On October 2, 2023 Takeda (TSE:4502/NYSE:TAK) reported that, following discussions with the U.S. Food and Drug Administration (FDA), it will be working with the FDA towards a voluntary withdrawal of EXKIVITY (mobocertinib) in the U.S. for adult patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) locally advanced or metastatic non-small cell lung cancer (NSCLC) whose disease has progressed on or after platinum-based chemotherapy (Press release, Takeda, OCT 2, 2023, View Source [SID1234635586]). Takeda intends to similarly initiate voluntary withdrawal globally where EXKIVITY is approved and is working with regulators in other countries where it is currently available on next steps.

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This decision was based on the outcome of the Phase 3 EXCLAIM-2 confirmatory trial, which did not meet its primary endpoint and thus did not fulfill the confirmatory data requirements of the Accelerated Approval granted by the U.S. FDA nor the conditional marketing approvals granted in other countries.

The EXCLAIM-2 trial was a Phase 3, multicenter, open-label study designed to investigate the safety and efficacy of EXKIVITY as a monotherapy versus platinum-based chemotherapy in first-line EGFR Exon20 insertion+ locally advanced or metastatic NSCLC. No new safety signals were observed in the EXCLAIM-2 trial. Full data from the trial will be presented at an upcoming medical meeting or published in a peer-reviewed journal.

"Our steadfast commitment to pursue solutions for people with high unmet needs led us to develop and launch EXKIVITY as the first oral therapy designed for patients with EGFR Exon20 insertion+ metastatic NSCLC," said Awny Farajallah, MD, head of Global Medical Affairs Oncology at Takeda. "We have been fortunate to witness the impact EXKIVITY has had on this previously underserved population and are encouraged to see the advancements made since its approval to introduce new therapies for these patients. We hope that findings from the EXCLAIM-2 study will inform future research and development for this disease."

Takeda is committed to ensuring patients receiving EXKIVITY can maintain access, as appropriate and in consultation with their healthcare provider. We are actively assessing access mechanisms with regulatory authorities. Patients currently being treated with EXKIVITY should consult their healthcare provider. For questions related to ongoing access, please contact us at [email protected].

Takeda will continue to assess the impact of the withdrawal and update our full-year, consolidated forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023), as appropriate and necessary.

About EXKIVITY (mobocertinib)
EXKIVITY is a first-in-class, oral tyrosine kinase inhibitor (TKI) specifically designed to selectively target epidermal growth factor receptor (EGFR) Exon20 insertion mutations. EXKIVITY was approved based on the results from the platinum-pretreated population in the Phase 1/2 trial of EXKIVITY in patients with EGFR Exon20 insertion mutation-positive (insertion+) metastatic NSCLC. For more information about EXKIVITY in the U.S., healthcare professionals may visit www.exkivity-update.com. For the U.S. Prescribing Information, including the Boxed Warning, please visit www.exkivity-update.com/prescribing-information.

About EGFR Exon20 Insertion+ NSCLC
Non-small cell lung cancer (NSCLC) is the most common form of lung cancer, accounting for approximately 85% of the estimated 2.2 million new cases of lung cancer diagnosed each year worldwide, according to the World Health Organization.1-2 Patients with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) NSCLC make up approximately 1-2% of patients with NSCLC, and the disease is more common in Asian populations compared to Western populations.3-7 This mutation carries a worse prognosis than other EGFR mutations, as EGFR tyrosine kinase inhibitors (TKIs) – which do not specifically target EGFR Exon20 insertions – and chemotherapy provide limited benefit for these patients.

Takeda is committed to continuing research and development to meet the needs of the lung cancer community through the discovery and delivery of transformative medicines.

On October 2, 2023 Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, reported that Abbey Jenkins, President and Chief Executive Officer, will present its corporate highlights at the annual Cell & Gene Meeting on the Mesa to be held October 10-12 in Carlsbad, California, and livestreamed globally (Press release, Gamida Cell, OCT 2, 2023, View Source [SID1234635585]). Ms. Jenkins will also participate in a panel titled "A record setting year for cell and gene therapies – how do we keep the momentum going?" at the event.

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During Gamida Cell’s corporate presentation, Ms. Jenkins will share commercial launch updates for Omisirge (omidubicel-onlv), the company’s allogeneic stem cell therapy, and an overview of the market opportunity.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is an annual three-day conference featuring more than 100 presentations by companies highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

Virtual attendance is available and includes a livestream of Gamida Cell’s presentation and the ability to view all conference sessions on-demand. Please visit View Source for more information.

The following are details regarding Gamida Cell’s presentation at the conference:

Date: Wednesday, October 11
Time: 4:45 – 5:00 p.m. PT
Location: Aseptic Technologies Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

The following are details regarding Ms. Jenkins’ panel participation at the conference:

Panel: A record setting year for cell and gene therapies – how do we keep the momentum going?
Date: Wednesday, October 11
Time: 3:15 – 4:15 p.m. PT
Location: Aseptic Technologies Ballroom, Park Hyatt Aviara Resort, 7100 Aviara Resort Dr., Carlsbad, CA 92011

Complimentary attendance at this event is available for credentialed investors and members of the media only. Investors should contact Savannah Bryant at [email protected] and interested media should contact Stephen Majors at [email protected].

Omisirge Indication

Omisirge is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.

Important Safety Information for Omisirge

BOXED WARNING: INFUSION REACTIONS, GRAFT VERSUS HOST DISEASE, ENGRAFTMENT SYNDROME, AND GRAFT FAILURE

Infusion reactions may be fatal. Monitor patients during infusion and discontinue for severe reactions. Use is contraindicated in patients with known allergy to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin or bovine material.
Graft-versus-Host Disease may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD.
Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids.
Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery.
Contraindications

OMISIRGE is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine products.

Warnings and Precautions

Hypersensitivity Reactions

Allergic reactions may occur with the infusion of OMISIRGE. Reactions include bronchospasm, wheezing, angioedema, pruritis and hives. Serious hypersensitivity reactions, including anaphylaxis, may be due to DMSO, residual gentamicin, Dextran 40, human serum albumin (HSA) and bovine material in OMISIRGE. OMISIRGE may contain residual antibiotics if the cord blood donor was exposed to antibiotics in utero. Patients with a history of allergic reactions to antibiotics should be monitored for allergic reactions following OMISIRGE administration.

Infusion Reactions

Infusion reactions occurred following OMISIRGE infusion, including hypertension, mucosal inflammation, dysphagia, dyspnea, vomiting, and gastrointestinal toxicity. Premedication with antipyretics, histamine antagonists, and corticosteroids may reduce the incidence and intensity of infusion reactions. In patients transplanted with OMISIRGE in clinical trials, 47% (55/117) patients had an infusion reaction of any severity. Grade 3-4 infusion reactions were reported in 15% (18/117) patients. Infusion reactions may begin within minutes of the start of infusion of OMISIRGE, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion. Monitor patients for signs and symptoms of infusion reactions during and after OMISIRGE administration. When a reaction occurs, pause the infusion and institute supportive care as needed.

Graft-versus-Host Disease

Acute and chronic GvHD, including life-threatening and fatal cases, occurred following treatment with OMISIRGE. In patients transplanted with OMISIRGE Grade II-IV acute GvHD was reported in 58% (68/117). Grade III-IV acute GvHD was reported in 17% (20/117). Chronic GvHD occurred in 35% (41/117) of patients. Acute GvHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Patients treated with OMISIRGE should receive immunosuppressive drugs to decrease the risk of GvHD, be monitored for signs and symptoms of GvHD, and treated if GvHD develops.

Engraftment Syndrome

Engraftment syndrome may occur because OMISIRGE is derived from umbilical cord blood. Monitor patients for unexplained fever, rash, hypoxemia, weight gain, and pulmonary infiltrates in the peri-engraftment period. Treat with corticosteroids as soon as engraftment syndrome is recognized to ameliorate symptoms. If untreated, engraftment syndrome may progress to multiorgan failure and death.

Graft Failure

Primary graft failure occurred in 3% (4/117) of patients in OMISIRGE clinical trials. Primary graft failure, which may be fatal, is defined as failure to achieve an absolute neutrophil count greater than 500 per microliter blood by Day 42 after transplantation. Immunologic rejection is the primary cause of graft failure. Monitor patients for laboratory evidence of hematopoietic recovery.

Malignancies of Donor Origin

Two patients treated with OMISIRGE developed post-transplant lymphoproliferative disorder (PTLD) in the second-year post-transplant. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. The etiology is thought to be donor lymphoid cells transformed by Epstein-Barr virus (EBV). Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. One patient treated with OMISIRGE developed a donor-cell derived myelodysplastic syndrome (MDS) during the fourth-year post-transplant. The natural history is presumed to be the same as that for de novo MDS. Monitor life-long for secondary malignancies. If a secondary malignancy occurs, contact Gamida Cell at (844) 477-7478.

Transmission of Serious Infections

Transmission of infectious disease may occur because OMISIRGE is derived from umbilical cord blood. Disease may be caused by known or unknown infectious agents. Donors are screened for increased risk of infection, clinical evidence of sepsis, and communicable disease risks associated with xenotransplantation. Maternal and infant donor blood is tested for evidence of donor infection. See full Prescribing Information, Warnings and Precautions, Transmission of Serious Infections for list of testing performed. OMISIRGE is tested for sterility, endotoxin, and mycoplasma. There may be an effect on the reliability of the sterility test results if the cord blood donor was exposed to antibiotics in utero. Product manufacturing includes bovine-derived reagents. All animal-derived reagents are tested for animal viruses, bacteria, fungi, and mycoplasma before use. These measures do not eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Test results may be found on the container label and/or in accompanying records. If final sterility results are not available at the time of use, Quality Assurance will communicate any positive results from sterility testing to the physician. Report the occurrence of transmitted infection to Gamida Cell at (844) 477-7478.

Transmission of Rare Genetic Diseases

OMISIRGE may transmit rare genetic diseases involving the hematopoietic system because it is derived from umbilical cord blood. Cord blood donors have been screened to exclude donors with sickle cell anemia, and anemias due to abnormalities in hemoglobins C, D, and E. Because of the age of the donor at the time cord blood collection takes place, the ability to exclude rare genetic diseases is severely limited.

ADVERSE REACTIONS

The most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion reaction.

Please see full Prescribing Information, including Boxed Warning.

On October 2, 2023 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA testing, reported the submission of the first module of its premarket approval (PMA) application to the U.S. Food and Drug Administration (FDA) for Signatera, Natera’s personalized and tumor-informed molecular residual disease (MRD) test, as a companion diagnostic (CDx) assay for patients with muscle-invasive bladder cancer (MIBC) (Press release, Natera, OCT 2, 2023, View Source [SID1234635584]).

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This module, submitted on September 28, included the required documentation regarding Natera’s manufacturing and quality control systems, which the Company expects will support all future indications for Signatera as well as other Natera products. The remaining modules for the Signatera MIBC indication, including software, analytical and clinical validation data, are expected to be submitted through 2025 upon completion of the ongoing registrational trial.

"With this initial step towards a PMA for Signatera, we are continuing to build on our longstanding engagement with the FDA," said Steve Chapman, chief executive officer of Natera. "This milestone reflects the significant efforts of our team in developing an FDA-grade quality and manufacturing system, a strong foundation designed to support future regulatory submissions across disease indications for Signatera and across product lines."

Signatera has previously been granted four Breakthrough Device Designations by the FDA, including the CDx claim in MIBC submitted in the PMA module package.

About Signatera

Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease (MRD) assessment in patients previously diagnosed with cancer. The test is available for both clinical and research use, and has been granted four Breakthrough Device Designations by the FDA for multiple cancer types and indications. The Signatera test is personalized and tumor-informed, providing each individual with a customized blood test tailored to fit the unique signature of clonal mutations found in that individual’s tumor. Signatera is intended to detect and quantify cancer left in the body, at levels down to a single tumor molecule in a tube of blood, to help identify recurrence earlier and optimize treatment decisions. The test has not been cleared or approved by the US Food and Drug Administration (FDA).

On October 2, 2023 Nerviano Medical Sciences S.r.l., a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc., a wholly owned subsidiary of NMS Group, focused on the discovery and early development of oncology drugs and the largest oncological R&D company in Italy, reported that it will present initial data from the PARP1 inhibitor Phase 1 studies at the upcoming AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), being held October 11-15 in Boston, MA (Press release, Nerviano Medical Sciences, OCT 2, 2023, View Source [SID1234635583]).

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The PARPA Phase 1 studies, PARPA-293-001 and PARPA-293-002 are evaluating NMS-03305293 (NMS-293), an oral, brain-penetrant PARP-1 selective inhibitor as single agent or in combination with temozolomide, in relapsed/refractory selected solid tumor types (PARPA-293-001) or patients with High-grade gliomas (PARPA-293-002, including glioblastoma). On September 14, 2022, NMS Group entered into a worldwide option agreement with Merck KGaA, Darmstadt, Germany for the development and commercialization of NMS-293.

Details for the plenary and poster presentations are as follows:

Title: Initial Results from 2 Phase I Studies of NMS-03305293, a Selective PARP1 Inhibitor
Abstract number: 37568
Poster number: LB_A12
Session: Poster Session A
Session date and time: Thursday, October 12 | 12:30 p.m. – 4:00 p.m. ET
Session location: Level 2, Exhibit Hall D

On October 2, 2023 Aviko Radiopharmaceuticals, a Deerfield Management-founded biotechnology company developing medicines to unlock the potential of boron neutron capture therapy (BNCT), and Neutron Therapeutics, a leading developer of accelerator-based neutron systems for targeted radiation therapy of solid tumors, reported an exclusive, strategic partnership with Leo Cancer Care, a company developing upright patient positioning systems for radiation therapy (Press release, Aviko Radiopharmaceuticals, OCT 2, 2023, View Source [SID1234635582]). The goal of the partnership between the three companies is to expand BNCT as a potential treatment for different types of cancer.

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"We believe that upright radiotherapy results in a better experience for patients compared to supine radiotherapy. We are excited to collaborate with Aviko and Neutron as we advance BNCT to potentially treat a variety of solid tumors."

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Leo Cancer Care’s upright patient positioning system will be integrated into Neutron’s nuBeam therapy platform, which is a compact, in-hospital neutron source designed to replace legacy nuclear reactors for BNCT. The companies will speak about their initiative today at 3:45 p.m. PT at booth #4207 at the Annual Meeting of the American Society for Radiation Oncology (ASTRO), which is being held in San Diego.

"The incorporation of Leo’s upright patient positioning system with Neutron Therapeutics’ nuBeam neutron accelerator has the potential to increase the versatility and impact of BNCT for patients, physicians, treatment centers and the overall healthcare system," said Dave Greenwald, Ph.D., chief executive officer of Aviko and vice president of business development at Deerfield. "This strategic partnership is an important moment for our three companies as we work together to build a complete suite of both therapies and technologies that have the potential to provide boron neutron capture therapy to patients in need."

The BNCT treatment under development by Aviko Radiopharmaceuticals and Neutron Therapeutics involves an investigational non-toxic boron medicine that is designed to accumulate in cancer cells. The boron medicine remains inert until it is irradiated by low-energy neutrons at the site of the tumor, releasing alpha particles that destroy cancerous cells. Positioning the patient in relation to the neutron beam is important to support patient safety and the clinical activity of BNCT. Traditionally, patients have been supine when receiving BNCT and other forms of radiation therapy. Leo Cancer Care has developed an upright patient positioning system that is designed to allow for greater efficiency and precision when delivering neutrons to the tumor compared to supine positioning systems.

"With our upright patient positioning system, all areas of the body can be positioned close to the neutron source to receive a precise delivery of neutrons at the site of the tumor," said Stephen Towe, chief executive officer of Leo Cancer Care. "We believe that upright radiotherapy results in a better experience for patients compared to supine radiotherapy. We are excited to collaborate with Aviko and Neutron as we advance BNCT to potentially treat a variety of solid tumors."

Disclaimer

The products being developed by Aviko Radiopharmaceuticals, Neutron Therapeutics and Leo Cancer Care are in development and are limited to investigational use. These products have not received marketing authorization from the FDA and are not commercially available.

About Boron Neutron Capture Therapy (BNCT)

Boron neutron capture therapy (BNCT) is a precision medicine approach that is being advanced to treat cancer by destroying cancerous cells while minimizing the impact to surrounding healthy tissues. BNCT uses a non-toxic boron medicine that is administered to the patient. The medicine is designed to accumulate in cancer cells while being quickly cleared from normal tissues in the body. The boron medicine remains inert until it is irradiated at the site of the tumor with low-energy neutrons, releasing alpha particles that can destroy cancerous cells. BNCT may also have applications in treating other diseases. Currently there are 21 BNCT clinics worldwide, primarily in Asia and Europe.