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.

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"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 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported that Sanofi will be transitioning its rights and obligations related to SAR445136, a zinc finger nuclease gene-edited cell therapy candidate in development by Sangamo and Sanofi for the treatment of sickle cell disease (SCD), back to Sangamo over the first half of 2022 (Press release, Sangamo Therapeutics, JAN 6, 2022, View Source [SID1234607774]). The Companies are collaborating on an orderly transition, while Sangamo explores options to advance the program, including seeking a new collaboration partner. This transition follows Sanofi’s termination for convenience of the Global Research, Development and Commercialization Collaboration and License Agreement (Collaboration Agreement) between the Companies to develop genomic medicines for hemoglobinopathies. Sanofi has elected to transition the SCD program to Sangamo following a recent change in Sanofi’s cell therapy strategy.

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"Although the preliminary Phase 1/2 clinical data for the autologous sickle cell treatment are encouraging, Sanofi has made the decision to terminate the collaboration on the SAR445136 program, which is consistent with our strategy to focus on universal genomic medicine approaches," said John Reed, M.D. Ph.D., Global Head of Research and Development at Sanofi. "Sangamo has been a good partner and this decision is not a reflection on the potential of the SAR445136 program. We continue to view them as a pioneer in the area of genomic medicines and will explore other possible collaboration opportunities as we work together to transition the autologous sickle cell program back to Sangamo."

"We remain committed to progressing this program and believe SAR445136 has the potential to relieve people living with sickle cell disease of some of their most challenging symptoms. We appreciate Sanofi’s collaboration in advancing the SAR445136 program and presenting promising preliminary proof-of-concept clinical data. We expect the Phase 1/2 study to be completed as planned, for final patients in the study to be dosed in the third quarter of 2022, and for discussions regarding potential future clinical trials to continue with health authorities," said Sandy Macrae, CEO of Sangamo. "We will vigorously investigate alternative options to bring this genomic medicine forward to patients."

Sanofi notified Sangamo of its termination for convenience on December 30, 2021. Sangamo expects the Phase 1/2 PRECIZN-1 study of SAR445136 to be completed as planned. Sangamo expects that Sanofi will continue to pay the costs of the Phase 1/2 PRECIZN-1 study until the termination date of June 28, 2022, as contemplated by the Collaboration Agreement.

About the PRECIZN-1 Study

PRECIZN-1 is an ongoing first-in-human, open label, single arm, multi-site Phase 1/2 study in up to eight patients with SCD evaluating the safety and tolerability of cell therapy candidate SAR445136. The therapeutic product is manufactured by ex vivo gene editing of a patient’s own (autologous) hematopoietic stem cells using non-viral delivery of zinc finger nuclease technology targeting the BCL11a gene erythroid-specific enhancer (ESE) to increase endogenous fetal hemoglobin (HbF) production. SAR445136 has received Fast Track Designation from the FDA and Orphan Medicinal Product from the EMA.

Preliminary proof-of-concept results presented at ASH (Free ASH Whitepaper) 2021 as of the cutoff date September 22, 2021 showed that no adverse events (AEs) related to investigational SAR445136 were reported through 91 weeks for the longest-treated patient and through 26 weeks for the most recently treated patient. All four treated patients experienced increases in total hemoglobin, fetal hemoglobin and percent F cells and none required blood transfusions post engraftment. Total hemoglobin stabilized by Week 26 after treatment with SAR445136 in all four patients. Most AEs reported in the screening, mobilization, apheresis and conditioning periods were SCD-related events. One serious adverse event of sickle cell anemia with crisis (vaso-occlusive crisis or VOC) was reported approximately nine months after treatment with SAR445136 in one patient, and no other SCD-related events were reported in the four patients post-infusion. The poster presentation is available on Sangamo’s website in the Investors and Media section under Events and Presentations.

On January 6, 2022 23andMe Holding Co. (Nasdaq: ME) ("23andMe"), a leading consumer genetics and research company, reported the first participant has been dosed in a Phase 1 clinical trial evaluating 23ME-00610 for the treatment of advanced solid tumors (Press release, 23andMe, JAN 6, 2022, View Source [SID1234606743]). 23ME-00610 is 23andMe’s first wholly-owned immuno-oncology (I/O) antibody to enter the clinic. The target for the new investigational antibody, CD200R1, was identified as a promising immuno-oncology target through 23andMe’s proprietary genetic and health survey database.

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"This is an important milestone for 23andMe in our mission to help people access, understand and benefit from the human genome," said Anne Wojcicki, CEO and co-founder, 23andMe. "When we started our Therapeutics group, our goal was to find new medicines validated by human genetics for people with serious unmet medical needs. That’s why we’re excited to move 23ME-00610 into the clinic to potentially help people with cancer who are in need of new treatment options."

The Phase 1 clinical trial is designed to evaluate the safety, tolerability and pharmacokinetics of 23ME-00610 in patients with locally advanced or metastatic solid tumors whose disease has progressed after standard of care treatment. 23ME-00610 is part of 23andMe’s larger drug discovery program of targets validated by human genetics across a spectrum of disease areas, including oncology, immunology, respiratory, and cardiometabolic diseases.

"Our approach to drug discovery is driven by human genetics, and we have an incredibly large database from which to select and advance genetically validated targets more efficiently, and with a potentially higher probability of success, than traditional drug discovery methods currently allow," said Kenneth Hillan, Head of Therapeutics at 23andMe. "23ME-00610 is an exciting example of how we are translating our data into investigational therapeutics."

23andMe has approximately 12 million customers, approximately 80% of whom consent to participate in research. 23andMe scientists study aggregate, de-identified genetics of these participants, alongside more than 4 billion health survey answers. Using its large database of genetically linked health traits and machine learning applied to its proprietary I/O genetic signature, 23andMe is able to pinpoint areas of the genome that contain targets for cancer therapeutics based on human genetics, including the 23ME-00610 target.

23andMe’s Immuno-oncology genetic signature
Using its genetic data, 23andMe can identify immune-related genes that are expected to have an impact on cancer biology. Specifically, germline genetics can reveal which of the immune-related genes harbor genetic variants that also alter an individual’s predisposition for developing cancer. 23andMe has developed an analytical approach, termed the immuno-oncology (I/O) genetic signature, to identify evidence for genetic variants that increase immune function while decreasing cancer risk. Using this approach, 23andMe scientists discovered that three components of the CD200R1 pathway exhibit an I/O genetic signature, including the CD200R1 receptor, the CD200 ligand, and DOK2, a mediator of downstream signaling from CD200R1. Following this genetic insight, 23andMe subsequently generated data consistent with CD200R1’s role in inhibiting anti-tumor responses in immune cells.

About CD200R1 and 23ME-00610
The CD200–CD200R1 axis is an immunological checkpoint that plays a pivotal role in maintenance of immune tolerance. CD200R1 is an inhibitory receptor expressed on T cells and myeloid cells while CD200, the ligand for CD200R1, is highly expressed on certain tumors. Binding of tumor associated CD200 to CD200R1 leads to immune suppression and decreased immune cell killing of cancer cells.

23ME-00610 is a high-affinity humanized monoclonal antibody that is designed to bind to the CD200R1 receptor and prevent the interaction of CD200 and CD200R1. Preclinical data indicate that this mechanism has the potential to reinvigorate tumor-exhausted T-cells and myeloid cells to restore their ability to kill cancer cells.

About the Phase 1 23ME-00610 Study
The first-in-human, multi-center, open-label clinical trial will determine the safety and tolerability of 23ME-00610 in people with locally advanced or metastatic solid malignancies that have progressed after standard therapy. This study will also evaluate preliminary anticancer activity, and the pharmacokinetic and pharmacodynamic profile of 23ME-00610 to identify the optimal dose and schedule for further clinical studies. After the Phase I dose escalation phase is completed, 23andMe will seek to enroll people with specific tumor types to evaluate the potential for anticancer efficacy and changes in pharmacodynamic endpoints in the expansion phase.

Participants with ECOG performance status 0-1, and with histologically diagnosed locally advanced or metastatic solid malignancy that has progressed after standard therapy for the specific tumor type are eligible. Additionally, adolescents 12 years and older with histologically diagnosed locally advanced, or metastatic solid malignancy are eligible for enrolment in the expansion phase.

R&D Day Event Information
To discuss 23ME-00610 and other developments from its Therapeutics group in more detail, the company will host a virtual R&D Day event from 8:00 a.m. to 11:30 am Pacific Time on Tuesday, January 18, 2022. The webcast event can be accessed on the day of the event at View Source A webcast replay will be available at the same address for a limited time within 24 hours after the event.

On January 6, 2022 Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN), a biopharmaceutical company with a portfolio of innovative, late-stage product candidates, reported preliminary net product revenue of NURTEC ODT (rimegepant) for the fourth quarter of 2021 (Press release, Biohaven Pharmaceutical, JAN 6, 2022, View Source [SID1234598503]).

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Based on preliminary unaudited financial information, the Company reported approximately $190 million in net product revenue from sales of NURTEC ODT in the fourth quarter of 2021 and approximately $462 million in net product revenue for 2021. There were no material changes in channel inventory days over the course of the year.

The foregoing information reflects the Company’s estimate with respect to net product revenue for NURTEC ODT based on currently available information, which is preliminary and unaudited, is not a comprehensive statement of the Company’s financial results and is subject to completion of the Company’s financial closing procedures. The Company’s final results, which will be issued upon completion of its closing procedures, may vary from these preliminary estimates.

Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven commented, "NURTEC ODT has changed the treatment paradigm for migraine with its dual-acting, acute and preventive indications. NURTEC ODT established itself as the market leading novel migraine therapy in 2021 and generated an impressive $462 million in net product revenue for the year. The dual indications offer healthcare providers and their patients a single treatment they can customize to treat and prevent migraine attacks. We remain steadfast in our commitment to the migraine community to support programs that drive awareness of migraine and the importance of effective treatment to reduce disability."

Approximately 40 million people in the U.S. and more than one billion individuals worldwide suffer from migraine. Migraine is the third most prevalent illness in the world and the World Health Organization classifies migraine as one of the 10 most disabling medical illnesses.

BJ Jones, Chief Commercial Officer, Migraine & Common Disease, added, "Every day we hear from patients and healthcare providers about the benefits of using NURTEC ODT to help stop a migraine attack quickly and prevent the next one. The overwhelming number of patients that share the positive impact that NURTEC ODT has for them
continues to inspire our team to do more to help those suffering from migraine. Our team is dedicated to continuing to deliver NURTEC ODT to as many patients as possible, especially during times of stress in the pandemic."
NURTEC ODT 75 mg is the only migraine medication approved as a dual therapy for both acute and preventive treatment, and is the first oral CGRP receptor antagonist approved for the preventive treatment of migraine. NURTEC ODT targets a key driver of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. A single dose of NURTEC ODT can deliver fast pain relief with some patients returning to normal function by 60 minutes and has a lasting effect of up to 48 hours for many patients. NURTEC ODT can be taken up to once daily, up to 18 doses/month, as needed to stop migraine attacks or taken every other day to help prevent migraine attacks and reduce the number of monthly migraine days. NURTEC ODT does not have addiction potential and is not associated with medication overuse headache or rebound headache.

NURTEC ODT was approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine in February 2020 and for the preventive treatment of episodic migraine in May 2021 with commercial launch commencing within weeks of the approvals.

On January 6, 2022 Galapagos NV (Euronext & NASDAQ: GLPG) reported that it will participate in the 40th Annual J.P. Morgan Healthcare Conference on January 10-13, 2022 (Press release, Galapagos, JAN 6, 2022, View Source [SID1234598491]).

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Onno van de Stolpe, CEO, will present on Thursday, January 13 at 14.15 CET (8.15 a.m. ET). The presentation will be a live audio webcast and can be accessed via the following link. A replay of the webcast will be available on the Galapagos’ website at www.glpg.com.