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.

On May 14, 2024 Calidi Biotherapeutics Inc. (NYSE American: CLDI) ("Calidi"), a clinical-stage biotechnology company developing a new generation of targeted virotherapies, reported its first quarter 2024 operating and financial results and reviewed recent business highlights (Press release, Calidi Biotherapeutics, MAY 14, 2024, View Source [SID1234643275]).

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"Calidi’s current focus is on raising additional capital and implementing continued cost cutting measures in order to advance our promising pipeline of novel immunotherapies designed to successfully target and eliminate solid tumors across the body. We plan to present three posters at ASCO (Free ASCO Whitepaper) including an update from our ongoing trial with City of Hope for recurrent high-grade glioma," said Allan Camaisa, CEO and Chairman of the Board at Calidi Biotherapeutics. "In parallel to our clinical progress, Calidi has strengthened our preclinical programs through our powerful R&D engine, unveiled our novel systemic delivery platform to target diverse tumor types, and executed a capital raise to strengthen our balance sheet. We believe these efforts will help propel the company through important milestones including an interim clinical update from our ongoing Phase 1 trial and the initiation of the Phase 1 trial with Northwestern University anticipated in the third quarter of 2024, subject to additional capital being raised."

First Quarter 2024 and Recent Corporate Developments

Announced upcoming poster presentations at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The posters will include an update from the Phase 1 trial in collaboration with City of Hope for CLD-101 in recurrent high-grade glioma. Additional posters will focus on preclinical data from the company’s RTNova (CLD-400) and CLD-201 platforms.
Presented new data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (AACR) (Free AACR Whitepaper) supporting CLD-101 and CLD-201’s first-in-class use of stem cells to protect and potentiate antitumor virotherapies. The results of experiments, conducted in collaboration with City of Hope, provide further detail around the unique stem cell-based mode of delivery and the therapeutic potential at the core of Calidi’s novel technology.
Highlighted the company’s novel RTNova systemic platform, a targeted enveloped virotherapy, that has demonstrated preclinically the ability to resist inactivation by human serum and an ability to target diverse tumor types. This novel therapeutic approach has the potential to be available to a diverse patient population with different forms of cancer while remaining cost-effective and easy to administer.
Closed public offering of 15,197,500 shares of common stock and accompanying warrants, with gross proceeds of $6.1 million.
First Quarter 2024 Financial Results

The company reported a net loss of $7.2 million, or $0.20 per share, for the three months ended March 31, 2024, compared to a net loss of $6.5 million, or $0.75 per share, for the same period in 2023.

Research and development expenses were $2.7 million for the three months ended March 31, 2024, compared to $2.6 million for the comparable period in 2023, respectively.

General and administrative expenses were $4.0 million for the three months ended March 31, 2024, compared to $2.8 million for the comparable period in 2023, respectively.

The company had approximately $1.2 million in cash and $0.2 million in restricted cash as of March 31, 2024, compared to $1.9 million in cash and $0.2 million in restricted cash as of December 31, 2023.

On May 14, 2024 Defence Therapeutics Inc. ("Defence" or the "Company"), (CSE: DTC, OTCQB: DTCFF, FSE: DTC), a Canadian biopharmaceutical company developing novel immune-oncology vaccines and drug delivery technologies, reported the issuance of several new patents, as well as new allowances of patent applications belonging to different Patent Families in Defence’s vast and diverse portfolio (Press release, Defence Therapeutics, MAY 14, 2024, View Source;utm_medium=rss&utm_campaign=defence-therapeutics-strengthens-and-expands-global-patent-protection-on-key-technologies [SID1234643216]).

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Defence’s vaccine enhancer platform technology based on simple admixture of Accum and variants thereof with specific antigens, is further expanded by the recent allowance of Defence’s US application no. 18/169,440 by the USPTO. The newly allowed subject-matter, which includes valuable composition-of-matter claims directed to Accum or variants thereof as standalone agents for enhancing antigen presentation, or in admixture with of any vaccine antigen, elegantly compliments Defence’s previously granted US patent no. 11,612,651 covering methods of improving polypeptide antigen immunogenicity. Importantly, this Patent Family is the first family broadly covering Defence’s anticancer ARM vaccine platform. Recent pre-clinical studies using Defence’s ARM-002 vaccine suggest potent antitumor responses in in vivo melanoma, lymphoma, and pancreatic cancer models when combined with the anti-PD-1 immune-checkpoint inhibitor, paving the way for upcoming Phase I clinical trials.

Defence is also pleased to announce the securing of a new patent in Australia protecting its ADC platform technology based on the ability of the original Accum to form potent antibody-drug conjugates leading to targeted cellular accumulation of a payload. Granted on February 1, 2024, Australian patent no. 2017233725 broadly protects not only Defence’s original Accum but also important variants thereof, which further strengthens the company’s patent protection in this valuable and growing therapeutic space. This represents a significant milestone in the future development and commercialization of novel ADCs for treating and detecting many cancers, and adds to Defence’s previous granted patents in the United States (US 11,352,437), Japan (JP 7,126,956), and Israel (IL 261765).

In addition, Defence’s vaccine platform technology based on covalent conjugation of Accum and variants thereof to specific antigens, which already included US patent no. 11,291,717, now includes Canadian patent no. 3,201,103 granted on December 12, 2023, as well as Australian patent no. 2021402007 granted on March 14, 2024. Furthermore, counterpart Japanese application no. 2023-535891 was recently allowed by the Japanese Patent Office and the Japanese patent is expected to be granted imminently. These patents include claims broadly covering a method of improving polypeptide antigen immunogenicity by covalently conjugating any antigen to Accum or any variant thereof, and also include independent composition-of-matter claims covering potential vaccine compositions.

"We are thrilled to announce the issuance of these patents and allowances with broad claims covering our ADC and vaccine platforms, which underscore our relentless pursuit of innovation and our unwavering commitment to improving human health" says Mr. Plouffe, CEO and president of Defence.

Acceleration of examination of the remaining applications in both of Defence’s vaccine platform Patent Families via the Patent Prosecution Highway (PPH) program is currently being pursued based on these broad issued and allowed claims. The issuance of new patents in these families is therefore expected shortly.

On May 14, 2024 Nykode Therapeutics ASA (OSE: NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported that it has expanded its collaboration with MSD (Merck & Co., Inc., Rahway, NJ, USA) to include a clinical trial collaboration and supply agreement for a phase 2 trial evaluating Nykode’s wholly-owned lead candidate, VB10.16, in combination with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) (Press release, Nykode Therapeutics, MAY 14, 2024, View Source [SID1234643233]). This study will enroll HPV16-positive high-risk patients with locally advanced cervical cancer undergoing chemoradiotherapy.

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VB10.16 is a potentially first-in-class off-the-shelf therapeutic cancer vaccine specifically designed to treat HPV16-induced malignancies. The cancer vaccine is built on Nykode’s technology platform of targeting antigens to antigen presenting cells. Nykode has reported promising data from the phase 2 VB C-02 trial (NCT04405349) in heavily pre-treated cervical cancer patients receiving VB10.16 in combination with atezolizumab with a median overall survival not yet reached (estimated to 25 months at the time of analysis). These data support Nykode’s ongoing efforts with the phase 2 VB-C-04 trial (NCT06099418) in second-line recurrent/metastatic cervical cancer. Furthermore, the VB10.16 clinical development program is expanding into new indications, including head and neck cancer, for which Nykode has an existing clinical trial collaboration and supply agreement with MSD for the ongoing VBC-03 trial (NCT06016920) evaluating VB10.16 in combination with KEYTRUDA in patients with HPV16-positive, PD-L1-positive, recurrent or metastatic head and neck squamous cell carcinoma.

Agnete Fredriksen, EVP, Chief Scientific Officer and Business Development, commented, "We are excited to expand our clinical development to include earlier stages of cervical cancer where there is a significant unmet medical need, and we see a huge potential for cancer vaccines. We are thrilled to continue building on our already established, highly valued collaboration with the experienced and talented team at MSD, a global leader in immuno-oncology."

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.