On May 14, 2024 Celularity Inc. (NASDAQ: CELU) ("Celularity"), a regenerative and cellular medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, reported that the Journal for ImmunoTherapy of Cancer, a peer-reviewed online journal of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), published data highlighting the potential advantages of Celularity’s T-Cell platform for future immunotherapies including chimeric antigen receptor (CAR-T) therapies (Press release, Celularity, MAY 14, 2024, View Source [SID1234643253]).

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The data compared CAR-T cells derived from Celularity’s proprietary T cell platform with CAR-T cells derived from healthy adult peripheral blood mononuclear cells (PBMCs). CAR-T cells generated from its T-Cell platform retained stemness, maintained longer telomeres, and were more resistant to both exhaustion and immune checkpoint upregulation. Additionally, an attenuated cytokine response was observed without loss of cytotoxicity. This translated into improved and prolonged in vivo efficacy and persistence compared with healthy, adult PBMC-derived CAR-T.

"These findings help differentiate the placenta as a source of cells for immunotherapy and demonstrate the valuable potential of our placental platform for allogeneic CAR-T products," said Dr. Robert Hariri, founder, Chairman and CEO of Celularity. "As the field of cellular immunotherapy moves towards delivering ‘one-size-fits-all’ allogeneic products, the stemness advantages inherent in our platform have the potential to translate into improved persistence and durable activity with an enhanced safety profile, offering promising prospects for developing more effective CAR-T therapies in the future. Moreover, we believe that our placental platform offers a level of scalability and consistency in manufacturing which can significantly impact the economics of delivering these therapies in the future. Through our state-of-the-art manufacturing and technical infrastructure, Celularity can be an ideal development and manufacturing partner in the cellular medicine industry."

On May 14, 2024 Moffitt Cancer Center, a world-renowned cancer treatment and research center, and Fulgent Pharma, a subsidiary of Fulgent Genetics, Inc. (NASDAQ: FLGT) and a leading nanobiotechnology company specializing in innovative cancer therapeutics, reported a strategic partnership aimed at advancing cancer treatment through clinical development and pre-clinical co-development initiatives (Press release, Fulgent Pharma, MAY 14, 2024, View Source [SID1234643274]). This collaboration combines Moffitt’s cutting-edge clinical capabilities with Fulgent’s groundbreaking nanotherapeutics and genomics platforms in an effort to accelerate the development of personalized cancer therapies. Under the agreement, Moffitt will provide Fulgent with priority access to its clinical expertise and resources, with the aim of expediting the advancement of Fulgent’s clinical pipeline. This priority access at Moffitt includes prioritized clinical trial activation, enhanced patient screening, and data sharing designed to get investigational therapies to patients in need more quickly and efficiently.

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Fulgent Pharma, a leader in nanoencapsulation technology, has transformed paclitaxel, a chemotherapy drug known for its poor solubility, into a soluble counterpart suitable for intravenous injection called FID-007. In Phase 1 clinical studies conducted with FID-007 to date, substantial tumor reduction across various cancer types, including breast, head and neck, lung, bile duct, and pancreatic cancers, has been observed.

Additionally, the partnership will focus on co-developing next-generation personalized treatment options for cancer. Moffitt will leverage its broad scientific and immunology expertise, access to tissue samples and nonclinical research resources, while Fulgent will contribute its novel and proprietary nano-particle-based drug development platform along with its broad spectrum genetic and genomic testing capabilities.

"Pooling our unique strengths, Moffitt and Fulgent will strive to create new precision oncology therapies customized to meet the specific needs of each patient, thereby improving future outcomes," said Fulgent Pharma President and CSO Ray Yin, Ph.D.

"This partnership aligns with Moffitt’s mission to contribute to the prevention and cure of cancer. We are looking forward to working with Fulgent to accelerate the translation of scientific discoveries into lifesaving treatments for our patients," said Moffitt President and CEO Patrick Hwu, M.D.

"We are excited to collaborate with Moffitt Cancer Center, a global cancer research and treatment leader," said Ming Hsieh, CEO of Fulgent Genetics. "By combining Moffitt’s clinical and research expertise with Fulgent’s innovative nanotherapeutic platform and growing patent portfolio, we believe we can make significant strides in advancing personalized cancer treatment options."

On May 14, 2024 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported first quarter financial results and provided a business update (Press release, Cyclacel, MAY 14, 2024, View Source [SID1234643215]).

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"We are excited to report that we have begun enrolling patients in the Phase 2, proof of concept (PoC) stage of our 065-101 study of fadraciclib, our oral CDK2/9 inhibitor, and are on track to deliver key readouts this year," said Spiro Rombotis, President and Chief Executive Officer. "Receipt of $8.0 million gross proceeds in a private placement together with existing resources support our ongoing clinical program. Pharmacokinetic, pharmacodynamic, safety and anticancer activity data from the Phase 1, dose escalation stage of 065-101 in patients with advanced solid tumors and lymphoma will be presented at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Data to date suggest that fadraciclib is differentiated from other next generation CDK inhibitors."

"Having determined the recommended Phase 2 dose for fadraciclib we are now enrolling patients in the Phase 2 PoC stage of 065-101" said Brian Schwartz, M.D., interim Chief Medical Officer. "We are initially concentrating on the biomarker cohort which is enrolling patients prospectively selected for CDKN2A/CDKN2B alterations to be followed by patients with T-cell lymphoma. There are no approved medicines for patients with CDKN2A/CDKN2B alterations. Including currently opened trial sites, we expect a total of up to seven sites will participate with the majority in the United States. We are encouraged about fadra’s prospects and look forward to presenting emerging data from the 065-101 study later in the year."

Key Upcoming Milestones for 2024

· Report final data from dose escalation stage and RP2D determination from the 065-101 study of oral fadraciclib in patients with advanced solid tumors and lymphoma at the ASCO (Free ASCO Whitepaper) 2024 Annual Meeting
· Report interim data from initial cohorts in Phase 2 proof-of-concept stage of 065-101 study with oral fadraciclib in patients with advanced solid tumors and lymphoma

Financial Highlights

As of March 31, 2024, pro forma cash and cash equivalents totalled $9.9 million, including proceeds from this month’s private placement and $0.8 million received for the United Kingdom research & development tax credit. Cash and cash equivalents as of March 31, 2024, totalled $2.8 million, compared to $3.4 million as of December 31, 2023.

Net cash used in operating activities was $0.5 million for the three months ended March 31, 2024, which includes $2.9 million received in March in respect of the United Kingdom research & development tax credit, compared to $6.9 million for the same period of 2023. The Company estimates that its current cash resources will fund planned programs into the fourth quarter of 2024.

Research and development (R&D) expenses were $2.8 million for the three months ended March 31, 2024, as compared to $5.7 million for the same period in 2023. R&D expenses relating to fadraciclib were $1.8 million for the three months ended March 31, 2024, as compared to $4.1 million for the same period in 2023 due to a decrease in clinical trial and other non-clinical expenditures. R&D expenses related to plogosertib were $1.0 million for the three months ended March 31, 2024, as compared to $1.4 million for the same period in 2023 due to a decrease in manufacturing and other non-clinical expenditures.

General and administrative expenses remained relatively flat at approximately $1.6 million for each of the three months ended March 31, 2024 and 2023.

Total other expenses, net, for the three months and year ended March 31, 2024, were $0.1 million, compared to $0.2 million for the same period of the previous year.

United Kingdom research & development tax credits for the three months March 31, 2024, were $1.4 million, which includes $0.8 million related to the 2023 claim which was received in May 2024, compared to $1.3 million for the same period of the previous year and are directly correlated to qualifying research and development expenditure.

Net loss for the three months March 31, 2024, was $2.9 million (including stock-based compensation expense of $0.2 million), compared to $5.8 million (including stock-based compensation expense of $0.4 million) for the same period in 2023.

Conference call information:

Call: (888) 632-3384 / international call: (785) 424-1794

Archive: (800) 938-1584 / international archive: (402) 220-1542

Code for live and archived conference call is CYCCQ124. Webcast link

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.

On May 14, 2024 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering nanoparticle-based therapeutic approaches to expand treatment possibilities for patients with cancer and other major diseases, reported that the U.S. Food and Drug Administration ("US FDA") issued a Study May Proceed Letter for a randomized Phase 2 study evaluating NBTXR3 for the treatment of patients with stage 3, unresectable non-small cell lung cancer ("NSCLC") (Press release, Nanobiotix, MAY 14, 2024, View Source [SID1234643232]). An IND to support this trial was submitted by the global trial sponsor, Johnson & Johnson Enterprise Innovation Inc., a Johnson & Johnson company.

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"The NBTXR3 collaboration established by our global licensing agreement with Janssen Pharmaceutica NV continues to make progress toward our goal of reaching millions of patients with cancer around the world. The US FDA acceptance of the protocol for this new Phase 2 study has the potential to expand the NBTXR3 development pipeline to a new indication where innovation could potentially provide important outcomes," said Louis Kayitalire, MD, chief medical officer at Nanobiotix. "We look forward to continuing to prepare for the launch of the study."

About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. Its proof-of-concept was achieved in soft tissue sarcomas for which the product received a European CE mark in 2019. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

Radiotherapy-activated NBTXR3 is being evaluated across multiple solid tumor indications as a single agent or in combination with anti-PD-1 immune checkpoint inhibitors, including in NANORAY-312—a global, randomized Phase 3 study in locally advanced head and neck squamous cell cancers. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the Phase 3 study.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a collaboration strategy to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several Phase 1 and Phase 2 studies evaluating NBTXR3 across tumor types and therapeutic combinations. In 2023, Nanobiotix announced a license agreement for the global co-development and commercialization of NBTXR3 with Janssen Pharmaceutica NV.

On May 14, 2024 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported first quarter 2024 financial results and business updates (Press release, Precigen, MAY 14, 2024, View Source [SID1234643254]).

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"We are excited to share the pivotal Phase 2 data for our PRGN-2012 study in patients with RRP at ASCO (Free ASCO Whitepaper) and look forward to providing additional details regarding the results at our planned conference call following the presentation. We remain on track for a PRGN-2012 rolling BLA submission in the second half of 2024 and we are actively moving ahead with our commercial readiness efforts in anticipation of a potential launch of PRGN-2012 in 2025," said Helen Sabzevari, PhD, President and CEO of Precigen. "Based on the competitive advantages of PRGN-2012, including a favorable route of administration, safety profile and the efficacy demonstrated in the clinical trial results so far, we believe, PRGN-2012 has the potential to be the first-in-class and best-in-class treatment for RRP patients. We anticipate PRGN-2012 to overwhelmingly be the treatment of choice for RRP patients, if approved, as indicated by our commissioned research of healthcare providers and key opinion leaders which found PRGN-2012’s competitive advantages highly compelling."

"With multiple milestones anticipated in 2024 and 2025, we remain steadfastly committed to a strategy of sound financial management," said Harry Thomasian Jr., CFO of Precigen. "We are evaluating various financing opportunities to strengthen our balance sheet as we prepare our lead asset, PRGN-2012, for potential commercial launch in 2025."

Key Program Highlights

AdenoVerse

· PRGN-2012 in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 was the first to receive Breakthrough Therapy Designation and an accelerated approval pathway for RRP from the FDA. PRGN-2012 received Orphan Drug Designation from the US Food and Drug Administration (FDA) and Orphan Drug Designation from the European Commission.

o PRGN-2012 is currently under investigation in a Phase 1/2 pivotal single-arm study in adult patients with RRP (clinical trial identifier: NCT04724980).

o Results from the Phase 1 portion of the Phase 1/2 study were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS).

§ PRGN-2012 demonstrated overall safety and clinically meaningful benefit with 50% of patients (N=12) in Complete Response, which is defined as no surgeries needed during the 12-month period following PRGN-2012 treatment completion. All Complete Responses were durable and ongoing more than two years after PRGN-2012 treatment.

§ 83% of patients had a reduction in RRP surgeries in the 12-month period after PRGN-2012 treatment compared to 12 months pre-treatment.

§ Correlative data support expansion of peripheral HPV 6 and HPV 11–specific T cell immunological responses as the underlying mechanism of action for PRGN-2012.

o PRGN-2012 is built using the Company’s differentiated gorilla adenovector that allows for repeat dosing. The redosing potential of AdenoVerse has been highlighted in clinical studies where repeat administrations of PRGN-2009 and PRGN-2012 gene therapies led to enhancement of antigen-specific T cell immune responses without generation of significant neutralizing antibodies in contrast to other viral vectors.

o Results from the pivotal Phase 2 study of PRGN-2012 for the treatment of RRP, including immunological responses, will be presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in a late-breaking oral presentation titled, "PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients." Scott M. Norberg, DO, Associate Research Physician, Center for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, will deliver the presentation on June 3, 2024 at 8:30 AM CT.

o The Company plans to host a conference call on June 3, 2024 to discuss in detail the PRGN-2012 pivotal study results presented and provide business updates.

o FDA confirmed that the ongoing Phase 1/2 single arm study will serve as pivotal and no additional randomized, placebo-controlled trial will be required to support submission of a Biologics License Application (BLA). A rolling BLA submission under an accelerated approval pathway is anticipated in the second half of 2024. Based on FDA guidance, the Company is on track to initiate a confirmatory study prior to submission of the BLA.

o Commercial readiness preparations are underway for a potential launch in 2025.

o The Company and the Recurrent Respiratory Papillomatosis Foundation will co-sponsor the inaugural RRP Awareness Day on June 11, 2024. The multi-stakeholder event will raise awareness and bring together individuals living with RRP, caregivers, clinicians, and government officials to encourage new connections and build community among those interested in and affected by RRP. The inaugural event will be hybrid with in-person participation at the National Press Club in Washington DC and a webcast for virtual participation.

· PRGN-2009 in OPSCC and Cervical Cancer: PRGN-2009 is an investigational off-the-shelf AdenoVerse gene therapy designed to activate the immune system to recognize and target HPV-associated cancers.

o The Phase 2 study of PRGN-2009 in combination with pembrolizumab in newly diagnosed patients with HPV-associated oropharyngeal squamous cell carcinoma (OPSCC) is enrolling patients (clinical trial identifier: NCT05996523).

§ An abstract titled, "Phase II trial of immunotherapeutic HPV vaccine PRGN-2009 with pembrolizumab before standard treatment in subjects with newly diagnosed HPV-associated oropharyngeal cancer" will be presented as a trial-in-progress poster presentation on June 2, 2024 from 9:00 AM to 12:00 PM CT at ASCO (Free ASCO Whitepaper).

o The Phase 2 randomized, open-label study of PRGN-2009 in combination with pembrolizumab in patients with HPV-associated recurrent/metastatic cervical cancer is active and recruiting patients (clinical trial identifier: NCT06157151).

§ An abstract titled, "A Phase 2 study to evaluate efficacy and safety of PRGN-2009, a novel gorilla adenovirus-based immunotherapy, in combination with pembrolizumab versus pembrolizumab monotherapy in patients with recurrent or metastatic cervical cancer" will be presented as a trial-in-progress poster presentation on June 3, 2024 from 9:00 AM to 12:00 PM CT at ASCO (Free ASCO Whitepaper).

UltraCAR-T

· PRGN-3006 in AML/MDS: PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a safety/kill switch. PRGN-3006 has been granted Orphan Drug Designation in patients with acute myeloid leukemia (AML) and Fast Track Designation in patients with relapsed/refractory (r/r) AML by the FDA.

o PRGN-3006 is currently under investigation in a Phase 1b dose expansion clinical trial (clinical trial identifier: NCT03927261) for the treatment of patients with r/r AML or higher-risk myelodysplastic syndromes (MDS).

o An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024.

o PRGN-3005 in Ovarian Cancer: PRGN-3005 is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a safety/kill switch.

o The Phase 1b dose expansion portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT03907527).

o PRGN-3007 in Advanced ROR1+ Hematological and Solid Tumors: PRGN-3007, based on the next generation UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a safety/kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.

o The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing (clinical trial identifier: NCT05694364).

o A preliminary Phase 1 dose escalation data presentation is anticipated by the end of 2024.

Financial Highlights

o Cash, cash equivalents, and short-term investments totaled $44.8 million as of March 31, 2024.

o Selling, general, and administrative (SG&A) costs decreased 13% compared to the three months ended March 31, 2023.

o Property, plant, and equipment, net, increased $5.5 million from December 31, 2023 primarily due to the build-out of our cGMP manufacturing facility.

First Quarter 2024 Financial Results Compared to Prior Year Period

Research and development expenses increased $2.1 million, or 17%, compared to the three months ended March 31, 2023. Salaries, benefits, and other personnel costs increased $1.5 million due to an increase in the hiring of employees throughout 2023 to support the growth in the Company’s clinical development activities as well as increased fees paid to consultants and contract research organizations in the first quarter of 2024 compared to the same period in 2023.

SG&A expenses decreased $1.5 million, or 13%, compared to the three months ended March 31, 2023, primarily driven by a reduction in stock compensation and insurance expenses in the first quarter of 2024 compared to same period in 2023. In addition, the costs associated with PRGN-2012 commercial readiness increased compared to the same period in 2023.

Total revenues decreased $0.8 million, or 43%, compared to the three months ended March 31, 2023. This decrease was due to the reduction in products and services performed at Exemplar. Gross margin on product and services also declined in the current period primarily as a result of the decreased revenues at Exemplar.

Net Loss was $23.7 million, or $(0.10) per basic and diluted share, compared to net loss of $22.7 million, or $(0.10) per basic and diluted share, in period ended March 31, 2023.