On March 30, 2021 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 its financial results for the year ended December 31, 2020 and the corporate progress. In 2020, the Company achieved total revenue of RMB 3843.8 million (Press release, Innovent Biologics, MAR 30, 2021, View Source [SID1234577391]). In addition to the first commercial product TYVYT (sintilimab injection), the Company successfully added three more antibody drugs to the commercial portfolio with the NMPA approval of BYVASDA (bevacizumab biosimilar), SULINNO (adalimumab biosimilar) and HALPRAZA (rituximab biosimilar), becoming the only biopharmaceutical company that successfully launched four antibody drugs in China in only nine years since inception.

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Dr. Michael Yu, Founder, Chairman and CEO of Innovent, stated, "2020 is a landmark year for Innovent. In the past year, despite the challenges of COVID-19, we minimized the impact on our business and achieved remarkable milestones. We ensured product supply with the non-stop operation of our production line even during the peak of pandemic in the beginning of 2020. Production capability expanded significantly from 5,000L to 24,000L. With the NRDL coverage, we more than doubled the sales of Tyvyt with even higher volume growth. We kept advancing our valuable assets rapidly in China and globally, with six NDAs submitted, four NDAs approved, five assets in Phase 3 or pivotal Phase 2 stage, and 14 assets in the early clinical stage. We also accelerated our global footprint with six assets under global development and a series of international collaborations achieved. More importantly, we’ve upgraded our R&D platform to strive toward global innovation for the next stage."

"With the conclusion of the ninth year since incepted, we are now starting the final year of our first decade. Up to now we have built Innovent from a biotech start up to a biopharmaceutical company with a fully integrated functional platform. However, this is just a start. Our journey to excellence is still in the infancy stage. Heading into the next decade, we are committed to moving Innovent to become a global innovative biopharmaceutical company, capturing the substantial potential of the rising innovative drug market both in China and globally, as well as and delivering tremendous value for our patients, employees, and shareholders."

Business Highlights during 2020 to Year-to-Date 2021

Four commercial product portfolio with three new drugs approved; RMB 2,367.5 million product revenue in 2020

After the first product TYVYT (sintilimab injection) was approved in 2018, BYVASDA (bevacizumab biosimilar), SULINNO (adalimumab biosimilar) and HALPRAZA (rituximab biosimilar) were approved by the NMPA during 2020, making Innovent the only biopharmaceutical company that successfully launched four antibody drugs in China in only nine years since inception.
Total product revenue reached RMB 2,367.5 million for the year ended 31 December 2020, an increase of 133.0% compared to RMB 1,015.9 million in the prior year.
The leading product TYVYT (sintilimab injection) generated RMB2,289.8 million in revenue for the year ended 31 December 2020. This represents an increase of 125.4% from RMB1015.9 million in the prior year.
In addition, TYVYT (sintilimab injection) was approved as the first line treatment for non-squamous non-small cell lung cancer (NSCLC) in February 2021. Three more supplemental new drug applications (sNDA) for TYVYT (sintilimab injection) were under NMPA review with expected approval by 2021 or early 2022, including: 1) the first line treatment for squamous NSCLC; 2) the first line treatment for hepatocellular carcinomas in combination with BYVASDA (bevacizumab biosimilar), and 3) the second line treatment of squamous NSCLC.
Robust pipeline of 23 diverse-staged assets

The company has built up a highly differentiated and competitive pipeline consisting of 23 clinical stage valuable assets that are being developed globally, including four assets approved, five assets under Phase 3 or pivotal studies and 14 assets in other clinical stage. The 23 assets cover major areas including monoclonal antibodies, bispecific antibodies, CAR-T and small molecules etc.
Five assets in Phase 3 or pivotal clinical trial

In addition to the four commercial products, the Company owns a total of five assets in Phase 3 or pivotal clinical studies that are expected to be launched within the next several years, including:

IBI-310 (CTLA-4 monoclonal antibody)
IBI-375 (FGFR inhibitor)
IBI-376 (PI3Kδ inhibitor)
IBI-306 (PCSK9 monoclonal antibody)
IBI-326 (BCMA CAR-T)
Comprehensive and differentiated pipeline in oncology and immunology

Supported by the Company’s deep understanding in immunology (IO) and the unique strength in monoclonal antibody and bispecific antibody, the Company’s IO assets have high degree of differentiation and lead the development progress even in the global market. The comprehensive pipeline cover most of the promising IO targets including PD-1, CD47, LAG-3, TIGIT etc.

In CD47 area: the Company owns three differentiated assets in the area. The potential best-in-class CD47 monoclonal antibody IBI-188 have completed Phase 1a dose escalation and started Phase 1b in 2020. The Company plans to enter phase III or pivotal clinical trial this year for 1L MDS for IBI-188. IBI-322 is a first-in-class PD-L1/CD47 bispecific antibody under Phase 1 study in China and the US. The Company plans to obtain proof-of-concept (PoC) data for IBI-322 in 2021. The Company also has the potential best-in-class SIRPα monoclonal antibody under pre-clinical research stage.
In LAG-3 area: the Company’s LAG-3 monoclonal antibody IBI-110 is the first LAG-3 approved for IND in China. The Company has completed phase 1b enrollment for IBI-110 and will obtain PoC data in 2021. The potential first-in-class PD- L1/LAG-3 bispecific antibody has also received IND approval recently, and the Company will start Phase 1 study in 2021.
In TIGIT area: the TIGIT monoclonal antibody IBI-939 has entered Phase 1b study, with plan to obtain PoC data in 2021. The Company also submitted the IND application for its PD-1/TIGIT bispecific antibody IBI-321 recently, and plans to start clinical study in 2021.
Besides, in 2020, the PD-1/PD-L1 bispecific antibody IBI-318 has finished Phase 1a study and entered multiple Phase 1b studies to explore the potential of IBI-318 in different cancer indications.
Featured non-oncology pipeline with significant progress

The Company’s featured pipeline in non-oncology cover high potential areas with unmet medical needs, including autoimmunity, metabolism, cardiovascular and ophthalmology. In particular, two unique assets with Best-in-Class or First-in-Class potentials made significant progress in 2020, including:

IBI-302 (VEGF/complement fusion protein): The Company has completed the Phase 1a study with optimistic preliminary data for the treatment of wet age-related macular degeneration ("wet AMD"). The Company plans to start Phase 2 clinical study in 2021.
IBI-362 (OXM3): The Company is conducting Phase 1b studies in both obesity objects and diabetic patients. IBI-362 has shown very promising effect on glucose control and weight reduction. The Company plans to start Phase 2 clinical studies in 2021.
Entered strategic collaborations with world-class partners

The company has made a series of collaborations with international and regional partners during the year of 2020. In particular:

The strategic expanded licensing out agreement with Lilly on the exclusive rights of TYVYT (sintilimab injection) outside of China marked the first major step in bringing our innovative portfolio to the global market.
The collaboration with Roche on the discovery and development of bispecific antibodies and multiple cell therapies shows the recognition of our drug discovery and R&D capabilities by a global top-tier pharmaceutical company, and could also further enrich our potential First-in-Class pipeline down the road.
Significant expansion in manufacturing capacity

In 2020, the Company expanded the manufacturing capacity from 5,000L to a total of 24,000L production capacity to support the production needs for both commercial product and clinical stage candidates in the pipeline.
In 2020, the Company has also started the construction of a new manufacturing facility that is designed to house additional twelve 3,000L production capacities, which once completed, will expand the production capacity to a total of 60,000L.
Continuous enlargement of talent pool; upgrade of R&D toward global innovation

In the year of 2020, the talent team increase from about 2,000 employees as at 31 December 2019 to more than 3,200 employees as at 31 December 2020.
In particular, the Company appointed Dr. Yongjun Liu, a renowned world class scientist and successful leader in biopharmaceutical industry as the president of the Company, responsible for our global R&D, portfolio strategy, business development as well as international operation.
In order to achieve the strategic goal of global innovation, the Company is upgrading the R&D to a fully functional structure with global scope, global talents and global vision.
Fruitful capital market achievements

In 2020, the "B" marker was also successfully removed from the Company’s stock name.
The stock was also included in the Hang Seng Composite Index and the Stock Connect in 2020.
During 2020 to year to the date of 2021, the Company has successfully raised a total of approximately US$1.3 billion fund from three rounds of new share placements, backed by strong subscription of well-known international and regional investors.
As the end of February 2021, the Company has approximately US$1.8 billion cash on hand, providing a strong support to our drug R&D, potential business collaboration, production facility expansion and increased international operation needs.
Financial highlights for the year ended December 31, 2020

Total revenue was RMB3,843.8 million for the year ended 31 December 2020, representing an increase of 266.9% from RMB1047.5 million in the prior year. The growth was mainly driven by the strong product growth as well as increase in license fee and service income in 2020.
Product revenue increased by 133.0% to RMB2,367.5 million in 2020 from RMB1,015.9 million in 2019. This was mainly driven by the strong growth of our leading product Tyvyt coupled with revenue contribution of three newly approved antibody drugs in the second half of 2020.
R&D expenses increased by 43.0% to RMB1,851.5 million for 2020. The R&D expenses were spent on progressing clinical trials of late-stage and prioritized assets towards our robust pipeline globally, and expanding collaboration and licensing programs to further enhance our all-rounded R&D capabilities.
Loss and total comprehensive expenses decreased by 42.0% to RMB998.4 million. This reduction in loss was primarily driven by higher product sales as well as license fee income from the upfront payment for the out-license of Tyvyt to Lilly in ex China region.

On March 30, 2021 Applied BioMath (www.appliedbiomath.com), the industry-leader in applying systems pharmacology and mechanistic modeling, simulation, and analysis to de-risk drug research and development, reported a collaboration with Asher Biotherapeutics for systems pharmacology modeling in solid tumors (Press release, Applied BioMath, MAR 30, 2021, View Source [SID1234577390]). "We have experience that development of a biotherapeutic has the best chance of success when the R&D process is supported by mathematical modeling," said Lioudmila Tchistiakova, Entrepreneur in Residence at Third Rock Ventures. "We have chosen to collaborate with Applied BioMath given their proven track record in this space," said Ivana Djuretic, PhD, Chief Scientific Officer of Asher Biotherapeutics. "This collaboration will enable us to confidently predict pharmacokinetics, receptor occupancy, and pharmacodynamics of our lead molecule."

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Applied BioMath employs a rigorous fit-for-purpose model development process which quantitatively integrates knowledge about therapeutics with an understanding of its mechanism of action in the context of human disease mechanisms. Their approach employs proprietary algorithms and software that were designed specifically for systems pharmacology model development, simulation, and analysis. "Systems pharmacology modeling’s impact is extremely effective when leveraged early on in the R&D process," said Dr. John Burke, PhD, Co-Founder, President, and CEO of Applied BioMath. "Incorporating modeling early on helps the project team gain thorough understanding of their lead molecule which in turn helps increase the likelihood of success in the clinic."

On March 30, 2021 Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS), a biopharmaceutical company focused on the discovery and development of novel anti-infective therapies to treat life-threatening infections, reported financial and corporate results for the fourth quarter ended December 31, 2020 (Press release, Aridis Pharmaceuticals, MAR 30, 2021, View Source [SID1234577389]).

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Fourth Quarter Highlights and Recent Developments

Announced positive preclinical efficacy data for AR-711, a potential inhaled, self-administered, at-home monoclonal antibody treatment ("mAb") for non-hospitalized mild-to-moderate COVID-19 patients. A clinical Phase 1/2 study is expected to be launched in 2H 2021.
Received concurrence from the U.S. Food and Drug Administration ("FDA") to streamline AR-501’s Phase 2 clinical trial design and to expand the originally planned Phase 2a protocol design into a Phase 2a/2b study for treating chronic lung infections in patients with cystic fibrosis (CF). Enrollment completion is expected in late 2021.
Continued enrolling global Phase 3 clinical trial of AR-301 in patients with ventilator associated pneumonia (VAP) including patients who presented with S. aureus VAP as a secondary infection to COVID-19. Interim futility analysis is expected in 2H 2021 and top-line data expected in 1H 2022.
Entered into a ʎPEX out-licensing and product discovery agreement with Kermode Biotechnologies, Inc. on vaccines and mAbs for zoonotic viruses, which are animal viruses that have the ability to infect humans.
Licensed the CRISPR gene editing technology from the Broad Institute of MIT and Harvard. CRISPR is a key component of the APEX mAb discovery and production platform technology.
Executed a Registered Direct offering with gross proceeds of approximately $8.5 million in the fourth quarter and approximately $7.0 million in March 2021.
"During the fourth quarter and over the recent months, we achieved multiple important milestones that impact our clinical and corporate profile, highlighted by the addition of an inhaled at-home COVID-19 treatment (AR-712) to our portfolio of product candidates and entering into an ʎPEX out-licensing and product discovery agreement with Kermode Biotechnologies for zoonotic viruses," commented Vu Truong, Ph.D., Chief Executive Officer of Aridis Pharmaceuticals. "Additionally, we reached concurrence with the FDA to streamline and thus expedite the clinical and regulatory process for AR-501’s Phase 2 program in cystic fibrosis, and bolstered our balance sheet with the $15.5 million financing. These important achievements have helped position us for continued growth as we head into 2021."

COVID-19 Program Update

AR-712: During the quarter, Aridis announced the development of a highly potent fully human mAb against SARS-CoV-2 virus. AR-712 is a cocktail of two mAbs, AR-711 and AR-713, designed to lower the barrier to treatment coverage of non-hospitalized COVID-19 patients by using a convenient, self-administered inhaled dosage presentation. The two mAbs that comprise AR-712 were discovered from convalescent COVID-19 patients and target the receptor-binding domain (RBD) region of the spike protein of the original SARS-CoV2 virus and its newly emerging variants including the currently prevalent strain ‘E484K’ associated with the South Africa, Brazil, and Japan variants.

In an animal challenge study with golden Syrian hamsters, inhaled AR-711 successfully eliminated all detectable SARS-CoV-2 virus at substantially lower doses than parenterally administered (injected) COVID-19 mAb. The AR-712 mAbs are engineered to be long-acting in blood for up to six to twelve months and are stabilized using a proprietary formulation designed to protect the mAbs from the physical stresses imparted by commercial nebulizer delivery devices on protein drugs. The potency of AR-712 and its direct delivery to the lungs by inhaled administration may facilitate significant dose sparing not achievable by parenteral administration. A proprietary formulation enables AR-712 to be deliverable using a variety of commercially available nebulizers that can be self-administered on an outpatient basis, thus lowering the barrier to COVID-19 therapeutic treatment. Clinical trials for AR-712 are expected to commence 2H 2021.

AR-701: During the quarter, Aridis continued to characterize this cocktail of fully human mAbs discovered from its in-house ʎPEX mAb discovery platform that is directed at multiple envelope proteins of the SARS-CoV-2 virus. AR-701 is intended to treat hospitalized, moderate to severe patients, which complements AR-712’s focus on milder non-hospitalized patients.

Clinical Program Update

AR-301: AR-301 is being evaluated in Phase 3 clinical study as an adjunctive treatment to standard of care antibiotics in S. aureus infected ventilator associated pneumonia (VAP) patients. Thus far, the pace of the trial has continued to be impacted by the protracted COVID-19 pandemic. The Phase 3 interim futility analysis from the ongoing pivotal trial is now expected to be reported in 2H 2021 and top line data by 1H 2022. It’s important to note that COVID-19 patients on prolonged mechanical ventilation in the intensive care unit (ICU) are prone to secondary infections (also called ‘superinfections’) by opportunistic pathogens such as bacteria. Superinfection is a reported complication in COVID-19 patients, which exacerbates morbidity and the rate of mortality. The AR-301 Phase 3 study allows for the enrollment of patients with baseline characteristics which are inclusive of certain COVID-19 patients. While AR-301 is not an agent to treat SARS-CoV-2 virus itself, it can potentially reduce the morbidity associated with secondary S. aureus pneumonia, which is a coronavirus complication and a contributing cause of death in such patients.

The trial, which was initiated in the first quarter of 2019, is expected to enroll 240 patients at approximately 160 clinical centers in 22 countries. Participating clinical centers that are activated continue to follow standard stringent clinical protocols and procedures for critically ill VAP patients, as is standard in the U.S. and Europe. The trial represents the first ever Phase 3 superiority clinical study evaluating immunotherapy with a fully human monoclonal antibody to treat acute pneumonia in the intensive care unit setting. Details of the study can be viewed on www.clinicaltrials.gov using identifier NCT03816956.

AR-501: During the quarter, the Company announced an agreement with the FDA to simplify AR-501’s Phase 2 trial design for the treatment of chronic lung infections associated with cystic fibrosis (CF). After reporting (June 2020) positive Phase 1 safety data in healthy adults who were exposed to a single ascending dose (SAD) or a multiple ascending dose (MAD) regimen, Aridis proposed, and the FDA has now agreed, to streamline AR-501’s forthcoming Phase 2a clinical trial in CF patients by removing the SAD and only conducting a MAD regimen. The FDA also concurred with the Company’s proposal to expand the originally planned Phase 2a protocol design into a Phase 2a/2b study. This Phase 2a/2b design will enable seamless and efficient advancement of the study from Phase 2a into Phase 2b using the same clinical study protocol. The data from the Phase 2a will inform the dose selection and sample size expansion to achieve statistical significance in efficacy in Phase 2b.

AR-501 is being developed in collaboration with the CF Foundation and has been granted Orphan Drug Designation (ODD), Fast Track and Qualified Infectious Disease Product (QIDP) designations by the FDA. In addition, the European Medicines Agency (EMA) granted ODD to AR-501. The original Phase 1/2a clinical trial was a randomized, double-blinded, placebo-controlled SAD and MAD trial investigating the safety and PK of inhaled AR-501 in healthy volunteers and cystic fibrosis patients with chronic bacterial lung infections. Details of the original Phase 1/2a clinical trial can be viewed on www.clinicaltrials.gov using identifier NCT03669614. The new Phase 2a/b study design will be available on clinicaltrials.gov within the next quarter.

Corporate Update

A key development was the closing of an $8.5 million financing which occurred on October 14th. The proceeds from this registered direct offering and concurrent private placement, strengthens the Company’s balance sheet during the fourth quarter to prioritize the continued advancement of AR-301’s Phase 3 VAP clinical trial, while allocating the requisite resources to AR-501’s Phase 2b cystic fibrosis clinical trial, and the ongoing development of novel COVID-19 therapies such as AR-701 and AR-711.

Fiscal 2020 Fourth Quarter Results:

Cash: Total cash and cash equivalents as of December 31, 2020 was $8.2 million. The Company completed a registered direct financing in March 2021 and received gross proceeds of approximately $7.0 million.
Revenues: Revenue was approximately $1 million for both periods ended December 31, 2020 and 2019. There was no revenue for both quarters ended December 31, 2020 and 2019.
Research and Development Expenses: Research and development expenses decreased by approximately $7.1 million from $24.1 million for the year ended December 31, 2019 to $17.0 million for the year ended December 31, 2020. The year over year decrease was primarily due to the following: a decrease in spending on clinical trial activities and drug manufacturing expenses for the Phase 2 study of our AR-105 program that was terminated during 2019; a decrease in drug manufacturing expenses for the Phase 3 study of our AR-301 program; a decrease in spending on clinical trial activities for the Phase 1/2a study of our AR-501 program because the Phase 1 portion of the study ended in the second quarter of 2020; a decrease in spending on other research and development activities; and a decrease in personnel, consulting and other related costs. These decreases were partially offset by an increase in spending on research and development activities for our COVID-19 program.
There was no material difference in research and development expenses for the quarter ended December 31, 2020 when compared to the same period in 2019.
General and Administrative Expenses: General and administrative expenses increased by approximately $419,000 from $6.0 million for the year ended December 31, 2019 to $6.4 million for the year ended December 31, 2020 due primarily to increases in directors’ and officers’ related liabilities insurance expense, professional service fees, and rent expense due to the Company entering into a new facility lease during the fourth quarter of 2020. These increases were partially offset by a decrease in patent related fees and Delaware franchise taxes. General and administrative expenses incurred in the quarter ended December 31, 2020 were approximately $1.6 million, an increase of approximately $204,000 over the same period in 2019 which was due primarily to increases in professional service fees, personnel related expenses, including stock-based compensation, and rent expense due to the Company entering into a new facility lease during the fourth quarter of 2020, partially offset by a decrease in Delaware franchise taxes.
Interest Income, net: Interest income, net decreased by approximately $280,000 from $357,000 for the year ended December 31, 2019 to $77,000 for the year ended December 31, 2020. Interest income, net for the quarter ended December 31, 2020 decreased by approximately $82,000 over the same period in 2019. These decreases are primarily due to lower interest rates and a lower average cash balance during 2020 as compared to 2019.
Share of Loss from Equity Method Investment: Loss from equity method investment decreased by approximately $942,000 from approximately $951,000 for the year ended December 31, 2019 to $9,000 for the year ended December 31, 2020 as our share of loss from our minority interest in the JV Agreement with Shenzhen Hepalink Pharmaceutical Group Co., Ltd., calculated under the equity method was limited to the reduction of the net book value of the investment to zero as of March 31, 2020. Loss from equity method investment decreased by $41,000 for the quarter ended December 31, 2020 when compared to the same period in 2019 which was due to there being no share of losses from our equity method investment recorded in the fourth quarter of 2020 as the net book value of the investment has been zero since March 31, 2020.
Net Loss: The net loss for the year ended December 31, 2020 was $22.3 million, or $2.44 net loss per share, compared to a net loss of $29.7 million, or $3.51 net loss per share per share, for the year ended December 31, 2019. The net loss for the quarter ended December 31, 2020 was $5.8 million, or $0.59 net loss per share, compared to a net loss of approximately $5.6 million, or $0.63 net loss per share, for the quarter ended December 31, 2019. The weighted average common shares outstanding was approximately 9.2 million and approximately 8.5 million for the year ended 2020 and 2019, respectively.

On March 30, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the U.S. Food and Drug Administration (FDA) approved a revised label for Vyxeos (daunorubicin and cytarabine) to include a new indication to treat newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged one year and older (Press release, Jazz Pharmaceuticals, MAR 30, 2021, View Source [SID1234577388]). The approval of Vyxeos for this indication is supported by safety data from two single-arm trials: AAML1421, conducted by the Children’s Oncology Group (COG) and CPX-MA-1201, conducted by Cincinnati Children’s Hospital (CCH) and evidence of effectiveness from an adequate and well-controlled study in adults.

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"At Jazz Pharmaceuticals, we believe all patients living with complex conditions deserve solutions, and work diligently to expand the science behind our therapies to ensure the greatest number of patients can benefit from our medicines," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. "While pediatric patients represent a relatively small percentage of total AML patients, there is a critical need for more effective therapies in this setting. With the expansion of the Vyxeos label to include the pediatric population, Jazz demonstrates our continued commitment to broadening our cancer research and focusing on the people for whom we can have the greatest impact."

Safety and pharmacokinetics of Vyxeos in children and young adults were established in two clinical studies that enrolled patients with AML or relapsed/refractory hematologic malignancies. Thirty-eight pediatric patients aged one to 21 years of age with AML in first relapse were enrolled in the Phase 1/2 AAML1421 study conducted by COG, and 27 patients aged one to 19 years with relapsed/refractory hematologic malignancies were enrolled in the Phase 1 CPX-MA-1201 study conducted by CCH. Both studies found no differences in the safety profile based on age. 1 The use of Vyxeos for this indication is supported by evidence of effectiveness from study CPX351-301 in adult patients.

Vyxeos has a Boxed Warning as it cannot be substituted with other daunorubicin and/or cytarabine-containing products. In the Phase 3 study, the most common adverse reactions (incidence ≥ 25%) were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders and vomiting.2

"The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients," said Dr. Edward Anders Kolb, M.D., director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children and chair of myeloid disease committee at COG. "Jazz has been a wonderful partner in pediatric drug development and we are grateful for the continued work being done to provide safe and effective therapies for children."

About Vyxeos (daunorubicin and cytarabine)
Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor, that is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older. For more information about Vyxeos in the United States, please visit View Source

More information about Vyxeos, including Full Prescribing Information, BOXED Warning and Medication Guide, is available here.

Important Safety Information
WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products.

VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients.

VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles, or legs
unusual tiredness
VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
VYXEOS contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

About AML
Acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets.3 It can sometimes spread to other parts of the body including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles.4 AML is a relatively rare disease representing 1.1 percent of all new cancer cases.5 It is estimated that more than 19,500 people will be diagnosed with AML in the United States this year with the potential for more than 11,000 people to die from the disease.6 The median age at diagnosis is 68 years old,6 with rising age associated with a progressively worsening prognosis.7 AML in children makes up a small portion of the overall AML population (4.5% occurs in patients < 20 years old). Further, t-AML and AML-MRC in pediatric AML are very rare subtypes of this group accompanied by poor prognosis.5 There is also a reduced tolerance for intensive chemotherapy as patients age.8 AML has the lowest survival rate of any other form of leukemia.5 Patients with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes may have a particularly poor prognosis.9-11 A hematopoietic stem cell transplant may be a curative treatment option for patients.12

On March 30, 2021 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 that the Center for Drug Evaluation (CDE) of China’s Nation Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation (BTD) for Parsaclisib (IBI376) for the treatment of patients with relapsed/refractory follicular lymphoma (FL) (Press release, Innovent Biologics, MAR 30, 2021, View Source [SID1234577387]). IBI376 is a phosphatidylinositol 3-kinase delta (PI3Kδ) inhibitor originally discovered by Incyte. Innovent owns the rights to develop and commercialize Parsaclisib in greater China.

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NMPA grants Breakthrough Therapy Designation to new medicines that are intended to treat serious conditions and where clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint. The BTD designation for Parsaclisib is based on the results observed in an ongoing Phase 2 study for the treatment of adults with relapsed or refractory FL being conducted in China (CTR2019239). Parsaclisib is currently in Phase 2 pivotal trials for the treatment of adults with relapsed or refractory follicular or with marginal zone lymphoma.

In clinical studies, Parsaclisib has demonstrated potent and rapid anti-lymphoma activity with promising safety, efficacy, and persistence. Preliminary clinical results of the CITADEL-203 study in relapsed or refractory FL presented at the 62st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) held in 2020 highlighted the safety profile, efficacy, and the durability of response of Parsaclisib (Abstract 2935). Study results showed that in the dosing group (N=95), Parsaclisib monotherapy achieved an objective response rate of 75%, with median PFS of 15.8 months in relapsed or refractory FL patients.

Dr. Hui ZHOU, Vice President of Medical Science and Strategy Oncology of Innovent, stated: "The breakthrough therapy designation from NMPA indicated that Parsaclisib possesses great potential in treating relapsed or refractory follicular lymphoma and we are hopeful that this product can help benefit more patients in the future."

About Parsaclisib (IBI376)

Parsaclisib is an investigational novel oral inhibitor of phosphatidylinositol 3-kinase delta (PI3Kδ) isoforms. PI3Kδ is an important anticancer target implicated in malignant B-cell growth, survival and proliferation which has demonstrated potency and selectivity in preclinical studies and has potential therapeutic utility in the treatment of patients with hematologic malignancies such as lymphoma. Parsaclisib is currently under evaluation as a monotherapy in several ongoing Phase 2 trials as treatment for non-Hodgkin lymphomas (follicular, marginal zone and mantle cell); and autoimmune hemolytic anemia. Pivotal trials of Parsaclisib in combination with ruxolitinib for the treatment of patients with myelofibrosis are also underway; and there are plans to initiate a trial to evaluate Parsaclisib in combination with tafasitamab for non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia (CLL).

In December 2018, Innovent and Incyte entered into a strategic collaboration for three clinical-stage product candidates discovered and developed by Incyte, including Parsaclisib (PI3Kδ inhibitor). Under the terms of the agreement, Innovent has received the rights to develop and commercialize Parsaclisib and two other assets in Mainland China, Hong Kong, Macau and Taiwan.

About Follicular Lymphoma

Follicular lymphoma (FL) is a B-cell cancer that originates from the uncontrolled division of specific types of B-cells known as centrocytes and centroblasts. Although it is classified as indolent lymphoma, and the current immunochemotherapy has achieved good efficacy, it still often relapses following by aggressive diseases, which may lead to death within 1 to 2 years. There is an unmet medical need for treatment options for recurrent or refractory follicular lymphoma.

About Breakthrough Therapy Designation

In recent years, in order to encourage innovation to address unmet clinical needs, China has established a rapid drug review and approval pathway. A Breakthrough Therapy Designation (BTD) is intended to facilitate and expedite development and review of an investigational drug to treat serious disease or condition when preliminary clinical evidence indicates that the drug has demonstrated substantial improvement over current therapies. The BTD will not only qualify a drug candidate to receive status for rapid review by the CDE, but also allow the sponsor to obtain timely advice and communications from the CDE to accelerate the approval and launch in order to address the unmet clinical needs of patients at an accelerated pace.