On May 30, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAN-3110, a next generation oncolytic viral immunotherapy, for the treatment of recurrent high-grade glioma (rHGG) (Press release, Candel Therapeutics, MAY 30, 2024, View Source [SID1234643880]). Glioblastoma (GBM) is the most common and aggressive form of high-grade glioma.

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CAN-3110 was previously granted Fast Track Designation by the FDA for the treatment of rHGG. Candel is currently evaluating CAN-3110 in a multi-institutional phase 1b clinical trial in rHGG. Results from Arm A of the ongoing phase 1b clinical trial in rHGG exploring the clinical and biomarker activity of a single dose administration of CAN-3110 were published in Nature, demonstrating a strong anti-tumoral response associated with extended survival.1 The Company will present data on the feasibility and safety of multiple doses of CAN-3110 in patients with rHGG, supported by the Break Through Cancer Foundation, in a trials-in-progress poster presentation at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.

"Building on the momentum of the FDA’s Fast Track Designation, recently granted to this program, the Orphan Drug Designation for CAN-3110 further reinforces the potential of this therapy and underscores the urgent need for novel and effective treatments for patients with rHGG," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "This designation not only reinforces our commitment to offering new hope and potential patient treatment options, but it also enables us to leverage development incentives and accelerate our efforts to evaluate new indications in the clinic. We are continuing our work in the phase 1b clinical trial of CAN-3110 and look forward to sharing further clinical updates in the second half of 2024."

E. Antonio Chiocca, M.D., Ph.D., Chair of the Department of Neurosurgery at Brigham and Women’s Hospital, Professor at Harvard Medical School, and Principal Investigator on the phase 1b clinical trial, said: "We are grateful to the FDA for recognizing the urgent need for new treatments in rHGG. Patients, and their families, affected by this disease, face immense challenges that the standard of care and conventional therapies have failed to adequately address. The early clinical data suggests that CAN-3110’s unique dual mechanism of action, combining oncolysis and immune activation, has the potential to overcome these challenges for rHGG patients."

About Orphan Drug Designation

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Orphan Drug Designation provides certain financial incentives to support clinical development, and the potential for up to seven years of marketing exclusivity for the product for the designated orphan indication in the United States if the product is ultimately approved for its designated indication.

About CAN-3110

CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) oncolytic viral immunotherapy candidate designed with dual activity for oncolysis and immune activation in a single therapeutic. Its activity is designed to be conditional to the expression of Nestin in cancer cells. CAN-3110 is being evaluated in a phase 1b clinical trial in patients with recurrent high-grade glioma (rHGG). In October 2023, the Company announced that Nature published results from this ongoing clinical trial. CAN-3110 was well tolerated with no dose-limiting toxicity reported. In the clinical trial, the investigators observed median overall survival after a single CAN-3110 injection of more than 12 months in this therapy-resistant condition.1 The Company and academic collaborators are currently evaluating the effects of multiple CAN-3110 injections in rHGG, supported by the Break Through Cancer Foundation. CAN-3110 has previously received FDA Fast Track Designation for the treatment of rHGG.

Details on the CAN-3110 ASCO (Free ASCO Whitepaper) poster are as follows:

The trials-in-progress poster presentation will focus on cohort C of the ongoing phase 1b clinical trial of CAN-3110 in patients with rGBM, the most common form of rHGG. Previously presented data showed the ability of a single CAN-3110 injection to double median overall survival (mOS) in the rGBM population, as compared to contemporary control cohorts. Patients presenting with seropositivity to HSV1, reached mOS of 14 months, largely exceeding expected survival of 6 to 9 months or less for this population.

In cohort C, supported by the Break Through Cancer Foundation, two cohorts of 12 patients will receive up to six injections of CAN-3110 over a four-month period. Cohort C is currently exploring the safety and tolerability of CAN-3110 in patients with rGBM. Patients in cohort C are treated with up to six doses of CAN-3110 delivered by stereotactic injections on days 0, 15, 30, 60, 90 & 120, along with concomitant biopsies over the four-month treatment period.

Two sub-cohorts (1&2) of patients who will receive 1×107 pfu or 1×108 pfu per injection of CAN-3110 have been planned for six patients per cohort, using a Bayesian optimal interval (BOIN) design for dose ranging.

Six patients have accrued, completing cohort 1; no dose-limiting toxicities or severe adverse events were observed.
More than 300 core biopsies were obtained from all six patients across the planned time points.
Biopsies were processed for "-omic" analyses, including single-cell RNA sequencing, proteomics/phophoproteomic/immunopeptidomics, metabolomics, spatial transcriptomics, and cell profiling.
ASCO Presentation details are as follows:

Trials-in-Progress Poster Presentation Title: Longitudinal stereotactic injections of oncolytic immunoactivating rQNestin34.5v.2 (CAN-3110) with concomitant biopsies for "-omic" analyses in recurrent glioblastoma (GBM)
Presenter: David A. Reardon, MD, Professor of Medicine at Harvard Medical School; Clinical Director, Center for Neuro-Oncology at Dana Farber Cancer Institute
Session Title: Poster Session – Central Nervous System Tumors
Session Date/Time: Saturday, June 1, 2024; 9:00 AM – 12:00 PM CT

On May 30, 2024 Oncotelic Therapeutics, Inc (OTCQB:OTLC) reported its CEO- Dr. Vuong Trieu will be participating at the BIO International 2024 meeting on June3-6, San Diego (Press release, Oncotelic, MAY 30, 2024, View Source [SID1234643879]). The BIO International Convention is the largest and most comprehensive event for biotechnology, representing the full ecosystem of biotech with more than 18,500 industry leaders from across the globe.

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On May 30, 2024 Blueprint Medicines Corporation (Nasdaq: BPMC) reported multiple upcoming datasets across two key conferences that reinforce the significant real-world burden of systemic mastocytosis (SM), and highlight the durable clinical outcomes of AYVAKIT/AYVAKYT (avapritinib) across the spectrum of the disease (Press release, Blueprint Medicines, MAY 30, 2024, View Source [SID1234643878]). The presentations, which build on over a decade of pioneering research with clinical experts and patient advocates, will be reported at the following meetings:

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European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024, May 31 to June 3
European Hematology Association 2024 (EHA24) Hybrid Congress, June 13 to 16
"Our data reinforce the efficacy and safety of prolonged durations of AYVAKIT therapy, and highlight the urgency to diagnose and treat patients living with systemic mastocytosis," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "Based on unprecedented clinical datasets and the strength of our ongoing commercial launches in the U.S. and Europe, we have made significant progress toward establishing AYVAKIT as the global standard of care. We look forward to continued collaboration with the SM community to redefine what ‘well-controlled’ means for patients across the spectrum of the disease."

PIONEER: With a median follow-up of more than two years, AYVAKIT showed durable efficacy and a favorable safety profile in patients with ISM. Safety data were consistent for the small number of patients (~10 percent) who dose escalated to 50 mg once daily.

AYVAKIT led to sustained improvements in total symptom score and all symptom domains (assessed by the Indolent Systemic Mastocytosis Symptom Assessment Form), as well as quality of life (assessed by the Mastocytosis Quality of Life Score).
AYVAKIT had a favorable safety and tolerability profile, with patients receiving up to four-plus years of treatment. The most common treatment-related adverse event (AE) was edema, with the majority reported as Grade 1, and the rate of treatment-related AEs leading to discontinuations remained low (3 percent).
PATHFINDER: With a median follow-up of more than three years, the median overall survival (OS) for AYVAKIT has not been reached in patients with advanced SM.

The median OS has not yet been reached regardless of disease subtype or prior therapy, reflecting the durable clinical benefits of AYVAKIT. In advanced SM, the historical median OS has ranged from less than six months to about 3.5 years.1
The overall response rate2 was 73 percent, and the rate of complete remission with full or partial hematologic recovery was 29 percent.
AYVAKIT had a favorable benefit/risk profile consistent with previously reported data. Common treatment-related AEs were periorbital edema, thrombocytopenia, peripheral edema and anemia.
PRISM: The largest European study to evaluate patient perspectives in SM reinforces the significant burden of disease, highlighting the urgency to treat.

The Perceptions, Realities & Insights on SM (PRISM) Survey – a collaborative research project led by Blueprint Medicines and involving disease experts and patient organizations across seven countries – further illustrates the substantial impact of SM on patients’ quality of life.
Patients reported debilitating symptoms, impaired physical and mental health functioning, meaningful impacts on the ability to work, and significant polypharmacy use.
Healthcare providers broadly recognized the burden of SM; 65 percent reported the disease affected patients’ lives "quite a bit" or "a great deal," and 58 percent reported patients lost employment opportunities.
At the start of their respective oral and poster sessions, data presentations will be made available in the "Science―Publications and Presentations" section of Blueprint Medicines’ website.

EAACI Congress 2024

Oral Presentation: Safety of Avapritinib in Indolent Systemic Mastocytosis (ISM): Longer Term Follow-up from the PIONEER Study (Abstract 000223)
Oral Presentation: The Burden of Indolent Systemic Mastocytosis in Europe: Results from the PRISM Patient Survey (Abstract 000405)
Oral Presentation: Identifying KIT D816V Mutation in Patients with Evidence of Systemic Mast Cell Activation (MCA) and Enriched for Hereditary Alpha-Typtasemia (HaT): Results from the PROSPECTOR Clinical Trial (Abstract 000403)
Poster Presentation: Healthcare Provider Perspectives on Management of European Patients with Systemic Mastocytosis (Abstract 000413)
EHA2024 Congress

Oral Presentation: Avapritinib in Patients with Advanced Systemic Mastocytosis (AdvSM): Efficacy and Safety Analysis from the Phase 2 PATHFINDER Study with 3-Year Follow-up (Abstract S224)
Poster Presentation: The Burden of Systemic Mastocytosis in Europe: Results from the PRISM Patient Survey (Abstract P1676)
ePoster Presentation: Patient Diagnostic Journey of Systemic Mastocytosis in Europe: Results from the PRISM Survey (Abstract P2292)
About Systemic Mastocytosis

Systemic mastocytosis (SM) is a rare disease driven by the KIT D816V mutation in about 95 percent of cases. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms across multiple organ systems. The vast majority of those affected have indolent systemic mastocytosis (ISM). A broad range of symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, frequently persist in patients with ISM despite treatment with multiple symptom-directed therapies. This burden of disease can lead to a profound, negative impact on quality of life. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Until 2023, there were no approved therapies for the treatment of ISM.

A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival.

About AYVAKIT (avapritinib)

AYVAKIT (avapritinib) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with ISM, adults with advanced SM, including ASM, SM-AHN and MCL, and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Under the brand name AYVAKYT (avapritinib), this medicine is approved by the European Commission for the treatment of adults with ISM with moderate to severe symptoms inadequately controlled on symptomatic treatment, adults with ASM, SM-AHN and MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. The therapy is not recommended for the treatment of patients with low platelet counts (less than 50,000/µL).

Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT.

To learn about ongoing or planned clinical trials, contact Blueprint Medicines at [email protected] or 1-888-BLU-PRNT (1-888-258-7768). Additional information is available at blueprintclinicaltrials.com or clinicaltrials.gov.

Important Safety Information

Intracranial Hemorrhage—Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In Advanced SM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study.

Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia.

Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH.

Permanently discontinue AYVAKIT if ICH of any grade occurs. In Advanced SM patients, a platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in Advanced SM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction.

Cognitive Effects—Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 28% of 148 Advanced SM patients (3% were Grade ≥3), and 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC (<1% were Grade 3). Depending on the severity and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.

Photosensitivity—AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment.

Embryo-Fetal Toxicity—AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks after the final dose.

Adverse Reactions—The most common adverse reactions (≥20%) in patients with Advanced SM were edema, diarrhea, nausea, and fatigue/asthenia.

The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.

Drug Interactions—Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided in patients with Advanced SM, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers.

To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA-1088 or View Source

Please click here to see the full Prescribing Information for AYVAKIT.

On May 30, 2024 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that Jack Khattar, President and CEO of Supernus Pharmaceuticals, will participate in a fireside chat at the Jefferies Global Healthcare Conference on Thursday, June 6, 2024, at 1:30 p.m. ET at the Marriott Marquis Hotel in New York City (Press release, Supernus, MAY 30, 2024, View Source [SID1234643877]).

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Investors interested in arranging a meeting with company management during the conference should contact the Jefferies conference coordinator. A live audio webcast of the presentation can be accessed here or by visiting Events & Presentations in the Investor Relations section on the Company’s website at www.supernus.com. An archived replay of the webcast will be available for 60 days on the Company’s website following the conference.

On May 30, 2024 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported that the Company’s CEO, Brian M. Culley, will present at the 2024 BIO International Convention, on Tuesday June 4th, 2024, at 2:30pm in Theater 3 (Press release, Lineage Cell Therapeutics, MAY 30, 2024, View Source [SID1234643875]). Company representatives are also hosting meetings with potential partners and collaborators to discuss opportunities for strategic alliances across Lineage’s novel pipeline of cell therapy transplant programs. The BIO International Convention is the world’s largest gathering of the biotechnology industry, along with industry-leading investor and partnering meetings held around the world, and is taking place June 3-6, 2024, at the San Diego Convention Center in San Diego, CA.

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About BIO

BIO is the world’s largest advocacy association representing biotechnology companies, academic and research institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products. Corporate members range from entrepreneurial companies developing a first product, to Fortune 500 multinationals. BIO also represents state and regional biotech associations, service providers to the industry, and academic centers. Our members help foster a healthy economy by creating good-paying, biotechnology jobs. We also host the largest cost-savings program in the life sciences industry, BIO Business Solutions, which saves nearly $700 million in aggregate for 4,500+ companies every year. For more information visit www.bio.org or follow the organization on X/Twitter @IAmBiotech.