On June 1, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, reported the activation of the initial clinical sites to screen and enroll patients in the company-sponsored Phase 1/2 study evaluating a cryopreserved formulation of GDA-201, a readily available cell therapy candidate for the treatment of follicular and diffuse large B cell lymphomas (NCT05296525) (Press release, Gamida Cell, JUN 1, 2022, View Source [SID1234615381]). On April 26, 2022, Gamida had announced that the U.S. Food and Drug Administration (FDA) cleared its investigational new drug (IND) application and removed the clinical hold for a cryopreserved formulation of GDA-201.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to be screening patients for enrollment in our company-sponsored Phase 1/2 clinical study of our novel, cryopreserved formulation of GDA-201, which has the potential to address the significant unmet need that exists for patients with follicular and diffuse large B cell lymphomas having relapsed or refractory disease," said Ronit Simantov, M.D., chief medical and scientific officer of Gamida Cell. "As described in previously announced clinical data, an investigator-sponsored (IS) study evaluating the fresh formulation of GDA-201 demonstrated encouraging results in heavily pretreated patients with lymphoma. With the initiation of enrollment, we look forward to dosing the first patient in our clinical study of the novel cryopreserved formulation of GDA-201."

GDA-201 leverages Gamida Cell’s proprietary NAM technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity (ADCC). In an investigator-sponsored Phase 1/2 study in patients with relapsed or refractory lymphoma, treatment with the fresh formulation of GDA-201 with rituximab demonstrated significant clinical activity. Of the 19 patients with non-Hodgkin lymphoma (NHL), 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%. The most common Grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia. At the December 2021 Annual Meeting of American Society of Hematology (ASH) (Free ASH Whitepaper), two-year follow-up data were reported on outcomes and cytokine biomarkers associated with survival. The data demonstrated a median duration of response of 16 months (range 5-36 months) and an overall survival at two years of 78% (95% CI, 51%–91%). In the IS study, GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 19 patients with NHL and 16 patients with multiple myeloma.

The study of the cryopreserved formulation of GDA-201 is currently open to enrollment at Henry Ford Health (Detroit, MI) and the Masonic Cancer Center at the University of Minnesota; additional sites will be added in the coming months and updated in Clinicaltrials.gov (NCT05296525). The Phase 1 portion of the study is designed to evaluate the safety of GDA-201 in patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL), marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in two patient cohorts, FL and DLBCL/HGBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR-T or stem cell transplant.

About NAM Technology

Our NAM-enabling technology, supported by positive Phase 3 data, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the US and EU, which is the patient population that will be studied in the GDA-201 Phase 1/2 clinical trial.

For more information about GDA-201, please visit View Source For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

On June 1, 2022 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported the pricing of an underwritten public offering of 10,000,000 shares of its common stock at a public offering price of $4.75 per share (Press release, Concert Pharmaceuticals, JUN 1, 2022, View Source [SID1234615398]). The gross proceeds to Concert, before deducting underwriting discounts and commissions and estimated offering expenses payable by Concert, are expected to be approximately $47.5 million. Concert has granted the underwriters a 30-day option to purchase up to an additional 1,500,000 shares of common stock at the public offering price, less underwriting discounts and commissions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

All shares in the offering are being sold by Concert. The offering is expected to close on or about June 6, 2022, subject to the satisfaction of customary closing conditions.

Concert intends to use the net proceeds from the offering, together with its current cash and cash equivalents, to complete clinical development supporting the filing of its New Drug Application for CTP-543 for the treatment of moderate to severe alopecia areata in adults, conduct pre-commercial activities related to CTP-543 and support its pipeline development, working capital needs and other general corporate purposes.

Jefferies and Truist Securities are acting as joint book-running managers for the offering. JMP Securities, A Citizens Company, and Mizuho Securities are acting as lead managers, and H.C. Wainwright & Co. is acting as co-manager for the offering.

The offering is being made only by means of a written prospectus supplement and prospectus forming part of a shelf registration statement previously filed with the Securities and Exchange Commission (SEC) and declared effective on November 16, 2020. A preliminary prospectus supplement relating to the offering was filed with the SEC. The final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and accompanying prospectus may also be obtained, when available, by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected] or Truist Securities, Inc., Attention: Prospectus Department, 3333 Peachtree Road NE, 9th floor, Atlanta, Georgia 30326, by telephone at (800) 685-4786, email: [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, 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 registration or qualification under the securities laws of any such state or other jurisdiction.

On June 1, 2022 Almac Discovery, a research driven drug discovery company and member of the Almac Group, reported the nomination of a new pre-clinical candidate molecule (ADP-c389), a novel Protein Drug Conjugate (PDC) targeting the tumour-associated antigen ROR1, for the treatment of refractory solid and haematological cancers (Press release, Almac, JUN 1, 2022, View Source [SID1234615313]). This follows an extensive profiling exercise in which ADP-c389 has demonstrated an excellent preclinical efficacy profile in a range of models, a large therapeutic index and robust preclinical development potential.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This nomination represents the first candidate molecule to be generated using Almac Discovery’s novel PDC platform. The platform combines the use of single chain antigen binding domains with Almac’s proprietary linker:payload and conjugation technologies to produce first and best in class compounds that target unmet needs in oncology.

ROR1 is a novel tumour-associated antigen and was selected as the first target for the platform due to its selective expression pattern on different tumour types including triple-negative breast cancer, lung cancer, ovarian and colorectal cancer, and its functional role in tumorigenesis, disease progression and drug resistance. With ADP-c389 in hand, the Almac Discovery team are now also exploring additional clinical applications for the molecule in areas of high unmet need.

Dr Graham Cotton, Head of Protein Therapeutics at Almac Discovery, said: "The nomination of this candidate drug represents a significant value inflection point in in the development of our PDC platform and an important step forward in our search for new treatments for solid tumours. This particular molecule has arisen from our strategic collaboration with Elasmogen Ltd, (Aberdeen UK), with whom we have worked to identify a selective, high affinity single domain binding molecule derived from their shark Variable New Antigen Receptor (VNAR) platform. With this, and other differentiating features included in the design of this compound, we are confident that ADP-c389 has the potential to progress to a Best-in-Class drug against this clinically relevant target."

Dr Alan Lamont, VP Business Development and Licensing added: "We are now seeking to identify a partner to facilitate clinical development of this asset and take this molecule through the next stages of development."

On June 1, 2022 Aadi Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company focused on developing and commercializing precision therapies for genetically defined cancers with alterations in mTOR pathway genes, reported members of the Aadi senior management team will participate in the Jefferies Global Healthcare Conference, to be held in New York, NY June 8-10, 2022 (Press release, Aadi Bioscience, JUN 1, 2022, View Source [SID1234615329]). Founder, Chief Executive Officer and President, Neil Desai, Ph.D. is scheduled to present live during a fireside chat at 10am ET on Thursday, June 9, 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the presentation will be available on the "Event Calendar" page within the Investors & News section of the Company’s website at www.aadibio.com. A replay of the webcast will be available for approximately 30 days following the presentation.

On June 1, 2022 EXACT Therapeutics AS ("EXACT-Tx", Euronext Growth: EXTX), a clinical-stage biopharmaceutical company with a mission to enhance the therapeutic efficacy of medicines through ultrasound-mediated drug delivery, reported the presentation of preclinical results of Acoustic Cluster Therapy (ACT) in a murine brain model, at the Annual International Symposium for Therapeutic Ultrasound (ISTU) Conference being held on 7th -10th June 2022 in Toronto, Canada (Press release, Exact Therapeutics, JUN 1, 2022, View Source [SID1234615347]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The ISTU is a non-profit organization founded in 2001 to increase and diffuse the knowledge of therapeutic ultrasound to the scientific and medical community and facilitate the translation of therapeutic ultrasound techniques into the clinical area for the benefit of patients worldwide.

The work presented by EXACT-Tx at the ISTU demonstrates the possibility of using ACT to help deliver therapeutics to the brain, addressing a significant unmet medical need with close to 98% of drugs in development unable to cross the blood-brain barrier (BBB).

Dr Masha Strømme, Executive Chair EXACT-Tx said: "These data sets have deepened our understanding of the mechanism behind ACT-induced increased BBB permeability and enhanced accumulation of co-injected molecules, and also given us new insights on the spatial and temporal extravasation pattern induced by ACT, important for understanding the mechanism of this therapeutic strategy for drug delivery to the brain."