On January 12, 2022 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported that pipeline and corporate updates at the 40th Annual J.P. Morgan Healthcare Conference (Press release, Precigen, JAN 12, 2022, View Source [SID1234605464]). Helen Sabzevari, PhD, President and CEO of Precigen, presented a summary of 2021 achievements and set forth Precigen’s goals for 2022.

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Precigen’s presentation included the following pipeline announcements:

PRGN-3006 UltraCAR-T in Acute Myeloid Leukemia (AML)

Overview: 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 kill switch. PRGN-3006 UltraCAR-T is under evaluation in a Phase 1/1b clinical trial for the treatment of patients with relapsed or refractory AML or higher-risk myelodysplastic syndromes (MDS). Trial subjects receive the PRGN-3006 infusion either without prior lymphodepletion (Cohort 1) or following lymphodepleting chemotherapy (Cohort 2). PRGN-3006 UltraCAR-T has been granted Orphan Drug Designation in patients with AML by the FDA.
Program Updates: Precigen announced enrollment completion for Dose Level 3 of the lymphodepletion cohort. Interim data for patients treated in Dose Levels 1-3 of the non-lymphodepletion cohort and Dose Levels 1-2 of the lymphodepletion cohort were recently presented at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The dose escalation phase of the study is now complete for both the lymphodepletion and non-lymphodepletion cohorts and the Company plans to initiate a multicenter expansion phase of the study at Dose Level 3 with lymphodepletion in the first half of 2022. The Company plans to incorporate a repeat dosing regimen in the expansion phase. Additional Phase 1/1b data is expected in 2022.
PRGN-3005 UltraCAR-T in Ovarian Cancer

Overview: PRGN-3005 UltraCAR-T is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, which is highly expressed on ovarian tumors with limited normal tissue expression, mbIL15, and a kill switch. PRGN-3005 UltraCAR-T is under evaluation in a Phase 1/1b clinical trial for the treatment of patients with advanced, recurrent platinum-resistant ovarian cancer. Trial subjects receive PRGN-3005 either via intraperitoneal (IP) (Arm A) or intravenous (IV) (Arm B) infusion.
Program Updates: Precigen announced enrollment completion for Dose Level 3 of the IV arm, completing enrollment in both the IP and IV arms in the dose escalation phase of the study. Interim data for patients treated in Dose Levels 1-3 of the IP arm were recently presented at the Company’s 2021 R&D Virtual Event. The Company has received FDA clearance to incorporate lymphodepletion at Dose Level 3 of the IV arm and will initiate the multicenter expansion phase of the study, incorporating redosing.
PRGN-3007 Next Generation UltraCAR-T with Intrinsic PD-1 Inhibition

Overview: PRGN-3007, based on the next generation of the UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to simultaneously express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression. ROR1 is aberrantly expressed in multiple hematological tumors, including chronic lymphocytic leukemia (CLL), mantle cell leukemia (MCL), acute lymphoblastic leukemia (ALL), and diffuse large B-cell lymphoma (DLBCL) and solid tumors, including breast adenocarcinomas such as triple negative breast cancer (TNBC), pancreatic cancer, ovarian cancer, and lung adenocarcinoma. ROR1 is minimally expressed in healthy adult tissues.
Program Updates: Precigen plans to initiate dosing in the Phase 1 study in ROR1+ hematological (CLL, MCL, ALL, DLBCL) and solid (TNBC) tumors in 2022.
PRGN-2012 AdenoVerse Immunotherapy in Recurrent Respiratory Papillomatosis (RRP)

Overview: PRGN-2012 is an investigational off-the-shelf (OTS) AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with HPV 6 or HPV 11 for treatment of RRP. PRGN-2012 is currently under evaluation in a Phase 1 clinical trial under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI). The Phase 1 trial is designed to follow 3+3 dose escalation of PRGN-2012 as an adjuvant immunotherapy following standard-of-care surgical removal of visible papillomas in adult patients with RRP. PRGN-2012 has been granted Orphan Drug Designation in patients with RRP by the FDA.
Program Updates: Precigen announced enrollment completion for the Phase 1 dose escalation and expansion cohorts of the Phase 1 study. Interim data for the Phase 1 study were recently presented at the Company’s 2021 R&D Virtual Event. The Company plans to seek FDA guidance on a rapid regulatory strategy for PRGN-2012 in RRP given the positive interim results and significant unmet patient need. Additional Phase 1 expansion data is expected in the second half of 2022.
PRGN-2009 AdenoVerse Immunotherapy in HPV-associated Cancers

Overview: PRGN-2009 is an OTS investigational immunotherapy utilizing the AdenoVerse platform designed to activate the immune system to recognize and target HPV-positive (HPV+) solid tumors. PRGN-2009 is currently under evaluation in a Phase 1/2 clinical trial under a CRADA with the NCI. The Phase 1 trial is evaluating safety and response of PRGN-2009 as a monotherapy (Arm A) and in combination with M7824 (Arm B) in previously treated patients with recurrent or metastatic HPV-associated cancers.
Program Updates: Precigen announced enrollment completion in the Phase 1 monotherapy arm. Enrollment is ongoing in the Phase 1 combination arm and the Phase 2 monotherapy arm in newly diagnosed OPSCC patients. Interim data for patients in the Phase 1 monotherapy and combination arms treated at Dose Levels 1-2 were recently presented at the Company’s 2021 R&D Virtual Event. Additional Phase 1 data for both arms is expected in 2022. The Company plans to seek FDA guidance on a rapid regulatory strategy for PRGN-2009 given the positive interim results and significant unmet patient need. The Company also plans to initiate a Phase 2 study in advanced HPV-associated cancer indications in combination with an approved anti-PD-1 checkpoint inhibitor.
AG019 ActoBiotics

Overview: AG019 is an investigational therapy designed to induce oral immune tolerance to reverse type 1 diabetes (T1D) and is currently under clinical evaluation for the treatment of early-onset T1D.The Phase 1b/2a clinical trial is evaluating AG019 as a monotherapy and in combination with teplizumab (PRV-031), which is currently under investigation in the PROTECT Phase 3 study for the treatment of newly diagnosed T1D.
Program Updates: Precigen announced the completion of the Phase 1b/2a clinical trial. Positive results from the trial were presented last year at the Federation of Clinical Immunology Societies (FOCIS) Virtual Annual Meeting and European Association for the Study of Diabetes (EASD) 57th Annual Meeting. The Company plans to initiate discussions with the FDA and European Medicines Agency (EMA) for Phase 2/3 clinical trial design for AG019 in T1D.
"Precigen made significant clinical progress in 2021 across our pipeline programs. We were able to meet the major clinical goals we outlined at JPM last year, and exceeded some goals such as the rapid progress made for PRGN-2012 in RRP. Interim data presented in 2021 across three platforms – UltraCAR-T, AdenoVerse, ActoBiotics – and five clinical programs continue to produce positive results" said Helen Sabzevari, PhD, President and CEO of Precigen, "In 2022, we will continue to advance our clinical programs with a concentration on rapid paths to licensure for programs addressing high unmet patient needs."

Precigen’s J.P. Morgan presentation is available on the Company website in the Events & Presentations section at investors.precigen.com/events-presentations.

On January 12, 2022 ICON plc, (NASDAQ: ICLR), a world-leading clinical research organisation powered by healthcare intelligence, reported its financial guidance for the year ended December 31, 2022 (Press release, ICON, JAN 12, 2022, View Source [SID1234605455]). For the full year 2022, revenue is expected to be in the range of $7,770 – $8,050 million, representing growth of 43 – 46% and adjusted earnings per share is expected to be in the range of $11.55 – $11.95, representing growth of 21 – 23%, over Full Year 2021 revenue and adjusted earnings per share guidance, respectively x. Adjusted earnings per share to exclude amortization, stock compensation, foreign exchange and transaction-related / integration-related adjustments.

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CEO Steve Cutler commented, "As the world’s largest and most comprehensive CRO, ICON is well positioned to lead the market as the healthcare intelligence partner of choice in clinical development. We saw strong demand from customers that resulted in a trailing twelve month book to bill of 1.3x and total backlog of $18.6 billion at the end of the third quarter 2021. With our solid performance in 2021 coupled with continued strength in the broader industry environment, we expect growth of 43 – 46% in revenue to a range of $7,770 – $8,050 million and adjusted earnings per share to increase by 21 – 23% to a range of $11.55 – $11.95 for the full year 2022."

The full year 2022 financial guidance assumes:

US dollar to Euro exchange rate of $1.15.
An effective tax rate of circa 16.5%.
Days Sales Outstanding of circa 25 – 30 days.
Circa $1bn free cash flow and capital expenditures of circa $150 million.
No share repurchase or M&A activity included in the above guidance.
With respect to 2021, the company reaffirmed its current guidance of revenue in the range of $5,430 – $5,530 million and adjusted earnings per share in the range of $9.55 – $9.75.

On January 12, 2022 Adagene presented the Corporate Presentation (Presentation, Adagene, JAN 12, 2022, View Source [SID1234598649]).

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On January 12, 2022  OncoHost, a global leader in next-generation precision oncology for improved personalized cancer therapy, reported results from its ongoing multicenter clinical trial, PROPHETIC (Press release, OncoHost, JAN 12, 2022, View Source [SID1234598648]). These interim results confirm that through proteomic analysis of just one blood test before treatment, OncoHost’s AI platform, PROphet, can predict response to cancer treatment for non-small cell lung cancer (NSCLC) patients with remarkably high accuracy at three months, six months and one year.

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"These results are statistically and clinically significant, giving us great hope for the future of precision oncology. The value that this validation will provide, both to cancer patients and their clinicians, is insurmountable," said Dr. Ofer Sharon, CEO of OncoHost. "With just one blood test pre-treatment, we will now be empowered with a level of knowledge that is currently non-existent. Understanding the response probability trajectory for a full twelve months before treatment initiation, combined with insights on resistance associated pathways and proteins, has the potential to allow physicians to make critical clinical decisions earlier, all based on personalized patient data."

PROphet is a first-of-its-kind diagnostic platform that combines proteomic analysis with AI to predict patient response to immunotherapy and identify resistance associated processes with just one blood test pre-treatment. The device employs high-throughput protein analysis technology, scanning approximately 7,000 proteins in a patient’s plasma using the SomaScan platform by SomaLogic (NASDAQ-GM: SLGC), combined with proprietary bioinformatic and machine learning-based algorithms, to identify proteomic patterns that are predictive of patient outcome. PROphet also identifies potential drug targets, advancing the development of novel therapeutic strategies and providing clinicians with potential combination strategies to overcome treatment resistance.

With a focus on patients diagnosed with advanced stages of melanoma or non-small cell lung cancer (NSCLC) receiving immunotherapy, OncoHost’s clinical trials have shown PROphet to have high accuracy. OncoHost continues to open additional clinical trial sites around the world and will be expanding its research to further cancer indications including ovarian cancer, head and neck cancers and urogenital cancers.

The full results of the analysis will be published later this year in a peer-reviewed paper. This announcement comes ahead of OncoHost’s official launch of PROphet in 2022.

On January 12, 2022 I-Mab (the "Company") (Nasdaq: IMAB), a clinical-stage biopharmaceutical company committed to the discovery, development, and commercialization of novel biologics, reported that the first patient had been dosed in its China phase 2 study (NCT05145907) of efineptakin alfa (also known as TJ107) in combination with pembrolizumab (Keytruda) in patients with advanced solid tumors (Press release, I-Mab Biopharma, JAN 12, 2022, View Source [SID1234598647]). The study will follow a "basket" trial design to include selected tumor types, including triple-negative breast cancer (TNBC) and squamous cell cancer of the head and neck (SCCHN).

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Efineptakin alfa, the world’s first and only long-acting recombinant human interleukin-7 (rhIL-7) developed as a T lymphocyte-booster for cancer-related immunotherapy, has distinct advantages over other cytokines such as human IL-2. Efineptakin alfa combined with immune checkpoint inhibitors, such as PD-(L)1 therapies, has a synergistic effect as it increases the number of circulating anti-tumor T cells for tumor suppression. It has been tested as monotherapy and in combination with checkpoint inhibitors to treat advanced solid tumors in the U.S., South Korea and China, with encouraging clinical results.

"As the majority of cancer patients either do not respond or respond poorly to current PD-(L)1 therapies, there are intense attempts to identify an effective agent that can work synergistically with PD-(L)1 antibodies to increase the probability of treatment success," said Professor Ye Guo, Deputy Director of Department of Oncology at Shanghai East Hospital and the principal investigator of the study. "Studies have shown that efineptakin alfa has the potential to address this unmet need. We look forward to further validating the safety and efficacy profile of efineptakin alfa in patients with solid tumors."

"The initiation of efineptakin alfa’s phase 2 study is another example that I-Mab’s pipeline is not only innovative but also mature with a majority of clinical programs being in the advanced clinical development stage," said Dr. Andrew Zhu, President of I-Mab. "Efineptakin alfa is the world’s first rhIL-7 designed to cater to the therapeutic needs of cancer patients, so we are mindful of the importance of this therapy and are committed to expediting the clinical development of a potentially transformative solution for patients in need."

I-Mab is also evaluating efineptakin alfa in another phase 2 clinical trial (NCT04600817) in lymphopenic patients with newly diagnosed glioblastoma multiforme (GBM) who have received standard concurrent chemoradiotherapy. By leveraging the two ongoing clinical studies, the Company aims to rapidly advance the clinical development of efineptakin alfa for approval in Greater China.

About Efineptakin alfa

Efineptakin alfa, also known as TJ107/GX-I7/NT-I7, is the world’s first and only long-acting recombinant human interleukin-7 (rhIL-7), known to boost T lymphocytes by increasing their number and functions. It emerged from Genexine’s proprietary hyFc platform for the discovering of long-acting biologics. I-Mab has acquired exclusive rights from Genexine to develop and commercialize efineptakin alfa in Greater China. Efineptakin alfa may have utility in cancer treatment-related lymphopenia (low blood lymphocyte levels), a common condition that occurs in cancer patients who have received chemotherapy or radiation therapy, for which there is no approved treatment. Efineptakin alfa has also been shown to synergize with a PD-1 antibody in various tumor animal models potentially through increased T-lymphocyte activation and proliferation.