full Prescribing Information for RYBREVANT.

Cardiff Oncology to Present at the 35th Annual Piper Sandler Healthcare Conference

On November 20, 2023 Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers, reported that company management will participate in a fireside chat and 1×1 investor meetings at the 35th Annual Piper Sandler Healthcare Conference, which is taking place at the Lotte New York Palace in New York from November 28-30, 2023 (Press release, Cardiff Oncology, NOV 20, 2023, View Source [SID1234637870]).

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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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Details of the fireside chat can be found below.

Presenter: Mark Erlander, CEO
Date: 11/29/2023
Time: 10:00 – 10:25 AM ET in Soho Track

Interested parties can register for and access the live webcast for Piper Sandler by visiting the "Events" section of the Cardiff Oncology website. The webcast replay will be available after the conclusion of the presentation.

Cofactor Genomics’ Immunotherapy Predictive Diagnostic, OncoPrism, Completes Validation and Moves Into Clinical Use in Head and Neck Cancer Patients

On November 20, 2023 Cofactor Genomics, the company bridging the precision medicine gap, reported successful validation of the OncoPrism immunotherapy predictive diagnostic assay, enabling physicians to use the test to help guide treatment decisions for patients with metastatic or recurrent squamous cell carcinoma of the head and neck (HNSCC) (Press release, Cofactor Genomics, NOV 20, 2023, View Source [SID1234637868]). The successful readout is announced on the heels of the yearly national PREDAPT Summit, where the participating institutions from across the country gathered and reviewed the study results. The assay was validated in independent HNSCC patient cohorts receiving anti-PD-1 inhibitors pembrolizumab and nivolumab both as a single agent and in combination with chemotherapy. This milestone clears the assay for nationwide clinical use in an interventional setting where doctors can now incorporate OncoPrism to help guide patient treatment in a landscape of multiple therapies, each benefiting a subset of the patient population.

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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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"This is a proud day for all those working tirelessly to bring precision medicine to immunotherapy and improve the outcome and quality of care for patients. We have learned not only from the study’s validation results but from physicians across the nation who have had significant input on the study and the OncoPrism assay these last 3 years," stated Jarret Glasscock, Cofactor’s CEO.

This validation announcement follows a publication earlier this year in the Journal of Cancer Research and Clinical Oncology, where Cofactor along with more than 20 participating institutions reported on OncoPrism’s capability to predict HNSCC patients’ response to anti-PD-1 immune checkpoint inhibitors. Cofactor’s broader work, on the technology’s ability to predict response to immune checkpoint inhibitors (ICI) across multiple cancers, was recognized by the Nature Publishing Group as the second most downloaded oncology paper of the year.

While the validation of OncoPrism in HNSCC patients was the focus of this week’s announcement, Cofactor’s national PREDAPT clinical trial is aimed at evaluating OncoPrism in 11 cancers including lung, head and neck, colorectal, breast, cervical, liver, kidney, esophageal and bladder. US healthcare systems supporting and participating in the PREDAPT trial collectively provide care to approximately 20% of the total US patient population.

VBI Vaccines Presents Additional Biomarker Data from Phase 1/2a Study of VBI-1901 in Recurrent GBM at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting

On November 20, 2023 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that additional biomarker data from the Phase 1/2a study of VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate, in recurrent glioblastoma (GBM) patients, were highlighted in a poster presentation at the 28th Annual Meeting and Education Day of the Society for Neuro-Oncology (SNO), held on Friday, November 17, 2023 (Press release, VBI Vaccines, NOV 20, 2023, View Source [SID1234637864]).

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New proof-of-mechanism data demonstrated that vaccination with VBI-1901 was associated with T-cell activation capable of trafficking across the blood-brain barrier to the tumor microenvironment, an area which is known to be highly immunosuppressive and difficult to infiltrate. The additional peripheral biomarker data assessed levels of C4G – a protein fragment produced when cytotoxic T-cells, or killer T-cells, migrate into and throughout the tumor microenvironment – high baseline levels of which have been associated with longer overall survival in studies conducted in metastatic melanoma patients [Prakash et al., 2014].1 After vaccination with VBI-1901 in the Phase 1/2a study in recurrent GBM patients, the data showed there were higher blood/plasma levels of C4G in patients who achieved partial tumor responses than in patients with stable or progressive disease. Additionally, higher levels of C4G after vaccination with VBI-1901 have been associated with longer progression-free survival.

"We are very pleased to share these significant mechanistic findings at SNO," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "The GBM tumor microenvironment is notoriously immunosuppressive and difficult to penetrate, which is why most treatment needs to be administered intratumorally, and why there are currently so few effective treatment options for patients. We believe that these data continue to enrich the potential for VBI-1901 to have meaningful impact in recurrent and primary GBM, and we look forward to data from the next phases of development in each setting."

Data Highlights from Phase 1/2a Study Data in Recurrent GBM Patients- VBI-1901 10µg + GM-CSF Study Arms

(n=16)

44% disease control rate achieved (n=7/16) – disease control rate is defined as stable disease (SD) + partial tumor response (PR) + complete tumor response (CR)
2 PRs were observed – 1 patient was on treatment for more than 28 months (2.33 years), surviving at least 40 months (3.33 years) as of August 1, 2023, with a maximum tumor reduction of 93% relative to baseline
5 additional patients demonstrated SD for a sustained period of time
All patients with a tumor response (PR or SD) (n=7/16) reached a minimum survival of 12 months
Median overall survival (mOS) was 12.9 months, comparing favorably to 8-month mOS for monotherapy standard-of-care2
Ongoing Phase 2b Study Design in Recurrent GBM Patients

Multi-center, randomized, controlled, open-label study in up to 60 patients with first recurrent GBM

Patients will be randomized in a 1:1 ratio across two study arms:
Intradermal VBI-1901 + GM-CSF: 10 µg dose every 4 weeks until clinical disease progression
Active comparator: monotherapy standard-of-care – either intravenous carmustine or oral lomustine, every 6 weeks until disease progression or intolerable toxicity
Endpoints include:
Safety and tolerability
Overall survival (OS) – median and overall
Tumor response rate (TRR)
Progression-free survival (PFS)
Immunologic responses
Reduction in corticosteroid use relative to baseline
Change in quality of life compared to baseline
The U.S. Food and Drug Administration (FDA) has considered demonstration of a statistically significant improvement in overall survival relative to a randomized control arm to be clinically significant and has recognized this as criteria to support the approval of new oncology drugs.3

For more information about the Phase 2b study, visit clinicaltrials.gov and reference trial identifier: NCT03382977.

About GBM and VBI-1901

Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 14,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. The FDA has granted VBI-1901 Fast Track Designation and Orphan Drug Designation for the treatment of recurrent glioblastoma. These designations are intended to provide certain benefits to drug developers, including more frequent meetings with the FDA, and Accelerated Approval and Priority Review, if relevant criteria are met, among other benefits.

Ultragenyx to Participate at Investor Conferences in November

On November 20, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported that management, will participating in two upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, NOV 20, 2023, View Source [SID1234637862]).

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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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6th Annual Evercore ISI HealthCONx (Miami, FL)

Tuesday, November 28, 2023, management will host 1×1 meetings.

Piper Sandler 35th Annual Healthcare Conference (New York City, NY)

Wednesday, November 29, 2023, at 10:00am, Emil Kakkis, M.D., Ph.D., CEO and President, will participate in a fireside chat and host 1×1 meetings.
The live and archived webcast of the fireside chat will be accessible from the company’s website at View Source