UroGen Pharma to Present at Evercore ISI 5th Annual Virtual HealthCONx Conference

On November 21, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it will participate in a virtual fireside chat at the upcoming Evercore ISI 5th Annual HealthCONx Conference on November 29, 2022 at 3:55 PM ET (Press release, UroGen Pharma, NOV 21, 2022, https://investors.urogen.com/news-releases/news-release-details/urogen-pharma-present-evercore-isi-5th-annual-virtual-healthconx [SID1234624278]).

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A live audio webcast will be available in the "Events and Presentations" section of UroGen’s investor website. A replay will be available for approximately 30 days.

Novo Nordisk A/S – Share repurchase programme

On November 21, 2022 Novo Nordisk reported that initiated a share repurchase programme in accordance with Article 5 of Regulation No 596/2014 of the European Parliament and Council of 16 April 2014 (MAR) and the Commission Delegated Regulation (EU) 2016/1052 of 8 March 2016 (the "Safe Harbour Rules") (Press release, Novo Nordisk, NOV 21, 2022, View Source [SID1234624277]). This programme is part of the overall share repurchase programme of up to DKK 24 billion to be executed during a 12- month period beginning 2 February 2022.

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Under the programme initiated 4 November 2022, Novo Nordisk will repurchase B shares for an amount up to DKK 3.8 billion in the period from 7 November 2022 to 30 January 2023.

With the transactions stated above, Novo Nordisk owns a total of 28,282,803 B shares of DKK 0.20 as treasury shares, corresponding to 1.2% of the share capital. The total amount of A and B shares in the company is 2,280,000,000 including treasury shares.

Novo Nordisk expects to repurchase B shares for an amount up to DKK 24 billion during a 12- month period beginning 2 February 2022. As of 18 November 2022, Novo Nordisk has since 2 February 2022 repurchased a total of 26,593,617 B shares at an average share price of DKK 785.54 per B share equal to a transaction value of DKK 20,890,241,156.

Treadwell Therapeutics Announces Presentations at the 2022 San Antonio Breast Cancer Symposium Featuring a Clinical Trial Update on the CFI-402257 and CFI-400945 programs

On November 21, 2022 Treadwell Therapeutics, a clinical-stage biotechnology company developing novel medicines for highly aggressive cancers, reported that four abstracts highlighting CFI-402257 and CFI-400945, the Company’s potent and selective inhibitors of TTK and PLK4, respectively, have been accepted for presentation at the 2022 San Antonio Breast Cancer Symposium (SABCS) being held from December 6-10, 2022 at the Henry B. Gonzalez Convention Center in San Antonio, Texas (Press release, Treadwell Therapeutics, NOV 21, 2022, View Source [SID1234624276]).

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"We are grateful that several abstracts highlighting our TTK and PLK4 inhibitor programs have been selected for presentation," said Dr. Michael Tusche, Treadwell co-CEO. "We look forward to additional data from these studies, as we further characterize the utility of our agents in various breast cancer settings."

Information on the four abstracts:

Abstract Title: An Update to a Phase I Trial of CFI-402257, an oral TTK Inhibitor, in Patients with Advanced Solid Tumors with HER2-Negative Breast Cancer Expansion Cohorts

Abstract Title: CCTG IND.236: A Phase 1b Trial of Combined CFI-402257 and Weekly Paclitaxel in Patients with HER2 Negative (HER2-) Advanced Breast Cancer (aBC)

Abstract Title: CCTG IND.237: A phase II study of CFI-400945 in patients with advanced/metastatic HER2-negative breast cancer

Abstract Title: CCTG IND.239: A Phase 2 Study of Combined CFI-400945 and Durvalumab in Patients with Advanced

FogPharma Announces $178 Million Series D Financing to Advance Pipeline of First-in-Class Helicon Polypeptide Therapeutics Targeting Major Cancer Drivers

On November 21, 2022 FogPharma, a biopharmaceutical company pioneering a new class of precision medicines that could ultimately prove applicable to the vast majority of therapeutic targets, including those previously considered "undruggable," reported a $178 Million Series D financing (Press release, FogPharma, NOV 21, 2022, View Source [SID1234624275]). The financing round includes new investors ARCH Venture Partners, Milky Way Investments and Fidelity Management & Research Company and existing investors VenBio Partners, Deerfield Management, GV, Cormorant Asset Management, funds and accounts advised by T. Rowe Price Associates, Inc., Invus, Farallon Capital Management, HBM Healthcare Investments, Casdin Capital, and PagsGroup, also participated.

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Proceeds from the Series D financing will be used to advance and accelerate FogPharma’s growing pipeline of hyperstabilized α-helical (Helicon) polypeptide therapeutics, a proprietary new class of drugs designed to overcome the limitations of today’s precision medicines with broad applicability to the vast majority of disease targets and therapeutic areas. FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor with potential applicability to significant cancer patient populations, is expected to enter clinical development in mid-2023. In addition, FogPharma is advancing other first-in-class programs against important, biologically validated cancer targets that have remained elusive to other approaches including TEAD, NRAS, Pan-KRAS, ERG and Cyclin E1.

"FogPharma continues to make rapid progress on our moonshot mission to achieve universal druggability – a world where no targets are off-limits to medicine," said Gregory Verdine, Ph.D., founder, chairman and chief executive officer of FogPharma. "We believe that Helicon polypeptides, a compelling new therapeutic modality, represent the future of precision medicine. We are thrilled by the support of our investors and will continue to build our platform capabilities, product pipeline which aims to address a significant percentage of cancer patient populations, and our phenomenal team across all levels as we aim to create one of the most impactful new classes of drugs in history."

In connection with the Series D Financing, Rick Klausner, M.D., has been appointed to FogPharma’s board of directors. In addition, Dr. Verdine has been appointed as chairman of the board.

"The team at FogPharma is building an unprecedented new therapeutic modality and robust pipeline with the potential to make a meaningful difference in the lives of cancer patients," said Dr. Klausner. "I am excited to join the board of directors and be part of something special – particularly at this important time as FogPharma continues to impressively scale its science, team, operations and infrastructure, with the goal of advancing its first Helicon polypeptide therapeutic into the clinic."

Dr. Klausner is currently the founder and chief scientist of Altos Labs and founder and chairman of Lyell Immunopharma. Dr. Klausner was founder and director of Juno Therapeutics and founder and director of GRAIL. He is also the chairman of Sonoma Biotherapeutics and co-founder and chairman of Lifemine Therapeutics. Previously, Dr. Klausner served as senior vice president, chief medical officer and chief opportunity officer of Illumina Corporation and as executive director for global health for the Bill and Melinda Gates Foundation. Dr. Klausner was appointed by Presidents Clinton and Bush as the eleventh director of the U.S. National Cancer Institute (NCI) between 1995 and 2001. Dr. Klausner served as chief of the Cell Biology and Metabolism Branch of the National Institute of Child Health and Human Development as well as a past president of the American Society of Clinical Investigation. He has served in senior advisory roles to the U.S., Norwegian, Qatari and Indian governments.

About FogPharma’s Universal Druggability Platform and Helicon Polypeptide Therapeutics

Existing drug classes are limited in both reach and applicability, with more than 80% of known human protein disease targets considered "undruggable" because they are beyond the reach of both antibodies and small molecules.

FogPharma’s Helicon peptide drug discovery engine integrates directed evolution, proprietary α-helix conformational hyperstabilization chemistry, highly multiplexed drug optimization technology, artificial intelligence including deep learning and machine learning, structure-based drug discovery, cancer genomics and biology, and multiscale manufacturing to rapidly discover Helicon polypeptide therapeutics. This novel therapeutic modality combines the targeting strength and specificity of antibodies with the broad tissue distribution, intracellular target engagement and oral dosing optionality of small molecules to address the limitations of today’s precision medicines and reach the most difficult targets – achieving universal druggability.

About FOG-001

FogPharma’s lead Helicon polypeptide development candidate, FOG-001, a first-and-only-in-class direct TCF-blocking β-catenin inhibitor. Dysregulation of the Wnt/β-catenin signaling pathway has been shown to occur in at least 20% of all human cancers. In the U.S. alone, FOG-001 has the potential to become a new treatment option for >1 million patients suffering from a broad range of intractable cancers.

In biochemical and cellular studies, FOG-001 has been shown to potently, precisely and selectively disrupt the interaction of β-catenin with its obligate downstream transcription factor, TCF. Preclinical studies have demonstrated the ability of FOG-001 to cause tumor growth inhibition and regression by disrupting β-catenin-dependent signaling.

FOG-001 is the inaugural member of FogPharma’s TCF-Catenix family of direct-acting β-catenin antagonists and combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: FOG-001 acts inside the cell, where it directly binds the key oncogenic driver β-catenin; and FOG-001 blocks TCF-β-catenin engagement at the most downstream node in the canonical Wnt pathway, thus abrogating the signal transmission mechanism by which most, if not all, known Wnt pathway mutations are believed to drive oncogenesis.

FogPharma plans to submit an investigational new drug (IND) application for FOG-001 to the U.S. Food and Drug Administration (FDA) and initiate clinical development by mid-2023.

Caribou Biosciences Announces FDA Clearance of IND Application for CB-011, an Allogeneic Anti-BCMA CAR-T Cell Therapy for the Treatment of Relapsed or Refractory Multiple Myeloma

On November 21, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported that it has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for CB-011, a genome-edited allogeneic anti-BCMA CAR-T cell therapy with immune cloaking (Press release, Caribou Biosciences, NOV 21, 2022, View Source [SID1234624274]). The CaMMouflage Phase 1 clinical trial, a multicenter, open-label study to evaluate the safety and efficacy of a single dose of CB-011 in adult patients with relapsed or refractory multiple myeloma (r/r MM), is expected to initiate patient enrollment for treatment at dose level 1 (50×106 CAR-T cells) in early 2023.

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"Clearance of our second IND application represents another key milestone for our pipeline of promising allogeneic cell therapies designed to have enhanced persistence of antitumor activity," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "CB-011 is designed with an immune cloaking strategy to reduce rejection of the cell therapy by a patient’s T and NK cells and we use Caribou’s highly precise and specific Cas12a chRDNA genome-editing technology to manufacture this product candidate. We are excited to develop CB-011 as an off-the-shelf cell therapy that may reach a broader number of patients with multiple myeloma than are currently being served, and we look forward to initiating patient enrollment in the CaMMouflage trial in early 2023."

CB-011 is the second allogeneic cell therapy advancing into clinical development from Caribou’s CAR-T cell platform targeting hematologic malignancies. Caribou’s first allogeneic cell therapy, CB-010, an allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout, is being evaluated in the ongoing ANTLER Phase 1 clinical trial in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). Encouraging safety and antitumor activity for CB-010 at dose level 1 have been reported from the ANTLER trial.

"CAR-T cell therapies have shown great promise for treating patients with relapsed or refractory multiple myeloma," said Sundar Jagannath, M.D., director of the Multiple Myeloma Center of Excellence at Tisch Cancer Institute, Mount Sinai Hospital, New York. "Allogeneic, or ‘off-the-shelf,’ CAR-T cell therapies would provide a great option for patients with multiple myeloma, helping to overcome the need for bridging therapies as well as variable quality and manufacturing timelines of autologous CAR-T cells."

CB-011 is the first allogeneic anti-BCMA CAR-T cell therapy, to Caribou’s knowledge, that is engineered to improve persistence of antitumor response through an immune cloaking genome-

editing approach that removes the B2M protein and inserts a B2M–HLA-E fusion protein. CB-011 has four edits implemented via Caribou’s Cas12a CRISPR hybrid RNA-DNA (chRDNA) technology:
•Edits 1 and 2 – A humanized anti-BCMA CAR is site-specifically inserted into the TRAC gene to target cancer cells, thereby knocking out expression of the T cell receptor to reduce the risk of graft versus host disease (GvHD).
•Edits 3 and 4 – A B2M–HLA-E peptide fusion gene is site-specifically inserted into the B2M gene of the CAR-T cells to prevent recognition and rejection by patient T cells and blunt rejection by NK cells. These edits knock out endogenous B2M expression, eliminating endogenous HLA class I presentation and reducing T cell-mediated rejection, while enabling expression of B2M–HLA-E needed to inhibit NK cell-mediated rejection.

About the CaMMouflage Trial
The CaMMouflage Phase 1 trial is an open-label, multicenter clinical trial designed to evaluate CB-011 in adults with relapsed or refractory multiple myeloma (r/r MM). Part A, a 3+3 dose escalation design, will evaluate the safety and tolerability of CB-011 at multiple dose levels and will be utilized to determine the maximum tolerated dose and/or the recommended Phase 2 dose. Part B is the dose expansion portion with the primary objective of determining tumor response after a single dose of CB-011. CaMMouflage will include patients who have had 3 or more prior lines of therapy and will exclude patients who have received a BCMA-targeted therapy within the last 3 months and/or any prior CAR-T cell therapy.

Caribou plans to initiate patient enrollment in the CaMMouflage trial to treat patients with a single administration of CB-011 at dose level 1 (50×106 CAR-T cells) in early 2023.

About Multiple Myeloma
Multiple myeloma (MM) is the second most common hematologic malignancy in the United States. According to the National Cancer Institute, an estimated 160,000 people in 2019 were living with MM and an estimated 34,000 individuals are diagnosed with MM each year in the United States. Approximately 50% of patients with MM relapse and have their cancer return after first line treatment or are refractory and do not respond to current treatments.

About Caribou’s Novel Next-Generation CRISPR Platform
CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA technology to carry out multiple edits at high efficiency, including multiplex gene insertions, to develop CRISPR-edited therapies.