Johnson & Johnson submits supplemental Biologics License Application to U.S. FDA seeking approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj)-based regimen for the treatment of patients with transplant-eligible, newly diagnosed multiple myeloma

On January 30, 2024 Johnson & Johnson reported the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval of a new indication for DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide and dexamethasone (D-VRd) for induction and consolidation treatment and with lenalidomide (D-R) for maintenance treatment of adult patients who are newly diagnosed with multiple myeloma (NDMM) and are eligible for autologous stem cell transplant (ASCT) (Press release, Johnson & Johnson, JAN 30, 2024, View Source;johnson-submits-supplemental-biologics-license-application-to-us-fda-seeking-approval-of-darzalex-faspro-daratumumab-and-hyaluronidase-fihj-based-regimen-for-the-treatment-of-patients-with-transplant-eligible-newly-302048440.html [SID1234639725]).

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This submission is supported by data from the Phase 3 PERSEUS (NCT03710603) study evaluating D-VRd induction and consolidation therapy, ASCT, and D-R maintenance therapy, compared to bortezomib, lenalidomide and dexamethasone (VRd), ASCT, and lenalidomide (R) maintenance. Results from the primary analysis showed that the study met its primary endpoint of progression-free survival (PFS), reducing the risk of disease progression or death by 58 percent (Hazard Ratio [HR], 0.42; 95 percent Confidence Interval [CI] 0.30-0.59; P<0.0001).1 Treatment with D-VRd and ASCT followed by D-R maintenance also increased the depth of response with higher rates of complete response (CR) or better, stringent complete response (sCR) and minimal residual disease (MRD) negativity compared to treatment with VRd, ASCT and R maintenance. Overall, 64 percent of patients who entered the maintenance phase in the D-VRd arm were able to discontinue treatment with DARZALEX FASPRO after achieving a complete response or better and sustained MRD-negativity following at least two years of D-R maintenance in accordance with the protocol. The overall safety profile of D-VRd followed by D-R maintenance was consistent with the known safety profiles for DARZALEX FASPRO, VRd and R.

"We are committed to changing the course of multiple myeloma through building combination regimens such as D-VRd with complementary mechanisms of action. The DARZALEX FASPRO-based quadruplet therapy demonstrated a clinically significant reduction in the risk of progression or death for transplant-eligible, newly diagnosed patients with multiple myeloma," said Craig Tendler, M.D., Vice President, Clinical Development, Diagnostics, and Global Medical Affairs, Johnson & Johnson Innovative Medicine. "Patients are most likely to experience their deepest and most durable responses during the first line of treatment with D-VRd. This regimen has the potential to improve long-term outcomes for newly diagnosed patients and we look forward to working with the FDA on the review of this application."

Data from the PERSEUS study were presented as a late-breaking oral presentation (LBA–1) at the 2023 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and simultaneously published in The New England Journal of Medicine.

About the PERSEUS study

The PERSEUS study is being conducted in collaboration with the European Myeloma Network as the sponsor. PERSEUS is an ongoing, randomized, open-label, Phase 3 study comparing the efficacy and safety of D-VRd and ASCT followed by D-R maintenance vs VRd and ASCT followed by R maintenance in patients with transplant-eligible NDMM. The primary endpoint is PFS, and secondary endpoints include overall CR or better rate, overall MRD-negativity (in patients with CR or better), and overall survival. DARZALEX FASPRO was discontinued after at least 24 months of D-R maintenance therapy in patients who had a CR or better and had sustained MRD–negative status for at least 12 months. The median age is 61.0 (range, 32-70) years for patients in the D-VRd arm and 59.0 (range, 31-70) years for patients in the VRd arm. The study is being conducted in 14 countries in Europe and Australia.

About multiple myeloma

Multiple myeloma is a blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.2 In multiple myeloma, these malignant plasma cells proliferate and replace normal cells in the bone marrow.3 Multiple myeloma is the second most common blood cancer worldwide and remains an incurable disease.4 In 2024, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 will die from the disease.5 People with multiple myeloma have a 5-year survival rate of 59.8 percent.5 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.6,7

About DARZALEX FASPRO

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) received U.S. FDA approval in May 2020 and is approved for eight indications in multiple myeloma, three of which are for frontline treatment in newly diagnosed patients who are transplant eligible or ineligible.8 It is the only subcutaneous CD38-directed antibody approved to treat patients with multiple myeloma. DARZALEX FASPRO is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE drug delivery technology.

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive license to develop, manufacture and commercialize daratumumab.

INDICATIONS

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj) is indicated for the treatment of adult patients with multiple myeloma:

In combination with bortezomib, melphalan, and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant
In combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for autologous stem cell transplant and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy
In combination with bortezomib, thalidomide, and dexamethasone in newly diagnosed patients who are eligible for autologous stem cell transplant
In combination with pomalidomide and dexamethasone in patients who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor
In combination with carfilzomib and dexamethasone in patients with relapsed or refractory multiple myeloma who have received one to three prior lines of therapy
In combination with bortezomib and dexamethasone in patients who have received at least one prior therapy
As monotherapy in patients who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
For more information, visit www.DARZALEX.com.

DARZALEX FASPRO IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

DARZALEX FASPRO is contraindicated in patients with a history of severe hypersensitivity to daratumumab, hyaluronidase, or any of the components of the formulation.

WARNINGS AND PRECAUTIONS

Hypersensitivity and Other Administration Reactions

Both systemic administration-related reactions, including severe or life-threatening reactions, and local injection-site reactions can occur with DARZALEX FASPRO. Fatal reactions have been reported with daratumumab-containing products, including DARZALEX FASPRO.

Systemic Reactions

In a pooled safety population of 898 patients with multiple myeloma (N=705) or light chain (AL) amyloidosis (N=193) who received DARZALEX FASPRO as monotherapy or in combination, 9% of patients experienced a systemic administration-related reaction (Grade 2: 3.2%, Grade 3: 1%). Systemic administration-related reactions occurred in 8% of patients with the first injection, 0.3% with the second injection, and cumulatively 1% with subsequent injections. The median time to onset was 3.2 hours (range: 4 minutes to 3.5 days). Of the 140 systemic administration-related reactions that occurred in 77 patients, 121 (86%) occurred on the day of DARZALEX FASPRO administration. Delayed systemic administration-related reactions have occurred in 1% of the patients.

Severe reactions included hypoxia, dyspnea, hypertension, tachycardia, and ocular adverse reactions, including choroidal effusion, acute myopia, and acute angle closure glaucoma. Other signs and symptoms of systemic administration-related reactions may include respiratory symptoms, such as bronchospasm, nasal congestion, cough, throat irritation, allergic rhinitis, and wheezing, as well as anaphylactic reaction, pyrexia, chest pain, pruritus, chills, vomiting, nausea, hypotension, and blurred vision.

Pre-medicate patients with histamine-1 receptor antagonist, acetaminophen, and corticosteroids. Monitor patients for systemic administration-related reactions, especially following the first and second injections. For anaphylactic reaction or life-threatening (Grade 4) administration-related reactions, immediately and permanently discontinue DARZALEX FASPRO. Consider administering corticosteroids and other medications after the administration of DARZALEX FASPRO depending on dosing regimen and medical history to minimize the risk of delayed (defined as occurring the day after administration) systemic administration-related reactions.

Ocular adverse reactions, including acute myopia and narrowing of the anterior chamber angle due to ciliochoroidal effusions with potential for increased intraocular pressure or glaucoma, have occurred with daratumumab-containing products. If ocular symptoms occur, interrupt DARZALEX FASPRO and seek immediate ophthalmologic evaluation prior to restarting DARZALEX FASPRO.

Local Reactions

In this pooled safety population, injection-site reactions occurred in 8% of patients, including Grade 2 reactions in 0.7%. The most frequent (>1%) injection-site reaction was injection-site erythema. These local reactions occurred a median of 5 minutes (range: 0 minutes to 6.5 days) after starting administration of DARZALEX FASPRO. Monitor for local reactions and consider symptomatic management.

Neutropenia

Daratumumab may increase neutropenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing information for background therapies. Monitor patients with neutropenia for signs of infection. Consider withholding DARZALEX FASPRO until recovery of neutrophils. In lower body weight patients receiving DARZALEX FASPRO, higher rates of Grade 3-4 neutropenia were observed.

Thrombocytopenia

Daratumumab may increase thrombocytopenia induced by background therapy. Monitor complete blood cell counts periodically during treatment according to manufacturer’s prescribing information for background therapies. Consider withholding DARZALEX FASPRO until recovery of platelets.

Embryo-Fetal Toxicity

Based on the mechanism of action, DARZALEX FASPRO can cause fetal harm when administered to a pregnant woman. DARZALEX FASPRO may cause depletion of fetal immune cells and decreased bone density. Advise pregnant women of the potential risk to a fetus. Advise females with reproductive potential to use effective contraception during treatment with DARZALEX FASPRO and for 3 months after the last dose.

The combination of DARZALEX FASPRO with lenalidomide, thalidomide, or pomalidomide is contraindicated in pregnant women because lenalidomide, thalidomide, and pomalidomide may cause birth defects and death of the unborn child. Refer to the lenalidomide, thalidomide, or pomalidomide prescribing information on use during pregnancy.

Interference With Serological Testing

Daratumumab binds to CD38 on red blood cells (RBCs) and results in a positive indirect antiglobulin test (indirect Coombs test). Daratumumab-mediated positive indirect antiglobulin test may persist for up to 6 months after the last daratumumab administration. Daratumumab bound to RBCs masks detection of antibodies to minor antigens in the patient’s serum. The determination of a patient’s ABO and Rh blood type are not impacted.

Notify blood transfusion centers of this interference with serological testing and inform blood banks that a patient has received DARZALEX FASPRO. Type and screen patients prior to starting DARZALEX FASPRO.

Interference With Determination of Complete Response

Daratumumab is a human immunoglobulin G (IgG) kappa monoclonal antibody that can be detected on both the serum protein electrophoresis (SPE) and immunofixation (IFE) assays used for the clinical monitoring of endogenous M-protein. This interference can impact the determination of complete response and of disease progression in some DARZALEX FASPRO-treated patients with IgG kappa myeloma protein.

ADVERSE REACTIONS

In multiple myeloma, the most common adverse reaction (≥20%) with DARZALEX FASPRO monotherapy is upper respiratory tract infection. The most common adverse reactions with combination therapy (≥20% for any combination) include fatigue, nausea, diarrhea, dyspnea, insomnia, headache, pyrexia, cough, muscle spasms, back pain, vomiting, hypertension, upper respiratory tract infection, peripheral sensory neuropathy, constipation, pneumonia, and peripheral edema.

The most common hematology laboratory abnormalities (≥40%) with DARZALEX FASPRO are decreased leukocytes, decreased lymphocytes, decreased neutrophils, decreased platelets, and decreased hemoglobin.

Please click here to see the full Prescribing Information.

Results of the Phase III INSPIRE Study on Qilu Pharmaceutical’s Iruplinalkib Published in the Journal of Thoracic Oncology

On January 30, 2024 The interim analysis results from the phase III INSPIRE study of iruplinalkib developed by Qilu Pharmaceutical, which focuses on the first-line treatment of locally advanced or metastatic anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), reported to have recently been featured in the Journal of Thoracic Oncology (Impact Factor=20.4), the official journal of the International Association for the Study of Lung Cancer (IASLC) (Press release, Qilu Pharmaceutical, JAN 30, 2024, View Source [SID1234639724]). The publication of the findings in the prestigious international journal underscores the global academic community’s strong recognition of the clinical value of these results, while significantly enhancing the community’s comprehension of the INSPIRE study and iruplinalkib.

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Iruplinalkib is a next-generation ALK receptor tyrosine kinase inhibitor (TKI) that effectively targets both the wild-type and mutant ALK fusion genes. The study, conducted at 40 centers throughout China and led by Prof. Shi Yuankai from the Cancer Hospital of Chinese Academy of Medical Sciences, showed that iruplinalkib significantly extended progression-free survival (PFS) and demonstrated enhanced intracranial anti-tumor activity relative to the control group.

The INSPIRE study, a randomized, open-label, multicenter Phase III clinical trial compared the efficacy and safety of iruplinalkib against crizotinib in locally advanced or metastatic ALK-positive NSCLC patients who had not previously been treated with an ALK inhibitor. Key findings were presented at the 2023 World Conference on Lung Cancer.

Between September 4, 2019 and December 2, 2020, the trial enrolled 292 patients diagnosed with locally advanced or metastatic ALK-positive NSCLC, including 81 with central nervous system (CNS) metastases.

The results of the INSPIRE study, as published in the Journal of Thoracic Oncology and assessed by the Independent Review Committee (IRC) as of November 13, 2022, indicate that the median PFS was significantly longer in the iruplinalkib group (27.7 months) compared to the crizotinib group (14.6 months), with a hazard ratio (HR) of 0.34 (98.02% CI: 0.23-0.52), demonstrating a 66% reduction in disease progression or death risk. The PFS outcomes assessed by investigators were consistent with those determined by the IRC. As of the data cut-off, the median follow-up time for overall survival (OS) was 26.7 months for the iruplinalkib group and 25.9 months for the control group. The median OS had not yet been reached.

Subgroup analyses and additional secondary endpoints further supported iruplinalkib’s superior efficacy. Iruplinalkib led to longer duration of tumor response compared to the control group (median duration of response: 26.8 months versus 12.9 months; HR: 0.31). In patients with CNS metastases at baseline, the iruplinalkib group exhibited a higher intracranial objective response rate (ORR). The intracranial ORR was 57.9% in the iruplinalkib group versus 25.6% in the control group The intracranial complete response (CR) rate was 31.6% compared to 2.6% in the control group. Furthermore, among those with measurable CNS metastases at baseline, the intracranial ORR for the iruplinalkib group reached 90.9%.

In terms of safety, although patients in the iruplinalkib group underwent treatment for a longer duration than those in the control group (23.9 months versus 12.9 months), the frequency of severe adverse reactions (grade 3 or 4) was comparable between the two groups, with 51.7% in the iruplinalkib group versus 49.7% in the control group.

Following the positive outcomes of the INSPIRE study, iruplinalkib was approved by the China National Medical Products Administration (NMPA) in January of this year for use as a single-agent therapy in treating locally advanced or metastatic ALK-positive NSCLC. Longer follow-up of the study is in progress. Updated results will be presented at upcoming academic conferences.

FDA Grants Orphan Drug Designation (ODD) Status to NeoImmuneTech’s NT-I7 for Advanced Pancreatic Cancer Treatment

On January 30, 2024 NeoImmuneTech, Inc. (NIT), a T cell-focused therapeutics company, reported that the U.S. Food and Drug Administration (FDA) has granted NT-I7 (efineptakin alfa) (rhIL-7-hyFc) Orphan Drug Designation (ODD) for the treatment of pancreatic cancer (Press release, NeoImmuneTech, JAN 30, 2024, View Source [SID1234639723]).

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Pancreatic cancer is an aggressive tumor-type associated with extremely poor prognosis. It is the third deadliest cancer in the US and European Union with a combined annual mortality of 139,000.[1],[4] Advanced pancreatic cancer has a five-year relative-survival-rate of 3%. Pancreatic cancer has been labeled as a "silent killer" because it is normally asymptomatic in the early stages which implies that it is often diagnosed at a late stage when tumors are highly resistant to treatment modalities. This underscores the critical need for new and more effective therapeutic approaches.2

Dr. Luke Oh, Ph.D., President of NeoImmuneTech, Inc. said: "We are excited that the FDA granted NT-I7 an ODD in the treatment of pancreatic cancer. This decision adds further credibility to our existing evidence that NT-I7 has the potential to bring a much-needed therapy option to people suffering from pancreatic cancer. We look forward to continuing our collaboration with FDA, as we explore the therapeutic benefits of combining NT-I7 with other anti-cancer treatments such as immunotherapies for patients with pancreatic cancer."

NT-I7 has been studied in several robust phase I and II clinical trials and has demonstrated the potential to amplify T cells across the subsets, boost the immune system, and enhance the anti-tumor response in people with pancreatic cancer and other solid tumors.[5]

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent.

About Pancreatic Cancer
Pancreatic cancer is a malignant, aggressive tumor of the pancreas. More than 90% of cases develop in the exocrine tissue of the pancreas, which makes enzymes to digest food. The less common endocrine tumors, commonly referred to as pancreatic neuroendocrine tumors (NETs), develop in hormone-producing cells.[1} Whilst pancreatic cancer’s low prevalence rate qualifies it as a rare cancer, the aggressiveness of the disease and its high death rate means that patients diagnosed have a very poor prognosis. Every year, 157,107 new patients are diagnosed with pancreatic cancer in the US and Europe Union combined. [1],[4]

About NT-I7 (efineptakin alfa) (rhIL-7-hyFc)
NT-I7 (efineptakin alfa) is the only clinical-stage long-acting human IL-7, and is being developed in oncologic and immunologic indications, where T cell amplification and increased functionality may provide clinical benefit. IL-7 is a fundamental cytokine for naïve and memory T cell development and for sustaining immune response to chronic antigens (as in cancer) or foreign antigens (as in infectious diseases). NT-I7 exhibits favorable PK/PD and safety profiles, making it an ideal combination partner. NT-I7 is being studied in multiple clinical trials in solid tumors and as vaccine adjuvant. Studies are being planned for testing in hematologic malignancies, additional solid tumors and other immunology-focused indications.

ITM and Alpha-9 Oncology Announce Global Supply Agreement to Support Alpha-9’s Clinical Radiopharmaceutical Development Program

On January 30, 2024 ITM Isotope Technologies Munich SE (ITM), a leading radiopharmaceutical biotech company, and Alpha-9 Oncology (Alpha-9), a clinical stage biotechnology company developing differentiated and highly targeted radiopharmaceuticals, reported the signature of a global master clinical supply agreement to support the development of Alpha-9’s Radiopharmaceutical Therapy (RPT) pipeline candidates for the treatment of cancer (Press release, ITM Isotopen Technologien Munchen, JAN 30, 2024, View Source [SID1234639722]). Under the terms of the agreement, ITM will supply its medical radioisotope, non-carrier-added Lutetium-177 (n.c.a. 177Lu) for Alpha-9’s Lutetium-based candidates.

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"Entering this strategic collaboration with Alpha-9 underscores our belief in the value of developing established beta-emitters such as Lutetium-177, to advance the radiopharmaceutical industry and maximize patient benefit," commented Steffen Schuster, CEO of ITM. "Our position as the world’s largest supplier of n.c.a. Lutetium-177 make us an ideal partner for Alpha-9. We look forward to working with a company that shares our vision of improving cancer care and outcomes for patients globally."

"A stable and reliable supply of high-quality isotopes is key to the successful development of our pipeline, and partnering with ITM is a valuable next step in building out our proprietary offering of differentiated and precise radiopharmaceuticals," said David Hirsch, M.D., Ph.D., CEO of Alpha-9.

ITM will supply Alpha-9 with its n.c.a. 177Lu for the clinical development of Alpha-9 radiopharmaceutical candidates comprising n.c.a. 177Lu combined with undisclosed targeting molecules. ITM holds a U.S. Drug Master File (DMF) with the Food and Drug Administration (FDA) for n.c.a. 177Lu and has marketing authorization in the EU (brand name EndolucinBeta).

About Radiopharmaceutical Therapy (RPT)

Radiopharmaceutical Therapy (RPT) is an emerging class of cancer therapeutics, which seeks to deliver radiation directly to the tumor while minimizing radiation exposure to normal tissue. Targeted radiopharmaceuticals are created by linking a therapeutic radioisotope such as Lutetium-177 or Actinium-225 to a targeting molecule (e.g., peptide, antibody, small molecule) that can precisely recognize tumor cells and bind to tumor-specific characteristics, such as receptors on the tumor cell surface. As a result, the radioisotope accumulates at the tumor site and decays, releasing a small amount of ionizing radiation, with the goal of destroying tumor tissue. The precise localization enables targeted treatment with potentially minimal impact to healthy surrounding tissue.

Rgenta Therapeutics Joins The Leukemia & Lymphoma Society Therapy Acceleration Program®

On January 30, 2024 Rgenta Therapeutics ("Rgenta" or the "Company") reported that it was selected by The Leukemia & Lymphoma Society (LLS) as a new Therapy Acceleration Program (TAP) portfolio company, and provided with strategic funding to support preclinical and clinical advancement of the Company’s therapeutics in hematologic malignancies utilizing its RNA-targeting small molecule platform to target MYB, an oncogenic transcription factor (Press release, Rgenta Therapeutics, JAN 30, 2024, View Source;lymphoma-society-therapy-acceleration-program-302047063.html [SID1234639721]).

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"We are honored by the support and recognition of LLS’s Therapy Acceleration Program. LLS’s strategic investment in Rgenta continues to bolster our confidence in the potential of our pipeline of oral, small-molecule RNA-targeting medicines," commented Simon Xi, Ph.D., CEO of Rgenta. "We are dedicated to advancing these best-in-class RNA-targeting small molecules and believe they have the potential to make a significant impact in the treatment of multiple high-value disease areas."

"We are excited to partner with LLS TAP to tackle historically undruggable oncogenes implicated in hematologic malignancies using our small molecule RNA-targeting platform", said Travis Wager, Ph.D., President & CSO. "We look forward to tapping into the extensive clinical expertise and resources from LLS to accelerate the development of our RNA-targeting MYB inhibitor molecules."

"LLS has a long-standing history of helping to accelerate development of breakthrough blood cancer treatments, and we’re particularly excited when work like Rgenta’s focuses on key mechanisms in blood cancer that have historically been extremely difficult to target with drugs" said Lore Gruenbaum, Ph.D., Vice President of LLS TAP. "This new partnership is an exciting addition to LLS TAP’s portfolio of companies that are working to provide new hope to all blood cancer patients."