On April 20, 2021 Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) reported that the companies have amended their collaboration agreement to develop, manufacture and commercialize CTX001, an investigational CRISPR/Cas9-based gene editing therapy that is being developed as a potentially curative therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) (Press release, CRISPR Therapeutics, APR 20, 2021, View Source [SID1234578250]). With this revised agreement, Vertex will deploy the breadth of its established global capabilities and proven experience in manufacturing, development, regulatory, and commercialization to maximize the potential for CTX001 to transform the lives of tens of thousands of patients in the U.S., Europe and other countries. CRISPR Therapeutics will continue to support the development of CTX001 and invest in further innovation to maximize its potential.

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Under the terms of the amended agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 with support from CRISPR Therapeutics. Vertex will be responsible for 60% of program costs and will receive 60% of profits from future sales of CTX001 worldwide, representing a 10% increase in program economics compared to the previous agreement. CRISPR will be responsible for 40% of costs and will receive 40% of profits. Additionally, CRISPR will receive a $900 million upfront payment, with potential for a $200 million payment upon the first regulatory approval of CTX001.

"Cell and genetic therapies are key to our strategy of developing transformative therapies for serious diseases, and this agreement is an important next step in cementing our leadership in these modalities as we bring forward our broad gene and cell-based therapeutics portfolio. As we take the lead on CTX001, we want to acknowledge the foundational contributions by the team at CRISPR Therapeutics," said Jeffrey Leiden, M.D., Ph.D., Executive Chairman of Vertex. "Our increased investment in our partnership with CRISPR is based on the compelling clinical profile of CTX001, which shows its potential to be a durable cure for patients with SCD and TDT, and the rapid progress that we and our partners at CRISPR have made toward registration and commercialization. We see a significant commercial opportunity for CTX001, and we believe we will be able to further enhance that opportunity by fully leveraging the breadth of Vertex’s capabilities – including our established and proven R&D and commercialization expertise in serious diseases – to bring CTX001 to more patients around the world, more quickly."

"Working with Vertex, we have made tremendous progress with CTX001, the first CRISPR/Cas9-based therapy to demonstrate proof of concept in the clinic and together we have broken new ground in the treatment of genetic diseases. We have now dosed more than 30 patients with CTX001, with longest follow-up beyond two years, and we are on track to complete enrollment in both clinical trials this year," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Given the transformative results and momentum that we have generated with this program, we are adopting a new operating model to enable a globally coordinated launch of CTX001, leveraging Vertex’s best-in-class global capabilities and leadership in development, manufacturing, and commercialization to enable this medicine to reach all patients that can benefit from it as quickly as possible. We remain deeply committed to the Sickle Cell and Thalassemia patient communities and look forward to continued success in our partnership with Vertex."

The transaction is subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program.

On April 20, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported updated positive data from the Phase 1 dose escalation portion of the ongoing Phase 1/2 AUGMENT-101 trial of SNDX-5613 in patients with mixed lineage leukemia rearranged (MLLr) and nucleophosmin (NPM1c) mutant relapsed/refractory (R/R) acute leukemias (Press release, Syndax, APR 20, 2021, View Source [SID1234578249]). SNDX-5613 is the Company’s highly selective, oral menin inhibitor. Information on how to access the live video webcast and accompanying slide presentation can be found below.

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"Data reported today further support the potential for SNDX-5613 to induce clinically meaningful responses in patients with genetically-defined acute leukemias," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Notably, robust clinical activity, including multiple complete responses with no evidence of minimal residual disease (MRD-), were observed in heavily pretreated MLLr and NPM1c patients. We have identified a candidate recommended Phase 2 dose (RP2D) and expect to commence the pivotal Phase 2 portion of the trial by the end of the second quarter."

"Genetically-defined acute leukemias, including those harboring MLLr and NPM1c mutations, represent a disease area with a particularly poor prognosis and few effective treatment options," said Eytan M. Stein, M.D., Assistant Attending Physician and Director, Program for Drug Development in Leukemia, Department of Medicine at Memorial Sloan Kettering Cancer Center, and the trial’s principal investigator. "With five-year survival rates in MLLr and NPM1c mutant acute leukemias of 50% or less, novel treatments that can offer clinically-meaningful benefit are desperately needed. I am excited to present evidence that underscores previous observations that SNDX-5613 has the potential to disrupt the treatment paradigm for this disease."

As of a March 12, 2021 data cutoff date, 43 patients with a median of three prior therapies, such as prior stem cell transplant, venetoclax and chemotherapy, were dosed in the Phase 1 portion of the AUGMENT-101 trial. A total of 31 patients were evaluable for efficacy at the time of the data cutoff date, with the remaining patients either not yet at their initial efficacy assessment (n=4) or not harboring either the MLLr or NPM1c mutation (n=8). The overall response rate1 (ORR) among evaluable patients was 48% (n=15), with 67% (n=10) of these responders achieving MRD negative status, with four of these patients proceeding to receive stem cell transplant. The ORR in evaluable patients harboring an MLL-rearrangement (n=24), was 54% (n=13), and in evaluable patients harboring an NPM1c mutation (n=7), was 29% (n=2).

A candidate RP2D of 226 mg every 12 hours was identified for patients who are not receiving a concomitant strong CYP3A4 inhibitor, and 113 mg every 12 hours for patients on a concomitant strong CYP3A4 inhibitor treatment. Eighteen patients treated at the RP2D were efficacy-evaluable and response results observed at the RP2D were consistent with the overall population.

Across all patients enrolled in the trial as of the data cutoff date (n=43), SNDX-5613 was generally well-tolerated, with no discontinuations due to treatment-related adverse events observed in heavily pretreated patients. The only grade 3 or greater related adverse events occurring in at least 5% of patients were QT prolongation, anemia, and differentiation syndrome. Among all patients treated at the candidate RP2D (n=22) as of the data cutoff date, 9% of patients (n=2) experienced grade 3 QT prolongation.

About AUGMENT-101

AUGMENT-101 is a Phase 1/2 open-label trial designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of orally administered SNDX-5613. The Phase 1 dose escalation portion of AUGMENT-101 was separated into two cohorts based on concomitant treatment with a strong CYP3A4 inhibitor. Arm A enrolled patients not receiving a strong CYP3A4 inhibitor, while Arm B enrolled patients receiving a strong CYP3A4 inhibitor. The pivotal Phase 2 portion of the trial will evaluate efficacy, as defined by complete response rate (per International Working Group response criteria), across three expansion cohorts: MLLr acute lymphoblastic leukemia (ALL), MLLr acute myeloid leukemia (AML), and NPM1c mutant AML.

Conference Call and Webcast Details

The Company will host a conference call and webcast today, Tuesday, April 20, 2021 at 8:00 a.m. ET. The presentation will feature the trial’s principal investigator, Eytan M. Stein, M.D., Assistant Attending Physician and Director, Program for Drug Development in Leukemia, Department of Medicine at Memorial Sloan Kettering Cancer Center.

The live video webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

Conference ID: 3129568
Domestic Dial-in Number: (877) 474-0326
International Dial-in Number: (918) 922-6881
Live webcast: View Source

For those unable to participate in the live event, a replay will be available on the Investors section of the Company’s website, www.syndax.com.

About SNDX-5613

SNDX-5613 is a potent, selective, small molecule inhibitor of the menin-MLL binding interaction that is being developed for the treatment of mixed lineage leukemia rearranged (MLLr) acute leukemias including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), and NPM1c mutant AML. In preclinical models of MLLr acute leukemias, SNDX-5613 demonstrated robust, dose-dependent inhibition of tumor growth, resulting in a marked survival benefit. Menin-MLL interaction inhibitors have also demonstrated robust treatment benefit in multiple preclinical models of NPM1c mutant AML, which represents the most frequent genetic abnormality in adult AML. SNDX-5613 is currently being evaluated in the Company’s AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory (R/R) acute leukemias. SNDX-5613 was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with AML.

About Mixed Lineage Leukemia Rearranged Acute Leukemias

Rearrangements of the MLL gene give rise to mixed lineage leukemia rearranged (MLLr) acute leukemias known to have a poor prognosis, with less than 25% of adult patients surviving past five years. MLL rearrangements produce fusion proteins that require interaction with the protein called menin to drive leukemic cancer growth. Disruption of the menin-MLLr interaction has been shown to halt the growth of MLLr leukemic cells. MLLr leukemias, which are routinely diagnosed through currently available cytogenetic or molecular diagnostic techniques, occur in approximately 80% of infant acute leukemias and up to 10% of all acute leukemias. There are currently no approved therapies indicated for MLLr leukemias.

About NPM1c Mutant Acute Myeloid Leukemia

NPM1c mutant acute myeloid leukemia (AML), which is distinguished by point mutations in the NPM1c gene that drive the leukemic phenotype, is the most common type of cytogenetically normal adult AML and represents approximately 30% of all adult AML cases. This subtype of AML has a five year overall survival rate of approximately 50%. Similar to mixed lineage leukemia rearranged (MLLr) leukemias, NPM1c mutant AML is highly dependent on the expression of specific developmental genes shown to be negatively impacted by inhibitors of the menin-MLL interaction. NPM1c mutant AML is routinely diagnosed through currently available screening techniques. There are currently no approved therapies indicated for NPM1c mutant AML.

On April 20, 2021 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a preclinical-stage biopharmaceutical company focused on the discovery, development and commercialization of irreversible small molecules to treat patients with genetically defined cancers, reported the closing of its initial public offering of 9,000,000 shares of its common stock at a public offering price of $17.00 per share (Press release, Biomea Fusion, APR 20, 2021, View Source [SID1234578248]). All of the shares of common stock were offered by Biomea. Biomea’s common stock began trading on The Nasdaq Global Select Market on April 16, 2021 under the ticker symbol "BMEA." The gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Biomea, were $153.0 million. In addition, Biomea has granted the underwriters a 30-day option to purchase up to an additional 1,350,000 shares of common stock at the initial public offering price, less the underwriting discounts and commissions and offering expenses.

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J.P. Morgan Securities LLC, Jefferies LLC and Piper Sandler & Co. are acting as joint book-running managers for the offering.

Registration statements relating to the shares sold in this offering were filed with the Securities and Exchange Commission and became effective on April 15, 2021. The offering was made only by means of a prospectus, copies of which may be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attn: Prospectus Department, by telephone at (800) 747-3924, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On April 20, 2021 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that it will webcast its Quarterly Update Conference Call for the first quarter 2021 on Monday, May 10 at 4:30 p.m. ET / 1:30 p.m. PT. Dr. Helen Torley, president and chief executive officer, will lead the call (Press release, Halozyme, APR 20, 2021, View Source [SID1234578247]). On the same date, Halozyme will release financial results for the first quarter ended March 31, 2021 following the close of trading.

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To register for this conference call, please use this link: View Source After registering, you will receive an email confirmation that includes dial in details and unique conference call codes for entry. Registration is open through the live call. However, to ensure you are connected for the full call, we suggest registering a day in advance or at minimum 10 minutes before the start of the call.

The call will be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit the Investors page of www.halozyme.com approximately 15 minutes prior to the call to register, download and install any necessary audio software. A telephone replay will be available for two weeks after the call by dialing (800) 585-8367 (domestic callers) or (416) 621-4642 (international callers) using replay ID number 1584694.

On April 20, 2021 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) Application to initiate a Phase 1b dose escalation study evaluating VIP152, a highly selective PTEFb/CDK9 inhibitor, in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and Richter syndrome (RS) (Press release, Vincerx Pharma, APR 20, 2021, View Source [SID1234578246]).

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"The IND clearance for VIP152 in CLL is an important milestone for Vincerx, marking our first IND clearance and now second clinical program for what we believe is the most selective CDK9 inhibitor in clinical development," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. "Preclinical data for VIP152 show highly selective, ATP-independent, inhibition of CDK9 which translates to robust on-target activity across key gene targets. Most importantly, we believe this differentiated profile leads to encouraging early clinical activity, with demonstrated durable single-agent activity in hematologic malignancies and heavily pretreated solid tumors. This new dose-escalation study in CLL and Richter syndrome, expected to initiate before year end, builds upon our planned Phase 1b expansion cohort study in MYC-driven hematologic malignancies and solid tumors, which is on track to begin patient dosing in Q2 2021. We are proud of our rapid progress and look forward to continued execution as we advance VIP152 through our targeted oncology clinical programs to address a broad range of aggressive, resistant cancers."

The Phase 1b dose-escalation study will evaluate VIP152 in patients with relapsed/refractory CLL who have failed a Bruton tyrosine kinase inhibitor (BTKi) and venetoclax. Part 1 of the study will enroll CLL patients treated with ³2 prior regimens including either a BTKi or venetoclax. Part 2 of the study will consist of a CLL Phase 1b expansion which will enroll 20 patients with CLL relapsed/refractory to venetoclax and BTKi, and a RS Phase 1b expansion which will enroll 20 patients with CLL transformed to diffuse large B cell lymphoma (DLBCL) who have relapsed after, or been refractory to, at least 1 prior line of therapy for DLBCL and having MYC overexpression/ amplification/translocation. The Company expects to initiate the Phase 1b dose-escalation study in 2H 2021.

The Phase 1b dose-escalation in CLL and RS builds upon Vincerx’s ongoing first-in-human (FIH) study in patients with advanced cancer. Part 2 of the FIH study is on-track to begin patient dosing in 2Q 2021 and will consist of two expansion arms. Arm 1 will enroll up to 30 patients with relapsed/refractory aggressive lymphoma including DLBCL, transformed follicular lymphoma, or blastoid mantle cell lymphoma. Arm 2 will enroll up to 40 patients with advanced solid tumors, including patients with ovarian cancer, triple negative breast cancer, castration-resistant neuroendocrine prostate cancer, and any other solid tumor with MYC aberration. All patients must have confirmed MYC overexpression or translocation.