On August 7, 2023 Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, reported financial results and provided business updates for the second quarter ended June 30, 2023 (Press release, Prime Medicine, AUG 7, 2023, View Source [SID1234633880]).

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"In recent months, we continued to advance our diversified portfolio of Prime Editing programs while also executing against a strategic partnering strategy aimed at further expanding the broad therapeutic potential of Prime Editing," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "PM359, our product candidate for the treatment of CGD, is progressing well, and at the ASGCT (Free ASGCT Whitepaper) Annual Meeting in May, we presented new preclinical data demonstrating its ability to reproducibly correct the CGD disease-causing mutation in CD34+ cells ex vivo with no detectable off-target editing. These findings further support our belief in the potential of this program to change the trajectory of this recurrent debilitating condition, and we look forward to sharing additional in vitro and in vivo data on this program and others later this year."

Dr. Gottesdiener continued, "Also in the second quarter, we entered into a research collaboration with Cimeio Therapeutics, gaining access to Cimeio’s powerful CD117 immunotherapy technology for genetic diseases. We are pleased to be working together to evaluate combining Prime Editing enabled protective shielding with multiplexed therapeutic edits to potentially reduce the toxicity and increase the efficiency of existing HSC transplant regimens. These combined technologies may enable us to more gently and effectively treat a wider range of genetic diseases than currently possible, and future applications may include selection of in vivo edited HSCs, which could allow for the treatment of genetic diseases without transplantation. This partnership reflects the tremendous breadth of our Prime Editing technology, as well as our commitment to leveraging its versatility, precision and efficiency to improve the care and treatment of patients worldwide."

Recent Business Updates

Pipeline

•Prime Medicine continued to advance its strategic pipeline of eighteen programs and remained on track to initiate investigational new drug (IND)-enabling studies of PM359, its development candidate for chronic granulomatous disease (CGD), in 2023.
•In May 2023, Prime Medicine presented new preclinical data at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting. These data further demonstrated the potential for Prime Editing to correct the causative mutation of CGD and showcased the potential application of the Prime Assisted Site-Specific Integrase Gene Editing (PASSIGE) platform to generate multiplex-edited CAR-T cells for the treatment of certain cancers and immune diseases. Read the full data here.

Corporate

•In June 2023, Prime Medicine announced a research collaboration with Cimeio Therapeutics to develop Prime Edited Shielded Cell and Immunotherapy Pairs (SCIP) for genetic diseases, acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The goal of the research is to reduce the toxicity of

conditioning regimens and introduce new therapeutic options to meaningfully expand the utility of hematopoietic stem cell (HSC) transplant and enable the in vivo selection of edited HSCs to potentially remove the need for transplantation entirely. Under the terms of the agreement, Prime Medicine will develop a Prime Editor for Cimeio’s CD117 shielding variant that will then be evaluated by both companies; if the research collaboration is successful, the companies will grant exclusive license options to each other for their technologies. If the companies exercise their exclusive license options, they will each be eligible to receive economics on net sales of licensed products.

Anticipated Upcoming Milestones

Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:

Pipeline

•Initiate investigational new drug (IND)-enabling studies for PM359 in CGD in 2023.
•Expand preclinical proof-of-concept in vivo data, with plans to share data from in vivo rodent studies and large animal studies from several programs in the second half of 2023.
•Share in vitro preclinical data in additional liver, eye and neuromuscular programs.
•Complete first IND filing as early as 2024 and additional IND filings anticipated in 2025.

Platform

•Continue to develop and optimize non-viral and viral delivery systems and share additional proof-of-concept data from in vivo rodent and large animal studies in the second half of 2023.
•Further demonstrate superior off-target profiles for Prime Editing programs.
•Expand Prime Editing using proprietary recombinase technologies for new and existing programs.
•Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.

Second Quarter 2023 Financial Results:

•R&D Expenses: Research and development (R&D) expenses were $34.6 million for the three months ended June 30, 2023, as compared to $18.9 million for the three months ended June 30, 2022. This increase was primarily due to increases in lab supplies, personnel, and facilities costs as the company continues to expand and build out its R&D activities and function.
•G&A Expenses: General and administrative (G&A) expenses were $10.7 million for the three months ended June 30, 2023, as compared to $7.4 million for the three months ended June 30, 2022. This increase was primarily due to an increase in professional and consultant costs and personnel costs primarily attributable to the build-out of the company’s G&A team to support our R&D function.
•Net Loss: Net loss was $42.4 million for the three months ended June 30, 2023, as compared to $29.3 million for the three months ended June 30, 2022.
•Cash Position: As of June 30, 2023, cash, cash equivalents, investments and restricted cash were $221.1 million, as compared to $263.0 million as of March 31, 2023.

Financial Guidance

Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of June 30, 2023, will be sufficient to fund its anticipated operating expenses and capital expenditure requirements into 2025.

On August 7, 2023 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") and Poseida Therapeutics, Inc. (NASDAQ: PSTX, CEO: Mark Gergen, "Poseida") reported a strategic investment to support the advancement of Poseida’s commitment to redefining cancer cell therapy (Press release, Poseida Therapeutics, AUG 7, 2023, View Source [SID1234633879]).

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Under the terms of the transaction agreements, Astellas will invest a total of $50 million, including $25 million to acquire 8,333,333 shares of common stock of Poseida (approximately 8.8% of the outstanding common stock of Poseida) at $3.00 per share in a private placement and a one-time $25 million payment for a right of exclusive negotiation and first refusal to license one of Poseida’s clinical stage programs: P-MUC1C-ALLO1, an allogeneic CAR-T cell therapy in development for multiple solid tumor indications. In addition, Poseida has granted Astellas a board observer seat, which includes the ability to attend Poseida’s scientific advisory board meetings, and certain notice rights related to any potential change of control of Poseida.

Astellas has established the Focus Area Approach for its research and development strategy. One of Astellas’ Primary Focuses within the strategy is immuno-oncology. Astellas is committed to developing next-generation immuno-oncology drugs using multi-functional platforms. Its portfolio includes oncolytic viruses, bispecific immune cell engagers, small molecules, and cell therapy platforms.

Poseida is engaged in the research and development of cell and gene therapies for cancer and rare genetic diseases by leveraging its proprietary genetic editing platforms. In oncology, Poseida has a broad pipeline of allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors, including P-MUC1C-ALLO1, which is in Phase 1 development for the treatment of multiple solid tumor indications.

"By leveraging our extensive expertise, experience in cancer biology and unique technologies, we are focused on reinvigorating the immune system’s ability to discover, disarm and destroy cancers in more patients. We are pursuing this ambitious goal through innovative and multifunctional modality platforms, using the capabilities at our global R&D sites as well as through partnership with external expert partners," said Adam Pearson, Chief Strategy Officer, Astellas.

"We believe that this investment fits strategically with our long-term vision of expanding our capability in immuno-oncology and will ultimately lead to the development of new therapeutics for patients in need of cancer immunotherapy."

"We are excited to enter this strategic relationship with Astellas, a premier biopharmaceutical company that shares our long-term vision that cell and gene therapies represent an exciting growth area for the development of innovative medicines for improving patient care," said Mark Gergen, Poseida’s Chief Executive Officer. "This investment further validates the potential of our proprietary genetic engineering technology platform and cell therapy approach, and we look forward to working with Astellas to advance our shared vision and explore future opportunities for collaboration to further unlock that potential."

About P-MUC1C-ALLO1

P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in Phase 1 development for multiple solid tumor indications. Poseida believes P-MUC1C-ALLO1 has the potential to treat a wide range of solid tumors derived from epithelial cells, such as breast, ovarian, colorectal, lung, pancreatic and renal carcinomas, as well as other cancers expressing a cancer-specific form of the Mucin 1 protein, or MUC1-C. P-MUC1C-ALLO1 is designed to be fully allogeneic, with genetic edits to eliminate or reduce both host-vs-graft and graft-vs-host alloreactivity. Poseida has demonstrated the elimination of tumor cells to undetectable levels in preclinical models of both breast and ovarian cancer. Additional information about the Phase 1 study is available at www.clinicaltrials.gov using identifier: NCT05239143.

On August 7, 2023 Keros Therapeutics, Inc. ("Keros" or the "Company") (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta ("TGF-ß") family of proteins, reported a business update and announced financial results for the quarter ended June 30, 2023 (Press release, Keros Therapeutics, AUG 7, 2023, View Source [SID1234633877]).

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"We are excited to share our initial development plans for KER-065—an activin receptor ligand trap that is designed to increase skeletal muscle and bone mass, increase fat metabolism and reduce fibrosis—in Duchenne muscular dystrophy ("DMD"), a debilitating disease with a serious unmet need, and look forward to moving this important program into the clinic in the first quarter of 2024," said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer of Keros. "As highlighted by the progression of KER-065 and the initiation of our Phase 2 clinical trial of KER-012 in patients with pulmonary arterial hypertension ("PAH"), we continue to execute on our strategy of developing our pipeline of differentiated assets designed to harness the powerful biology of the TGF-ß family of proteins."

Recent Program Highlights

•KER-050 for the treatment of ineffective hematopoiesis to address cytopenias
◦Following recommendation by the Safety Review Committee, dosing for Cohort 4 of Keros’ ongoing Phase 2 clinical trial of KER-050 in patients with myelofibrosis-associated cytopenias was initiated at 4.5 mg/kg in both combination and monotherapy arms. Keros expects to announce dose escalation data from and initiate Part 2 of this trial in the second half of 2023.
•KER-047 for the treatment of functional iron deficiency
◦Keros initiated an open-label Phase 2 clinical trial in myelodysplastic syndromes ("MDS") and myelofibrosis patients with functional iron deficiency. The Company expects to report initial data from this trial in the first half of 2024.
•KER-012 for the treatment of PAH and for the treatment of cardiovascular disorders
◦In July 2023, Keros announced that the U.S. Food and Drug Administration cleared the Company’s investigational new drug application to conduct a randomized, double-blind, placebo-controlled, global Phase 2 clinical trial, which Keros refers to as the TROPOS trial, of KER-012 in combination with background therapy in patients with PAH.
◦The Company is hosting a conference call and webcast tomorrow, August 8, 2023, at 8:00 a.m. Eastern time to provide an overview of the TROPOS trial design.
•KER-065 for the treatment of neuromuscular disease
◦KER-065, which was nominated out of Keros’ proprietary library of activin receptor type II ligand traps, is being developed for the treatment of neuromuscular diseases, with an initial focus on DMD.
◦The Company plans to commence a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate the safety and tolerability of single and multiple ascending doses of KER-065 in healthy volunteers in the first quarter of 2024.

Second Quarter 2023 Financial Results

Keros reported a net loss of $37.5 million in the second quarter of 2023 as compared to a net loss of $27.3 million in the second quarter of 2022. The increase of $10.2 million for the second quarter was largely due to increased research and development efforts as well as additional investments to support the achievement of Keros’ clinical and corporate goals.

Research and development expenses were $32.5 million for the second quarter of 2023 as compared to $23.3 million for the same period in 2022. The increase of $9.3 million was primarily due to additional research and development efforts, manufacturing activities and personnel expenses to support the advancement of Keros’ pipeline.

General and administrative expenses were $8.8 million for the second quarter of 2023 as compared to $7.4 million for the same period in 2022. The increase of $1.4 million was primarily due to increase in personnel expenses and other external expenses to support Keros’ organizational growth.

Keros’ cash and cash equivalents as of June 30, 2023 was $322.0 million compared to $279.0 million as of December 31, 2022. Keros expects that the cash and cash equivalents it had on hand at June 30, 2023 will enable Keros to fund its operating expenses and capital expenditure requirements into the fourth quarter of 2025.

On August 7, 2023 IO Biotech, Inc. (Nasdaq: IOBT), a clinical biopharmaceutical company developing novel, immune-modulating cancer vaccines based on its T-win technology platform, reported that it has entered into a definitive securities purchase agreement for a private placement that is expected to result in gross proceeds of approximately $75 million, before deducting offering expenses (Press release, IO Biotech, AUG 7, 2023, View Source [SID1234633876]). The private placement includes participation from new and existing investors, including Lundbeckfonden BioCapital; Kurma Growth Opportunities Fund; Vivo Capital; Armistice Capital; Marshall Wace; Samsara BioCapital; Novo Holdings; Stonepine Capital Management; PFM Health Sciences, LP; HBM Healthcare Investments (Cayman) Limited; Pivotal Life Sciences; Sunstone Life Science Ventures; Logos Capital; Altamont Pharmaceutical Holdings, LLC; and The Red Hook Fund LP; among others. Members of the company’s management team also participated in this transaction.

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"The continued strong support that we have received from our existing investors and the enthusiasm from an impressive group of new healthcare investors are testaments to the potential of IO102-IO103, to the strength of the data generated to date from the Phase 1/2 study in metastatic melanoma and from our ongoing Phase 2 basket study (IOB-022/KND38)," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "With the proceeds of this financing, we expect to have sufficient capital to fund our operations into the fourth quarter of 2025, supporting the continued development of IO102-IO103 through critical clinical milestones from our pivotal Phase 3 trial in first-line advanced melanoma, as well as from our Phase 2 studies."

Under the securities purchase agreement, the investors have agreed to purchase 37,065,647 shares of the company’s common stock and accompanying warrants to purchase up to an aggregate of 37,065,647 shares of common stock, at a combined purchase price of $2.025 per share and accompanying warrant. Each accompanying warrant will represent the right to purchase one share of the company’s common stock at an exercise price of $2.47 per share. The warrants will be exercisable for a period of three years and six months following the date of issuance.

Morgan Stanley and Piper Sandler acted as joint placement agents for the financing.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state.

About IO102-IO103

IO102-IO103 is an investigational immune-modulating cancer vaccine designed to target the immunosuppressive mechanisms mediated by the proteins indoleamine 2,3-dioxygenase (IDO) and PD-L1. The company is currently conducting a Phase 3 trial (IOB-013/KN-D18; NCT05155254) evaluating IO102-IO103 in combination with pembrolizumab in first-line advanced melanoma patients. The company is also conducting a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709), a non-comparative, open label trial to investigate the safety and efficacy of IO102-IO103 in combination with pembrolizumab for the first-line treatment of each of the following advanced cancers: non-small cell lung cancer (NSCLC) and squamous cell carcinoma of the head and neck (SCCHN).

On August 7, 2023 Ikena Oncology, Inc. (Nasdaq: IKNA, "Ikena"), a targeted oncology company forging new territory in patient-directed cancer treatment, and Pionyr Immunotherapeutics, Inc. (Pionyr), a privately-held, clinical-stage biotechnology company, reported the closing of Ikena’s acquisition of Pionyr in an all-stock transaction (Press release, Ikena Oncology, AUG 7, 2023, View Source [SID1234633875]). Ikena acquired all of Pionyr’s assets, including approximately $43 million in net cash, in exchange for shares of IKNA stock, in a combination of common stock and non-voting convertible preferred stock priced at $7.15 per share.

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"Ikena has made incredible strides this year, and we are glad to welcome the Pionyr shareholders to the Ikena team as we head into a pivotal time for our targeted oncology programs," said Mark Manfredi, Ph.D., Chief Executive Officer of Ikena. "The capital from this acquisition allows us to accelerate and expand our clinical efforts in IK-930 and further support our comprehensive clinical plan for IK-595 as we continue to build value through both depth and breadth in our pipeline. We are looking forward to sharing more updates from across our business and pipeline, including initial clinical data from IK-930 later this year."

The transaction further strengthens Ikena’s financial position as the Company advances development of its targeted oncology programs. The new capital will enable Ikena to expand and accelerate its targeted oncology programs, including both IK-930, Ikena’s clinical program targeting the Hippo pathway with initial clinical data expected this year, and IK-595, a MEK-RAF complexing inhibitor expected to have an IND submitted this year. The new Ikena shareholders include New Enterprise Associates, SV Health Investors, Sofinnova Investments, Vida Ventures, and Gilead Sciences. Gilead Sciences acquired an equity stake in Pionyr through their 2020 option-to-acquire agreement.

"We have been highly rigorous and selective in our search for a buyer. Our attraction to Ikena was driven by not only the potential of their targeted oncology programs, but by their company culture and the way their team has managed capital and developed their pipeline," commented Steven P. James, Pionyr’s Chief Executive Officer. "On behalf of our investors, board of directors, and the Pionyr executive team, I want to thank and congratulate the entire Pionyr team for their efforts."

Further details on the transaction are described below.

Transaction and Approval

•
The transaction was unanimously approved by the boards of directors from both companies and closed following signing of the definitive agreement

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Ikena’s acquisition of Pionyr was structured as a stock-for-stock transaction whereby all of Pionyr’s outstanding common stock was exchanged for a combination of Ikena common stock and non-voting convertible preferred stock

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Ikena acquired all of Pionyr’s assets, including cash, cash equivalents, and investments as well as all intellectual property and clinical and preclinical assets

Pionyr Programs

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Ikena plans to include Pionyr’s programs as part of Ikena’s partnering portfolio, and to pursue strategic business development opportunities, including out-licensing

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The transaction includes contingent value rights (CVRs) providing the legacy Pionyr shareholders with rights to 50% of the net proceeds, outside of royalties, for any potential monetization of the Pionyr programs within the next two years

Additional Details

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Pionyr shareholders were collectively issued approximately 1.8 million shares of Ikena common stock with the remainder of the shares issued as Ikena non-voting convertible preferred stock

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Pionyr’s valuation for the transaction was determined solely by net cash available at closing

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The transaction price of $7.15 per share represents a premium to both Ikena’s twenty-day volume-weighted average ending on the transaction date and its most recent underwritten public offering

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As a result of the transaction, the Pionyr shareholders collectively hold approximately 12% of Ikena’s outstanding shares

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Ikena’s management team and board of directors remain unchanged