On September 09, 2021 Vor Biopharma, a clinical-stage cell and genome engineering company, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to VOR33, the Company’s lead engineered hematopoietic stem cell (eHSC) therapeutic candidate for the treatment of acute myeloid leukemia (AML) (Press release, PureTech Health, SEP 10, 2021, View Source [SID1234587506]).

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VOR33 consists of CRISPR genome-edited hematopoietic stem and progenitor cells that have been engineered to lack CD33. Once infused, VOR33 is designed to protect patients’ healthy cells from anti-CD33 therapies, such as VCAR33 or Mylotarg (gemtuzumab ozogamicin). VOR33 is intended to replace standard of care transplants for AML patients who are at high risk of relapse and has the potential to seamlessly integrate into current transplant settings.

"Receiving Fast Track designation is an important milestone for Vor, which signals the FDA’s recognition of the serious and life-threatening medical condition of patients facing acute myeloid leukemia and the potential of VOR33 to address this unmet medical need," said Robert Pietrusko, PharmD, Vor’s Chief Regulatory and Quality Officer. "We will continue to work closely with the FDA to expedite the development of VOR33, which is now actively enrolling in its Phase 1/2a clinical trial for AML patients who currently have limited treatment options. We continue to remain on-track to report VOR33’s initial clinical data in the first half of 2022."

VOR33 is the lead product candidate of Vor’s novel scientific platform, which has the mission to create next-generation, treatment-resistant transplants that unlock the potential of targeted cancer therapies by leveraging advances in cell therapy and gene editing. Vor is currently exploring the use of its genome engineered hematopoietic stem cell platform in combination with multiple therapeutic modalities.

Fast Track designation is intended to facilitate development and expedite review of products designed to treat serious and life-threatening conditions with unmet medical needs. The designation is granted upon the FDA’s review of data that demonstrate this potential, along with a product development program that is adequately designed to address the unmet medical need. Therapeutic candidates receiving Fast Track designation may be eligible for priority review and accelerated approval if certain conditions are met.

On September 9, 2021 PAQ Therapeutics ("PAQ"), a biotechnology company harnessing the power of autophagy to restore health and cure disease, and Insilico Medicine ("Insilico"), an end-to-end artificial intelligence (AI)-driven drug discovery company, reported that the companies have entered into a collaboration agreement to develop small molecule therapies designed to hijack the autophagy pathway to address novel disease targets (Press release, PAQ Therapeutics, SEP 9, 2021, View Source [SID1234649950]).

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Under the terms of the agreement, both parties will leverage Insilico’s small molecule compound generation platform Chemistry42 and other AI-powered drug discovery technologies in combination with PAQ’s autophagosome-tethering compound (ATTEC) technology platform to discover novel small molecules with the potential to catalyze and selectively enhance the degradation of disease-causing entities through autophagy.

Autophagy is the natural cellular degradation mechanism that removes unnecessary or dysfunctional components through a lysosome-dependent mechanism. With its powerful ATTEC technology platform, PAQ expands the therapeutic potential of autophagy to target not only disease-causing proteins, but also lipids, pathogens, and other substrates with the goal of restoring health. Insilico’s AI-powered drug discovery platforms utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques to speed the generation of new molecular structures with specific properties, improving the efficiency and success of drug discovery.

"PAQ’s autophagy-dependent approach supports a pivotal moment in the evolution of drug discovery and design by expanding the ability to target the disease-causing substrates underlying diseases with limited or no treatment options," said Feng Ren, Ph.D., Chief Scientific Officer and Head of Drug R&D at Insilico. "Insilico is pleased to partner with PAQ Therapeutics to leverage our AI-driven drug discovery platforms in advancing drug R&D of ATTECs to address unmet medical needs."

"As part of PAQ’s ongoing efforts to therapeutically harness autophagy-dependent degradation, one key next step is to integrate cutting-edge technologies like AI that support our overall hit-finding and drug discovery strategy," said Nan Ji, Ph.D., Co-founder, President and CEO, PAQ Therapeutics. "We are excited to leverage Insilico’s deep expertise and proven track record in AI-driven drug discovery during a pivotal time in our company’s growth and progress."

On September 9, 2021 PharmAbcine Inc. (KOSDAQ: 208340ks), a clinical-stage biotech company focusing on the development of next-generation antibody therapeutics, reported it submitted a Patent Cooperation Treaty (PCT) application for the combination therapy of olinvacimab, the Company’s anti-VEGFR2 antibody, and KEYTRUDA (pembrolizumab), MSD’s anti-PD-1 antibody, for the treatment of cancer (Press release, PharmAbcine, SEP 9, 2021, View Source [SID1234649188]).

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The PCT application is an international application that preserves the priority date. The PCT is an international treaty that has over 150 Contracting States worldwide. It assists applicants in seeking patent protection internationally for their inventions, helps patent offices with their patent granting decisions, and facilitates public access to a wealth of technical information relating to those inventions.

The application, which was submitted jointly with MSD, is based on the Phase Ib olinvacimab and pembrolizumab combo trial in metastatic Triple-Negative Breast Cancer (mTNBC). According to the interim result presented at San Antonio Breast Cancer Symposium (SABCS) 2020, the combo therapy showed encouraging efficacy data, including 50% Overall Response Rate (ORR) and 67% Disease Control Rate (DCR) in the high-dose olinvacimab (16mg/kg) cohort (n=6pts).

mTNBC is a highly malignant type of cancer that shows a high recurrence rate within the first five years after the diagnosis. mTNBC accounts for 15-20% of all breast cancers and shows a 5-year survival rate of approximately 11%. Unlike some other breast cancers, mTNBC does not express estrogen or progesterone receptors or human epidermal growth factor receptor 2 (HER2), and it does not respond to existing cancer drugs designed to target these markers. mTNBC is very difficult to treat, and there are very few FDA approved treatment options for these patients.

Olinvacimab, an anti-VEGFR2 (Vascular Endothelial Growth Factor Receptor 2) fully human antibody, is the Company’s leading pipeline and is undergoing multiple global clinical trials. In early September 2021, PharmAbcine initiated a Phase II olinvacimab and pembrolizumab combo study for the treatment of mTNBC (metastatic Triple-Negative Breast Cancer) in Australia. Also, a Phase II olinvacimab mono study for bevacizumab-nonresponding rGBM (recurrent glioblastoma multiforme) patients at multiple sites in both US and Australia and two Phase Ib olinvacimab and pembrolizumab combo trials in mTNBC and rGBM in Australia are still ongoing.

"Submission of this patent application will be our first step in securing the novelty of the combo therapy." said Dr. Jin-San Yoo, CEO of PharmAbcine. "We will move onto getting our patent secured in multiple countries with great needs for a cancer treatment."

On September 9, 2021 Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported dosing of the first patient in the HERKULES-2 Phase 1b/2 trial evaluating ERAS-007 in combination with various agents in patients with advanced non-small cell lung cancer (NSCLC) (Press release, Erasca, SEP 9, 2021, View Source [SID1234639383]).

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"As the foundation of Erasca’s lung cancer platform, HERKULES-2 is a master protocol designed to inhibit multiple oncogenic drivers of the RAS/MAPK pathway to address high unmet needs in lung cancer. Initially focused on patients with mutant EGFR or KRAS NSCLC, HERKULES-2 will further progress to evaluate other combinations targeting additional subtypes of NSCLC," said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. "Erasca’s series of HERKULES trials also includes tissue-specific master protocols in gastrointestinal cancers and hematological malignancies as well as a tissue-agnostic trial, tailored to evaluate promising combinations to inhibit oncogenic signaling and prevent the emergence of resistance."

HERKULES-2 will initially examine the safety, tolerability, and preliminary efficacy of ERAS-007 in combination with osimertinib (TAGRISSO) in patients with advanced NSCLC harboring an epidermal growth factor receptor mutation (EGFRm). After a recommended dose is determined, the Phase 2 expansion portion will further evaluate the safety and efficacy of the combination in patients whose disease has developed resistance to osimertinib, a setting in which there are currently no approved targeted therapies. Future sub-studies of HERKULES-2 will explore ERAS-007 or the SHP2 inhibitor ERAS-601 in combination with other agents in patients with different mutational subtypes, including a KRAS G12C mutation.

ERAS-007, a potential best-in-class inhibitor of the extracellular signal-regulated kinases (ERK), targets the terminal node of the RAS/MAPK pathway. The broad applicability of ERAS-007 across a wide range of indications and tumor types was recently highlighted in a preclinical study published in Cell Reports Medicine, supporting durable ERK blockade and potent antiproliferative efficacy in both solid tumor and hematological malignancy cell lines. ERAS-007 demonstrated preferential anti-tumor activity for tumor types harboring mutant BRAF, KRAS, NRAS, or HRAS, as well as robust inhibitory activity across a range of mutant KRAS subtypes.

About ERAS-007
ERAS-007 is a potential best-in-class ERK1/2 inhibitor being investigated alone or in combination with different inhibitors targeting upstream nodes of the MAPK pathway as part of Erasca’s MAPKlamp strategy. The extracellular signal-regulated kinases (ERK), ERK1 and ERK2, belong to a family of serine-threonine kinases that regulate cellular signaling and comprise the terminal node of the RAS/MAPK pathway. The broad therapeutic potential of ERAS-007 is being investigated initially across four HERKULES clinical trials that span multiple tumor types and include both monotherapy and combinations with approved and investigational agents, such as RTK, SHP2, RAS, RAF, and/or cell cycle inhibitors. HERKULES-1, a Phase 1b/2 clinical trial for ERAS-007 as a single agent and in combination with the SHP2 inhibitor ERAS-601 (together, Erasca’s first MAPKlamp) in advanced solid tumors, and HERKULES-2, a Phase 1b/2 clinical trial for ERAS-007 in combination with various agents in patients with NSCLC, are currently enrolling patients. HERKULES-3, a Phase 1b/2 clinical trial for ERAS-007 in combination with various agents in patients with gastrointestinal cancers, is expected to begin by year-end. HERKULES-4, a Phase 1b/2 clinical trial for ERAS-007 in combination with various agents in patients with hematologic malignancies, is anticipated to begin in the first quarter of 2022.

On September 9, 2021 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company engineering a novel class of injectable biologics designed to selectively engage and modulate targeted T cells directly within the patient’s body, reported that it will take part in two fireside chats, Baird’s 2021 Global Healthcare Conference, September 14-15 and the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit, September 20-23, 2021 (Press release, Cue Biopharma, SEP 9, 2021, View Source [SID1234608272]). Both conferences will be held virtually.

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During the fireside chats, Cue Biopharma will provide a data update highlighting its lead clinical program, CUE-101, representative of the IL-2 based CUE-100 series for the treatment of second line and beyond patients with HPV+ recurrent/metastatic head and neck cancer. The discussion will also focus on the Company’s latest developments and pipeline progress.

Presentation Details:
Baird’s 2021 Global Healthcare Conference
Date and Time: Tuesday, September 14, 3:45 – 4:15 p.m. EDT
The webcasted fireside chat will be hosted on the conference website and available only to conference participants. Please visit View Source for more information.

Oppenheimer Fall Healthcare Life Sciences & MedTech Summit
Date and Time: Wednesday, September 22, 1:15 – 1:55 p.m. EDT
A live and archived webcast of the fireside chat will be available on the Events page in the Investors and Media section of the Company’s website at www.cuebiopharma.com. The webcast will be archived for 30 days.

About Baird’s 2021 Global Healthcare Conference
Baird’s 2021 Global Healthcare Conference brings institutional and private equity investors together with senior management from over 100 public and privately held companies. The conference will feature companies across the following sectors: Biotechnology, Healthcare Supply Chain & Pharma Services, Healthcare Information Technology, Life Sciences & Diagnostics, Medical Technology and Facilities & Services.

About Oppenheimer Fall Healthcare Life Sciences & MedTech Summit
Oppenheimer & Co. Inc. hosts multiple conferences a year covering many different industries. These conferences bring together corporate leaders, financial sponsors and institutional investors to explore market and sector trends. September 20-22 will feature presentations and one-on-one meetings in a virtual format with a select group of public companies in the Healthcare Life Sciences and MedTech arena. Thursday, September 23 will be the Private Company Day within the Summit and will feature a select group of emerging biotechnology and life science private companies. Investors will have the opportunity to meet virtually with management teams to discuss in detail key therapeutic programs in development and recent corporate updates.