On June 26, 2024 Verrica Pharmaceuticals Inc. (the "Company") reported to have entered into an amendment (the "Amendment") to its Credit Agreement dated as of July 26, 2023 (the "Credit Agreement"), by and between the Company, as borrower, OrbiMed Royalty & Credit Opportunities IV, LP, as a lender, each other lender that may from time to time become a party thereto, and OrbiMed Royalty & Credit Opportunities IV, LP, as administrative agent for the lenders (Filing, Verrica Pharmaceuticals, JUN 26, 2024, View Source [SID1234644596]). As previously disclosed, payments of the principal amount of borrowings under the Credit Agreement, together with a repayment premium and other fees, are not required under the Credit Agreement unless the Company’s net revenue attributable to YCANTHTM does not equal or exceed specified amounts for specified test periods as set forth in the Credit Agreement. Pursuant to the Amendment, the parties agreed to modify the commencement of such revenue test so that such revenue test now begins on September 30, 2024. In connection with the Amendment, the Company agreed to pay the lenders an amendment fee of $500,000.

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Except as set forth in the Amendment, the remaining terms of the Credit Agreement, as amended to date, remain unchanged. The foregoing description of the terms of the Amendment are not intended to be complete and are qualified in their entirety by reference to the Amendment, which the Company expects to file as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarter ending June 30, 2024.

On June 26, 2024 AstraZeneca reported that Tagrisso (osimertinib) with the addition of pemetrexed and platinum-based chemotherapy has been approved in China for the 1st-line treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) whose tumours have exon 19 deletions or exon 21 (L858R) mutations (Press release, AstraZeneca (Australia), JUN 26, 2024, View Source [SID1234644537]).

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The approval by China’s National Medical Products Administration (NMPA) was based on the results from the FLAURA2 Phase III trial published in The New England Journal of Medicine, and results from a prespecified exploratory subgroup analysis of efficacy and safety in Chinese patients, which was presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Asia Congress 2023.

In the overall trial population, Tagrisso with the addition of chemotherapy reduced the risk of disease progression or death by 38% by investigator assessment compared to Tagrisso monotherapy, which is the 1st-line global standard of care (hazard ratio [HR] 0.62; 95% confidence interval [CI] 0.49-0.79; p<0.0001). Median progression-free survival (PFS) was 25.5 months for patients treated with Tagrisso plus chemotherapy, an 8.8-month improvement versus Tagrisso monotherapy (16.7 months).

PFS results by blinded independent central review (BICR) in the overall trial population were consistent with results by investigator assessment, showing 29.4 months median PFS with Tagrisso plus chemotherapy, a 9.5-month improvement over Tagrisso monotherapy (19.9 months) (HR 0.62; 95% CI 0.48-0.80; p=0.0002).

While the overall survival (OS) remained immature at the second interim analysis (41% maturity), an encouraging trend towards an OS benefit was observed with Tagrisso plus chemotherapy versus Tagrisso alone (HR 0.75; 95% CI 0.57-0.97). The trial continues to assess OS as a key secondary endpoint.

Efficacy results from the China cohort of FLAURA2 were broadly consistent with the overall trial. By investigator assessment, Tagrisso with the addition of chemotherapy reduced the risk of disease progression or death by 44% compared to Tagrisso monotherapy (HR 0.56; 95% CI 0.34-0.92). Median PFS was 27.4 months for patients treated with Tagrisso plus chemotherapy, a 5.1-month improvement versus Tagrisso monotherapy (22.3 months).

PFS results in the China cohort by BICR were consistent with the results by investigator assessment, showing 33.2 months median PFS with Tagrisso plus chemotherapy, an 11.2-month improvement over Tagrisso monotherapy (22.0 months) (HR 0.58; 95% CI 0.34-1.01).

A trend towards an OS benefit was also observed in the China cohort for Tagrisso plus chemotherapy versus Tagrisso alone (HR 0.97; 95% CI 0.45-2.06).

Each year in China, there are over one million people diagnosed with lung cancer, accounting for more than a third of the world’s lung cancer patients.1,2 Among those with NSCLC, the most common form of lung cancer, approximately 40% of patients in China have tumours with an EGFR mutation.3 Additionally, the majority of patients with NSCLC are diagnosed with advanced disease.4

Professor Ying Cheng, MD, Director of Jilin Lung Cancer Diagnosis and Treatment Centre, and principal investigator in China said: "The approval of osimertinib with the addition of chemotherapy in China is critical for the treatment of the largest population of patients with EGFR-mutated lung cancer worldwide. These patients will now have a choice of two highly effective osimertinib-based options for 1st-line treatment, allowing physicians to tailor their approach to their patients. This is especially important for those with a poorer prognosis, such as cancer that has spread to the brain or those who have L858R mutations."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: "Tagrisso with the addition of chemotherapy in FLAURA2 has not only shown unprecedented progression-free survival in the overall trial population, but also among Chinese patients, reducing the risk of disease progression by nearly half. This approval reinforces Tagrisso as a backbone therapy in EGFR-mutated lung cancer and acknowledges its important role, as monotherapy or with chemotherapy, in addressing the high prevalence of this disease in Asian countries and China, specifically."

The safety profile of Tagrisso with the addition of chemotherapy was generally manageable and consistent with the established profiles of the individual medicines. Adverse event (AE) rates were higher in the Tagrisso plus chemotherapy arm, driven by well-characterised chemotherapy-related AEs. Discontinuation rates of Tagrisso due to AEs were 11% for Tagrisso plus chemotherapy and 6% for monotherapy.

Tagrisso is approved as monotherapy in more than 100 countries including in the US, EU, China and Japan. Approved indications include for 1st-line treatment of patients with locally advanced or metastatic EGFRm NSCLC, locally advanced or metastatic EGFR T790M mutation-positive NSCLC, and adjuvant treatment of early-stage EGFRm NSCLC. In addition to this new approval in China, Tagrisso with the addition of chemotherapy is also approved in the US and several other countries for 1st-line treatment of patients with locally advanced or metastatic EGFRm NSCLC.

Notes

Lung cancer
Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths.5 Lung cancer is broadly split into NSCLC and small cell lung cancer.6 Each year there are an estimated 2.4 million people diagnosed with lung cancer globally, with 80-85% of patients diagnosed with NSCLC, the most common form of lung cancer.5-8

Approximately 10-15% of NSCLC patients in the US and Europe, and 30-40% of patients in Asia have EGFRm NSCLC.9-11 Patients with EGFRm NSCLC are particularly sensitive to treatment with an EGFR-tyrosine kinase inhibitor (EGFR-TKI) which blocks the cell-signalling pathways that drive the growth of tumour cells.12

FLAURA2
FLAURA2 is a randomised, open-label, multi-centre, global Phase III trial in the 1st-line treatment of patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) EGFRm NSCLC. Patients were treated with Tagrisso 80mg once-daily oral tablets with the addition of chemotherapy (pemetrexed (500mg/m2) plus cisplatin (75mg/m2) or carboplatin (AUC5)) every three weeks for four cycles, followed by Tagrisso with pemetrexed maintenance every three weeks.

The trial enrolled 557 patients in more than 150 centres across more than 20 countries, including in the US, Europe, South America and Asia. The primary endpoint is PFS. The trial is ongoing and will continue to assess the secondary endpoint of OS.

Tagrisso
Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI with proven clinical activity in NSCLC, including against central nervous system (CNS) metastases. Tagrisso (40mg and 80mg once-daily oral tablets) has been used to treat nearly 800,000 patients across its indications worldwide and AstraZeneca continues to explore Tagrisso as a treatment for patients across multiple stages of EGFRm NSCLC.

There is an extensive body of evidence supporting the use of Tagrisso in EGFRm NSCLC. Tagrisso is the only targeted therapy to improve patient outcomes in early-stage disease in the ADAURA Phase III trial , locally advanced stages in the LAURA Phase III trial and late-stage disease in the FLAURA Phase III trial and FLAURA2 Phase III trial.

As part of AstraZeneca’s ongoing commitment to treating patients as early as possible in lung cancer, Tagrisso is also being investigated in the neoadjuvant setting in the NeoADAURA Phase III trial with results expected later this year and in the early-stage adjuvant resectable setting in the ADAURA2 Phase III trial.

The Company is also researching ways to address tumour mechanisms of resistance through the SAVANNAH and ORCHARD Phase II trials, and the SAFFRON Phase III trial, which test Tagrisso plus savolitinib, an oral, potent and highly selective MET TKI, as well as other potential new medicines.

On June 26, 2024 AbbVie (NYSE: ABBV) reported that the U.S. Food and Drug Administration (FDA) has approved EPKINLY (epcoritamab-bysp) as the first and only T-cell engaging bispecific antibody administered subcutaneously for the treatment of adults with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of prior therapy (Press release, AbbVie, JUN 26, 2024, View Source [SID1234644557]). This indication is approved under the FDA’s Accelerated Approval program based on overall response rate (ORR) and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

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"The FDA approval of EPKINLY offers a new treatment option for relapsed or refractory follicular lymphoma, particularly following failure of other therapies," said Mariana Cota Stirner, M.D., Ph.D., vice president, therapeutic area head for hematology, AbbVie. "EPKINLY treatment has shown deep and durable responses for many patients. Subcutaneous dosing offers convenience, and EPKINLY can be given to patients without mandatory hospitalization using a 3-step-up dosing regimen. We believe that EPKINLY has the potential to be a core therapy in the treatment of multiple B-cell malignancies and furthers our company mission to advance research to raise standards of care for patients with cancer."

Epcoritamab is being co-developed by AbbVie and Genmab as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization.

FL is typically an indolent (or slow-growing) form of non-Hodgkin’s lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL accounting for 20-30% of all cases.2 About 15,000 people develop FL each year in the U.S.3 and it is considered incurable with current standard of care therapies.4 Patients often relapse and, with each relapse the remission and time to next treatment is shorter.5 Over time, transformation to DLBCL, an aggressive form of NHL associated with poor survival outcomes, can occur in more than 25% of FL patients.6

"Patients with relapsed or refractory follicular lymphoma face significant treatment challenges, and there is currently no clear standard of care treatment available across practice settings," said Jeff Sharman, M.D., Disease Chair, Hematology Research, Sarah Cannon Research Institute (SCRI) at Willamette Valley Cancer Institute in Eugene, Oregon. "The responses observed in the follicular lymphoma cohort of the EPCORE NHL-1 clinical trial, as well as in patients with relapsed or refractory DLBCL from the trial, show the potential of EPKINLY to serve as an important treatment option for these patients."

"People living with follicular lymphoma are in need of additional options when their cancer returns," said Lee Greenberger, Ph.D., Chief Scientific Officer at The Leukemia & Lymphoma Society. "Today’s approval is welcome news for patients, as it provides another tool in the physician arsenal for this difficult-to-treat form of cancer."

FDA approval is based on results from the Phase 1/2 EPCORE NHL-1 clinical trial, which evaluated the safety and efficacy of EPKINLY in 127 adult patients with R/R FL who previously received a median of three lines of therapy and with 70% having double refractory disease. Results and safety findings include:

The study results showed an ORR – the study’s primary endpoint − of 82%, a complete response (CR) rate of 60% and a partial response (PR) rate of 22%.
At a median follow-up of 14.8 months among responders, more than half of patients who responded to treatment in the study remained responsive to treatment (i.e., median duration of response was not reached).
Safety was evaluated in a total of 213 patients:
The safety of EPKINLY at a target dose of 48 mg was evaluated in EPCORE NHL-1, a single-arm study of 127 patients who received a 2-step up dosage schedule.
The most common (≥20%) adverse reactions were injection site reactions, cytokine release syndrome (CRS), COVID-19, fatigue, upper respiratory tract infection, musculoskeletal pain, rash, diarrhea, fever, cough, and headache.
A separate dose optimization cohort evaluated 86 patients at the recommended 3-step dosage schedule for CRS mitigation. For the first full dose of this 3-step regimen, mandatory hospitalization was not required. There were no grade 3 CRS observed in patients with FL who received EPKINLY with the 3 step-up dosage schedule.
The prescribing information has a Boxed Warning for serious or life-threatening CRS and immune effector cell-associated neurotoxicity syndrome (ICANS). Warnings and precautions include infections, cytopenias, and embryo-fetal toxicity. Please see additional Important Safety Information, below.

About the Phase 1/2 EPCORE NHL-1 Trial
EPCORE NHL-1 is an open-label, multi-center safety and preliminary efficacy trial of epcoritamab that consists of three parts: a dose escalation part; an expansion part; and an optimization part. The trial was designed to evaluate subcutaneous epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin’s lymphoma (B-NHL), including FL after two or more lines of systemic therapy. In the expansion part, additional patients were enrolled to further explore the safety and efficacy of epcoritamab in three cohorts of patients with different types of relapsed/refractory B-NHLs who have limited therapeutic options. This cohort generated pivotal data from patients with FL and DLBCL. The optimization part evaluates the potential for alternative step-up dosing regimens to help further minimize Grade 2 cytokine release syndrome (CRS) and mitigate Grade ≥3 CRS. The primary endpoint of the expansion part was ORR as assessed by an IRC. Secondary efficacy endpoints included duration of response, complete response rate, duration of complete response, progression-free survival, and time to response as determined by the Lugano criteria. Overall survival, time to next therapy, and rate of minimal residual disease negativity were also evaluated as secondary efficacy endpoints. The primary endpoint of the optimization part was the rate of ≥ Grade 2 CRS events and all grade CRS events from first dose of epcoritamab through 7 days following administration of the second full dose of epcoritamab.

NCCN Clinical Practice Guidelines
The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) for "B-Cell Lymphomas" were recently updated (Version 2.2024) to add EPKINLY as a Category 2A, preferred recommendation for third-line and subsequent therapy for patients with FL.

About EPKINLY (epcoritamab-bysp)
EPKINLY is a prescription medicine used to treat adults with certain types of diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, or follicular lymphoma (FL) that has come back or that did not respond to previous treatment after receiving 2 or more treatments. EPKINLY is approved based on patient response data. Studies are ongoing to confirm the clinical benefit of EPKINLY. It is not known if EPKINLY is safe and effective in children.

Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.7

Epcoritamab (approved under the brand name EPKINLY in the United States and TEPKINLY in the European Union) has received regulatory approval in certain lymphoma indications in several countries.

AbbVie will continue to pursue regulatory submissions for epcoritamab across international markets. Both Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy and in combination across lines of therapy in a range of hematologic malignancies. Please visit clinicaltrials.gov for more information.

EPKINLY (epcoritamab-bysp) U.S. IMPORTANT SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

Important Warnings—EPKINLY can cause serious side effects, including:

Cytokine release syndrome (CRS), which is common during treatment with EPKINLY and can be serious or life-threatening. To help reduce your risk of CRS, you will receive EPKINLY on a step-up dosing schedule (when you receive 2 or 3 smaller step-up doses of EPKINLY before your first full dose during your first cycle of treatment), and you may also receive other medicines before and for 3 days after receiving EPKINLY. If your dose of EPKINLY is delayed for any reason, you may need to repeat the step-up dosing schedule.
Neurologic problems that can be life-threatening and lead to death. Neurologic problems may happen days or weeks after you receive EPKINLY.
People with DLBCL or high-grade B-cell lymphoma should be hospitalized for 24 hours after receiving their first full dose of EPKINLY on day 15 of cycle 1 due to the risk of CRS and neurologic problems.

Tell your healthcare provider or get medical help right away if you develop a fever of 100.4°F (38°C) or higher; dizziness or lightheadedness; trouble breathing; chills; fast heartbeat; feeling anxious; headache; confusion; shaking (tremors); problems with balance and movement, such as trouble walking; trouble speaking or writing; confusion and disorientation; drowsiness, tiredness or lack of energy; muscle weakness; seizures; or memory loss. These may be symptoms of CRS or neurologic problems. If you have any symptoms that impair consciousness, do not drive or use heavy machinery or do other dangerous activities until your symptoms go away.

EPKINLY can cause other serious side effects, including:

Infections that may lead to death. Your healthcare provider will check you for signs and symptoms of infection before and during treatment and treat you as needed if you develop an infection. You should receive medicines from your healthcare provider before you start treatment to help prevent infection. Tell your healthcare provider right away if you develop any symptoms of infection during treatment, including fever of 100.4°F (38°C) or higher, cough, chest pain, tiredness, shortness of breath, painful rash, sore throat, pain during urination, or feeling weak or generally unwell.
Low blood cell counts, which can be serious or severe. Your healthcare provider will check your blood cell counts during treatment. EPKINLY may cause low blood cell counts, including low white blood cells (neutropenia), which can increase your risk for infection; low red blood cells (anemia), which can cause tiredness and shortness of breath; and low platelets (thrombocytopenia), which can cause bruising or bleeding problems.
Your healthcare provider will monitor you for symptoms of CRS, neurologic problems, infections, and low blood cell counts during treatment with EPKINLY. Your healthcare provider may temporarily stop or completely stop treatment with EPKINLY if you develop certain side effects.

Before you receive EPKINLY, tell your healthcare provider about all your medical conditions, including if you have an infection, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. If you receive EPKINLY while pregnant, it may harm your unborn baby. If you are a female who can become pregnant, your healthcare provider should do a pregnancy test before you start treatment with EPKINLY and you should use effective birth control (contraception) during treatment and for 4 months after your last dose of EPKINLY. Tell your healthcare provider if you become pregnant or think that you may be pregnant during treatment with EPKINLY. Do not breastfeed during treatment with EPKINLY and for 4 months after your last dose of EPKINLY.

In DLBCL or high-grade B-cell lymphoma, the most common side effects of EPKINLY include CRS, tiredness, muscle and bone pain, injection site reactions, fever, stomach-area (abdominal) pain, nausea, and diarrhea. The most common severe abnormal laboratory test results include decreased white blood cells, decreased red blood cells, and decreased platelets.

In follicular lymphoma the most common side effects of EPKINLY include injection site reactions, CRS, COVID-19, tiredness, upper respiratory tract infections, muscle and bone pain, rash, diarrhea, fever, cough, and headache. The most common severe abnormal laboratory test results include decreased white blood cells and decreased red blood cells.

These are not all of the possible side effects of EPKINLY. Call your doctor for medical advice about side effects. You are encouraged to report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch or to Genmab US, Inc. at 1-855-4GENMAB (1-855-443-6622).

Please see Medication Guide, including Important Warnings.

Globally, prescribing information varies; refer to the individual country product label for complete information.

About AbbVie in Oncology
At AbbVie, we are committed to transforming standards of care for patients living with difficult-to-treat cancers. We are advancing a dynamic pipeline of investigational therapies across a range of cancer types in both blood cancers and solid tumors. We are focusing on creating targeted medicines that either impede the reproduction of cancer cells or enable their elimination. We achieve this through various, targeted treatment modalities including Antibody Drug Conjugates (ADCs), Immuno-Oncology, and bi-specific and CAR-T platforms. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potential breakthrough medicines.

Today, our expansive oncology portfolio is comprised of approved and investigational treatments for a wide range of blood and solid tumors. We are evaluating more than 20 investigational medicines across some of the world’s most widespread and debilitating cancers. As we work to have a remarkable impact on people’s lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit us at View Source

On June 25, 2024 Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported that Steven Kelly, President and Chief Executive Officer, will participate in a fireside chat at the Stifel 2024 Cell Therapy Forum on Tuesday, July 9th at 9:10 am ET (Press release, Carisma Therapeutics, JUN 25, 2024, View Source [SID1234644540]).

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An audio webcast of the event will be available on the Company’s Investor Events section of the Investor Relations webpage and will be archived for a limited time following the event.

On June 25, 2024 Exsilio Therapeutics ("Exsilio"), a biotechnology company developing genomic medicines for a broad range of diseases, reported its emergence from stealth with $82 million in Series A financing (Press release, Exsilio Therapeutics, JUN 25, 2024, View Source [SID1234644541]). The funding was co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight. Exsilio was seed-funded by OrbiMed.

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Exsilio will use proceeds from the financing to advance its genomic medicines based on naturally occurring, programmable genetic elements that can precisely insert new genes into a cell through mRNA intermediates. Exsilio’s interdisciplinary team has built a platform that combines predictive in silico modeling and wet lab-based experimentation to discover and engineer such elements for integration of therapeutic genes into safe harbor sites. Because Exsilio’s medicines are encoded in mRNA, they can be delivered using existing lipid nanoparticle (LNP) platforms that are safe, efficient, scalable, and cost-effective, and can be redosed and titrated with curative intent.

"mRNA-based medicines allow for a software-like approach to creating new medicines," said Tal Zaks, M.D., Ph.D., who serves as Exsilio’s Chairman and Interim Chief Executive Officer. "Exsilio’s approach leverages the advantages of mRNA and goes a step further by encoding genes that integrate permanently, offering the possibility of curative rather than transient effects. The ability to insert whole genes with a repeatable and titratable approach should allow us to treat genetic diseases irrespective of the patient’s individual mutation. This financing will help us advance our genomic medicines and select promising lead candidates so that we can bring much-needed new options to patients."

"We were captivated by Exsilio’s genomic medicines approach that stands to enable large-gene integration in a safe and redosable manner," said Aaron Nelson, Managing Director at Novartis Venture Fund and Exsilio Board Member. "Through this significant investment, Exsilio will be able to select and advance promising candidates for difficult-to-treat diseases."

"We believe that the vision of safely and durably integrating therapeutic genes into a patient’s genome requires using RNA-based payloads that can leverage clinically validated non-viral gene delivery technologies," said Henry Chen, Managing Partner of Delos Capital and Exsilio Board Member. "Exsilio is bringing together a singular group of people to help establish this new pillar of genomic medicine."