Poseida Therapeutics Presents Preliminary Results from Phase 1 Trial of P-PSMA-101 at the 6th Annual CAR-TCR Summit

On August 31, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported preliminary results from its Phase 1 clinical trial of P-PSMA-101, the Company’s solid tumor autologous CAR-T product candidate to treat patients with metastatic castrate-resistant prostate cancer (mCRPC) (Press release, Poseida Therapeutics, AUG 31, 2021, View Source [SID1234587035]). These data will be presented at the 6th Annual CAR-TCR Summit virtual meeting at 10:00am ET today in a presentation entitled, "P-PSMA-101 is a High-Tscm Autologous CAR-T Targeting PSMA Producing Exceptionally Deep and Durable Responses in Castration-Resistant Metastatic Prostate Cancer."

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"We are excited about the preliminary data from our Phase 1 trial of P-PSMA-101, which provides further evidence of the effectiveness of our CAR-T platform for solid tumor cancers," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida, who will present at the CAR-TCR Summit. "To date, other CAR-T therapeutics have not had much success outside of hematologic malignancies. The deep and durable responses in our trial demonstrate that CAR-T products have the potential to work well against solid tumors, even at low doses, when using the appropriate technology platform."

Efficacy:

As of the cutoff date, the study had enrolled a total of nine patients with mCRPC: five patients at Dose A who each received a single treatment of 0.25X10E6 cells/kg (an average of about 20M cells), and four patients at Dose B, who each received a single treatment of 0.75X10E6 cells/kg (an average of about 60M cells). All patients received a lymphodepletion regimen consisting of 30 mg/m2 fludarabine + 300 mg/m2 cyclophosphamide. Patients were heavily pre-treated, having received an average of six prior lines of therapy with a median time since diagnosis of 6.4 years.

Key findings included:

Five patients dosed showed measurable declines in PSA levels

Three patients treated showed a greater than 50% decline in PSA levels and had concordant improvements in PSMA-PET imaging

One patient demonstrated evidence of complete tumor elimination and remains in a durable response of greater than five months at the time of this presentation

"This innovative Poseida PSMA-directed CAR T cell platform has demonstrated a robust anti-tumor response in patients with metastatic castration resistant prostate cancer," commented Susan F. Slovin, M.D., Ph.D., Associate Vice Chair of Academic Administration at Memorial Sloan Kettering Cancer Center and investigator on the trial. "This is the first time that I have seen such impressive responses with an immunotherapy product. The responses of my patients in the trial are far beyond my expectations."

Safety and Tolerability:

P-PSMA-101 demonstrated a favorable safety and tolerability profile. After a previously reported case of Macrophage Activation Syndrome (MAS) exacerbated by patient non-compliance, only three cases of possible Cytokine Release Syndrome (CRS) were observed, which were all low grade (1/2) and were managed well with early treatment. No cases of neurotoxicity (CRES/ICANS) were observed as of the cutoff date.

The Phase 1 trial is an open label, multi-center, 3+3 dose-escalating study designed to assess the safety of P-PSMA-101 in up to 40 adult subjects with mCRPC. The primary objectives of this study are to determine the safety, efficacy, and maximum tolerated dose of P-PSMA-101. Additional information about the study is available at www.clinicaltrials.gov using identifier: NCT04249947.

"We believe the key to success in solid tumors is a product with a high percentage of desirable stem cell memory T cells (Tscm)," said Matthew Spear, M.D., Chief Medical Officer of Poseida. "In this study, we have demonstrated that a high-percentage Tscm CAR-T product can home to the bone marrow and, in at least one case, completely eliminate tumor. This bone marrow homing property may be particularly important for bone avid diseases such as prostate adenocarcinoma. Importantly, the favorable tolerability associated with our Tscm CAR-T products has carried over to prostate cancer where we have so far seen manageable cytokine release syndrome and no neurotoxicity."

Company-Hosted Conference Call and Webcast Information

Poseida’s management team will host a conference call and webcast today, August 31, 2021 at 11:00am ET. The dial-in conference call numbers for domestic and international callers are (866) 939-3921 and (678) 302-3550, respectively. The conference ID number for the call is 50220147. Participants may access the live webcast and the accompanying presentation materials on Poseida’s website at www.poseida.com in the Investors section under Events and Presentations. An archived replay of the webcast will be available for 30 days following the event.

Additional CAR-TCR Summit Highlights

Presentation: "Developing CAR-T Cells for Multiple Myeloma: From Autologous to Allogeneic"

Session Date/Time: Wednesday, September 1, 2021, 4:00pm ET

Presenter: Matthew Spear, M.D., CMO, Poseida Therapeutics

This presentation will outline Phase 1 and 2 development of the Company’s lead autologous P-BCMA-101 CAR-T therapy and insights that were used to develop a fully allogeneic version, P-BCMA-ALLO1 that is expected to enter the clinic soon. The presentation will be part of the afternoon session on the Clinical Management Track.

Presentation: "Advancing Nonviral Manufacturing for Multi-Product Allogeneic T-Cell Therapies"

Session Date/Time: Wednesday, September 1, 2021, 4:30pm ET

Presenter: Devon Shedlock, Ph.D., SVP Research & Development, Poseida Therapeutics

This presentation will discuss how Poseida’s piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System and Booster Molecule are used to manufacture multi-product, fully allogeneic T-cell therapies. The Company will also discuss how efficient multiplexed Cas-CLOVER gene editing exhibits low to no off-target editing or translocations as determined by next-generation sequencing, and how the Company’s Booster Molecule helps to protect against the "allo tax," maintaining a favorable high-stem cell memory T cell (Tscm) product and enabling up to hundreds of doses in a single manufacturing run. This presentation will be part of the afternoon session on the Manufacturing Track.

Presentation: "Developing ‘Off-the-Shelf’ CAR-T Cells for Bone Marrow Transplant Conditioning"

Session Date/Time: Thursday, September 2, 2021, 9:00am ET

Presenter: Nina Timberlake, Ph.D., Associate Director, Research (Gene Therapy), Poseida Therapeutics

This presentation will discuss leveraging the piggyBac DNA Delivery System and Cas-CLOVER Site-specific Gene Editing System to generate off-the-shelf fully allogeneic CAR-T cells to specifically target hematopoietic cells in the bone marrow. This potential therapeutic could be used as a non-myeloablative conditioning regimen for hematopoietic stem cell transplant or as a therapeutic for the treatment of acute myeloid leukemia (AML). The presentation will occur as part of the conference’s Focus Day, "CAR-TCR Beyond Oncology: Fundamental Biology & Mechanisms of Action Beyond Oncology."

The full presentations at the CAR-TCR Summit will be made available on Poseida’s website at their respective session times.

Eagle Pharmaceuticals Granted Additional Patent for Bendamustine Franchise

On August 31, 2021 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported that the U.S. Patent and Trademark Office has granted the Company U.S. Patent No. 11,103,483, entitled "Formulations of Bendamustine (Press release, Eagle Pharmaceuticals, AUG 31, 2021, View Source [SID1234587030])." Eagle is submitting the ‘483 patent for listing in the U.S. Food and Drug Administration’s Orange Book for both BENDEKA and BELRAPZO.

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Eagle had asserted several Orange Book-listed patents against Slayback Pharma LLC, Apotex Inc. et al, Mylan Laboratories Limited, and Fresenius Kabi USA, LLC, related to their respective abbreviated new drug applications referencing BENDEKA. On July 6, 2020, the District Court for the District of Delaware had held these asserted patents both valid and infringed. Apotex, Mylan, and Fresenius appealed this ruling. Previous to the appellate hearing, Eagle settled the Fresenius litigation. On August 13, 2021, the United States Court of Appeals for the Federal Circuit affirmed that the asserted patents were both valid and infringed. Both the asserted patents and the ‘483 patent expire in 2031.

"We are pleased that this appellate decision, as well as the newly issued patent, continue to strengthen Eagle’s intellectual property rights for both BENDEKA and BELRAPZO," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

Prescient (ASX:PTX) delivers a milestone-rich 2021, major strides made in OmniCAR development

On August 31, 2021 Prescient Therapeutics Limited (ASX:PTX) reported that it has completed another action-packed year with a sound financial position (Press release, Prescient Therapeutics, AUG 31, 2021, View Source;utm_medium=rss&utm_campaign=prescient-asxptx-delivers-a-milestone-rich-2021-major-strides-made-in-omnicar-development [SID1234587029]). The Company ended the year with a product pipeline of highly valuable cancer drugs, rapidly advancing towards several key milestones. The business remains well-funded to progress its development programs.

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During the financial year 2021 the principal activities of Prescient consisted of:

RELATED READ: Prescient Therapeutics in a sweet spot with significant progress in multiple cancer programs

Targeted Therapies: PTX-100 and PTX-200
Prescient made excellent progress in the development of PTX-100 and PTX-200 assets with both clinical programs achieving key development milestones on the back of excellent results and ongoing investigator support. Both PTX-100 and PTX-200 cancer therapies achieved important clinical milestones during the reporting period.

The PTX-100 Phase 1b basket trial, led by principal investigator Professor Miles H Prince, progressed to an increased dose, yielding encouraging results after the reporting period. Now, the Company plans to conduct an expansion cohort study for PTX-100, that will focus on T cell lymphomas.
The ongoing Phase 1b trial of PTX-200 with cytarabine in patients with relapsed and refractory acute myeloid leukemia (AML) has progressed to higher dose levels under an amended protocol based on robust safety and efficacy results at lower dose levels.
ALSO READ: Prescient takes its PTX-100 trial to next level after Phase 1b success

Cell Therapy Enhancements (CTE)
The CTE programs are designed to enhance both current-generation and next generation CAR-T approaches. Last year, Prescient consolidated the two CTE programs with collaborators Carina Biotech and the Peter MacCallum Cancer Center (Peter Mac). Now, these two programs are being undertaken at Peter Mac, signifying its strong relationship with Prescient.

OmniCAR Platform
The initiation of OmniCAR programs along with the completion of CAR-T manufacturing programs is considered as a significant highlight of the financial year 2021.

OmniCAR is a universal, modular CAR (Chimeric Antigen receptor) platform developed to overcome many constraints and challenges of current CAR-T therapy, particularly in the area of solid cancers.

Source: PTX Investor Presentation (4 August 2021)

After the strategic review led by Prescient and its Scientific Advisory Board (SAB), the Company identified three internal programs for OmniCAR including-

OmniCAR CD33 and CLL-1 for AML.
OmniCAR Her2 for Her2+ solid tumours including breast, ovarian and gastric cancers.
OmniCAR Her2 and EGFRviii for glioblastoma multiforme (GBM).
All three next-generation CAR-T programs of PTX are highly differentiated from other CAR-T programs and seek to utilise the capabilities of the OmniCAR platform to overcome the challenges of other CAR-T approaches.

In a crucial OmniCAR milestone, the Company has successfully completed the manufacturing of binders, OmniCAR T-cells as well as immunogenicity testing of the components of OmniCAR platform.

Commenting on this, Steven Yatomi-Clarke, CEO and Managing Director of PTX stated-

RELATED ARTICLE: Positive results from immunogenicity testing send Prescient’s shares higher

It is noteworthy to mention that Prescient’s OmniCAR programs have opened the way for collaborations and partnerships with leading research teams and companies. This will not only help Prescient in the development of new markets and indications for the technology, but also enable the Company to combine its capabilities in ways that have the potential to immensely improve cancer therapy.

Prescient strengthens its IP position
During the year, Prescient has expanded its intellectual property (IP) position with most of the groundbreaking insights and progress made being retained and owned by the Company. This is a significant aspect of Prescient’s business and generates enormous future value and opportunities for the Company.

A key development in strengthening the IP portfolio was the notice of allowance from the USPTO (United States Patent and Trademark Office) for new patents, covering methods for using a specific biomarker for stratifying breast cancer patients and identifying the ones who are most likely to respond to PTX-100 treatment. After the grant, the patent is anticipated to be valid until 2031.

RELATED READ: Another feather in Prescient Therapeutics’ (ASX:PTX) cap, bags new US Patent for PTX-100

Growing a highly talented and accomplished team
Prescient has continued to attract talented and experienced people to its team:

Appointing internationally renowned cancer and CAR-T expert Professor Phillip Darcy and renowned oncologist and CAR-T pioneer, Professor H. Miles Prince, AM, to its Scientific Advisory Board (SAB).
Prescient appointed US-based Dr Dan Shelly, a cell therapy expert, as its VP -Business Development & Alliances, to address enhanced BD activity.
Dr Rebecca Lim joined Prescient as Director of Scientific Affairs. Dr Lim is a cell therapy expert and has experience in initiation of several novel cell therapy clinical studies.
After the reporting period, PTX onboarded brain cancer surgeon Donald M. O’Rourke to its SAB and Leanne West as its Director of Clinical Affairs.
Furthermore, Prescient also has four full-time researchers at Peter Mac to drive its cell therapy enhancements and OmniCAR programs.

Bottom Line
Prescient Therapeutics is progressing well with its robust clinical development profile. It is currently undertaking several clinical trials that await further promising outcomes. Furthermore, Prescient is backed by highly experienced and skilled officials who will also support the Company’s developing profile in the most exciting field of cancer therapy.

On 31 August 2021, PTX shares were trading at AU$0.192, with a market capitalisation of AU$122.09 million.

To know more about Prescient Therapeutics Limited, click here.

To stay updated with PTX company activities and announcements, please update your details on their investor centre

NeuExcell Therapeutics Raises $10+ Million Series Pre-A Round To Continue Its Company Growth

On August 30, 2021 NeuExcell Therapeutics (www.neuexcell.com), a gene therapy company focusing on neurodegenerative diseases, reported a $10+ million Series Pre-A financing round (Press release, NeuExcell Therapeutics, AUG 30, 2021, View Source [SID1234638279]). The round was led by Co-Win Ventures, joined by other institutional investors YuanBio, Oriza Seed, Tsingyuan and InnoAngel.

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"We are honored to have this highly reputable group of investors join us," stated Peter Tombros, Board Chair at NeuExcell Therapeutics. "The investors’ experience and support will enable the Company to leverage our proprietary neuroregenerative gene therapy platform across multiple neurodegenerative indications. The strength of this financing validates the Company’s strategy and further validates our science within the biotech industry."

"We are so excited about the prospects of accelerating our innovative technology to bring effective therapies to millions of patients around the world," said Professor Gong Chen, the company’s co-founder and chief scientific advisor. "There is a desperate need for breakthrough therapies like ours."

"We view this as a great opportunity to invest in experienced leadership," stated Dr. Xin Huang, managing partner at Co-Win Ventures. "NeuExcell’s proprietary technology has the potential to serve as a platform to treat many neurodegenerative diseases, offering hope to groundbreaking new therapies to patients without adequate options today."

With the closure of this successful Pre-A round, the Company welcomes Dr. Xin Huang and Jonathan Sun to the Board of Directors.

About NeuExcell Therapeutics

NeuExcell is a privately held early-stage gene technology company, with its headquarters in Pennsylvania, USA and Shanghai, China. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen, the Company has developed a potentially disruptive neural repair technology through astrocyte-to-neuron conversion in vivo by introducing neural transcription factor(s) through adeno-associated virus (AAV)-based gene therapy. NeuExcell’s pipeline covers major neurodegenerative diseases such as Stroke, Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s Disease, Parkinson’s Disease, Traumatic Brain Injury, Spinal Cord Injury, and Glioma.

SpringWorks Therapeutics Enters into Research Collaboration with Leading Cancer Institute to Further Evaluate Nirogacestat as a BCMA Potentiator in Multiple Myeloma

On August 30, 2021 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that it has entered into a sponsored research collaboration with Dana-Farber Cancer Institute (Dana-Farber) (Press release, SpringWorks Therapeutics, AUG 30, 2021, View Source [SID1234591665]). The collaboration is designed to further investigate nirogacestat, SpringWorks’ investigational gamma secretase inhibitor (GSI), with anti-B-cell maturation antigen (BCMA) agents in a variety of preclinical multiple myeloma models. Constantine Mitsiades, M.D., Ph.D., Assistant Professor of Medicine at Dana-Farber and Harvard Medical School, will serve as the Principal Investigator for the research, which will utilize preclinical in vivo models from the Mitsiades Lab to simulate the multiple myeloma tumor microenvironment. In addition, the research will also deploy functional genomics approaches to explore determinants of response and mechanisms of resistance to GSI + BCMA combination therapy.

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Gamma secretase inhibition prevents the cleavage and shedding of BCMA from the surface of multiple myeloma cells. In preclinical models, nirogacestat has been shown to increase the cell surface density of BCMA and reduce levels of soluble BCMA, thereby enhancing the activity of BCMA-targeted therapies.1 To date, SpringWorks has entered into clinical collaborations with six industry partners to evaluate nirogacestat in combination with BCMA therapies across modalities.

"SpringWorks has demonstrated its commitment to understanding the science and advancing the clinical exploration of GSI + BCMA combination therapies," said Dr. Mitsiades. "I am pleased to collaborate with them towards the goal of improving clinical outcomes for patients with multiple myeloma while simultaneously enabling the advancement of the underlying science."

"We are delighted to be collaborating with the Mitsiades Lab at Dana-Farber to expand our understanding of nirogacestat’s mechanism of action when combined with BCMA therapies," said Badreddin Edris, Ph.D., Chief Operating Officer of SpringWorks. "Dr. Mitsiades and his colleagues have been at the forefront of developing cutting-edge translational models of multiple myeloma and we believe that this collaboration has the potential to yield novel insights that can help us to further refine our approach to improving outcomes for these patients."

Under the terms of the sponsored research agreement, SpringWorks will be responsible for funding the work and will retain an option to exclusively license any new intellectual property emerging from the research collaboration.

About Nirogacestat

Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors, which are rare and often debilitating and disfiguring soft-tissue tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

In addition, gamma secretase has been shown to directly cleave membrane-bound BCMA, resulting in the release of the BCMA extracellular domain, or ECD, from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat’s ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has six collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities, including with an antibody-drug conjugate, two CAR T cell therapies, two bispecific antibodies and a monoclonal antibody. SpringWorks has also formed research collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA therapies using a variety of preclinical multiple myeloma models.

Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.