DRAFT MEETING PACKAGE

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Northeastern University *

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6207

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Medicine

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Dec 6, 2023

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docx

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Uploaded by MinisterMagpie2727

LYMPHOCURE Dear [FDA Reviewer], I'm writing to request approval of our New medication Application (NDA) for Lymphocure, an experimental medication for the treatment of relapsed or refractory Acute Lymphocytic Leukemia (ALL). Lymphocure is a new immunotherapy drug that uses the immune system's capacity to target cancer cells and trigger immune-mediated death. We think Lymphocure has the potential to be of great help to individuals with relapsed or refractory ALL, for whom therapy choices are restricted. Our preclinical studies show that Lymphocure can bind to CD19 and CD20 receptors on cancer cells and promote immune- mediated death. Lymphocure was demonstrated to be effective in lowering tumor burden in both xenograft and syngeneic ALL models in both in vitro and in vivo settings. A single-arm, multicenter phase II investigation in patients with relapsed or refractory ALL is included in our planned clinical trial design. The study's primary aim is objective response rate (ORR), with secondary objectives including duration of response, progression-free survival (PFS), overall survival (OS), and safety. This trial design, we feel, is appropriate for assessing the effectiveness and safety of Lymphocure in this patient group. We conducted thorough preclinical toxicity investigations in rats and nonhuman primates, demonstrating Lymphocure's safety and tolerability at dosages up to ten times the intended therapeutic dose. Mild to moderate alterations in hematology and clinical chemistry parameters, including temporary declines in white blood cell counts and elevations in liver enzymes, were the predominant toxicological results. We appreciate the opportunity to submit our research and discuss the development of Lymphocure with the FDA. We think Lymphocure represents a substantial development in the treatment of relapsed or refractory ALL, and we look forward to collaborating with the FDA to provide this critical medicine to patients in need. Sincerely, Hema Anantha Harshitha Nalam Rushikesh Reddy Nandireddy Sahithi Swarna Sahithi Puja Uppaluru Sai Ram Yadlapalli Boston, MA Agenda: DRAFT MEETING PACKAGE 1

LYMPHOCURE Lymphocure introduction and summary: Provide a brief review of the medicine and its intended application for the treatment of relapsed or refractory ALL. Discuss the patient population's unmet medical needs and the possible advantages of Lymphocure. Toxicology research and preclinical data: Examine the preclinical data and toxicity studies done in rats and nonhuman primates, including Lymphocure's mode of action and the drug's safety and tolerability profile. Provide a summary of the planned Lymphocure clinical trial design, including the patient population, study objectives, and statistical analysis strategy. Discussion of any available comparison data with licensed medications for the treatment of relapsed or refractory ALL. Questions for the FDA: Request comments and direction from the FDA on critical development concerns such as study design, patient selection criteria, and clinical outcomes. Specific inquiries may include: Are the proposed criteria for inclusion and exclusion suitable for the patient population? Is ORR alone sufficient to demonstrate efficacy, or should other endpoints be considered? Should the clinical study contain a comparison arm, and if yes, which comparator is best? Following steps: Discuss the future stages in the development of Lymphocure, such as the timing for submitting new data and the FDA approval procedure. Conclusion: Thank you for your time and insight, and please repeat the company's commitment to discovering safe and effective therapies for patients with relapsed ALL. Company: DRAFT MEETING PACKAGE 2

LYMPHOCURE XYZ Pharmaceuticals, a biopharmaceutical firm focused on creating novel medicines for cancer and other critical diseases, is developing Lymphocure. The firm was established in 2005 and is based in San Francisco, California. Background: Lymphocure is a new small molecule inhibitor of BTK, a crucial enzyme in the B-cell receptor signaling pathway involved in B-cell survival and proliferation. Lymphocure is intended to decrease the proliferation and survival of malignant B-cells in patients with relapsed or refractory ALL by inhibiting BTK. Preclinical investigations have shown that Lymphocure is highly selective for BTK and has substantial anticancer action in vitro and in vivo against ALL cell lines. Furthermore, Lymphocure demonstrated a positive safety profile in preclinical toxicological investigations in rats and nonhuman primates. Lymphocure is now being tested in a phase 1 clinical trial in patients with relapsed or refractory ALL, with early findings expected in the next 6-12 months. The trial's goal is to assess Lymphocure's safety, tolerability, and preliminary effectiveness as a single drug in this patient group. If the phase 1 study findings are positive, the business intends to launch a pivotal phase 2/3 trial to support regulatory approval of Lymphocure. Product Information DRAFT MEETING PACKAGE 3

LYMPHOCURE Lymphocure, as a fictitious medicine, has no comparison research with licensed drugs. Potential comparison studies, depending on the mechanism of action and current data on other CD19-targeted treatments, might include: Blinatumomab vs. Lymphocure: Blinatumomab is a CD19-targeted therapeutic licensed for the treatment of relapsed or refractory ALL. In patients with relapsed or refractory ALL, a comparison trial might compare the safety and effectiveness of Lymphocure vs Blinatumomab. Lymphocure combined with chemotherapy vs. chemotherapy alone: The current standard of treatment for ALL is chemotherapy, and some chemotherapy regimens include CD19-targeted medicines. In newly diagnosed patients with ALL, a comparison research might compare the safety and effectiveness of adding Lymphocure to conventional treatment vs chemotherapy alone. Lymphocure vs Hematopoietic stem cell transplantation (HSCT): HSCT is a potentially curative therapeutic option for certain ALL patients, but it is fraught with dangers and problems. A research comparing the safety and effectiveness of Lymphocure against HSCT in patients with relapsed or refractory ALL who are suitable for both therapies might be conducted. These are only a few examples of prospective Lymphocure comparative studies in the treatment of ALL. The particular design of comparison studies would be determined by the study aims, patient population, and resources available. Lymphocure's distinct traits as a fictitious medicine may be identified by its supposed attributes and mode of action. Lymphocure is distinguished by its new method of action, which employs a bispecific antibody to target CD19-positive cancer cells. This method of action differs from existing treatments for Acute Lymphocytic Leukemia (ALL), which mostly include chemotherapy and hematopoietic stem cell transplantation. Another distinguishing feature of Lymphocure is its ability to elicit long-lasting reactions with low harm. Although CD19-targeted treatments have shown encouraging outcomes in the treatment of ALL, they can cause serious adverse effects such as cytokine release syndrome and neurotoxicity. Lymphocure's dual specificity for CD19 and T-cell receptors has the potential to improve the efficacy and reduce the toxicity of CD19-targeted treatment. Lymphocure might be a beneficial therapy option for individuals with relapsed or refractory ALL if it can demonstrate safety and effectiveness in clinical studies. Lymphocure is a good contender for FDA clearance due to its novel mode of action and potential for low toxicity, assuming it passes all regulatory standards for safety and efficacy. Finally, the FDA will base its judgment on the totality of clinical trial data, including the drug's safety, effectiveness, and potential advantages above existing treatment choices. DRAFT MEETING PACKAGE 4

LYMPHOCURE Description: Lymphocure is a novel treatment for Acute Lymphocytic Leukemia (ALL), a kind of blood cancer that affects lymphocytes, which are white blood cells. The medication targets and destroys malignant lymphocytes while leaving healthy cells alone. Phase I Clinical Trial Study Design: The goal of this study was to assess the safety and maximum tolerated dosage of Lymphocure in individuals with ALL. Participants: ALL patients (n = 20) Intervention: To examine safety and acceptability, administer Lymphocure at three different dosages (10mg, 25mg, and 50mg). Outcome measurements include adverse events, vital signs, laboratory tests, and electrocardiograms. Clinical Trial in Phase II: The goal of this study is to determine the effectiveness and safety of Lymphocure in individuals with ALL. Patients with ALL (n=100) participated. Intervention: To test effectiveness and safety, administer Lymphocure at the optimum dosage determined in Phase I. Outcome measurements include efficacy evaluations such as overall response rate, progression-free survival, and overall survival, as well as safety assessments such as adverse events, vital signs, laboratory tests, and electrocardiograms. The goal of the Phase III clinical trial is to validate the effectiveness and safety of Lymphocure in a broader group of patients with ALL. Patients with ALL (n=500) took part in the study. Intervention: To establish effectiveness and safety, administer Lymphocure at the optimum dosage determined in Phase II. Outcome measurements include efficacy evaluations such as overall response rate, progression-free survival, and overall survival, as well as safety assessments such as adverse events, vital signs, laboratory tests, and electrocardiograms. DRAFT MEETING PACKAGE 5

LYMPHOCURE Regulatory Affairs Landscape: Preclinical studies: Animal models are used to assess the safety and efficacy of Lymphocure before human trials. Before Phase I trials may begin, data from these research is submitted to regulatory authorities for assessment and permission. Application for an Investigational New Drug (IND): Requested approval from regulatory authorities to undertake Phase I clinical studies in people. This application contains information on the research design and safety procedures, as well as data from preclinical trials. A phase I clinical trial was conducted to evaluate the safety and maximum tolerated dosage of Lymphocure in ALL patients. Before Phase II trials may begin, data from this trial is submitted to regulatory authorities for assessment and approval. Lymphocure effectiveness and safety were evaluated in a phase II clinical trial in individuals with ALL. Before Phase III trials may begin, data from this trial is submitted to regulatory authorities for assessment and approval. Phase III Clinical Trial: Conducted to establish Lymphocure's effectiveness and safety in a broader group of ALL patients. Before the medicine may be licensed for commercialization, the data from this study is submitted to regulatory authorities for assessment and approval. NDA stands for New Drug Application. Lymphocure has been submitted to regulatory authorities for commercial authorisation. This application contains information from preclinical and clinical studies, as well as manufacturing, labeling, and post-marketing surveillance plans. Post-marketing surveillance: Ongoing monitoring of Lymphocure's safety and efficacy after it has been approved for marketing. This involves tracking adverse occurrences and performing more research. DRAFT MEETING PACKAGE 6

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