On January 6, 2022 Bolt Biotherapeutics, Inc. (Nasdaq: BOLT), a clinical-stage biotechnology company pioneering a new class of immuno-oncology agents that combine the targeting precision of antibodies with the power of both the innate and adaptive immune systems, reported that the first patient has been dosed in a new combination arm of the ongoing multi-center, multi-dose Phase 1/2 clinical trial of BDC-1001 (Press release, Bolt Biotherapeutics, JAN 6, 2022, View Source [SID1234618690]). This arm is evaluating BDC-1001 in combination with Bristol Myers Squibb’s PD-1 checkpoint inhibitor OPDIVO (nivolumab). In parallel, Bolt continues to advance the single-agent portion of the study, following the presentation of interim dose-escalation data at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology (ESMO I-O) Congress 2021. BDC-1001 is a HER2-targeting Boltbody immune-stimulating antibody conjugate (ISAC) (trastuzumab biosimilar conjugated to a toll-like receptor 7 and 8 agonist) in development for the treatment of patients with HER2-expressing solid tumors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to evaluate BDC-1001 in combination with nivolumab, a leading PD-1 checkpoint inhibitor. Pairing BDC-1001’s mechanism of action with the checkpoint inhibitor approach has the potential to yield a stronger, targeted modulation of the immune system. Initial safety and early efficacy findings reported from the ongoing monotherapy arm of the Phase 1/2 clinical trial make BDC-1001 a potentially promising candidate for the treatment of patients with HER2-expressing solid tumors," said Edith A. Perez, M.D., Chief Medical Officer of Bolt Biotherapeutics. "In the early clinical development of BDC-1001, our strategy is to follow the science, elucidating how this novel approach to engaging a patient’s immune system can eliminate tumors not addressed by currently available therapies. We look forward to investigating BDC-1001 in this first combination arm as we also continue investigation of its single-agent activity."

Bristol Myers Squibb will provide Opdivo for the combination dose escalation and combination dose expansion portions of the trial. Bolt Biotherapeutics is the study sponsor and will be responsible for costs associated with the trial execution.

About BDC-1001
BDC-1001 is a human epidermal growth factor receptor 2 (HER2) ISAC comprising a HER2-targeting biosimilar of trastuzumab conjugated with a non-cleavable linker to an innovative TLR7/8 agonist. It is currently being investigated in a Phase 1/2 clinical trial (NCT04278144) in patients with HER2-expressing solid tumors, including breast, gastroesophageal and colorectal. The trial is being conducted in four parts, and the dose-escalation monotherapy part will continue in parallel with the combination therapy study. Interim monotherapy data presented by Bolt at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Immuno-Oncology (ESMO I-O) Congress 2021 demonstrated a safe and well-tolerated profile with early clinical activity, supporting continued dose escalation and evaluation of a weekly dosing regimen.

About the Boltbody Immune-Stimulating Antibody Conjugate (ISAC) Platform
ISACs are a new category of immunotherapy that combines the precision of antibody targeting with the strength of the innate and adaptive immune systems. Boltbody ISACs comprise three primary components: a tumor-targeting antibody, a non-cleavable linker, and a proprietary immune stimulant to activate the patient’s innate immune system. By initially targeting a single marker on the surface of a patient’s tumor cells, an ISAC can create a new immune response by activating and recruiting myeloid cells. The activated myeloid cells start a feed-forward loop by releasing cytokines and chemokines, chemical signals that attract other immune cells and lower the activation threshold for an immune response. This reprograms the tumor microenvironment and invokes an adaptive immune response that targets the tumor, with the goal of durable responses for patients with cancer.

On January 6, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapies leveraging CRISPR-based technologies, reported its expected milestones and the following strategic priorities for 2022 (Press release, Intellia Therapeutics, JAN 6, 2022, View Source [SID1234598317]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Accelerating clinical validation of in vivo pipeline: Further characterize the safety and efficacy of NTLA-2001, including in patients with cardiomyopathy and complete enrollment of the Phase 1 study; establish the initial clinical profile of NTLA-2002 as a single-dose therapy for the treatment of hereditary angioedema (HAE).
Strategic pipeline expansion: Significantly progress in vivo and ex vivo pipeline, including determining the initial safety profile of NTLA-5001 for acute myeloid leukemia (AML), advancing in vivo insertion candidates and nominating multiple new development candidates.
Platform innovation: Broaden the Company’s industry-leading platform through expansion of Intellia’s genome editing, delivery and cell engineering capabilities.
"Unequivocally, 2021 was a landmark year for Intellia. We demonstrated that our proprietary CRISPR-based platform and LNP technology can turn revolutionary science into potentially transformational medicines. Our platform enables us to advance genome editing approaches, which maximizes our ability to target a multitude of life-threatening diseases," said Intellia President and Chief Executive Officer John Leonard, M.D. "As we begin 2022 with great momentum, we are poised to significantly expand our full-spectrum pipeline of potentially curative therapies with the nomination of at least two new in vivo candidates and our first allogeneic development candidate during the year. Importantly, we look forward to sharing additional data from the ongoing study of NTLA-2001 and interim results from the Phase 1/2 study of NTLA-2002, which we expect will further demonstrate the modularity of our genome editing platform."

Anticipated 2022 Milestones:

In Vivo Programs

NTLA-2001 for ATTR amyloidosis: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, and has the potential to be the first single-dose treatment for transthyretin (ATTR) amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Today, Intellia announced that the first patient in the cardiomyopathy arm of the Phase 1 study has been dosed with NTLA-2001. This follows the Company’s recent announcement that the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) approved a protocol amendment for the Company’s ongoing Phase 1 study of NTLA-2001 to include patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study now includes patients with ATTR-CM enrolled in new dose-escalation and expansion cohorts. The inclusion of the ATTR-CM patient population is in addition to the original Phase 1 study, which continues to evaluate NTLA-2001 in patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Intellia expects to complete enrollment of the Phase 1 study for both ATTRv-PN and ATTR-CM subjects in 2022.
Intellia intends to present additional interim clinical data of NTLA-2001 in ATTRv-PN patients from Part 1, the single-ascending dose portion, and to initiate Part 2, a single-cohort expansion, in the first quarter of 2022. Data to be presented at a company-sponsored event will be from all four ATTRv-PN dose cohorts in Part 1 and include safety and serum TTR knockdown for Cohorts 3 and 4, as well as an early look at durability across all cohorts.
NTLA-2002 for HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous suppression of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.
In December 2021, Intellia dosed the first patient in its second clinical study of a CRISPR-based therapeutic candidate evaluating NTLA-2002 for HAE. The first-in-human Phase 1/2 trial is expected to evaluate the safety, tolerability and activity of NTLA-2002 in adults with Type I or Type II HAE, and will continue to leverage insights gained from the development of NTLA-2001.
The Company expects to present interim data from the Phase 1/2 study in the second half of 2022. These results are expected to characterize the emerging safety and activity profile of NTLA-2002 and demonstrate preliminary proof-of-concept.
NTLA-3001 for AATD-associated lung disease: NTLA-3001 is Intellia’s wholly owned CRISPR-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001 with plans to file an IND or IND-equivalent in 2023. The Company also continues to explore additional editing strategies for AATD.
Ex Vivo Programs

NTLA-5001 for AML: NTLA-5001 is an investigational autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML).
In the fourth quarter of 2021, Intellia initiated screening of patients in the Phase 1/2a study of NTLA-5001 for patients with AML. The Company expects to dose its first patient in the coming weeks and enroll patients throughout the year. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its TCR-based platform.
Modular Platform and Pipeline Expansion

Platform Innovation: Intellia is expanding its industry-leading genome editing platform and scientific leadership through editing, delivery and cell engineering innovations that will enable broader in vivo and ex vivo applications.
Intellia plans to advance at least two new in vivo development candidates by the end of 2022.
The Company expects to nominate its first allogeneic ex vivo development candidate by the first half of 2022.
The Company plans to highlight additional advances to its proprietary technology capabilities, including both genome editing and delivery tools, at upcoming scientific conferences in 2022.
Corporate Updates:

In January, Intellia and Kyverna Therapeutics announced a licensing and collaboration agreement for the development of KYV-201, an allogeneic CD19 CART-cell therapy for the treatment of a variety of B cell-mediated autoimmune diseases. Intellia may exercise an option to lead U.S. commercialization for KYV-201 under a co-development and co-commercialization agreement.
In December 2021, Intellia appointed Derek Hicks as Executive Vice President, Chief Business Officer. Mr. Hicks joins Intellia with more than 25 years of combined business, leadership and biotechnology experience, having most recently served as Head of Business Development at Spark Therapeutics.
Cash Position

Intellia ended the fourth quarter of 2021 with approximately $1.1 billion in cash, cash equivalents and marketable securities.
Intellia’s Presentation at the 40th Annual J.P. Morgan Healthcare Conference

Intellia is scheduled to present virtually at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, at 2:15 p.m. ET. A live audio webcast of Intellia’s presentation can be accessed under the Events and Presentations page of the Investors & Media section on the company’s website at www.intelliatx.com. A replay of the webcast will be available on Intellia’s website for at least two weeks following the presentation.

On January 6, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported an update on its business performance, including preliminary net product sales results of INGREZZA (valbenazine) for 2021, and key clinical development milestones for 2022 and 2023 (Press release, Neurocrine Biosciences, JAN 6, 2022, View Source [SID1234598334]). Kevin Gorman, Chief Executive Officer of Neurocrine Biosciences, will discuss these updates as part of a webcast presentation at the 40th Annual J.P. Morgan Healthcare Conference to be held virtually on Monday, January 10 at 11:15 a.m. Eastern Time, followed by a Question and Answer session at approximately 11:35 a.m. Eastern Time.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Preliminary Fourth Quarter 2021 INGREZZA (valbenazine) Net Product Sales (Unaudited) Highlights

Unaudited preliminary fourth quarter 2021 INGREZZA net sales were approximately $301 million and $296 million on an inventory adjusted basis
Fourth quarter 2021 INGREZZA net sales and total prescriptions grew 25% and 32% respectively vs. fourth quarter of 2020
Quarterly growth driven by record patients on therapy exiting 2021
Commercial expansion to better meet the needs of healthcare professionals across diverse sites of care on track for completion by the end of Q1 2022
The Company plans to provide full-year 2022 INGREZZA sales guidance on its Q4 and FY 2021 Earnings Call on February 11, 2022
"Our fourth quarter and full-year results highlight INGREZZA’s return to growth in a challenging environment. We exited 2021 helping more patients with tardive dyskinesia than ever before. Furthermore, we now have 13 clinical programs in mid-to-late-stage studies which will generate important data readouts over the next two years," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "This year, our priorities are focused on INGREZZA and continuing to advance our broad pipeline. We are uniquely positioned to drive INGREZZA growth and reinvest in our pipeline to develop potential best-in-class medications that are focused on neurological, neuro-endocrine and psychiatric disorders for patients who deserve better treatment options."

Expected Future Milestones and Key Activities

Program

Indication

Milestones / Key Activities

Valbenazine*

(VMAT2 Inhibitor)

Chorea in Huntington Disease

File Supplemental New Drug Application in Second Half (2H) 2022

Adjunctive Treatment of Schizophrenia

Initiate 2nd Registrational Study in 2022; Top-Line Registrational Data in 2023

Dyskinesia Due to Cerebral Palsy

Top-Line Registrational Data in 2023

NBI-827104**

(Selective T-Type CaV

Channel Blocker)

Essential Tremor

Top-Line Phase 2 Data in Mid-2022

Rare Pediatric Epilepsy: CSWS

Top-Line Phase 2 Data in 2H 2022

NBI-1117568†

(Selective M4 Agonist)

Treatment of Schizophrenia

Initiate Phase 2 Study in 2022

New Chemical Entity

Neurological or Psychiatric Indication

Initiate Phase 1 Study in 2022

Crinecerfont

(CRF1 Receptor Antagonist)

Congenital Adrenal Hyperplasia (Adult)

Top-Line Registrational Data in 2023

Congenital Adrenal Hyperplasia (Pediatric)

Top-Line Registrational Data in 2023

NBI-1065845‡

(AMPA Potentiator)

Inadequate Response to Treatment in

Major Depressive Disorder

Phase 2 Data in 2023

NBI-1065846‡

(GPR-139 Agonist)

Anhedonia in Depression

Phase 2 Data in 2023

NBI-921352∝

(Selective NaV1.6 Channel
Blocker)

Focal Onset Seizure in Adults

Phase 2 Data in 2023

Key: VMAT2 = Vesicular Monoamine Transporter 2; CaV = Calcium Channel, Voltage-Gated; CSWS = Epileptic Encephalopathy with Continuous Spike and Wave During Sleep; M4= M4 Muscarinic Receptor; CFR1 = Corticotropin-Releasing Factor Type 1; AMPA = Alpha-Amino-3-Hydroxy-5-Methyl-4-Isoxazole Propionic Acid; GPR = Orphan G Protein Coupled Receptor; NaV1.6 = Sodium Channel, Voltage-Gated

Neurocrine Bioscience Partners: * Mitsubishi Tanabe Pharma Corporation has commercialization rights in East Asia;
** In-Licensed from Idorsia Pharmaceuticals; † In-Licensed from Sosei Group Corporation; ‡ Partnered with Takeda Pharmaceutical Company Limited; ∝ In-Licensed from Xenon Pharmaceuticals

About Tardive Dyskinesia (TD)
Tardive dyskinesia (TD) is an involuntary movement disorder characterized by uncontrollable, abnormal and repetitive movements of the torso, extremities and/or face, which can include hand or foot movements, rocking of the torso, lip smacking, grimacing, tongue protrusion, facial movements or blinking, as well as puckering and pursing of the lips. The condition is associated with taking certain mental health medicines such as antipsychotics, which are commonly prescribed to treat mental illnesses such as bipolar disorder, depression and schizophrenia. In patients with TD, these treatments are thought to result in irregular dopamine signaling in a region of the brain that controls movement. The symptoms of TD can be severe and are often persistent and irreversible. TD is estimated to affect approximately 600,000 people in the U.S.

About INGREZZA (valbenazine) Capsules
INGREZZA, a selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is an FDA-approved product indicated for the treatment of adults with tardive dyskinesia, a condition associated with uncontrollable, abnormal and repetitive movements of the face, torso and/or other body parts.

INGREZZA is thought to work by reducing the amount of dopamine released in a region of the brain that controls movement and motor function, helping to regulate nerve signaling in adults with tardive dyskinesia. VMAT2 is a protein in the brain that packages neurotransmitters, such as dopamine, for transport and release in presynaptic neurons. INGREZZA, developed by Neurocrine Biosciences, is novel in that it selectively inhibits VMAT2 with no appreciable binding affinity for VMAT1, dopaminergic (including D2), serotonergic, adrenergic, histaminergic or muscarinic receptors. Additionally, INGREZZA can be taken for the treatment of tardive dyskinesia as one capsule, once-daily, together with most psychiatric medications such as antipsychotics or antidepressants.

Important Information

Approved Use
INGREZZA (valbenazine) capsules is a prescription medicine used to treat adults with movements in the face, tongue, or other body parts that cannot be controlled (tardive dyskinesia).

It is not known if INGREZZA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Do not take INGREZZA if you:

are allergic to valbenazine, or any of the ingredients in INGREZZA.
INGREZZA may cause serious side effects, including:

Sleepiness (somnolence). Do not drive, operate heavy machinery, or do other dangerous activities until you know how INGREZZA affects you.
Heart rhythm problems (QT prolongation). INGREZZA may cause a heart problem known as QT prolongation.
Symptoms of QT prolongation may include:
• fast, slow, or irregular heartbeat

• shortness of breath

• dizziness or fainting

Tell your healthcare provider right away if you have a change in your heartbeat (a fast or irregular heartbeat), or if you faint.
Abnormal movements (Parkinson-like). Symptoms include: shaking, body stiffness, trouble moving or walking, or keeping your balance.
Before taking INGREZZA, tell your healthcare provider about all of your medical conditions including if you: have liver or heart problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins and herbal supplements.

The most common side effect of INGREZZA is sleepiness (somnolence). Other side effects
include changes in balance (balance problems, dizziness) or an increased risk of falls, headache, feelings of restlessness, dry mouth, constipation, and blurred vision.

These are not all of the possible side effects of INGREZZA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see accompanying INGREZZA full Product Information.

On January 6, 2022 RayzeBio, Inc., a targeted radiopharmaceutical company developing an innovative pipeline against validated solid tumor targets, reported that Ken Song, M.D., President and CEO of the Company, will be presenting at the 40th Annual J.P. Morgan Healthcare Conference being held virtually on Tuesday, January 11, 2022 (Press release, RayzeBio, JAN 6, 2022, View Source [SID1234598372]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

J.P. Morgan Healthcare Conference
Date: Tuesday, January 11, 2022
Time: 11:00 a.m. – 11:25 a.m. EST
Please click on the below link to find additional details about the J.P. Morgan Healthcare Conference
View Source

On January 6, 2022 Paula and Rodger Riney of St. Louis, MO, through the Paula and Rodger Riney Foundation, reported a $40 million grant to support multiple myeloma research at Dana-Farber Cancer Institute (Press release, Dana-Farber Cancer Institute, JAN 6, 2022, View Source [SID1234598389]). The grant represents the largest single award supporting multiple myeloma research in Dana-Farber’s history. The Paula and Rodger Riney Foundation has been a strong supporter of Dana-Farber and with this grant has cumulatively donated nearly $60 million to the Institute.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Multiple myeloma is a challenging cancer that forms in a type of white blood cell called a plasma cell. Dana-Farber has been at the forefront of multiple myeloma therapies over the past two decades, helping to convert myeloma from a fatal disease to a chronic condition for many patients. However, therapeutic resistance and drug-related toxicities continue to take a toll on many patients, underscoring the need for innovative treatments.

"The path to developing new treatments for multiple myeloma is through rigorous research. The most effective way to spur that research is in supporting the scientists doing the complex work. The Riney Family are generous and stalwart supporters, and through this grant and their previous support they continue to make a profound impact on scientific discovery and clinical care. Their leadership will help patients at Dana-Farber and around the world," said Laurie H. Glimcher, MD, President and CEO of Dana-Farber and the Richard and Susan Smith Professor of Medicine at Harvard Medical School.

"My own journey as a myeloma patient—and knowing how many others are also living with this disease—has led us to seek out the individuals, teams, and organizations that are on the leading edge of research," said Rodger Riney. "There is no time to waste in the pursuit of better understanding, treatment, and cures. My family and I feel grateful to be able to support Ken, Paul, and Nikhil and their teams at Dana-Farber who are making incredible inroads. We are humbled by the lifelong dedication that Ken, Paul, and Nikhil bring to myeloma patients suffering from this terrible disease. We hope this gift will inspire others to also support the tremendous work happening every day in Dana-Farber’s labs and clinics."

This new $40 million grant builds upon ongoing work and will deepen and expand approaches for addressing the most complex challenges in myeloma research and improving patient care. Specifically, this grant will:

Renew support for preclinical experiments to identify novel targets and develop new medicines and immune-based therapies for patients;
Fund clinical research designed to test novel myeloma therapies, alone and in combination with standard and experimental treatments, to improve patient outcomes; and,
Support to co-locate myeloma labs at Dana-Farber to facilitate greater cohesion and collaboration among members of the research team.
"I extend my heartfelt thanks to Paula and Rodger Riney for their unprecedented support of our research to develop novel treatments for multiple myeloma. This very generous grant will fast-forward our translation of basic discoveries to clinical trials, ultimately providing innovative treatments for patients and their families," said Ken Anderson, MD, program director at Dana-Farber’s Jerome Lipper Multiple Myeloma Center and LeBow Institute for Myeloma Therapeutics and Kraft Family Professor of Medicine at Harvard Medical School. Anderson will lead the research efforts supported by this grant in close partnership with Nikhil Munshi, MD, director of Basic and Correlative Science at the Jerome Lipper Multiple Myeloma Center and Kraft Family Chair at Dana-Farber. The grant will also provide support for clinical work led by Paul Richardson, MD, clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center and RJ Corman Professor of Medicine at Harvard Medical School.

The Rineys have a strong legacy of supporting multiple myeloma research at Dana-Farber and in 2019 gave a $16.5 million gift to establish the Riney Family Multiple Myeloma Initiative, which has driven groundbreaking research in record time. Examples of recent discoveries by Dana-Farber investigators include:

Bringing therapeutic antibodies, which help immune cells find and attack tumors, to patients with multiple myeloma.
Leading clinical studies demonstrating the remarkable therapeutic effects of CAR T-cells that have been engineered to target multiple myeloma.
Setting the stage for the development of innovative therapies that exploit the unique vulnerabilities of multiple myeloma cells.
Over the past two years, the Paula and Rodger Riney Foundation also made gifts totaling $2.6 million to establish the Riney Family Fund for COVID-19 and Multiple Myeloma Research at Dana-Farber, under Richardson’s direction.

These commitments provide powerful momentum for The Dana-Farber Campaign, an ambitious multi-year $2 billion fundraising effort to prevent, treat, and defy cancer by accelerating revolutionary science, extraordinary care, exceptional expertise, and essential opportunities.