On May 12, 2022 Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported two key executive appointments. Dr. Bruce Rogers has been named President of Morphic Therapeutic and Dr. Blaise Lippa, has been named Chief Scientific Officer (Press release, Morphic Therapeutic, MAY 12, 2022, View Source [SID1234614397]). Dr. Rogers previously served as Morphic’s Chief Scientific Officer and Dr. Lippa served as Morphic’s Senior Vice President and Head of Molecular Discovery.

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"Bruce Rogers has been an invaluable leader on the team that built the MInT discovery platform and has been a tireless champion of a science-driven enterprise focused on patients," commented Praveen Tipirneni, MD, Chief Executive Officer of Morphic Therapeutic. "Bruce has the scientific expertise, strategic vision plus innate sense of teamwork to drive Morphic to greater success as we advance MORF-057 in clinical studies for inflammatory bowel disease while reaping the benefits of the MInT platform in additional indications. On behalf of the Board of Directors and the entire Morphic team, it is my great pleasure to congratulate him on the well-deserved appointment to President and I look forward to our continued partnership as Morphic grows in its mission to create integrin medicines."

"I am extremely humbled by the opportunity to lead the Morphic Team that has made such tremendous scientific and clinical achievements in our short history. I believe that we still have a great deal to accomplish, and my objective is to expand upon our culture of teamwork, collaboration, tenacity, and hypothesis-driven challenge that characterizes Morphic today in our efforts to bring integrin targeted medicines to patients," commented Bruce Rogers, President of Morphic Therapeutic.

"Blaise is a chemistry-driven drug designer first, who utilized his broader scientific skills to significantly contribute to the expansion of our MInT platform, enabling for Morphic a sustained ability to discover small molecule drugs against targets where only biologics had succeeded before," continued Bruce. "Expanding his role to lead all of discovery research is a recognition of his capabilities, talents, and contributions to Morphic."

Blaise Lippa commented, "MORF-057, the first wholly owned candidate generated by the MInT Platform, has achieved great success thus far. However, the integrin receptor family and the MInT Platform provide enormous opportunities to expand Morphic’s portfolio of therapeutic programs. I am honored to lead the efforts of the Research team as we continue to push scientific boundaries to create truly transformative medicines that broadly impact human health."

Bruce Rogers has served as Morphic’s Chief Scientific Officer since 2016. Prior to Morphic, Dr. Rogers was the Head of Neuro-Opportunities at Pfizer, where he led a team focused on entrepreneurial approaches to long standing CNS biology challenges. Bruce spent over 16 years in positions of increasing responsibility within the medicinal chemistry organization at Pfizer and Pharmacia, and during his time there led multiple discovery and early clinical development teams, with his group advancing over a dozen small molecule candidates into clinical trials. Bruce has co-authored more than 60 scientific publications, reviews and abstracts and is a co-inventor on over 80 patents and patent applications. He holds a BA in chemistry from the University of Minnesota and a PhD in organic chemistry from the University of California. He was a National Institutes of Health postdoctoral fellow at the University of California at Irvine prior to joining the pharmaceutical industry.

Blaise Lippa previously served as Morphic’s Senior Vice President of Molecular Discovery and was part of the founding team at Morphic. Prior to this, Blaise was a Senior Director of Medicinal Chemistry at Cubist Pharmaceuticals for seven years until the company’s acquisition by Merck. There he led teams that advanced
multiple compounds to the clinic and served on a development team that achieved an NDA. Blaise began his career at Pfizer nine years earlier, where he conducted advanced drug design in multiple therapeutic indications, and initiated a project that led to the approved drug Daurismo (glasdegib) for leukemia. Blaise is an author of over 60 publications and patents. Blaise holds dual BSc degrees in chemistry and molecular biology from the University of Michigan, and a PhD from Stanford University.

On May 12, 2022 Volastra Therapeutics, an oncology company focused on exploiting chromosomal instability (CIN) as a vulnerability for cancer cells, reported that members of its management team are scheduled to participate in a fireside chat at the 2022 Guggenheim Synthetic Lethality Day on May 16th at 11:30 a.m. ET (Press release, Volastra Therapeutics, MAY 12, 2022, View Source [SID1234614417]).

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A live webcast of the fireside chat can be accessed in the News and Views section of the company’s website at View Source A replay of the webcast will be archived on the company’s website for 90 days.

On May 12, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported the details of three abstracts that it will present at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress (EHA2022 Congress), being held from June 9 – June 12 in Vienna, Austria (Press release, Gracell Biotechnologies, MAY 12, 2022, View Source [SID1234614436]). The abstracts highlight the clinical data from ongoing investigator-initiated trials (IITs) of BCMA/CD19 dual-targeting FasTCAR candidate GC012F in two indications of B-cell non-hodgkin’s lymphoma (B-NHL) and relapsed/refractory multiple myeloma (RRMM), and allogeneic TruUCAR candidate GC502 in B-cell acute lymphoblastic leukemia (B-ALL).

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"We are very excited to share our data for both our FasTCAR candidate GC012F in two indications of RRMM and B-NHL, and allogeneic TruUCAR candidate GC502 in B-ALL at the EHA (Free EHA Whitepaper)2022 Congress," said Dr. Martina Sersch, Chief Medical Officer of Gracell. "The new data, including the expanded indication of GC012F into B-NHL, demonstrates the potential of our platforms and provides further validation. The clinical data of BCMA/CD19 dual-targeting GC012F in the treatment of B-NHL shows promising early results, along with benefits of the next-day manufacturing enabled by the FasTCAR platform. The CD19/CD7 dual-directed CAR-T therapy GC502 is our second allogeneic candidate on our TruUCAR platform, demonstrating the potential wide applicability of the TruUCAR design."

BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of B-NHL

GC012F is an autologous CAR-T therapeutic candidate dual-targeting B cell maturation antigen (BCMA) and CD19. It is developed using Gracell’s proprietary FasTCAR platform which enables next-day manufacturing, and is currently being evaluated in IITs in China including in RRMM and B-NHL. GC012F is the first BCMA/CD19 dual-targeting CAR-T in human trials for B-NHL.

Gracell will present the early results of the first-in-human phase 1 IIT in China evaluating the safety and tolerability of GC012F in B-NHL patients. Three patients who had received a median of two prior lines of therapy were enrolled, all of which presented with bulky disease. As of the February 22, 2022 data cutoff date, the enrolled patients had received one single infusion of GC012F at three different doses of 3.7×104 cells/kg and 2-3×105 cells/kg.

All three patients had achieved a complete response (CR) confirmed by PET- CT at day 28 after GC012F infusion. At 3-month follow-up, both of the two assessable patients had ongoing response. No dose-limiting toxicities were observed and no immune effector cell-associated neurotoxicity syndrome (ICANS) were observed. CRS presented as Grade 1 in two patients and Grade 3 in one patient (duration of two days) with no Grade 4 or 5 events.

Details of the presentation are as follows:

Abstract title: First-in-human study of CD19/BCMA dual-targeting FasTCAR-T GC012F for patients with relapsed/refractory B-cell non-Hodgkin’s lymphoma
Session title: Poster session
Presentation time: Friday, June 10 from 4:30 – 5:45 PM CEST
BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of RRMM

Gracell will also present as an oral abstract presentation the updated results from the first-in-human IIT evaluating GC012F for the treatment of RRMM patients. This data is currently under embargo and will be published on the EHA (Free EHA Whitepaper)2022 Hybrid Congress website on Thursday, May 26 concurrently with ASCO (Free ASCO Whitepaper).

Details of the presentation are as follows:

Abstract title: Updated results of a multicenter first-in-human study of BCMA/CD19 dual-targeting FasTCAR-T GC012F for patients with relapsed/refractory multiple myeloma (RRMM)
Session title: Relapsed/refractory myeloma: BCMA-directed therapies
Presentation time: Sunday, June 12 from 11:30 AM – 12:45 PM CEST
Presentation location: Hall A2-A3
CD19/CD7 Dual-directed Allogeneic TruUCAR-T GC502 for the Treatment of B-ALL

GC502 leverages the novel dual-directed CAR design of Gracell’s proprietary TruUCAR platform, designed to generate high-quality allogeneic CAR-T cell therapies that can be administered "off-the-shelf" at lower cost and with faster patient’s access. TruUCAR-enabled GC502 utilizes the dual-directed CAR design with one CAR targeting CD19 on malignant cells and a second CAR targeting CD7 to suppress host-versus-graft rejection. An enhancer molecule is embedded in the basic construct of TruUCAR to enhance proliferation of TruUCAR T cells.

Between September 2021 and January 2022, four r/r B-ALL patients were enrolled and treated in an open-label, non-randomized, prospective IIT in China in two different dose levels and with two different formulations. Patients were heavily pretreated, and all had previously received either autologous or donor derived CD19 or CD19/CD22 targeted CAR-T therapy. As of the January 28, 2022 data cutoff date, all four patients had received a single dose of GC502, including one patient at dose level 1 (DL1) 1.0×107 cells/kg and three patients at dose level 2 (DL2) 1.5×107 cells/kg. Patients received a Flu/Cy based lymphodepletion regimen prior to treatment with GC502.

Three of four patients achieved minimal residual disease negative complete response or complete response with incomplete count recovery (MRD- CR/CRi), and one patient achieved a partial response at month one and subsequently received allogeneic hematopoietic stem-cell transplantation (allo-HSCT) on day 39.

Cytokine release syndrome (CRS) presented as Grade 2 and Grade 3 with no Grade 4 or 5 events. No immune effector cell-associated neurotoxicity syndrome (ICANS) or acute graft-versus-host disease (aGvHD) were observed.

Details of the presentation are as follows:

Abstract title: Early results of a safety and efficacy study of allogeneic TruUCARTM GC502 in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL)
Session title: Poster session
Presentation time: Friday, June 10 from 4:30 – 5:45 PM CEST
For more information about the EHA (Free EHA Whitepaper)2022 Hybrid Congress, visit www.ehaweb.org.

About GC012F

GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being evaluated in IIT studies in China for the treatment of multiple myeloma and B-cell non-Hodgkin’s lymphoma. GC012F simultaneously targets CD19 and BCMA to drive fast, deep and durable responses, which can potentially improve efficacy and reduce relapse in multiple myeloma and B-NHL patients.

About B-NHL

Non-Hodgkin’s lymphoma (NHL) is a group of blood cancers that developed from lymphocytes, most commonly derived from B cells (B-NHL). Globally, approximately 510,000 patients are diagnosed with NHL every year with about 80,470 patients expected to be diagnosed with NHL in the United States in 2022[1]. B-NHL accounts for approximately 85% of NHL diagnoses.

[1] Data source: American Cancer Society

About GC502

GC502 is a TruUCAR-enabled CD19/CD7 dual-directed, off-the-shelf allogeneic CAR-T product candidate that is being studied in an ongoing Phase 1 IIT in China for the treatment of B-cell malignancies. GC502 is manufactured using T cells from non-human leukocyte antigen (HLA) matched healthy donors. An enhancer molecule is embedded in the basic construct of TruUCAR to enhance proliferation of TruUCAR T cells. Optimized for CD19/CD7 dual-CAR functionality and in vivo durability, GC502 has demonstrated robust anti-tumor effects with potential to suppress host versus graft (HvG) rejection in preclinical models.

About B-ALL

Acute lymphoblastic leukemia (ALL) is a type of blood cancer characterized by proliferation of immature lymphocytes in the bone marrow, which can involve either T lymphocytes (T-ALL), or B lymphocytes (B-ALL). Globally, approximately 64,000 patients are diagnosed with ALL every year with an estimated 6,660 new cases to be diagnosed in the United States in 2022[2]. B-ALL accounts for 75% of ALL diagnoses in adults.

[2] Data source: American Cancer Society

About FasTCAR

CAR-T cells manufactured on Gracell’s proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, and, together with fast turnaround time, enables enhanced accessibility of cell therapies for cancer patients.

About TruUCAR

TruUCAR is Gracell’s proprietary technology platform and is designed to generate CAR-T cell therapies from high quality allogeneic T cells that can be administered "off-the-shelf" at lower cost and with improved accessibility of cell therapies for cancer patients. With differentiated design enabled by gene editing, TruUCAR is designed to control HvG as well as GvHD without the need for being co-administered with additional strong immunosuppressant after conventional lymphodepletion. The novel dual-CAR design allows tumor antigen-CAR moiety to target malignant cells, while the CD7 CAR moiety is designed to suppress rejection (HvG response) of allogeneic CAR-T cells by host T and NK cells (HvG).

On May 12, 2022 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the first quarter ended March 31, 2022 (Press release, Crinetics Pharmaceuticals, MAY 12, 2022, View Source [SID1234614481]).

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"We continue to build the foundation needed to establish Crinetics as the world’s leading endocrine company, as recent progress has strengthened our clinical dataset, balance sheet and leadership team," said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. "This is highlighted by CRN04777’s recent Phase 1 top-line results, which further demonstrated the molecule’s dose-dependent effects on biomarkers that are critically important to the pathophysiology and medical management of hyperinsulinism. Later this quarter, we expect to report data from a healthy volunteer study designed to evaluate CRN04894’s potential to counteract the effects of excess ACTH with a novel mechanism of action. The pharmacologic proof-of-concept these Phase 1 studies aim to achieve highlights the unique efficiency of our endocrine drug development paradigm. Looking forward, we will continue to combine the advantages of this paradigm with the efforts of our deeply talented drug discovery team to thoughtfully expand our pipeline as we work to advance our current clinical-stage candidates towards registration."

First Quarter 2022 and Recent Highlights

Reported positive top-line results from multiple-ascending dose (MAD) cohorts of the CRN04777 Phase 1 study. In March 2022, Crinetics announced positive data from the MAD cohorts of a Phase 1 study of CRN04777, a somatostatin receptor type 5 (SST5) agonist being developed as a treatment for congenital and syndromic hyperinsulinisms. The results built upon previously reported pharmacologic proof-of-concept data from the trial’s single-ascending dose (SAD) cohorts, as they showed strong dose-dependent suppression of fasting insulin as well as dose-dependent suppression of sulfonylurea-induced insulin secretion. Pharmacokinetic data indicated CRN04777 was orally bioavailable and supported a once daily dosing schedule. CRN04777 was shown to be well tolerated in the Phase 1 trial, with no dose discontinuations due to adverse events. Crinetics plans to initiate a Phase 2 study of CRN04777 in congenital hyperinsulinism patients in the second half of 2022, following discussions with global regulators.

Entered into strategic licensing agreement with Sanwa Kagaku Kenkyusho Co., Ltd. ("Sanwa") for the development and commercialization of paltusotine in Japan. Crinetics received $13 million upfront in connection with the license agreement and is also eligible to receive payments related to development, regulatory, and commercial milestones. In addition, Crinetics will be eligible to receive tiered royalties on net product sales in Japan should paltusotine receive marketing approval in Japan. In exchange, Sanwa was granted an exclusive right to develop and commercialize paltusotine in Japan and will assume all costs associated with clinical trials and regulatory applications in the territory. Crinetics retains all rights to develop and commercialize paltusotine outside of Japan.

Strengthened balance sheet with successful $125 million common stock offering. In April 2022, Crinetics completed an underwritten follow-on offering of 5,625,563 shares of its common stock at a price to the public of $22.22 per share, raising gross proceeds of approximately $125.0 million.

Strengthened company leadership with appointments to management team and Board of Directors. In the first quarter of 2022, Crinetics built upon its strong leadership and scientific expertise by appointing James Hassard to the role of chief commercial officer, Chris Robillard to the role of chief business officer, and Dr. Rogério Vivaldi Coelho and Caren Deardorf to the Board of Directors.
First Quarter 2022 Financial Results

Research and development expenses were $28.3 million for the three months ended March 31, 2022, compared to $17.6 million for the same period in 2021. The increase was primarily attributable to increased spending on manufacturing and development activities of $7.0 million associated with our clinical and nonclinical activities for paltusotine and our other clinical and preclinical programs, and an increase in personnel costs of $3.0 million, of which stock-based compensation was $1.4 million.

General and administrative expenses were $8.7 million for the three months ended March 31, 2022, compared to $5.3 million for the same period in 2021. The increase was primarily attributable to an increase in personnel costs of $2.1 million, of which stock compensation was $1.0 million.

Net loss for the three months ended March 31, 2022, was $34.6 million, compared to a net loss of $22.9 million for the same period in 2021.

Revenues were $3.1 million for the three months ended March 31, 2022, consisting of license revenue recognized from the Sanwa license agreement.

Unrestricted cash, cash equivalents and investments totaled $319.7 million as of March 31, 2022, compared to $333.7 million as of December 31, 2021. The $319.7 million in unrestricted cash, cash equivalents and investments does not include the $125.0 million in gross proceeds from the company’s April 2022 common stock offering. Based on its current plans, the company expects that current cash, cash equivalents and short-term investments will fund its current operating plan into the second half of 2024.

The company had 53,505,809 common shares outstanding as of May 9, 2022.

On May 12, 2022 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), a biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported financial results for the quarter ended March 31, 2022, and provided a business update (Press release, Diffusion Pharmaceuticals, MAY 12, 2022, View Source [SID1234615255]).

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Business Updates

Completed Dosing in Altitude Trial: The Company announced the last participant in this study of its lead product candidate, trans sodium crocetinate ("TSC"), completed dosing in April 2022 and topline data are expected in June 2022. Two abstracts, based on blinded, aggregated (placebo and treatment), interim physiological data from a subset of study participants, will be presented by the clinical investigators at the Undersea and Hyperbaric Medical Society’s Annual Scientific Meeting in Reno, Nevada being held from May 22-26, 2022.
Continued Enrollment in TSC ILD-DLCO Trial: Diffusion announced dosing of the first patients in December 2021 and enrollment continued during the first quarter of 2022. The Company currently anticipates completing dosing in the second half of 2022 and reporting topline results within two months of study completion.
Expanded Scientific Advisory Board: In February 2022, the Company announced the addition of five prominent radiation and medical oncologists to its Scientific Advisory Board ("SAB") to further support development of TSC, as an adjunctive treatment for hypoxic solid tumors.
Maintained Solid Cash Position: As of March 31, 2022, Diffusion reported $32.6 million in cash, cash equivalents, and marketable securities, which is expected to fund the Company’s operating expenses and capital expenditure requirements through 2023.
Regained Nasdaq Compliance: In May 2022, the Company announced that it had regained compliance with the Nasdaq Capital Market’s continued listing requirements, following the bid price of the Company’s common stock closing above $1.00 for 10 consecutive trading days.
"We believe 2022 has the potential to be a transformational year, as we anticipate the topline data readout from the Altitude Trial in June and the completion of patient dosing in our ILD-DLCO trial later this year," commented Robert Cobuzzi, Jr., Ph.D., President and Chief Executive Officer of Diffusion. "With our strong cash position, experienced development team, and the appointment of our new and distinguished SAB members, we believe Diffusion is now effectively positioned to launch our Hypoxic Solid Tumor Program aimed at evaluating TSC as an adjunctive treatment for hypoxic solid tumors. Our team worked hard during the first quarter, evaluating various options in designing an appropriate clinical study protocol and amending our oncology investigational new drug application for TSC to support a start date for the first trial in the program in the second half of 2022. We look forward to providing updates on these development activities that we hope will drive both patient and shareholder value."

Financial Results

Research and development expenses in the first quarter of 2022 were $2.4 million, compared to $2.9 million in the prior year period. A significant portion of this decrease was attributable to the timing of clinical trials and drug manufacturing, offset by an increase in salaries, wages and stock-based compensation related to increased headcount.
General and administrative expenses were $2.1 million during the first quarter of 2022 versus $1.7 million in the comparable quarter last year. The increase compared to the prior year period was mainly due to an increase in salaries, wages, and stock-based compensation related to increased headcount, as well as an increase in outside professional fees to support business activities during the quarter.
As of March 31, 2022, Diffusion had cash, cash equivalents, and marketable securities of $32.6 million, compared to $46.6 million as of March 31, 2021.