On May 12, 2021 Caelum Biosciences and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) reported that two abstracts on CAEL-101, a potentially first-in-class amyloid fibril targeted therapy, have been accepted for e-poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2021, taking place virtually from June 9 to 17, 2021 (Press release, Caelum Biosciences, MAY 12, 2021, View Source [SID1234579771]). Both presentations will include new data from the Phase 2 open-label dose escalation study evaluating the safety and tolerability of CAEL-101 in combination with standard-of-care therapy in AL amyloidosis. One will feature data studying safety and tolerability in AL amyloidosis patients treated with 1000 mg/m2 CAEL-101 in combination with cyclophosphamide-bortezomib-dexamethasone plus daratumumab. The second will include new data further supporting the selection of the 1000 mg/m2 dose for the ongoing Phase 3 study and demonstrating the possible stabilization of cardiac disease as assessed by Mayo staging.

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The abstracts accepted for e-poster presentations are listed below and are now available on the EHA (Free EHA Whitepaper) website. All e-poster presentations will be made available on the virtual congress platform on June 11, 2021 at 09:00 a.m. CEST (3:00 a.m. EDT) and will be available throughout the duration of the Congress.

Safety and Tolerability of CAEL-101 in Combination with Cyclophosphamide-Bortezomib-Dexamethasone and Daratumumab in Patients with AL Amyloidosis. Abstract #EP1017– e-poster presentation; session title: Myeloma and other monoclonal gammopathies – Clinical.

Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis in a Phase 2 Study for a Median of 38 Weeks. Abstract #EP1018 – e-poster presentation; session title: Myeloma and other monoclonal gammopathies – Clinical.

As was previously announced, the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101 in combination with SoC therapy in AL amyloidosis has begun. Enrollment is underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease (ClinicalTrials.gov Identifier: NCT04512235) and one in patients with Mayo stage IIIb disease (ClinicalTrials.gov Identifier: NCT04504825) – and will collectively enroll approximately 370 patients globally. The Phase 2 program continues with the ongoing evaluation of CAEL-101 in combination with SoC therapy plus daratumumab.

About CAEL-101
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a potential therapy for patients with AL amyloidosis.

About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

AL amyloidosis is a rare disease but is the most common form of systemic amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

On May 12, 2021 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported its first quarter 2021 financial results and corporate highlights (Press release, Kezar Life Sciences, MAY 12, 2021, View Source [SID1234579788]).

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"Last quarter saw excellent execution by the Kezar team across both of our programs. We look forward to sharing data this June from the completed Phase 1b portion of the MISSION study of KZR-616 in SLE patients, followed by an interim review of the Phase 2 portion of MISSION in LN patients in the fourth quarter. Our conviction continues to deepen that immunoproteasome inhibition has the potential to be a powerful, differentiated treatment approach for patients with autoimmune diseases," said John Fowler, Kezar’s Co-founder and Chief Executive Officer. "In addition, we are on track to submit an IND for KZR-261, our first-in-class protein secretion inhibitor, in mid-2021 and commence a Phase 1 study in solid tumors shortly thereafter."

Clinical Highlights & Updates

KZR-616: Selective Immunoproteasome Inhibitor

MISSION – Phase 1b/2 clinical trial in patients with systemic lupus erythematous (SLE) and lupus nephritis (LN), respectively (NCT03393013)

The last patient from the MISSION Phase 1b completed treatment in February 2021, and final clinical data from this 25-week safety and tolerability study of up to 75 mg weekly of KZR-616 in 47 patients with SLE will be presented during the European Congress of Rheumatology (EULAR 2021) in June.
The amended Phase 2 open-label portion of the MISSION trial in patients with active, proliferative lupus nephritis opened for enrollment in August 2020 and is actively recruiting. The primary efficacy endpoint for the trial is the proportion of patients achieving a renal response measured by a 50% or greater reduction in urine protein to creatinine ratio (UPCR) at six months.
Interim data are expected in late 2021, and topline data are expected in the first half of 2022.
PRESIDIO – Phase 2 clinical trial in patients with dermatomyositis (DM) and polymyositis (PM) (NCT04033926)

The PRESIDIO Phase 2, placebo controlled cross-over trial of KZR-616 in DM and PM is actively enrolling. Additionally, a 12-month open-label extension study is open to patients completing the 32-week placebo-controlled trial (NCT04628936).
Topline data are expected in the first half of 2022.
Protein Secretion Program

KZR-261 is a first-in-class protein secretion inhibitor which targets the Sec61 translocon and has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies.
Pending successful completion of drug product manufacturing, submission of an Investigational New Drug (IND) application for KZR-261 is anticipated in mid-2021. The Phase 1 clinical trial will evaluate pharmacokinetics, safety, and tolerability in patients with solid tumors as well as preliminary signs of efficacy in tumor-specific expansion cohorts. The trial is expected to commence shortly after the IND becomes effective.
Two abstracts featuring Kezar’s small molecule inhibitors of the Sec61 translocon were presented in April during the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Virtual Annual Meeting.
Corporate Update

In May 2021, Kezar was recognized as a winner of the 2021 Bay Area Best Places to Work, an awards program presented by the San Francisco Business Times and the Silicon Valley Business Journal.
Financial Results

Cash, cash equivalents and marketable securities totaled $142.3 million as of March 31, 2021, compared to $140.4 million as of December 31, 2020. The increase in cash, cash equivalents and marketable securities was primarily attributable to the net proceeds from the issuance of common stock under the "at-the-market" Sales Agreement with Cowen and Company, LLC, offset by cash used by the company in operations to advance its clinical-stage programs and preclinical research and development.
Research and development expenses for the first quarter of 2021 increased by $1.8 million to $9.3 million compared to $7.5 million in the first quarter of 2020. This increase was primarily related to advancing the KZR-616 clinical program in multiple indications and the protein secretion preclinical program.
General and administrative expenses for the first quarter of 2021 increased by $0.8 million to $3.8 million compared to $3.0 million in the first quarter of 2020. The increase was primarily due to an increase in personnel expenses and stock-based compensation as a result of an increase in headcount and salaries and an increase in the cost of directors’ and officers’ liability insurance.
Net loss for the first quarter of 2021 was $13.0 million, or $0.25 per basic and diluted common share, compared to a net loss of $10.0 million, or $0.30 per basic and diluted common share, for the first quarter of 2020.
Total shares of common stock outstanding were 48.1 million shares as of March 31, 2021. Additionally, there were outstanding pre-funded warrants to purchase 3.8 million shares of common stock at an exercise price of $0.001 per share and outstanding options to purchase 7.1 million shares of common stock at a weighted-average exercise price of $5.90 per share as of March 31, 2021.
About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b clinical trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from Kezar’s research and discovery efforts targeting protein secretion pathway. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered by Kezar through a robust medicinal chemistry campaign in which several scaffolds were progressed through the company’s proprietary platform evaluating Sec61 modulation. As a result, Kezar has established a broad library of protein secretion inhibitors. KZR-261 has demonstrated several encouraging properties that lead to its potential to be an anti-cancer agent for the treatment of solid and hematologic malignancies. An IND submission in solid tumors is expected to be filed in mid-2021.

About Lupus Nephritis

Lupus nephritis is one of the most serious complications of systemic lupus erythematosus. LN is a disease comprising a spectrum of vascular, glomerular and tubulointerstitial lesions and develops in approximately 50% of SLE patients within 10 years of their initial diagnosis. LN is associated with considerable morbidity, including an increased risk of end-stage renal disease requiring dialysis or renal transplantation and an increased risk of death. There are limited approved therapies for the treatment of LN. Management typically consists of induction therapy to achieve remission and long-term maintenance therapy to prevent relapse.

About Dermatomyositis and Polymyositis

Dermatomyositis and Polymyositis are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Approximately 30,000 to 120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.

On May 12, 2021 Sensei Biotherapeutics, Inc. (NASDAQ: SNSE), a clinical-stage immunotherapy company focused on the discovery and development of next generation therapeutics for cancer, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Sensei Biotherapeutics, MAY 12, 2021, View Source [SID1234579804]).

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"The first quarter was a productive time for Sensei with the successful execution of our IPO, continued progress with our pipeline, and expansion of our headcount to support anticipated growth," said John Celebi, President and Chief Executive Officer of Sensei Biotherapeutics. "As we approach several near-term events, we continue to believe in the strength of our proprietary ImmunoPhage platform to potentially generate multiple immune activating therapeutics and our monoclonal antibody and nanobody platform targeted to key immune pathways. We are looking forward to the presentation of more mature data from the Phase 1/2 clinical trial of SNS-301 at ASCO (Free ASCO Whitepaper), and to further explore the potential breadth and depth of our programs in collaboration with leading researchers. With the proceeds raised from our recent IPO, we are well capitalized to advance our current programs and our drug discovery operations."

Sensei Biotherapeutics is developing a pipeline of medicines designed to fulfill the substantial potential of the immune system to defeat cancer and other diseases. The company has developed two unique approaches – the ImmunoPhage platform, which leverages its proprietary knowledge and expertise in bacteriophage (widespread viruses that infect bacteria but not mammalian cells) immunology, and its monoclonal antibody and nanobody platform, which are comprised of unique human monoclonal antibodies and alpaca-derived nanobodies that are selectively active in the tumor microenvironment. Together, these platforms are designed to create a new class of personalized immuno-oncology medicines.

First Quarter and Recent Highlights:

Strengthened Immuno-Oncology Advisory Board and Board of Directors – In May 2021, Sensei announced that it appointed Maura Gillison, M.D., Ph.D., Professor of Medicine, Thoracic/Head and Neck Medical Oncology at MD Anderson Cancer Center, and Richard Ulevitch, Ph.D., Professor of Immunology and Chairman Emeritus at The Scripps Research Institute to its Immuno-Oncology Advisory Board. In April 2021, Sensei appointed Jessie M. English, Ph.D. to its Board of Directors.
Completed Upsized Initial Public Offering (IPO)– In February 2021, Sensei completed its initial public offering of 8,030,295 shares of common stock, inclusive of the exercise by the underwriters of their option to purchase 1,030,243 shares, at a public offering price of $19.00 per share. Gross proceeds were $152.6 million.
Anticipated Pipeline Milestones and Events:

ImmunoPhage Platform Updates

SNS-301: a first-in-class, bacteriophage-based immune activating agent targeting human aspartate β-hydroxylase (ASPH), a tumor associated antigen overexpressed in multiple tumor types – SNS-301 is being evaluated in a signal-seeking Phase 1/2 study in combination with pembrolizumab. The study is designed to have two cohorts and enroll 60 patients with locally advanced unresectable or metastatic SCCHN who did not achieve a response to prior treatment with PD-1 blockade or patients who did not receive prior therapy with PD-1 blockade. The company expects to add a third cohort by the end of 2021 to evaluate SNS-301 with HPV-specific E6/E7 ImmunoPhage.

American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 conference: An abstract titled: "Update on Safety and Efficacy of a Phase 1/2 Study of SNS-301 and Pembrolizumab in Patients with Advanced Squamous Cell Carcinoma of the Head and Neck (SCCHN)" has been accepted for poster presentation at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place virtually from June 4-8, 2021.
Phase 1/2 Topline Data Readout on Track: Sensei remains on track to report a substantial subset of data from the Phase 1/2 study by the end of 2021.
Enrollment Ongoing in Frontline Therapy Trial: Sensei announced that patient dosing has been initiated in the second cohort of the Phase 1/2 study evaluating SNS-301 as a first-line therapy in combination with pembrolizumab. The company continues to evaluate early enrollment trends in this cohort and the impact of COVID-19 on this clinical program.
Evaluation of New Opportunities for SNS-301 in the Neoadjuvant Setting: Sensei’s current Phase 1/2 studies in head and neck cancer are focusing only on SNS-301 in combination with pembrolizumab (Keytruda). Sensei has concluded its clinical trial collaboration with AstraZeneca for durvalumab (Imfinzi). Sensei is currently evaluating additional opportunities to study SNS-301 in the neoadjuvant setting following the results in February from AstraZeneca’s Phase 3 KESTREL trial of durvalumab for the treatment of first line recurrent or metastatic head and neck squamous cell carcinoma.
SNS-401: a first-in-class personalized ImmunoPhage cocktail being developed in collaboration with the University of Washington – SNS-401 is in preclinical development for the treatment of Merkel Cell Carcinoma, an aggressive form of skin cancer commonly caused by the Merkel Cell Carcinoma Polyoma Virus.

IND submission planned for first half of 2022: Sensei plans to submit an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for its SNS-401 program in the first half of 2022.
ImmunoPhage Platform and Phortress Library – Phortress is Sensei’s proprietary library of ImmunoPhages, derived from antigens found across multiple patient populations and tumor types. Sensei has manufactured more than 25 ImmunoPhages and continues to add inventory to this "off-the-shelf" library as well as drive technologic innovations to the ImmunoPhage platform itself.

Monoclonal Antibody Platform Update

VISTA (V-domain Ig suppressor of T cell activation) is an immune checkpoint that inhibits anti-tumor immune responses – SNS-VISTA is in preclinical development in collaboration with Adimab LLC.

IND-Enabling Studies on Track: Sensei announced it is on track to initiate IND-enabling studies for its SNS-VISTA program by year-end 2021.
Corporate Update

Sensei Biotherapeutics announced that it will move its corporate headquarters to the Seaport Innovation District in Boston, MA. The new space more than doubles the size of the company’s current research and development footprint. The company’s manufacturing operations will remain in Rockville, MD.

First Quarter 2021 Financial Results

Cash Position – Cash and cash equivalents were $169.4 million as of March 31, 2021, as compared to $16.6 million as of December 31, 2020. Sensei expects the current cash balance to fund operations at least into the second half of 2023.

Research and Development (R&D) Expenses – R&D expenses were $3.4 million for the quarter ended March 31, 2021, compared to $2.2 million for the quarter ended March 31, 2020. The increase in R&D expenses was primarily attributable to increased headcount to support Sensei’s research, development, and manufacturing activities.

General and Administrative (G&A) Expenses – G&A expenses were $4.6 million for the quarter ended March 31, 2021, compared to $1.9 million for the quarter ended March 31, 2020. The increase was primarily attributable to higher personnel costs, including stock-based compensation expense, to support Sensei’s business.

Net Loss – Net loss was $8.0 million, for the quarter ended March 31, 2021, compared to $4.7 million for the quarter ended March 31, 2020.

KEYTRUDA is a registered trademark of Merck, and IMFINZI is a registered trademark of the AstraZeneca group of companies.

On May 12, 2021 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, reported that 15 abstracts have been accepted at the upcoming 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the European Hematology Association (EHA) (Free EHA Whitepaper)’s (EHA) (Free EHA Whitepaper) 2021 Virtual Congress, including new and updated data from the CARTITUDE clinical development program being led by Janssen Research & Development, LLC (Janssen) for the investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy, ciltacabtagene autoleucel (cilta-cel) (Press release, Legend Biotech, MAY 12, 2021, View Source [SID1234579822]).

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"The Legend Biotech team, together with our collaborator Janssen, look forward to sharing the updated efficacy and longer-term safety data for cilta-cel," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "These exciting data and a wide range of other abstracts highlight our continued commitment and efforts to developing innovative treatments that will make a difference in the lives of those living with multiple myeloma."

Longer-term follow-up efficacy and safety results from the Phase 1b/2 CARTITUDE-1 study of cilta-cel in patients with relapsed/refractory multiple myeloma (Abstract #8005) will be featured in an oral presentation at the 2021 ASCO (Free ASCO Whitepaper) Meeting and as a poster presentation at EHA (Free EHA Whitepaper) (Abstract #EP964). Results from this study supported recent U.S. and European regulatory filings submitted by Legend Biotech’s collaborator, Janssen.

For the first time, data from Cohort A of the CARTITUDE-2 study evaluating the safety and efficacy of cilta-cel in patients with progressive multiple myeloma who have received 1-3 prior lines of therapy, will be presented, being featured as a poster presentation at ASCO (Free ASCO Whitepaper) (Abstract #8013) and in an oral presentation at EHA (Free EHA Whitepaper) (Abstract # S190). Poster presentations at both meetings will provide additional efficacy and safety information with cilta-cel in comparison to standard of care therapies and neurological adverse event mitigation measures.

Select abstracts from both Congresses are below.

ASCO Presentations (June 4-8, 2021)

Abstract No.

Title

Date/ Time

Abstract #8005

Oral Presentation

Ciltacabtagene autoleucel, a B-cell maturation antigen (BCMA)–directed chimeric antigen receptor T-cell (CAR-T) therapy, in relapsed/refractory multiple myeloma (R/R MM): Updated results from CARTITUDE-1​

Tuesday, June 8th 8:00-11:00 AM EDT

Abstract #8013

Poster Discussion

CARTITUDE-2: Efficacy and safety of ciltacabtagene autoleucel (cilta-cel), a BCMA-directed CAR T-cell therapy, in patients with progressive multiple myeloma (MM) after 1–3 prior lines of therapy​

Friday, June 4th @ 9:00AM EDT

Abstract #8028

Poster Presentation

Incidence, mitigation, and management of neurologic adverse events in patients with multiple myeloma (MM) treated with ciltacabtagene autoleucel (cilta-cel) in CARTITUDE-2​

Friday, June 4th @ 9:00 AM EDT

Abstract #8045

Poster Presentation

Comparison of outcomes with ciltacabtagene autoleucel (cilta-cel) in CARTITUDE-1 vs real-world standard of care (RW SOC) for patients (pts) with triple-class exposed relapsed/refractory multiple myeloma (RRMM)

Friday, June 4th @ 9:00 AM EDT

Abstract #8030

Poster Presentation

Cilta-cel vs. conventional treatment in patients with relapse/refractory multiple myeloma​

Friday, June 4th @ 9:00 AM EDT

Abstract #8041

Poster Presentation

LocoMMotion: A prospective, non-interventional, multinational study of real-life current standards of care in patients with relapsed/refractory multiple myeloma (RRMM) receiving ≥3 prior lines of therapy.

Friday, June 4th @ 9:00 AM EDT

The abstracts will be released on ASCO (Free ASCO Whitepaper) Meeting Library on May 19th, 2021 at 5:00 PM EDT.

EHA Presentations (June 9-17, 2021)

Abstract No.

Title

Date/ Time

Abstract #S190

Oral​

Efficacy and safety of the BCMA-directed CAR-T cell therapy, ciltacabtagene autoleucel, in patients with progressive multiple myeloma (MM) after 1–3 prior lines of therapy: Initial results from CARTITUDE-2

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP964

EPoster​

Updated CARTITUDE-1 results of ciltacabtagene autoleucel, a B-cell maturation antigen–directed chimeric antigen receptor T cell therapy, in relapsed/refractory multiple myeloma

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP1003

EPoster​

Incidence, mitigation, and management of neurologic adverse events in the phase 2 CARTITUDE-2 study of ciltacabtagene autoleucel in patients with multiple myeloma

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP987

EPoster​

A prospective, non-interventional, multinational study of real-life standard of care in patients with relapsed/refractory multiple myeloma receiving ≥3 prior lines of therapy: Interim data from LocoMMotion

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP990

EPoster​

Comparison of ciltacabtagene autoleucel versus conventional treatment in patients with relapsed/refractory multiple myeloma

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP1049

EPoster​

Ciltacabtagene autoleucel versus selinexor + dexamethasone in patients with relapsed/refractory multiple myeloma (RRMM) treated with ≥3 lines of prior therapy: A matching adjusted indirect comparison

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP978

EPoster​

Matching adjusted indirect comparison of ciltacabtagene autoleucel versus belantamab mafodotin in patients with relapsed/refractory multiple myeloma (RRMM) treated with ≥3 lines of prior therapy

Available starting Friday, June 11th @ 9:00AM CEST

Abstract #EP977

EPoster​

Comparison of outcomes with ciltacabtagene autoleucel (cilta-cel) in CARTITUDE-1 versus standard of care in triple-class exposed multiple myeloma patients in clinical trials of daratumumab

Available starting Friday, June 11th @ 9:00AM CEST

Abstract # EP972

EPoster​

Ciltacabtagene autoleucel for triple-class exposed multiple myeloma: Adjusted comparison of CARTITUDE-1 outcomes versus real world clinical practice observed in German registry

Available starting Friday, June 11th @ 9:00AM CEST

The abstracts are available on the EHA (Free EHA Whitepaper) website at: View Source

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multicenter study evaluating the safety and efficacy of cilta-cel in adults with relapsed and/or refractory with multiple myeloma who have received at least 3 prior lines of therapy or are double refractory to a proteasome inhibitor (PI) and immunomodulatory drug (IMiD), received a PI, an IMiD, and anti-CD38 antibody and documented disease progression within 12 months of starting the most recent therapy.1 The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the recommended Phase 2 dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2). The Phase 2 portion further evaluated the efficacy of cilta-cel with overall response rate as the primary endpoint.

About CARTITUDE-2

CARTITUDE-2 (NCT04133636) is an ongoing Phase 2 multicohort study evaluating the safety and efficacy of cilta-cel in various clinical settings. Cohort A included patients who had progressive multiple myeloma after 1–3 prior lines of therapy, including PI and IMiD, were lenalidomide refractory, and had no prior exposure to BCMA-targeting agents. The primary objective was percentage of patients with negative minimal residual disease (MRD).2

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.3 Although treatment may result in remission, unfortunately, patients will most likely relapse.4 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.5 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.6,7 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections. 8 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.9

About Cilta-cel

Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 outside of China and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed and/or refractory multiple myeloma and in earlier lines of treatment. Cilta-cel is a differentiated CAR-T therapy with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commercialize cilta-cel. In addition to a Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, cilta-cel received a BTD in China in August 2020. In addition, Orphan Drug Designation was granted for cilta-cel by the U.S. FDA in February 2019, and by the European Commission in February 2020. A Biologics License Application seeking approval of cilta-cel was submitted to the U.S. FDA and a Marketing Authorization Application was submitted to the European Medicines Agency.

On May 12, 2021 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it is scheduled to participate in the following upcoming events (Press release, Arrowhead Pharmaceuticals, MAY 12, 2021, View Source [SID1234579946]):

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2021 RBC Capital Markets Global Healthcare – May 18-20, 2021

May 18, 2021, 11:30 a.m. ET – Chris Anzalone, Ph.D., Arrowhead’s president and CEO, will participate in a fireside chat presentation

UBS Global Healthcare Virtual Conference – May 24-26, 2021

May 26, 2021, 3:00 p.m. ET – Chris Anzalone, Ph.D., Arrowhead’s president and CEO, will participate in a fireside chat presentation

A copy of the presentation materials and/or webcast links may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.