On August 6, 2024 Precigen, a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported a strategic reprioritization of the Company’s clinical portfolio and streamlining of resources, including a reduction of over 20% of its workforce, to focus on potential commercialization of the PRGN-2012 AdenoVerse gene therapy for the treatment of recurrent respiratory papillomatosis (RRP) (Press release, Precigen, AUG 6, 2024, View Source [SID1234646014]).

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These strategic changes substantially reduce required resources for non-priority programs and will enable the Company to focus on pre-commercialization efforts on PRGN-2012, including supporting submission of a rolling biologics license application (BLA) under an accelerated approval pathway anticipated in the second half of 2024, conducting the confirmatory clinical trial, and manufacturing of commercial product. Additionally, the Company will continue acceleration of commercial readiness efforts for a potential launch in 2025, led by the Company’s new Chief Commercial Officer, Phil Tennant.

Strategic prioritization will also include:

PRGN-2009 AdenoVerse Gene Therapy Clinical Trials: The Company plans to continue PRGN-2009 Phase 2 trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer. PRGN-2009 clinical trial enrollment at non-NCI clinical sites will be paused.
UltraCAR-T Clinical Programs: The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the US Food and Drug Administration (FDA), and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps. The Company will pause all other UltraCAR-T clinical programs, including PRGN-3005 and PRGN-3007. The Company will minimize UltraCAR-T spend and focus on strategic partnerships to further advance UltraCAR-T programs.
Preclinical Programs: The Company will pause all preclinical programs.
ActoBio: The Company has initiated a shutdown of its Belgium-based ActoBio subsidiary operations, including planned elimination of all ActoBio personnel. In conjunction with this shutdown, ActoBio’s portfolio of intellectual property will be made available for prospective transactions.
"We are on track toward our goal of submitting a rolling BLA for PRGN-2012 in the second half of this year and we are pleased to announce that the confirmatory clinical trial, an important step guided by the FDA to support an accelerated approval, has already been initiated and is actively enrolling patients," said Helen Sabzevari, PhD, President and CEO of Precigen. "These prioritization steps enhance our ability to rapidly prepare for potential commercialization of PRGN-2012, which if approved, we believe has the safety, efficacy, and route of administration profile to be the first and best-in-class therapy for RRP patients."

Please refer to the Company’s 8-K filing for additional details.

Precigen: Advancing Medicine with Precision
Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on X @Precigen, LinkedIn or YouTube.

AdenoVerse
Precigen’s AdenoVerse platform utilizes a library of proprietary adenovectors for the efficient gene delivery of therapeutic effectors, immunomodulators, and vaccine antigens designed to modulate the immune system. Precigen’s gorilla adenovectors, part of the AdenoVerse library, have potentially superior performance characteristics as compared to current competition. AdenoVerse gene therapies have been shown to generate high-level and durable antigen-specific T-cell immune responses as well as an ability to boost these responses via repeat administration. Superior performance characteristics and high yield manufacturing of AdenoVerse vectors leveraging UltraVector technology allows Precigen to engineer cutting-edge investigational gene therapies to treat complex diseases.

About PRGN-2012 AdenoVerse Gene Therapy
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 RRP. PRGN-2012 was the first to receive Breakthrough Therapy Designation and an accelerated approval pathway for RRP from the US Food and Drug Administration (FDA). PRGN-2012 received Orphan Drug Designation from the FDA and from the European Commission. 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). Pivotal data was presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting.

Trademarks
Precigen, AdenoVerse, UltraVector, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates.

On August 6, 2024 Elevation Oncology, Inc. (Nasdaq: ELEV), an innovative oncology company focused on the discovery and development of selective cancer therapies to treat patients across a range of solid tumors with significant unmet medical needs, reported financial results for the second quarter ended June 30, 2024, and highlighted recent business achievements (Press release, Elevation Oncology, AUG 6, 2024, View Source;utm_medium=rss&utm_campaign=elevation-oncology-reports-second-quarter-2024-financial-results-and-highlights-recent-business-achievements [SID1234645416]).

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"Today, we announced promising initial data from our Phase 1 trial evaluating monotherapy EO-3021, which reinforce clinical proof-of-concept and support EO-3021’s potential as a best-in-class Claudin 18.2 antibody drug conjugate. We are particularly excited to see encouraging anti-tumor activity in patients with Claudin 18.2-expressing gastric or GEJ cancer, as well as a differentiated safety profile, demonstrating the value of a targeted therapeutic approach and the benefit of EO-3021’s site-specific conjugation," said Joseph Ferra, President and Chief Executive Officer of Elevation Oncology. "These results support our broad clinical development program for EO-3021 and strengthen our conviction that EO-3021 can provide meaningful benefit to patients living with gastric or GEJ cancer."

Mr. Ferra continued, "Looking ahead, we remain on track to advance into monotherapy dose expansion and initiate the combination portion of our Phase 1 trial evaluating EO-3021 by year-end, report additional monotherapy data for EO-3021 in the first half of 2025, and nominate a development candidate from our HER3-ADC program in the second half of 2024. This year has been transformative for Elevation Oncology, marked by strong execution toward our goal of bringing important treatment options to patients with significant unmet medical needs, and we are eager to continue these efforts as we advance our pipeline of differentiated ADC therapies."

Recent Business Achievements

Earlier today, Elevation Oncology announced promising initial data from the dose escalation portion of the ongoing Phase 1 clinical trial of EO-3021 in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2, including gastric, gastroesophageal junction (GEJ), pancreatic or esophageal cancers. As of the data cutoff date of June 10, 2024:
EO-3021 was observed to be generally well-tolerated. No neutropenia or peripheral neuropathy/hypoesthesia, both known toxicities associated with monomethyl auristatin E (MMAE), were observed in the safety population of 32 patients treated with EO-3021.
In seven patients with Claudin 18.2 in ≥20% of tumor cells at IHC 2+/3+, the objective response rate (ORR) was 42.8% (three confirmed partial responses, one of which was confirmed following the June 10, 2024, data cutoff) and the disease control rate (DCR) was 71.4%, including two patients with stable disease (SD).
In eight patients with Claudin 18.2 in <20% of tumor cells at IHC 2+/3+, the ORR was 0% and the DCR was 50%, including four patients with SD.
A press release with further details regarding these results is available in the News Releases section of Elevation Oncology’s investor relations website at View Source
In June 2024, Elevation Oncology announced plans to expand its ongoing Phase 1 clinical trial of EO-3021 to include two combination cohorts evaluating EO-3021 for the treatment of advanced gastric or GEJ cancer. Pursuant to clinical supply agreements with Lilly and Company and GSK, respectively, Elevation Oncology will evaluate EO-3021 in combination with ramucirumab, a VEGFR2 inhibitor, in the second-line setting and in combination with dostarlimab, a PD-1 inhibitor, in the front-line setting.
Expected Upcoming Milestones

EO-3021:

Initiate dosing in combination portion of the ongoing Phase 1 clinical trial of EO-3021 by year-end 2024.
Share additional data from the ongoing Phase 1 clinical trial of monotherapy EO-3021, including from the dose expansion cohort, in the first half of 2025.
HER3-ADC:

Nominate development candidate from HER3-ADC program in the second half of 2024.
Second Quarter 2024 Financial Results

As of June 30, 2024, Elevation Oncology had cash, cash equivalents and marketable securities totaling $110.8 million, compared to $83.1 million as of December 31, 2023. The increase in cash reflects net proceeds of $44.2 million, which Elevation Oncology raised through its at-the-market (ATM) facility in the first half of 2024, partially offset by cash used to fund operating activities.

Research and development (R&D) expenses for the second quarter of 2024 were $6.6 million, compared to $6.0 million for the second quarter of 2023. The increase in R&D expenses in the second quarter of 2024 was primarily due to increased EO-3021 clinical trial expenses.

General and administrative (G&A) expenses for the second quarter of 2024 were $4.4 million, compared to $3.8 million for the second quarter of 2023. The increase in G&A expenses in the second quarter of 2024 was primarily due to increased professional fees, including costs for accounting and legal services, and increased utilization of consultants.

Net loss for the second quarter of 2024 was $10.5 million, compared to $10.1 million for the second quarter of 2023.

Financial Outlook

Elevation Oncology expects its existing cash, cash equivalents and marketable securities as of June 30, 2024 to be sufficient to fund its current operations into 2026.

Conference Call Information

Elevation Oncology will host a live conference call and webcast at 8:30 a.m. ET today to discuss the initial EO-3021 safety and efficacy data announced today. Participants may register for the conference call here. It is recommended that participants join the call ten minutes prior to the scheduled start.

A webcast of the call will also be available on the Events page of Elevation Oncology’s investor relations website at View Source The archived webcast will be available on the website approximately two hours after the conference call and will be available for 90 days following the call.

About EO-3021

EO-3021 (also known as SYSA1801) is a differentiated, clinical-stage antibody drug conjugate (ADC) with best-in-class potential comprised of an immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets Claudin 18.2. EO-3021 is site-specifically conjugated to the monomethyl auristatin E (MMAE) payload via a cleavable linker with a drug-to-antibody ratio (DAR) of 2. Claudin 18.2 is a specific isoform of Claudin 18 that is normally expressed in gastric epithelial cells. During malignant transformation, the tight junctions may become disrupted, exposing Claudin 18.2 and allowing them to be accessible by Claudin 18.2 targeting agents. Elevation Oncology is evaluating EO-3021 in a Phase 1 study (NCT05980416) in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2 including gastric, gastroesophageal junction, pancreatic or esophageal cancers.

Elevation Oncology has the exclusive rights to develop and commercialize EO-3021 in all global territories outside Greater China.

On August 6, 2024 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported second quarter 2024 financial results and corporate highlights (Press release, Relay Therapeutics, AUG 6, 2024, View Source [SID1234645432]).

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"In the second quarter, we made important progress continuing to advance our clinical programs, which we believe has positioned us well for multiple data readouts later this year. Additionally, we look forward to expanding the RLY-2608 development program, with the initiation of a new triplet combination with Pfizer’s novel investigative selective-CDK4 inhibitor atirmociclib by the end of the year," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics. "Looking further ahead, we are very excited by the pre-clinical programs we unveiled in June, including our first two genetic disease programs, which will be important in driving our continued growth and diversification."

Recent Corporate Highlights

RLY-2608 (ReDiscover study)

• RLY-2608 doublet: continued to enroll patients with PI3Kα-mutant, HR+, HER2- locally advanced or metastatic breast cancer in dose expansion cohorts of RLY-2608 400mg BID and 600mg BID in combination with fulvestrant

o
The next data update is expected in the fourth quarter of 2024 and will include approximately 100 patients across doses in the safety database and approximately 60 patients at the 600mg BID dose, including about 40 who have had the opportunity to be on RLY-2608 for at least 6 months
• RLY-2608 triplet:

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CDK4/6: continued enrollment of RLY-2608 + fulvestrant + ribociclib triplet combination in patients with PI3Kα-mutant, HR+, HER2- locally advanced or metastatic breast cancer
o
CDK4: announced a clinical trial collaboration to evaluate atirmociclib, Pfizer’s investigative selective-CDK4 inhibitor, in combination with RLY-2608 and fulvestrant in patients with PI3Kα-mutant, HR+, HER2- metastatic breast cancer. The RLY-2608 + atirmociclib + fulvestrant triplet combination is planned to begin by the end of 2024

• RLY-2608 monotherapy: continued to enroll patients with unresectable or metastatic solid tumors with a PI3Kα mutation in dose escalation portion of RLY-2608 monotherapy arm and reported partial responses in multiple tumor types

Migoprotafib (GDC-1971)​

• As previously disclosed, Genentech has terminated the collaboration agreement for the development and commercialization of migoprotafib

• The company will not continue development of migoprotafib

Pre-Clinical Programs

• Disclosed three new pre-clinical programs: vascular malformations, Fabry disease and NRAS

• Vascular Malformations

o
PI3Kα is the most common driver mutation among specific types of vascular malformations, which are a series of rare syndromes that occur due to atypical development of lymphatic and/or blood vessels and can become life-threatening, depending on what vessel(s) are involved
o
In the U.S., an estimated 170,000 people have one of the sub-types driven by a PI3Kα mutation, which include PIK3CA-related overgrowth spectrum, lymphatic malformations, venous malformations and cerebral cavernous malformations
o
A mutant selective PI3Kα inhibitor provides the opportunity for greater target coverage, leading to the potential for improved efficacy and better chronic tolerability
o
Relay Therapeutics plans to initiate clinical development of RLY-2608 in vascular malformations in the first quarter of 2025
• Fabry Disease

o
In Fabry disease, harmful levels of Gb3 accumulate in blood cells and tissues throughout the body, due to insufficient αGal enzyme activity, which can lead to a range of symptoms, including potentially life-threatening ones such as kidney failure, heart failure and stroke. In the U.S., approximately 8,000 people have Fabry disease
o
Relay Therapeutics has created the first investigational non-inhibitory chaperone for Fabry disease, which is designed to stabilize the αGal protein without inhibiting its activity, thus enabling greater Gb3 clearance across organs
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The company expects its non-inhibitory chaperone to enter the clinic in the second half of 2025
• NRAS

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In the U.S., an estimated 28,000 people are diagnosed annually with mutated NRAS solid tumors, which are a known oncogene driver in the RAS family and can lead to a number of cancers, including melanoma, colorectal and non-small-cell lung
o
Relay Therapeutics has created the first NRAS-selective inhibitor, which has been designed to address the liabilities of current pan-RAS inhibitors by only binding to NRAS, while sparing KRAS and HRAS
o
The company expects to initiate clinical development of its NRAS-selective inhibitor in the second half of 2025

Anticipated Upcoming Milestones

• Breast Cancer

o
RLY-2608 + fulvestrant data update in the fourth quarter of 2024
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RLY-2608 + fulvestrant + ribociclib initial safety data in the fourth quarter of 2024
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RLY-2608 + fulvestrant + atirmociclib clinical trial initiation by the end of 2024
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RLY-2608 + fulvestrant potential Phase 3 trial initiation in 2025
• Lirafugratinib: tumor agnostic data and regulatory update in the second half of 2024

• Pre-clinical

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Vascular malformations: RLY-2608 clinical trial initiation in the first quarter of 2025
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Fabry disease: clinical start in the second half of 2025
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NRAS: clinical start in the second half of 2025
Second Quarter 2024 Financial Results

Cash, Cash Equivalents and Investments: As of June 30, 2024, cash, cash equivalents and investments totaled $688.4 million compared to $750.1 million as of December 31, 2023. The company expects its current cash, cash equivalents and investments will be sufficient to fund its current operating plan into the second half of 2026.

R&D Expenses: Research and development expenses were $92.0 million for the second quarter of 2024, as compared to $88.2 million for the second quarter of 2023. The increase was primarily due to additional external costs in connection with the ReDiscover trial for RLY-2608.

G&A Expenses: General and administrative expenses were $20.1 million for the second quarter of 2024, as well as for the second quarter of 2023.

Net Loss: Net loss was $92.2 million for the second quarter of 2024, or a net loss per share of $0.69, as compared to a net loss of $98.5 million for the second quarter of 2023, or a net loss per share of $0.81.

On August 6, 2024 Bayer reported its Half-Year Financial Report 2024 (Presentation, Bayer, AUG 6, 2024, View Source [SID1234646084]).

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On August 06, 2024 COSCIENS Biopharma Inc. (formerly Aeterna Zentaris Inc.) (NASDAQ: AEZS) (TSX: AEZS) ("COSCIENS" or the "Company"), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products and active ingredients for healthcare and cosmetics industries, reported that the Company’s name has been changed from "Aeterna Zentaris Inc." to "COSCIENS Biopharma Inc.", effective as of August 6, 2024 (the "Name Change") (Press release, COSCIENS Biopharma, AUG 6, 2024, View Source;b=2533&ID=136661&m=rl&g=1592 [SID1234647477]).

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In addition, the Company announces that, effective as of August 9, 2024, the Company’s common shares (the "Common Shares") will begin trading on the Toronto Stock Exchange (the "TSX") and NASDAQ Capital Market (the "NASDAQ") under the trading symbol "CSCI" and will concurrently cease trading thereon under the former trading symbol "AEZS".

At the Company’s annual general and special meeting of shareholders held on July 16, 2024, the shareholders of the Company approved a special resolution authorizing the board of directors of the Company to effect the Name Change. Implementation of the Name Change remains subject to the final approval of the TSX and the NASDAQ.

There is no change in the capitalization of the Company in connection with the Name Change and no action will be required by existing shareholders or warrantholders of COSCIENS in connection with the Name Change. Certificates representing Common Shares and warrants of the Company (the "Warrants") will not be affected by the Name Change and will not need to be exchanged. The Company encourages any shareholder or warrantholder with any questions or concerns to contact the Company or to discuss any of the foregoing with their broker or agent.

The new CUSIP number for the Common Shares is 22112H101, and the new ISIN for the Common Shares is CA22112H1010. In addition, the new CUSIP number for the Warrants is 22112H119, and the new ISIN for the Warrants is CA22112H1192.