On February 15, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2023 (Press release, Agios Pharmaceuticals, FEB 15, 2024, View Source [SID1234640134]).

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"The past 12 months have been remarkable for Agios. We reported positive data across our industry-leading pipeline of PK activators, including Phase 3 data in non-transfusion-dependent thalassemia, Phase 2 data in sickle cell disease and clinical proof-of-concept in lower-risk MDS, and expanded our preclinical pipeline by in-licensing a novel siRNA program from Alnylam," said Brian Goff, chief executive officer at Agios. "In 2024, we expect two additional Phase 3 readouts, including the Phase 3 study of mitapivat in transfusion-dependent thalassemia, and are actively preparing for a potential U.S. launch in thalassemia in 2025. Together with our strong cash position, Agios is poised for significant near- and long-term growth as we progress toward of our vision of becoming a leading rare disease company."

Fourth Quarter 2023 & Recent Highlights

PYRUKYND U.S. Launch: Generated $7.1 million in U.S. net revenue for the fourth quarter of 2023, a 4 percent decrease from the third quarter of 2023, primarily driven by lower customer inventory levels at the end of the fourth quarter of 2023, partially offset by favorable gross-to-net adjustments. A total of 178 unique patients have completed prescription enrollment forms, representing an increase of 11 percent over the third quarter of 2023. A total of 109 patients are on PYRUKYND therapy, a 9 percent increase from the third quarter of 2023.
Thalassemia: Announced positive topline data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia. The study achieved its primary endpoint of hemoglobin response and achieved both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.
Sickle Cell Disease: Presented positive results from the Phase 2 portion of the RISE UP pivotal study of mitapivat at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The study achieved its primary endpoint of hemoglobin response and an improvement in annualized rates of sickle cell pain crises was observed.
Lower-risk Myelodysplastic Syndromes: Announced clinical proof-of-concept data in the open-label Phase 2a study of AG-946 for the treatment of anemia in lower-risk myelodysplastic syndromes (LR-MDS).
Earlier-stage Pipeline: Filed an Investigational New Drug Application (IND) for AG-181, Agios’ PAH stabilizer for the treatment of phenylketonuria (PKU).
Data presentations: Presented broad set of clinical and translational data at the 65th ASH (Free ASH Whitepaper) Annual Meeting & Exposition, including positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease, as noted above.
Anticipated 2024 Milestones

Thalassemia: Following the announcement of positive topline data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia in January 2024, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year) and file for FDA approval of mitapivat in thalassemia (year-end)
Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end)
Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat (mid-year). Report topline data from Phase 3 ACTIVATE kids-T study (year-end)
Lower-risk Myelodysplastic Syndromes: Dose first patient in Phase 2b study of AG-946 (mid-year)
Earlier-stage Pipeline: Dose the first patient in the Phase 1 study of AG-181 for the treatment of PKU (early 2024)
Fourth Quarter and Full Year 2023 Financial Results

Revenue: Net U.S. product revenue from sales of PYRUKYND was $7.1 million for the fourth quarter of 2023 compared to $4.3 million for the fourth quarter of 2022, and $26.8 million for the year ended Dec. 31, 2023 compared to $11.7 million for the year ended Dec. 31, 2022.

Cost of Sales: Cost of sales was $0.6 million for the fourth quarter of 2023 and $2.9 million for the full year ended Dec. 31, 2023.

Research and Development (R&D) Expenses: R&D expenses were $77.5 million for the fourth quarter of 2023 compared to $70.3 million for the fourth quarter of 2022, and $295.5 million for the year ended Dec. 31, 2023 compared to $279.9 million for the year ended Dec. 31, 2022. These changes reflect an increase in development costs for mitapivat and the up-front payment associated with the license agreement with Alnylam, partially offset by a reduction in expenses associated with the evolution of our research organization and the sale of our oncology business to Servier.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $35.3 million for the fourth quarter of 2023 compared to $32.8 million for the fourth quarter of 2022, and $119.9 million for the year ended Dec. 31, 2023 compared to $121.7 million for the year ended Dec. 31, 2022.

Net Income (Loss): Net loss was $95.9 million for the fourth quarter of 2023 compared to a net income of $36.5 million for the fourth quarter of 2022, and net loss was $352.1 million for the year ended Dec. 31, 2023 compared to $231.8 million for the year ended Dec. 31, 2022. The increase in net loss is due to the $127.9 million sale to Sagard in the fourth quarter of 2022 of our rights to future contingent payments associated with royalties on U.S. net sales of TIBSOVO.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of Dec. 31, 2023, were $806.4 million compared to $1.1 billion as of Dec. 31, 2022. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue, interest income and vorasidenib milestone will enable the company to fund its operating expenses and capital expenditures at least into 2026. This does not include cash inflows which could extend runway beyond 2026 including potential royalties or monetization of royalties from vorasidenib, commercializing mitapivat outside of the U.S. through one or more partnerships, or other potential strategic business or financial agreements.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2023 financial results and recent business highlights. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On February 15, 2024 Imugene Limited (ASX: IMU), a clinical stage immuno-oncology company, reported that its CD19 oncolytic virotherapy drug candidate onCARlytics (on-CAR-19, CF33-CD19 HOV4), has dosed the first patient in the intravenous monotherapy arm of the Phase 1 clinical trial (Press release, Imugene, FEB 15, 2024, https://mcusercontent.com/e38c43331936a9627acb6427c/files/7ffe1327-a379-db3f-e3a1-90fed6834c49/Phase_1_onCARlytics_Trial_Doses_First_Intravenous_Patient.pdf [SID1234640107]).

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Known as OASIS, the first-in-class clinical trial is targeting adult patients with advanced or metastatic solid tumours. The trial aims to evaluate the safety and efficacy of two routes of administration, intratumoural (IT) injection and intravenous (IV) infusion, either alone, or in combination with blinatumomab.

The trial is titled: "A Phase I, Dose Escalation and Dose Expansion, Safety and Tolerability Study of onCARlytics (CF33-CD19), Administered Intravenously or Intratumorally in Combination with Blinatumomab in Adults with Advanced or Metastatic Solid Tumors." See View Source

The combination arm of the study will include onCARlytics combined with CD19 targeting bispecific monoclonal antibody blinatumomab (marketed as Blincyto by Amgen).

onCARlytics has the potential to target and eradicate solid tumours that otherwise cannot be treated with Blincyto therapy alone.

OASIS is a dose escalation trial being conducted in the United States, with the first IV patient dosed at City of Hope in California. Additional sites are expected to open for recruitment across the US with 52 patients proposed to take part in the trial.

Imugene Managing Director and CEO Leslie Chong said: "We’re pleased to see onCARlytics continue to advance in the clinic after the first patient on the trial was dosed in late October, and this move into intravenous administration marks another milestone in our mission to bring innovative cancer treatments to patients in need. With the combination arm of the study still to come, we eagerly await the chance to see more on the potential onCARlytics holds."

onCARlytics is a CD19-expressing oncolytic virus that enters tumour cells and forces them to express the CD19 protein on the cell surface, presenting a target for CD19 targeting therapies.

On February 14, 2024 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported positive results from its diagnostic 64Cu-SAR-bisPSMA trial, COBRA (NCT05249127) (Press release, Clarity Pharmaceuticals, FEB 15, 2024, View Source [SID1234640087]).

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Dr Neal Shore MD, FACS Lead Principal Investigator in the COBRA trial, Medical Director of Carolina Urologic Research Centre, commented, "We are very enthusiastic regarding the data from the early phase COBRA trial as 64Cu-SAR-bisPSMA was confirmed to be safe and effective in detecting PC lesions in patients with BCR who came into the study with negative or equivocal scans using SOC imaging. The trial demonstrates a clinical advantage of same-day and next-day imaging in detecting additional lesions as well as for potentially detecting low prostate-specific membrane antigen (PSMA) expressing or smaller lesions. When detecting recurrence of PC, it is important to visualise small lesions, as reliable imaging of low volume tumor burden can affect treatment decision making. In the COBRA trial, we saw that 64Cu-SAR-bisPSMA positron emission tomography (PET) informed potential changes in the treatment plan in approximately half of the patients. This makes a difference for patients who may then have insight into the location of their disease recurrence and thereby proceed to treatment of their cancer. We look forward to additional data readouts from the trial and presenting the results at future international medical conferences. If the data from the COBRA trial can be substantiated in a registrational Phase 3 study, and if approved by the United States Food and Drug Administration (US FDA), this PET PSMA modality will impact care for patients with PC."

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are extremely excited with the initial findings from the COBRA trial as it further substantiates the many benefits of copper-64 (64Cu) and our optimised bisPSMA product in the diagnosis of PC. Combined with our clinical and pre-clinical trial data to date, this further validates SAR-bisPSMA as a potential best-in-class PSMA agent for the diagnosis (with 64Cu) and subsequent treatment (with copper-67 [67Cu]) of PC. We have also seen the performance of 64Cu-SAR-bisPSMA in the real-world setting through our compassionate use program, which demonstrated the ability of 64Cu-SAR-bisPSMA to detect lesions in patients with BCR and a negative or equivocal SOC imaging (including 18F-DCFPyL and 68Ga-PSMA-11). These cases also showed a higher number of lesions and lesions with higher maximum standardised uptake values (SUVmax) on next-day imaging. Therefore, we were already aware of the benefits of later-timepoint imaging, but the high number of lesions detected on same-day imaging that were negative or equivocal on SOC imaging (e.g. bone scan, CT or PSMA PET), and the almost doubling of the number of lesions with next-day imaging, is remarkable. The COBRA study now validates previous observations in a larger group of patients under trial conditions.

"Most importantly, the high rate of detection of PC in up to 80% of patients that were negative or equivocal on SOC imaging further brings to light the low sensitivity issues of current SOC imaging. We adhere to the highest standards and methods of clinical development as we pride ourselves on high quality science at Clarity. As such, the COBRA trial design was built to reflect the gold standard in clinical research and took a very conservative approach. Given we identified a large number of lesions with 64Cu-SAR-bisPSMA that were not visible with SOC scans, a number that was not anticipated when the protocol was prepared, it was not feasible nor ethical to verify all of these lesions with biopsy when caring for the patient. Therefore, our investigators were not able to confirm the true positivity of a very large number of lesions. Furthermore, among the patients that were excluded from the assessment of the efficacy endpoints because they had initiated systemic therapy while on study (which is not allowed according to the trial protocol), the majority had positive 64Cu-SAR-bisPSMA scans. As this was a first-in-human study in BCR PC patients, we have learnt significantly more about our product relative to current SOC imaging and will consider this potentially much higher level of detection and sensitivity of 64Cu-SAR-bisPSMA when structuring the Phase 3 trial design in this patient group.

"As can be seen from this study, and the clinicians reporting that they would change their treatment plan in response to this data, the increase in sensitivity with bisPSMA is incredibly important when assessing the return of PC in BCR patients. It is equally important for patients at initial staging of their disease, when they are considering their first course of definitive therapy, in order to understand whether their PC has metastasised or not from the primary lesion prior to prostatectomy. This is an immediate unmet medical need, which we are addressing with our Phase 3 CLARIFY trial2. Current SOC imaging fails to do this for a large percentage of men who eventually experience BCR of their PC. As such, there is an immediate opportunity for bisPSMA to address the entire market of PSMA imaging as we move closer to our ultimate goal of improving treatment outcomes of people with cancer.

"In addition to the clinical benefits, 64Cu-SAR-bisPSMA has a number of logistical and manufacturing advantages in comparison to other currently used PSMA agents. The ready-to-use product has been shipped to the trial sites in the US from a central manufacturing facility on-demand and on time, providing flexibility and reliability to the patients and their treating staff. This facilitates expanding the radiopharmaceutical field into the large oncology market, minimising logistical hindrances associated with the current generation of radio-diagnostics, and helping to focus on the needs of patients and their clinicians. Furthermore, the longer shelf-life of 64Cu-based products, coupled with the advantages of the SAR Technology, could offer access to PSMA PET for PC patients in underserved and broad geographical areas, which is one of the limitations of the approved PSMA agents due to their short half-life."

COBRA trial design & quality

The US-based COBRA study (Copper-64 SAR-bisPSMA in Biochemically Recurrent prostAte cancer) was a first-in-human trial of 64Cu-SAR-bisPSMA in patients with BCR of PC. It was a multi-centre, single-arm, non-randomised, Phase 1/2 diagnostic imaging study of 64Cu-labelled SAR-bisPSMA (64Cu-SAR-bisPSMA) administered to participants with BCR of PC following definitive therapy. The primary objectives of the trial were to investigate the safety and tolerability of 64Cu-SAR-bisPSMA as well as its ability to correctly detect recurrence of PC. Patients underwent PET/computed tomography (CT) scans on Day 0 and Day 1 (1-4h and 24±6h post-dose, respectively), which were interpreted by three blinded central readers. To determine the efficacy of 64Cu-SAR-bisPSMA imaging, the Day 0 and Day 1 PET/CT results of the central readers were assessed against a composite reference standard that was determined by an independent, blinded, central expert panel. The reference standard consisted of histopathology, follow-up SOC imaging and/or confirmed prostate specific antigen (PSA) response to focal therapy.

The design of the COBRA study followed advice from regulators to achieve the highest standards in clinical research in the BCR setting, in contrast to trials with other PSMA agents. Based on this guidance, the expert panel, who determined the reference standard, was blinded to the results of the 64Cu-SAR-bisPSMA scans and distinct from the central readers assessing the 64Cu-SAR-bisPSMA scans. This approach removed any potential biases in the assessment of the reference standard, which was not the case with previously approved PSMA tracers, where the reference standard was determined by readers having access to the PSMA scans investigated in the study, aiming to anatomically correlate the findings across the scans. Furthermore, a conservative approach was taken for the analysis of both co-primary endpoints. If a lesion identified on the 64Cu-SAR-bisPSMA scan was not biopsied and it was also not present on follow-up SOC imaging (a suboptimal reference standard with known low sensitivity), it was considered as false positive in the analysis by default. The higher standards in study design for COBRA compared to that used for other PSMA agents’ trials, along with various benefits intrinsic to 64Cu-SAR-bisPSMA not seen with currently approved products (e.g. higher uptake/retention and delayed imaging), make comparisons among other PSMA tracers and 64Cu-SAR-bisPSMA not viable. The methodology used in the COBRA trial underscores the ability of 64Cu-SAR-bisPSMA to correctly identify PC recurrence even when a higher level of rigour in study design is applied, making the transition of data to real world scenarios more probable.

Initial results from the COBRA trial

This study was the first-in-human trial of 64Cu-SAR-bisPSMA in patients with BCR of PC. Fifty-two patients with negative or equivocal SOC scans were enrolled and imaged, of whom 42 were included in the calculation of the efficacy endpoints. The median PSA at study entry was 0.9 ng/mL (range 0.25 – 17.60). This PSA range was considerably lower than in the registrational studies for the approved PSMA PET agents in BCR. Among the patients who did not complete the study as they proceeded to systemic therapy (which was not allowed according to the trial protocol), the majority had lesions identified on the 64Cu-SAR-bisPSMA scan, but they did not contribute towards the assessment of the efficacy endpoints. Results from COBRA showed for the first time that 64Cu-SAR-bisPSMA is safe and effective in detecting lesions in patients with BCR of PC who were negative or equivocal on SOC imaging (e.g. bone scan, CT or PET with approved PSMA imaging agents) at screening.

Next-day imaging – a key advantage

The possibility of performing next-day imaging is a feature not available to currently approved PSMA-targeted PET products and unique to 64Cu-based SAR diagnostics due to the optimal half-life of 64Cu and the ability of the SAR Technology to prevent leakage of copper isotopes from the radiopharmaceutical in-vivo. The COBRA trial confirmed the benefits of delayed imaging in this patient group as more lesions and more patients with a positive scan were identified on next-day imaging.

64Cu-SAR-bisPSMA was able to identify approximately 91% more lesions (median increase) on next-day imaging compared to same-day imaging (ranges across the readers for the total number of lesions: 53–80 on Day 0 vs. 82–153 on Day 1). The number of lesions in the pelvic LN region more than doubled on next-day imaging compared to same-day imaging (median increase of 108.3% across all readers comparing Day 0 vs. Day 1) (Figures 4 and 5). The ranges of CDR and patient-level detection rate (DR, defined as the proportion of participants with a positive 64Cu-SAR-bisPSMA PET/CT scan out of all scanned participants) were both higher on Day 1 compared to Day 0. The DR range on Day 0 was 44–58% (95% CI 30–71.8), increasing on Day 1 to 58–80% (95% CI 43.2–90).

About SAR-bisPSMA
SAR-bisPSMA derives its name from the word "bis", which reflects a novel approach of connecting two PSMA-targeting agents to Clarity’s proprietary sarcophagine (SAR) Technology that securely holds copper isotopes inside a cage-like structure, called a chelator. Unlike other commercially available chelators, the SAR Technology prevents copper leakage into the body. SAR-bisPSMA is a TCT that can be used with isotopes of copper-64 (Cu-64 or 64Cu) for imaging and copper-67 (Cu-67 or 67Cu) for therapy.

64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA are unregistered products. The data outlined in this announcement has not been assessed by health authorities such as the US Food and Drug Administration (FDA). A clinical development program is currently underway to assess the efficacy and safety of these products. There is no guarantee that these products will become commercially available.

About Prostate Cancer
Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of cancer death worldwide3. The American Cancer Institute estimates in 2024 there will be 299,310 new cases of prostate cancer in the US and around 35,250 deaths from the disease.

On February 14, 2024 Bristol Myers Squibb (NYSE: BMY) reported that it has priced a public offering (the "Offering") of senior unsecured notes in a combined aggregate principal amount of $13 billion (collectively, the "Notes") (Press release, Bristol-Myers Squibb, FEB 15, 2024, View Source [SID1234640084]). The Notes will be issued in nine tranches: (i) $500,000,000 in aggregate principal amount of floating rate notes due 2026, (ii) $1,000,000,000 in aggregate principal amount of 4.950% notes due 2026, (iii) $1,000,000,000 in aggregate principal amount of 4.900% notes due 2027, (iv) $1,750,000,000 in aggregate principal amount of 4.900% notes due 2029, (v) $1,250,000,000 in aggregate principal amount of 5.100% notes due 2031, (vi) $2,500,000,000 in aggregate principal amount of 5.200% notes due 2034 (the "2034 Notes"), (vii) $500,000,000 in aggregate principal amount of 5.500% notes due 2044 (the "2044 Notes"), (viii) $2,750,000,000 in aggregate principal amount of 5.550% notes due 2054 (the "2054 Notes") and (ix) $1,750,000,000 in aggregate principal amount of 5.650% notes due 2064 (the "2064 Notes"). Bristol Myers Squibb expects that the closing of the Offering will occur on February 22, 2024, subject to the satisfaction of customary closing conditions.

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Bristol Myers Squibb intends to use a portion of the net proceeds of the Offering to fund the cash consideration payable in connection with the previously announced proposed acquisitions of Karuna Therapeutics, Inc. ("Karuna") and RayzeBio, Inc. (collectively, the "Acquisitions") and the fees and expenses in connection therewith and with the Offering. Bristol Myers Squibb expects to use any remaining net proceeds from the Offering for general corporate purposes. The Offering is not conditioned upon the consummation of the Acquisitions; however, if Bristol Myers Squibb’s acquisition of Karuna is not completed on or before the later of (i) June 30, 2025 and (ii) the date that is five business days after any later date to which the "End Date" as set forth in the merger agreement relating to acquisition of Karuna may be extended pursuant to its terms, or Bristol Myers Squibb notifies the trustee in respect of the Notes that it will not pursue consummation of the acquisition of Karuna, then Bristol Myers Squibb will be required to redeem all outstanding Notes, other than the 2034 Notes, the 2044 Notes, the 2054 Notes and the 2064 Notes, at a special mandatory redemption price equal to 101% of the aggregate principal amount of such series of Notes, plus accrued and unpaid interest, if any, to, but excluding, the special mandatory redemption date.

Citigroup Global Markets Inc., BofA Securities, Inc., Wells Fargo Securities, LLC, and Mizuho Securities USA LLC are acting as joint lead managers and joint book-running managers for the Offering.

The Offering of the Notes is being made pursuant to an effective shelf registration statement (including a prospectus and preliminary prospectus supplement) (File No. 333-261623) filed with the U.S. Securities and Exchange Commission (the "SEC"). You may get these documents for free by visiting EDGAR on the SEC website at www.sec.gov. Alternatively, Bristol Myers Squibb, any underwriter or any dealer participating in the Offering will arrange to send you the prospectus and the preliminary prospectus supplement (or, if available, the prospectus supplement) if you request it by contacting Bristol Myers Squibb Investor Relations or Citigroup Global Markets Inc. at 1-800-831-9146, BofA Securities, Inc. at 1-800-294-1322, Wells Fargo Securities, LLC at 1-800-645-3751, or Mizuho Securities USA LLC at 1-866-271-7403.

On February 14, 2024 Elsie Biotechnologies, Inc. reported that GSK plc (LSE/NYSE: GSK) has exercised its option to a non-exclusive license to Elsie’s discovery platform, following a successful outcome of the research collaboration, announced in July 2023, to explore the platform capabilities (Press release, Elsie Biotechnologies, FEB 14, 2024, View Source [SID1234644171]). The option allows GSK to employ Elsie’s discovery platform and P(V) chemistry technologies in its own oligonucleotide drug discovery research.

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Kevin Green, Chief Operating Officer, Elsie Biotechnologies, said, "We are pleased that GSK has elected to license our platform technology following the research collaboration. GSK is a great innovator in the field of oligonucleotide therapeutics, and this license validates the potential of our discovery platform, which we believe can power next generation RNA medicines, optimized for safety, activity, and delivery."

Christine Donahue, VP, Molecular Modalities Discovery, GSK, said, "We are excited to continue our work with Elsie which aligns so closely with our focus at the intersection of science and tech. It builds on our industry-leading position in advanced platform technologies and exemplifies our commitment to the potential of oligonucleotide therapeutics."

Elsie’s discovery platform is an unparalleled ultra-high throughput proprietary process that allows for the complete evaluation of oligonucleotide chemical space. By applying proprietary encoding technology to oligonucleotide therapeutic candidates, all possible sequences or chemical modification patterns can be evaluated to increase activity, reduce toxicity, and improve delivery. Elsie also applies proprietary P(V) chemistry technologies, encompassing a suite of novel reagents and processes, to synthesize diverse oligonucleotide therapeutics with complete synthetic control.

Under the terms of the agreement, Elsie will receive a license payment from GSK and could receive additional development and commercial milestones on a target-by-target basis.