On March 30, 2022 Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. reported that the U.S. Food and Drug Administration (FDA) has accepted for priority review the New Drug Application (NDA) for futibatinib in the treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring FGFR2 gene rearrangements, including gene fusions (Press release, Taiho, MAR 30, 2022, View Source [SID1234611161]). Futibatinib is an investigational, oral, potent, selective and irreversible small-molecule inhibitor of FGFR1, 2, 3 and 4. The FDA provided an anticipated Prescription Drug User Fee Act (PDUFA) action date of September 30, 2022.

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Each year, approximately 8,000 individuals in the U.S. are diagnosed with CCA,1 a rare cancer of the bile ducts of the liver, and approximately 0.3-6 people per 100,000 individuals live with CCA worldwide.2 CCA is mainly seen in people 65 years of age or older,3 and treatment options are limited. Within the CCA patient population, approximately 10-16% have tumors with FGFR2 gene rearrangements,4,5,6,7,8 including gene fusions, which can form a hybrid gene and promote tumor proliferation. It is this subset of patients with CCA that encompasses the NDA for futibatinib.

"Given the lack of an accepted standard chemotherapy following the failure of first-line treatment,9 futibatinib could represent a significant opportunity for a targeted therapy in this subset of patients with CCA, which has driven our pursuit with this investigational compound," said Volker Wacheck, Vice President, Clinical Development, Taiho Oncology, Inc. "We look forward to working with the FDA as they consider the application for futibatinib under priority review."

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The NDA is based on data from the pivotal Phase 2b FOENIX-CCA2 trial in 103 patients with locally advanced or metastatic unresectable intrahepatic (inside the liver) CCA, harboring FGFR2 gene rearrangements including fusions who had received one or more prior lines of systemic therapy. Patients in the trial received futibatinib 20 mg once daily until disease progression or unacceptable toxicity. The trial’s primary endpoint was an objective response rate (ORR), which was 41.7% as assessed by independent central review. The key secondary endpoint of duration of response (DOR) demonstrated a median of 9.7 months (72% of responses ≥6 months). Common treatment-related adverse events (TRAEs) in the trial were hyperphosphatemia (85%), alopecia (33%) and dry mouth (30%). The only serious adverse reaction reported in more than one patient enrolled in the FOENIX-CCA2 trial was migraine (1.9%).

Results from the trial were presented in 2021 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Meeting. Based on these data, the FDA granted Breakthrough Therapy Designation (BTD) for futibatinib for the treatment of patients with previously treated locally advanced or metastatic CCA in 2021.

"This is a very important step towards our goal to deliver futibatinib to patients awaiting potential new treatment options," said Teruhiro Utsugi, Senior Managing Director at Taiho Pharmaceutical. "The Taiho group, working as one, will continue to do its utmost to deliver this agent to those in need."

About Futibatinib
Futibatinib (TAS-120) is an investigational, oral, potent, selective, and irreversible tyrosine kinase inhibitor of FGFR1, 2, 3 and 4 being studied as a potential treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including cholangiocarcinoma, who were previously treated with chemotherapy or other therapies. Futibatinib selectively and irreversibly binds to the ATP binding pocket of FGFR1-4 resulting in the inhibition of FGFR-mediated signal transduction pathways, reduced tumor cell proliferation and increased tumor cell death in tumors with FGFR1-4 genetic aberrations.

On March 28, 2022 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinicalstagecompany developing therapeutics designed to treat diseases in areas of high unmet need, reported the peer-reviewed publication of a Phase 1, multicenter, open-label, dose-escalation studyinvestigating the therapeutic potential of intravenous VCN-01 oncolytic adenovirus with or withoutstandard-of-care (SoC) chemotherapy (gemcitabine/nab-paclitaxel) in patients with advanced solidtumors (Press release, VCN Biosciences, MAR 30, 2022, View Source [SID1234611126]). The data, published in the Journal for ImmunoTherapy of Cancer, suggests that treatment withVCN-01 is feasible and has an acceptable safety profile, with encouraging biological and clinical activity.These findings provide valuable dose-finding context and inform the clinical development strategy forVCN-01.

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"The results in this publication support VCN-01 administration intravenously at doses ≥3.3x1012vp/patient, resulting in viral exposure in the primary tumor and liver metastases, replication within thetumor, and the potential to remodel the tumor matrix to further promote tumor inflammation," saidManel Cascallό, Ph.D., General Director of Synthetic Biologics’ European Subsidiary. "These clinical dataunderscore VCN-01’s differentiated mechanism of action and were used to inform our Phase 2 study inpatients with metastatic pancreatic adenocarcinoma, which is expected to initiate in the second half of2022. More broadly, these results will help guide our rapidly advancing clinical program for VCN-01 andfurther support the development of our novel OV platform."

In the Phase 1, multicenter, open-label, dose-escalation study (NCT02045602), researchers evaluatedthe administration of a single dose of VCN-01 alone, in patients with solid tumors (Part I), or incombination with SoC chemotherapy (gemcitabine/nab-paclitaxel) in patients with locally advanced ormetastatic, unresectable, pancreatic adenocarcinoma (Parts II and III). In Part II, the VCN-01 wasadministered on the same day as the first dose of chemotherapy (Concomitant Regimen) and in Part IIIthe VCN-01 was administered 7-days prior to the first dose of chemotherapy (Sequential Regimen). Theobjective was to determine the maximum tolerated dose (MTD)/recommended Phase 2 dose (RP2D)and dose-limiting toxicity (DLT) of the intravenous delivery of the replication-competent VCN-01adenovirus.

Overall, systemic VCN-01 was well tolerated in the patient population. The most common treatmentrelatedadverse events (AEs) were pyrexia, flu-like symptoms and increases in liver transaminases. These AEs were dose-dependent, reversible and consistent with AEs previously described for other adenovirusbasedproducts. In Part II, transient increases in neutropenia and thrombocytopenia were observedwhen VCN-01 in combination with gemcitabine/nab-paclitaxel was administered using the ConcomitantRegimen, and one patient suffered a fatal episode of enterocolitis and thrombocytopenia. The AE profilewas significantly reduced in Part III when VCN-01 and gemcitabine/nab-paclitaxel was administeredusing the Sequential Regimen, and was similar to the observed AE profile when VCN-01 wasadministered alone. There were no dose limiting toxicities observed in patients administered VCN-01using the Sequential Regimen. The investigators determined the RP2D was 1×1013 viral particles(vp)/patient in Part I and Part III, and 3.3×1012 vp/patient in Part II.

The Phase 1 study provided encouraging clinical results and further confirmed the proposed VCN-01mechanism of action. In patients with pancreatic adenocarcinoma, overall response rates were 50%(Part II) and 50% (Part III). VCN-01 viral genomes were detected in tumor tissue in five out of six biopsies(primary pancreatic tumor and liver metastases) on day eight. A second peak of virus concentrations inplasma and increased serum hyaluronidase levels suggest replication after IV injection in all patients.Higher peaks of hyaluronidase serum levels were associated with maximum tumor shrinkage andincreased levels of immune biomarkers (IFNγ, sLAG3, IL-6, IL-10) were found in sera after VCN-01administration. Several markers of tumor inflammation (including CD8 infiltration and indoleamine 2, 3-dioxygenase [IDO] upregulation) were described in tumor biopsies indicating that VCN-01 promotes achange in the tumor immune environment.

Synthetic Biologics anticipates the initiation of multiple international studies, including a Phase 2 clinicaltrial of intravenous VCN-01 in combination with SoC chemotherapy using the Sequential Regimen as afirst-line therapy in newly diagnosed metastatic pancreatic adenocarcinoma patients in the fourthquarter of 2022, as well as a Phase 2/3 pivotal trial of intravitreal VCN-01 as either an adjunct tochemotherapy or a potential rescue therapy in pediatric patients with advanced retinoblastoma in early2023.

On March 30, 2022 IGM Biosciences, Inc. (NASDAQ: IGMS) reported the pricing of its underwritten public offering of 8,695,653 shares of its non-voting common stock at a price to the public of $23.00 per share (Press release, IGM Biosciences, MAR 30, 2022, View Source [SID1234611125]). IGM expects to receive total gross proceeds of approximately $200.0 million from this offering, before deducting the underwriting discounts and commissions and estimated offering expenses payable by IGM. In addition, IGM has granted the underwriters a 30-day option to purchase up to an additional 1,304,347 shares of its voting common stock at the public offering price, less underwriting discounts and commissions. All of the shares in the offering will be sold by IGM. The offering is expected to close on or about April 1, 2022, subject to satisfaction of customary closing conditions.

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J.P. Morgan, BofA Securities, Stifel, and Guggenheim Securities are acting as joint book-running managers for the offering.

The securities in the offering will be offered by IGM pursuant to a Registration Statement on Form S-3, filed with the Securities and Exchange Commission (SEC) on August 9, 2021 and declared effective on August 19, 2021. A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and may be accessed for free through the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to this offering may also be obtained from: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected]; BofA Securities, Attention: Prospectus Department, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, North Carolina 28255, or via email: [email protected]; Stifel, Nicolaus & Company, Incorporated, One Montgomery Street, Suite 3700, San Francisco, CA 94104, Attn: Syndicate, or by phone at (415) 364-2720, or by email at [email protected]; or Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected].

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

On March 29, 2022 SYNSIGHT, a biotech company specialized in RNA-targeting small molecules drugs, reported a $1.5 million fundraising (Press release, SYNSIGHT, MAR 29, 2022, View Source [SID1234644755]). The financing was led by an international investor.

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SYNSIGHT is pleased to announce today a $1.5 million fundraising This financial opportunity was realized with a Chinese investor who well recognized SYNSIGHT’s potential, which is completed by financial help from Region Ile-de-France and BPIfrance.

This fundraising will allow SYNSIGHT to accelerate its growth and development. The company will continue to refine its innovative platform and expand the drug discovery platform potentials. Thus, SYNSIGHT’s advantage in RNA targeting will be even enhanced.

The fundraising will also advance the SYNSIGHT pipelines and expand its portfolio. Noted that the SYNSIGHT pipelines are innovative targets which could only be treated by SYNSIGHT’s indoor innovative platform mentioned above, these targets (neurogenerative, oncology and infectious diseases related) were considered to be undruggable since the lack of breakthrough RNA target platform.

The fundraising will allow SYNSIGHT to expand its team and welcome new experts: senior project manager, AI research scientist, senior cell biologist and High-Content Screening engineer.

On March 29, 2022 Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases reported 2021 annual results including its major achievements and progress (Press release, Innovent Biologics, MAR 29, 2022, View Source [SID1234611483]).

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Dr. Michael Yu, Founder, Chairman and CEO of Innovent, stated: "2021 is the 10th year since our company’s inception. Over the past decade, bearing the mission to develop and commercialize high-quality biopharmaceutical products that are affordable to ordinary people, Innovent has developed into a leading Chinese biopharmaceutical company with fully-integrated platform capability, strong execution with track record and a healthy financial position. For the past year, we are determined with the global innovation strategy and have made tremendous progress in all aspects including R&D, clinical development, CMC, commercialization and business collaboration. The company has rolled out development for a pipeline of 32 valuable assets in various clinic stages. We plan to expand commercial portfolio to over ten products in two years with continuous strong revenue growth, supported by our validated commercial platform capability and competitive cost advantage under large scale and high quality manufacturing facilities. Besides, we further fortified R&D structure and platform to expedite the development for over a dozens of clinical stage candidates with global potential and more preclinical projects with global innovation. For the year of 2022, as the inflection point for next decade of development, we will adhere to the company strategy of global innovation, strive to be a global premier biopharmaceutical company with steady growth prospects, and create sustainable value for patients, employees, shareholders and the society."

Business Highlight Overview

For the year of 2021, achieved RMB4,261million total revenue and RMB4,001million product revenue with an increase of 69.0% compared to the prior year.
For the year of 2021, commercial portfolio increased to six products with two innovative anti-cancer drugs approved.
Expanded strategic partnership with Eli Lilly in March 2022 added the seventh newly approved product and a ready-to-launch product at New Drug Application (NDA) stage.
TYVYT received three additional first-line indication approvals for major types of cancer and successfully included in the updated National Reimbursement Drug List (NRDL), also as the only PD-1 inhibitor globally with positive Phase 3 data in the first-line treatment for 5 major types of cancer.
6 molecules at NDA or late clinical stage.
7 molecules achieved positive Proof of Concept (PoC) data readout.
Acceleration of Global R&D and clinical development, to explore first-in-class (FIC) targets with full-functional global development platform and global talents.
About 10 strategic collaborations with global and regional partners in research, development and commercialization, unleashing the platform potential and explore synergic value of pipeline portfolio.
Commercial – Strong Track Record

Product revenue RMB4,001 million in 2021: an increase of 69.0% compared with the prior year.
Expansion of commercial portfolio into six approved products, including: TYVYT, BYVASDA, SULINNO, HALPRYZA, PEMAZYRE and NAILIKE by the end of 2021.
In March 2022, expansion of strategic collaboration with Eli Lilly, which showed further recognition by the global MNC: for Innovent to obtain sole commercialization right of the 7th approved product Cyramza (ramucirumab) and NDA-stage asset Retsevmo (selpercatinib), and the right of first negotiation for future potential commercialization of late-stage asset Pirtobrutinib (BTK inhibitor) in mainland China.
Broad coverage in commercial channels and networks with an experienced and professional sales and marketing team expanded to cover over 5,100 hospitals and 1,100 pharmacies across more than 320 cities, and a well-structured commercial team with nearly 3,000 people.
Pipeline – Expedited Speed to Launch

– TYVYT as leading brand in PD-(L)1 market:

Newly approved 3 additional indications got successfully included in the NRDL (non-squamous Non-Small Cell Lung Cancer [nsqNSCLC], sqNSCLC, hepatocellular carcinoma [HCC]).
The only PD-1 inhibitor with positive Phase 3 data in first-line treatments of five major types of cancer (1L nsqNSCLC, 1L sqNSCLC, 1L HCC, 1L esophageal squamous cell carcinoma [ESCC], 1L gastric cancer [GC]).
Multiple clinical datasets of TYVYT were published in world-renowned medical journals such as EClinicalMedicine, ESMO (Free ESMO Whitepaper) Open, BMJ etc.
– Six assets at late stage incl. NDA/pivotal trials:

Retsevmo (selpercatinib), NDA submitted
IBI-326 (BCMA CAR-T), planned NDA submission in 2022
IBI-306 (PCSK9), planned NDA submission in 2022
IBI-310 (CTLA-4), planned NDA submission in 2022
IBI-344 (ROS1/NTRK), ongoing pivotal Phase 2
IBI-376 (PI3Kδ), ongoing pivotal Phase 2[1]
– Seven innovative molecules achieved positive PoC data readout, plan to advance into the late stage in clinic:

IBI-188 (CD47): achieved preliminary PoC data readout in Phase 1b study for 1L MDS, promising efficacy data (ORR 83.3%) and a good safety profile. Plan to initiate Phase 3 study for 1L MDS in 2022.
IBI-362 (GLP-1/GCGR): showed robust efficacy in weight loss for Phase 1b in obesity, data published in EClinicalMedicine. Robust efficacy in blood glucose lowering effects in diabetics patients. Potentially the best GLP1-/GCGR dual agonist. Plan to initiate Phase 3 studies for obesity and type 2 diabetes in 2022.
IBI-326 (BCMA CAR-T): demonstrated impressive efficacy and safety in Phase 1/2 study, with improved in-vivo persistency and encouraging efficacy in patients with prior murine BCMA CAR-T treatment failure. Plan to submit NDA in 2022.
IBI-344 (ROS1/NTRK): ORR 90.5% in the crizotinib-naïve patient and ORR 43.8% in the crizotinib-treated patient group. Received NMPA BTD for ROS1+ NSCLC. Pivotal Phase 2 study ongoing.
IBI-310 (CTLA-4): achieved positive PoC in combination with sintilimab for CC and HCC. Plan to submit NDA for CC in 2022.
IBI-302 (VEGF/C): showed visual acuity improvement with edema reduction in Phase 1b study, ongoing Phase 2 study for nAMD.
IBI-112 (IL-23p19): observed significant efficacy signal in the Phase 2 study for psoriasis with long-acting potential. Plan to initiate Phase 3 study in 2022.
– Expedite the clinical development of multiple molecule clusters with global potentials, expected PoC data readout roll out in a well-schemed plan:

CD47 cluster: IBI-188 (CD47), IBI-322 (PD-L1/CD47), IBI-397 (SIRPα)
LAG-3 cluster: IBI-110 (LAG-3), IBI-323 (PD-L1/LAG-3)
TIGIT cluster: IBI-939 (TIGIT), IBI-321 (PD-1/TIGIT)
VEGF cluster: IBI-302 (VEGF/C), IBI-324 (VEGF/ANG-2), IBI-333 (VEGF-A/VEGF-C)
R&D: Infrastructure Set for Global Innovation

– Innovent US Lab established: primarily focused on disease mechanism study and technology-platform development, accelerating the translation of scientific discovery into the next-generation drug candidates.

– The Scientific Advisory Board (SAB) established: comprising three World-renowned scientists to provide insights of frontline global innovation in cancer biology and immunology and contribute scientific advice to our research and clinical pipelines.

– The CMC Advisory Board (CAB) established: comprising industry-leading experts to provide professional advice on production structure and process streamline, resources investment and strategic development.

– The global product development team expansion and infrastructure improvement: have built a full-functional global development team and expect to have 100+ employees in near term. Have established an effective global development platform and process which enables to cooperate seamlessly with China clinical development team of 1,000 people to carry out global development for our novel assets.

– Technology platform collaborations for global innovation:

The development of the proprietary ADC technologies with Synaffix.
The development of three new therapeutic immune-stimulating antibody conjugate (ISAC) candidates with Bolt Biotherapeutics.
The development of up to three enzyme specific inhibitors with Amagma Therapeutics.
The license-out of non-exclusive commercial right of fully-human BCMA CAR construct to SANA Biotechnology for certain in vivo gene therapy and ex vivo hypo-immune cell therapy application.
BD: Meaningful Collaboration to Succeed

– Established multiple co-development and co-commercialization collaboration to unlock assets potential:

Expanded strategic collaboration with Eli Lilly for the sole commercialization right of Cyramza (ramucirumab) and Retsevmo (selpercatinib), and right of first negotiation for future commercialization of pirtobrutinib (BTK inhibitor) in mainland China.
Taletrectinib (ROS1/NTRK) – a next-generation TKI designed to effectively target ROS1 and NTRK, co-development and co-commercialization with AnHeart in China.
NAILIKE (olverembatinib) – a novel third-generation BCR-ABL TKI, co-development and co-commercialization with Ascentage Pharma in China.
GFH925 (KRAS G12C inhibitor) – a novel, orally active, potent KRAS G12C inhibitor, co-development and co-commercialization with GenFleet Therapeutics in China and opt-in right for global development and commercialization.
Orismilast (PDE4 inhibitor) – a novel, orally active PDE4 inhibitor for broad target indications, granted sole development and sole commercialization right in China from UNION therapeutics A/S.
Manufacturing Facilities Upgradation

New Commercial Facility: successfully expanded the production capacity from 24,000L to 60,000L, which is one of the largest stainless steel production capacity in China.
Quality compliance to GMP and cost advantage further strengthen market competitiveness.
Financial Highlights (Non-IFRS measure)

Total revenue was RMB 4,261 million, including product revenue 4,001 million with an increase of 69.0% compared to the prior year.
Gross profit margin was 88.6%，an increase of 3.7% compared to the prior year.
R&D expenses were RMB2,116 million an increase of 23.2% compared to the prior year.
Loss for the year was RMB2,243 million.
Cash on hand and short-term financial assets at the end of 2021 was approximately USD1,415million, which enables strategical focus on the long-term development strategies.[2]
Note: [1] In January 2022, Incyte has withdrawn the application of parsaclisib in FL, MZL and MCL in the U.S. as a business decision and is not related to any changes in either the efficacy or safety of parsaclisib. We plan to have communication with China NMPA regarding the potential submission of PI3Kδ.

[2] The financial numbers mentioned above was based on non-IFRS measure which excluded certain non-cash items and non-recurring events. Detailed disclosure can be found at the Company’s annual result announcement.