On February 27, 2024 Nektar Therapeutics (Nasdaq: NKTR) reported that management is scheduled to present at the upcoming TD Cowen 44th Annual Health Care Conference in Boston on Tuesday, March 5, 2024 at 2:10 p.m. Eastern Time (Press release, Nektar Therapeutics, FEB 27, 2024, View Source [SID1234640544]).

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The presentation and Q&A session will be accessible via a webcast through a link posted on the Investor Events section of the Nektar website: View Source This webcast will be available for replay until April 5, 2024.

On February 27, 2024 Oragenics, Inc. (NYSE American: OGEN) ("Oragenics" or the "Company"), a company focused on developing unique, intranasal nanoparticle pharmaceuticals for the treatment of neurological disorders, reported the pricing of an underwritten public offering of 1,400,000 shares of its common stock at a public offering price of $1.50 per share, for gross proceeds of $2.1 million, before deducting underwriting discounts and offering expenses payable by the Company (Press release, Oragenics, FEB 27, 2024, View Source [SID1234640543]). In addition, the Company has granted the underwriters a 45-day option to purchase up to an additional 210,000 shares of its common stock at the public offering price, less discounts, to cover over-allotments, if any. The offering is expected to close on March 1, 2024, subject to satisfaction of customary closing conditions.

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The Company intends to use the net proceeds from the offering to fund the continued development of ONP-002, which is a unique neurosteroid drug compound intended to treat mild traumatic brain injuries also known as concussions, and for general corporate purposes and working capital.

ThinkEquity and Laidlaw & Company (UK) Ltd. are acting as joint book-running managers for the offering.

The securities are being offered and sold pursuant to a shelf registration statement on Form S-3 (File No. 333-269225), including a base prospectus, filed with the Securities and Exchange Commission (the "SEC") on January 13, 2023 and declared effective on January 25, 2023. The offering is being made only by means of a written prospectus. A final prospectus supplement and accompanying base prospectus describing the terms of the offering will be filed with the SEC and available on its website at www.sec.gov. Copies of the final prospectus supplement and the accompanying base prospectus relating to the offering may also be obtained, when available, from the offices of ThinkEquity, 17 State Street, 41st Floor, New York, New York 10004.

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

On February 27, 2024 Recursion (Nasdaq : RXRX), a leading clinical stage TechBio company decoding biology to industrialize drug discovery, reported business updates and financial results for its fourth quarter and fiscal year ended December 31, 2023 (Press release, Recursion Pharmaceuticals, FEB 27, 2024, View Source [SID1234640542]).

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"2023 was a year of remarkable progress for Recursion as we continued to demonstrate how combining technology, biology, chemistry, and patient data can industrialize drug discovery, and we look forward to the milestones ahead of us in 2024," said Chris Gibson, Ph.D., Co-founder and CEO of Recursion. "As we have watched the dynamics of our landscape, it appears that BioTech is increasingly evolving into TechBio, where it is imperative for life science companies to embrace digital nativity similar to how SaaS companies 10+ years ago evolved to being cloud-native in order to thrive. In this data-driven age, we believe the most important differentiator will be connected data in order to increasingly understand and treat the complexities of human disease. Recursion plans to continue leading the field in terms of data generation and aggregation."

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Summary of Business Highlights

•Platform
◦Causal AI Modeling and Additional Datasets: We have been training causal AI models leveraging over 20 petabytes of multimodal precision oncology patient data from Tempus to support the discovery of potential biomarker-enriched therapeutics at scale. By combining the forward genetics approach of Tempus

with the reverse genetics approach at Recursion, we believe we have an opportunity to improve the speed, precision, and scale of therapeutic development in oncology. This work has already resulted in a directed-oncology program against a novel gene/disease relationship in a large oncology indication. Recursion intends to operate both as a data generator and multimodal data aggregator. In the future, we intend to augment our dataset and hone the Recursion OS with germline genetic data, organoid technologies, and automated nano-synthesis technologies.
◦LOWE (Large Language Model-Orchestrated Workflow Engine): LOWE is an LLM agent that represents the next evolution of the Recursion OS. LOWE supports drug discovery programs by orchestrating complex wet and dry-lab workflows via natural language prompts. These workflows are the steps and tools available in the Recursion OS, from finding significant relationships across biology, chemistry, and patient-centric data to generating novel compounds and scheduling them for synthesis and experimentation. Through its natural language interface and interactive graphics, LOWE can put state-of-the-art AI tools into the hands of every drug discovery scientist.
•Pipeline
◦Cerebral Cavernous Malformation (CCM) (REC-994): Our Phase 2 SYCAMORE clinical trial is a randomized double-blind, placebo-controlled, safety, tolerability and exploratory efficacy study of REC-994 in participants with CCM. This trial was fully enrolled in June 2023 with 62 participants and the vast majority of participants who completed 12 months of treatment continue to elect to enter the long-term extension study. We expect to share Phase 2 data in Q3 2024.
◦Neurofibromatosis Type 2 (NF2) (REC-2282): Our adaptive Phase 2/3 POPLAR clinical trial is a randomized, two part study of REC-2282 in participants with progressive NF2-mutated meningiomas. Part 1 of the study is ongoing and is exploring two doses of REC-2282 in approximately 23 adults and 9 adolescents, with enrollment in adults expected to complete in H1 2024. We expect to share Phase 2 safety and preliminary efficacy data in Q4 2024.
◦Familial Adenomatous Polyposis (FAP) (REC-4881): Our Phase 1b/2 TUPELO clinical trial is an open label, multicenter, two part study of REC-4881 in participants with FAP. Part 1 is complete with FPI for Part 2 anticipated in H1 2024. We expect to share Phase 2 safety and preliminary efficacy data in H1 2025.
◦AXIN1 or APC Mutant Cancers (REC-4881): Our Phase 2 LILAC clinical trial is an open label, multicenter study of REC-4881 in participants with unresectable, locally advanced or metastatic cancer with AXIN1 or APC mutations. This study was initiated at the end of 2023, with FPI anticipated in Q1 2024. We expect to share Phase 2 safety and preliminary efficacy data in H1 2025.
◦Clostridioides difficile Infection (REC-3964): We conducted a Phase 1 healthy volunteer study to evaluate the safety, tolerability and PK of REC-3964 at increasing oral doses in comparison with placebo. REC-3964 was safe and well tolerated and there were no serious adverse events, deaths or TEAEs that led to discontinuation. REC-3964 is a first-in-class C. difficile toxin inhibitor and the first new chemical entity developed by Recursion, with promising preclinical efficacy data seen in relevant models (superiority versus bezlotoxumab). We expect to initiate a Phase 2 study in 2024.

◦RBM39 HR-Proficient Ovarian Cancers and Other Solid Tumors: RBM39 is a novel CDK12-adjacent target identified by the Recursion OS. We intend to position our lead candidate as a single agent for the potential treatment of HR-proficient ovarian cancers and other HR-proficient solid tumors. As a result of our strategic collaboration with Tempus, we are leveraging genomic data across all tumor types to identify clinical biomarkers for patient expansion. We are advancing our lead candidate through IND-enabling studies with IND submission expected in H2 2024.
◦Undisclosed Indication in Fibrosis (Target Epsilon): Phenotypic screening of human PBMCs identified novel and structurally diverse small molecules that reverse the phenotypic features of disease-state fibrocyte cells into those of healthy-state cells. The most promising compounds were confirmed as potent inhibitors of a novel target for fibrosis. This program originated under our initial fibrosis collaboration with Bayer and we have since in-licensed from Bayer all rights to this program which is now entering IND-enabling studies.
•Partnerships
◦Transformational Collaborations: We continue to advance efforts to discover potential new therapeutics with our strategic partners in the areas of undruggable oncology (Bayer) as well as neuroscience and a single indication in gastrointestinal oncology (Roche-Genentech). In the near-term, there is the potential for option exercises associated with partnership programs, option exercises associated with map building initiatives or data sharing and additional partnerships in large, intractable areas of biology or technological innovation.
◦Enamine: In December 2023, we entered a collaboration with Enamine to generate and design enriched compound libraries for the global drug discovery industry. By leveraging MatchMaker, a Recursion AI model, to identify compounds in the Enamine REAL Space (~36 billion chemical compounds) predicted to bind to high-value targets, we believe we can generate more powerful compound libraries for drug discovery purposes. Enamine may offer the resulting libraries to customers for purchase and will co-brand any libraries under both the Enamine and Recursion’s trademarks. This collaboration is an example of how select data layers can drive value in novel ways.

Additional Corporate Updates
•Letter to Shareholders: Recursion Co-Founder & CEO Chris Gibson, Ph.D. wrote an annual letter to shareholders which may be found in the 10-K report.
•L(earnings) Call: Recursion will host a L(earnings) Call on February 27, 2024 at 5:00 pm Eastern Time / 3:00 pm Mountain Time. A L(earnings) Call is Recursion’s take on interacting with a broad public audience around notable business developments. Recursion will broadcast the live stream from Recursion’s X (formerly Twitter), LinkedIn and YouTube accounts and analysts, investors and the public will be able to ask questions of the company.
•Chief Business Operations Officer: In February 2024, Recursion named Kristen Rushton, M.B.A. as Chief Business Operations Officer. Ms. Rushton has worked at Recursion for over 6 years, previously serving as Senior Vice President of Business Operations. Prior to Recursion, Ms. Rushton worked at Myriad Genetics and Myrexis.
•Annual Shareholder Meeting: Recursion’s Annual Shareholder Meeting will be held on June 3, 2024 at 10:00 am Eastern Time / 8:00 am Mountain Time.

Fourth Quarter and Fiscal Year 2023 Financial Results
•Cash Position: Cash and cash equivalents were $391.6 million as of December 31, 2023, compared to $549.9 million as of December 31, 2022.
•Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $10.9 million for the fourth quarter of 2023, compared to $13.7 million for the fourth quarter of 2022. Total revenue, consisting primarily of revenue from collaboration agreements, was $44.6 million for the year ended December 31, 2023, compared to $39.8 million for the year ended December 31, 2022. For the fourth quarter of 2023, the decrease compared to the prior period was due to the timing of workflows from our strategic partnership with Roche-Genentech. For the year ended December 31, 2023 compared to the prior year, the increase was due to revenue recognized from our Roche-Genentech collaboration, which has progressed from primarily cell type evaluation work to inference based Phenomap building and additional cell type evaluation work.
•Research and Development Expenses: Research and development expenses were $69.5 million for the fourth quarter of 2023, compared to $44.0 million for the fourth quarter of 2022. Research and development expenses were $241.2 million for the year ended December 31, 2023, compared to $155.7 million for the year ended December 31, 2022. The increase in 2023 research and development expenses compared to the prior year was due to increased platform costs as we have expanded and upgraded our capabilities in platform including our chemical technology, machine learning and transcriptomics platform.
•General and Administrative Expenses: General and administrative expenses were $30.5 million for the fourth quarter of 2023, compared to $19.8 million for the fourth quarter of 2022. General and administrative expenses were $110.8 million for the year ended December 31, 2023, compared to $81.6 million for the year ended December 31, 2022. The increase in 2023 general and administrative expenses compared to the prior year was primarily driven by an increase in salaries and wages of $12.4 million and increases in legal, software and depreciation expense.
•Net Loss: Net loss was $93.0 million for the fourth quarter of 2023, compared to a net loss of $57.5 million for the fourth quarter of 2022. Net loss was $328.1 million for the year ended December 31, 2023, compared to a net loss of $239.5 million for the year ended December 31, 2022.
•Net Cash: Net cash used in operating activities was $74.1 million for the fourth quarter of 2023, compared to net cash used in operating activities of $44.7 million for the fourth quarter of 2022. Net cash used in operating activities was $287.8 million for the year ended December 31, 2023, compared to net cash used in operating activities of $83.5 million for the year ended December 31, 2022. The difference was primarily driven by a $150.0 million upfront payment from Roche-Genentech in early 2022 and an increase in operating expenses in 2023.

On February 27, 2024 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported that the Company will present at TD Cowen’s 44th Annual Health Care Conference on Monday, March 4 at 10:30 a.m. ET. (Press release, Dynavax Technologies, FEB 27, 2024, View Source [SID1234640540]).

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The presentation will be webcast and may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source

On February 27, 2024 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical-stage biopharmaceutical company discovering and developing clinically differentiated small molecule therapeutics targeting fundamental biological pathways of cancers, reported financial results for the year ended December 31, 2023 , and highlighted recent corporate accomplishments (Press release, Zentalis Pharmaceuticals, FEB 27, 2024, View Source [SID1234640537]).

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"Zentalis is making remarkable progress in advancing our clinical development program for azenosertib, our potentially groundbreaking WEE1 inhibitor, across various tumor types," stated Kimberly Blackwell, M.D., Chief Executive Officer of Zentalis. "In November 2023, we disclosed promising clinical data demonstrating the potential of azenosertib as an effective monotherapy in ovarian cancer and uterine serous carcinoma. Our team is diligently executing a clinical strategy, which is firmly on track, designed to rapidly bring this promising therapeutic option to patients with gynecological cancers that continue to have a poor prognosis based on currently available treatment options. We anticipate an exciting and data-rich period ahead during the remainder of 2024 and into 2025 where we plan to share multiple clinical datasets to help further demonstrate the potential of azenosertib in multiple cancer types as a monotherapy and in combination."

Program Updates and Highlights

•Azenosertib development program updates. In November 2023, the Company announced an updated analysis of the ongoing Phase 1 clinical trial of azenosertib as a monotherapy in solid tumors (ZN-c3-001), which continued to show anti-tumor activity with intermittent dosing. In the same population of 19 platinum resistant or refractory ovarian cancer and uterine serous carcinoma (USC) patients that were included in the data reported on June 6, 2023, the objective response rate (ORR) was 37%. Median follow-up had increased and the median progression free survival (mPFS) had increased to 6.5 months. The Company also disclosed in November 2023, that, with additional safety-evaluable patients and follow-up, azenosertib continued to demonstrate a favorable safety and tolerability profile similar to or better than approved ovarian cancer products.

•Azenosertib development continues to progress across a broad array of tumor types. Azenosertib is being evaluated in more than 10 ongoing and planned clinical trials as a monotherapy and in combinations supported by compelling scientific rationales across a broad array of tumor types, including platinum resistant ovarian cancer (PROC), platinum sensitive ovarian cancer (PROC), BRAF mutant metastatic colorectal cancer, and other solid tumors. In addition, the Company is evaluating azenosertib and its BCL-2 inhibitor in patients with relapsed or refractory Acute Myeloid Leukemia (AML).

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•On track to disclose multiple clinical data readouts during 2024 and 2025 (anticipated milestones noted below) and on track to submit its first New Drug Application (NDA) for azenosertib in a gynecologic malignancy in 2026.

Corporate Updates

•Strengthened Leadership Team with the addition of Diana Hausman, M.D. as Chief Medical Officer, and Kyle Rasbach, Ph.D., Pharm.D. as Chief Business Officer. Dr. Hausman is an oncologist with extensive experience in all aspects of drug development and clinical strategy, including over a decade of experience as a Chief Medical Officer at various biopharma companies. During her career, she has contributed to the development of multiple cancer therapeutics, including small molecules, antibody drug conjugates, and immunotherapies. Dr. Rasbach joins from Eventide Asset Management, where he was a Portfolio Manager for Eventide’s healthcare and life sciences strategies, a Managing Director for Eventide Ventures, and a Senior Research Analyst for other Eventide investments. He previously held investment management and equity research roles at several other firms.

•Strengthened balance sheet with licensing of ROR1 antibody drug conjugate and proprietary technology platform to Immunome. Under the terms of the transaction, in January 2024 Zentalis received an up-front payment of $35 million in cash and Immunome common stock and remains eligible to receive up to $275 million of milestone payments and mid-to-high single-digit royalties.

•Hosted a webcast with gynecologic oncology academic expert highlighting the strength of the emerging clinical profile for azenosertib. On November 10, 2023, Zentalis participated in a webcast with Joyce F. Liu, M.D., MPH, to discuss azenosertib ovarian cancer clinical data.

Anticipated Upcoming Milestones

•1H 2024
•Final results of Phase 1 azenosertib + chemotherapy (gemcitabine) trial in osteosarcoma (ZN-c3-003)
•2H 2024
•Final results of Phase 1b azenosertib monotherapy trial in solid tumors (ZN-c3-001)
•Topline data from Phase 1/2 azenosertib + PARP inhibitor (niraparib) and azenosertib monotherapy trial in platinum resistant ovarian cancer in partnership with GSK (MAMMOTH, ZN-c3-006)
•Initial data from Phase 1 azenosertib + BEACON regimen (encorafenib + cetuximab) trial in BRAF mutant metastatic colorectal cancer in partnership with Pfizer (ZN-c3-016)
•Initial data from Phase 1 of azenosertib + ZN-d5 trial in R/R AML (ZN-d5-004C)
•Additional details on planned clinical trial of azenosertib in PSOC in the 1L maintenance setting
•1H 2025
•Topline data from Phase 2 azenosertib monotherapy trial in platinum resistant high-grade serous ovarian cancer (DENALI, ZN-c3-005)
•2H 2025
•Topline data from Phase 2 azenosertib monotherapy trial in recurrent or persistent USC (TETON, ZN-c3-004)
•2025

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•Initiate clinical trial of azenosertib in PSOC in the 1L maintenance setting
•2026
•First NDA for azenosertib in a gynecologic malignancy

Full Year 2023 Financial Results

•Cash, Cash Equivalents and Marketable Securities Position: As of December 31, 2023, Zentalis had cash, cash equivalents and marketable securities of $482.9 million. The Company believes that its existing cash, cash equivalents and marketable securities as of December 31, 2023 will be sufficient to fund its operating expenses and capital expenditure requirements into 2026. The December 31, 2023 cash, cash equivalents and marketable securities balance does not reflect the up-front payment from Immunome of $35 million in cash and Immunome stock, which was received in January 2024, as the Company’s agreement was executed and announced in January 2024.

•Research and Development Expenses: Research and development (R&D) expenses for the year ended December 31, 2023 were $189.6 million, compared to $172.7 million for the year ended December 31, 2022. The increase of $16.9 million was primarily due to a $7.5 million increase in expense relating to our cost sharing relationship with Zentera that was terminated in June 2023, a $7.4 million increase related to personnel expenses, of which $5.8 million related to non-cash stock-based compensation expense, a $2.8 million increase in facilities and overhead expenses and a $2.8 million increase in consulting expense. These increases were partially offset by decreases of $2.8 million and $0.9 million in collaborations expense and supplies/other expenses, respectively.

•General and Administrative Expenses: General and administrative expenses for the year ended December 31, 2023 were $64.4 million, compared to $54.6 million during the year ended December 31, 2022. The increase of $9.8 million was primarily attributable to a $4.9 million non-cash operating lease impairment charge, a $5.1 million increase related to personnel expenses, of which $3.7 million related to non-cash stock-based compensation expense, and $1.4 million related to outside services. This was partially offset by a $1.6 million decrease in facilities and overhead and other expense.

About Azenosertib
Azenosertib is a potentially first-in-class and best-in-class small molecule WEE1 inhibitor in development for the treatment of cancer. Inhibition of WEE1, a DNA damage response kinase, drives cancer cells into mitosis without being able to repair damaged DNA, resulting in cell death. Currently, there are no FDA-approved WEE1 inhibitors, and azenosertib has been designed for superior selectivity and pharmacokinetic properties. Azenosertib is being developed in therapeutic areas of high unmet need and is being evaluated as a monotherapy, in combination with chemotherapy, and in combination with molecularly targeted agents.