On April 28, 2021 Isofol Medical AB (publ), (Nasdaq First North Premier Growth Market: ISOFOL), reported that the Board of Directors has the intention to carry out an issue of shares of approximately SEK 400 million with preferential rights for the Company’s existing shareholders (the "Rights Issue") (Press release, Isofol Medical, APR 28, 2021, View Source [SID1234578622]). An extraordinary general meeting will be held around May 14, 2021 (the "EGM") to propose to resolve to authorize the Board of Directors to resolve on the Rights Issue and the terms thereof. The EGM is also proposed to resolve to authorize the Board of Directors to resolve on a directed share issue, corresponding to approximately SEK 100 million, with deviation from the shareholders’ preferential rights (the "Over-Allotment Option"), in order to meet potential additional demand from strategic investors and thereby broaden the shareholder base. The notice to the EGM will be announced through a separate press release.

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Summary
The Board of Directors announces its intention to propose an EGM to authorize the Board of Directors to carry out the Rights Issue and the potential Over-Allotment Option (provided that the Rights Issue is oversubscribed). The EGM scheduled to be held around May 14, 2021, will be announced through a separate press release.
The net proceeds from the Rights Issue and the potential Over-Allotment Option will be used to fund i) clinical development activities relating to the AGENT study beyond top-line results and activities for finalization of the NDA application, (ii) finalization of the development and validation of CMC, iii) global preparedness activities including the development of medical affairs and commercial launch packages, as well as continued partnering activities, iv) clinical development activities, including gene expression analysis and initiation of studies in additional indications and (v) general corporate purposes.
Subject to the EGM’s resolutions, the Rights Issue is fully guaranteed through subscription undertakings from current shareholders and guarantee commitments from current shareholders and external investors.
The potential Over-Allotment Option will be conditional upon the Rights Issue being oversubscribed.
Isofol’s CEO Ulf Jungnelius comments: "During the last year, Isofol has achieved several important milestones such as two partnership deals, a fully recruited AGENT study and in Q1 2021 a positive outcome of the interim analysis. Strengthening our financial position enables us to secure the financing needed to submit a New Drug Application to FDA and EMA, expected in H2 2022. Furthermore, it allows us to maintain a high pace in the pre-commercialization activities to ensure the prerequisites for a successful launch of arfolitixorin upon potential approval. We also see good potential to continue the development of gene expression analysis for arfolitixorin in order to bring new treatment options for cancer patients and to create value for our shareholders."

Background and intention
Isofol is a clinical stage biotech company developing arfolitixorin to improve the efficacy of standard of care chemotherapy for advanced colorectal cancer by increasing tumor response and progression free survival.

Arfolitixorin – the key active metabolite of widely used folate-based drugs – can potentially benefit more patients with advanced colorectal cancer as it does not require complicated metabolic activation to become effective. Arfolitixorin is currently being studied in the global Phase III AGENT study.

The AGENT study is a randomized, controlled, multi-centre study assessing the efficacy and safety of arfolitixorin, [6R]-5,10-methylene-THF acid (MTHF), compared to leucovorin, both used in combination with 5-FU, oxaliplatin, and bevacizumab, in first line metastatic colorectal cancer patients. Patients are randomized in a 1:1 ratio and the primary endpoint is overall response rate (ORR). The key secondary endpoints are progression free survival (PFS) and duration of response (DOR). Other secondary endpoints include overall survival (OS), number of curative metastasis resections, safety, and patient reported outcomes such as quality of life (QoL). Exploratory endpoints include pharmacokinetic (PK) measurements and level of gene expression of folate relevant genes in tumor cells. The study is designed to show superiority for arfolitixorin over leucovorin.

The AGENT study is fully recruited and is ongoing at approximately 90 sites in the U.S., Canada, Europe, Australia and Japan, where Isofol currently has 15 active sites.

Isofol raised approximately SEK 180 million in June 2020 through a rights issue and an over-allotment option. Since the June 2020 capital raise, Isofol has reached the mentioned and critical milestones for that capital raise, such as; the study is fully recruited, the interim result has been presented and the Company has signed licensing agreements with Solasia in Japan and Endo/Paladin in Canada. These licensing agreements are expected to positively affect Isofol’s financial position over time.

In March 2021 the independent Data Safety and Monitoring Board ("iDSMB") recommended continuation of the AGENT study with 440 patients, in accordance with the study design for arfolitixorin. The interim analysis was the fifth time the iDSMB has assessed safety data. Isofol views the iDSMB’s recommendation to continue the study without any amendments to the study protocols as an important signal that the treatment is safe. The treatment of enrolled patients will continue with follow-ups and repeated tumor measurements according to the study’s protocol. After 300 PFS events have taken place, either with tumor growth or that the patient has passed away, a data read out is initiated, with compilation and statistical analysis to present top line results. The Company expects these events to occur during the first and second halves of 2022.

The Board of Directors intends to carry out the Rights Issue and the potential Over-Allotment Option to ensure the continued and successful development of the Company, in accordance with its business plan and strategy. The intention of the Rights Issue and the potential Over-Allotment Option is primarily to fund i) clinical development activities relating to the AGENT study beyond top-line results and activities for finalization of the NDA application, (ii) finalization of the development and validation of CMC, iii) global preparedness activities including the development of medical affairs and commercial launch packages, as well as continued partnering activities, iv) clinical development activities, including gene expression analysis and initiation of studies in additional indications and (v) general corporate purposes.

Through the potential Over-Allotment Option, if exercised in full, the Company will receive an additional financing of approximately SEK 100 million before transaction costs. The potential Over-Allotment Option is conditional upon the Rights Issue being oversubscribed. The reason to deviate from the shareholders’ preferential rights is that the Board of Directors, in the event of strong interest from investors, wishes to further strengthen the Company’s capital as well as broaden the Company’s shareholder base with new strategic investors.

Subscription undertakings and guarantee commitments
The Rights Issue is fully guaranteed through subscription and guarantee commitments.

A number of investors have provided guarantee commitments, which together with subscription undertakings from several existing shareholders, including The Fourth Swedish National Pension Fund ("AP4"), in total represent SEK 400 million. In addition, certain shareholders including Handelsbanken Fonder and Swedbank Robur have expressed that they are supportive to the Rights Issue and that they intend to subscribe for their respective pro rata shares.

Members of the Board of Directors and Management, comprising Ulf Jungnelius, Pär-Ola Mannefred, Gustaf Albèrt and Anna Belfrage, who jointly hold approximately 0.4 percent of the Company’s outstanding shares have committed to subscribe their respective pro rata shares in the Rights Issue amounting to approximately SEK 1.8 million.

Shareholders representing in total approximately 12.3 percent of the shares and votes in the Company have undertaken to at the EGM vote to authorize the Board of Directors to carry out the Rights Issue and the potential Over-Allotment Option.

EGM and expected timetable for the Rights Issue
The Board of Directors convene the shareholders to the EGM through a separate press release. The EGM will take place around May 14, 2021. A detailed timetable and terms of the Rights Issue will be announced if the Board of Directors resolves on the Rights Issue.

Advisors
Carnegie Investment Bank AB (publ) and Pareto Securities AB act as Joint Bookrunners in connection with the Rights Issue and the potential Over-Allotment Option. Vinge law firm acts as legal adviser to Isofol, and Schjødt law firm acts as legal adviser to the Joint Bookrunners.

This is information that Isofol Medical AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 18:00 CEST on April 28, 2021.

About arfolitixorin
Arfolitixorin is Isofol’s proprietary drug candidate being developed to increase the efficacy of standard of care chemotherapy for advanced colorectal cancer. The drug candidate is currently being studied in a global Phase III study, AGENT. As the key active metabolite of the widely used folate-based drugs, arfolitixorin can potentially benefit more patients with advanced colorectal cancer, as it does not require complicated metabolic activation to become effective.

On April 28, 2021 BeiGene (NASDAQ: BGNE; HKEX: 06160) reported positive results from a planned interim analysis of the Phase 3 ALPINE trial comparing BRUKINSA (zanubrutinib) against ibrutinib in adults with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) (Press release, BeiGene, APR 28, 2021, View Source [SID1234578640]).

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BRUKINSA met the primary endpoint of the trial, demonstrating non-inferiority in objective response rate (ORR) by both investigator and independent review committee (IRC) assessments (p < 0.0001). The trial also demonstrated superior ORR with a statistically significant improvement in ORR for BRUKINSA vs. ibrutinib (p = 0.0006) by investigator assessment, as well as a numerically higher ORR but not statistically significant improvement by IRC (p = 0.0121 compared to the two-sided stringent statistical boundary of p < 0.0099 set for the interim analysis). The interim analysis from this fully-enrolled, ongoing trial is based on 415 of 652 patients followed for a minimum of 12 months.

Data pertaining to progression-free survival (PFS) in the 652 patients, a secondary endpoint of the trial, were immature at the data cutoff for the interim analysis. However, the descriptive summaries of PFS showed an early trend favoring BRUKINSA.

The trial also met a pre-specified secondary endpoint related to safety. Compared to ibrutinib, BRUKINSA demonstrated a statistically significant lower risk of atrial fibrillation or flutter, which is characterized by an irregular heartbeat that can lead to blood clots, stroke, heart failure and other heart-related complications. Overall, the safety profile of BRUKINSA was consistent with the previously seen profile in its clinical development program.

ALPINE is BeiGene’s second Phase 3 head-to-head trial comparing BRUKINSA to ibrutinib.

Jane Huang, M.D., Chief Medical Officer, Hematology of BeiGene said, "The interim results from this head-to-head trial demonstrated that, as a selective inhibitor designed to deliver sustained and continuous inhibition of BTK, BRUKINSA provides CLL patients with improvements in response and reduced rates of atrial fibrillation or flutter compared to ibrutinib. Data from this interim analysis, in addition to BRUKINSA’s comprehensive clinical program, provide important new information to support its benefit-risk profile."

BeiGene plans to consult with global regulatory authorities on next steps and present these data at an upcoming major medical conference. ORR per IRC will be further assessed at the planned final analysis, and patients will be followed for analyses on key secondary endpoints including PFS.

About Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

Chronic lymphocytic leukemia (CLL) is the most common form of leukemia in adults, with a global incidence of approximately 114,000 new cases in 2017.1,2 CLL affects white blood cells or lymphocytes in the bone marrow.1 Proliferation of cancer cells (leukemia) in the marrow result in reduced ability to fight infection and spread into the blood, which affects other parts of the body including the lymph nodes, liver and spleen.1,3 The BTK pathway is a known route that signals malignant B cells and contributes to the onset of CLL.4 Small lymphocytic lymphoma (SLL) is a non-Hodgkin’s lymphoma affecting the B-lymphocytes of the immune system, which shares many similarities to CLL but with cancer cells found mostly in lymph nodes.5

About ALPINE

ALPINE is a randomized, global Phase 3 trial (NCT03734016) comparing BRUKINSA against ibrutinib in previously treated patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

In the trial, a total of 652 patients were randomized into two arms with the first receiving BRUKINSA (160 mg orally twice daily) and the second receiving ibrutinib (420 mg orally once daily) until disease progression or unacceptable toxicity. The primary analysis of objective response rate (ORR), defined by pre-specified non-inferiority of BRUKINSA versus ibrutinib, was assessed by investigator and independent review committee (IRC) using the modified 2008 iwCLL guidelines with modification for treatment-related lymphocytosis for patients with CLL and per Lugano Classification for non-Hodgkin’s lymphoma for patients with SLL. There was hierarchical testing of non-inferiority followed by superiority in ORR as assessed by investigator and IRC. Key secondary endpoints include progression-free survival (PFS), duration of response, overall survival, and incidence of adverse events. The study is ongoing, with pre-specified endpoints of ORR and PFS to be evaluated at the planned final analysis expected in 2022.

About BRUKINSA

BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:

•For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;

•For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;

•For the treatment of chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;

•For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021); and

•For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021).

To-date, more than 30 marketing authorization applications in multiple indications have been submitted outside of the United States and China, covering the EU and more than 20 other countries.

*This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

**This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.

On April 28, 2021 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported that two trial in progress posters, on the Phase 1 INNATE clinical trial and the Phase 2 SELECT clinical trial, will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Annual Meeting being held June 4-8, 2021 (Press release, Jounce Therapeutics, APR 28, 2021, View Source [SID1234578658]).

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Poster Details:

Poster Title: Phase 1, First-in-Human trial of JTX-8064, an anti-LILRB2/ILT4 monoclonal antibody, as monotherapy and in combination with anti-PD-1 in adult patients with advanced solid tumors (INNATE)
Presenter: Kyriakos P. Papadopoulos, MD, South Texas Accelerated Research Therapeutics (START), San Antonio, TX
Session Title: Developmental Therapeutics – Immunotherapy
Abstract Number: TPS2672
Date and Time: Friday, June 4, 2021; 9:00am ET

Poster Title: Phase 2 Study of PD-1 Inhibitor JTX-4014 (Pimivalimab) Alone and in Combination with Vopratelimab, an ICOS Agonist, in Biomarker-selected Subjects with Metastatic NSCLC After One Prior Platinum-containing Regimen (SELECT)
Presenter: Oleh Kobziev, MD, Regional Center of Oncology, Kharkiv, 61070, Ukraine
Session Title: Lung Cancer – Non-Small Cell Metastatic
Abstract Number: TPS9137
Date and Time: Friday, June 4, 2021; 9:00am ET

About JTX-8064

JTX-8064 is a humanized IgG4 monoclonal antibody designed to specifically bind to Leukocyte Immunoglobulin Like Receptor B2 (LILRB2/ILT4) and block interactions with its ligands. JTX-8064 is the first tumor-associated macrophage candidate developed from Jounce’s Translational Science Platform. Preclinical data presented at the 2020 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Annual Meeting and the 2019 and 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meetings support the development of JTX-8064 as a novel immunotherapy to reprogram immune-suppressive macrophages and enhance anti-tumor immunity. A Phase 1 clinical trial named INNATE (NCT04669899), of JTX-8064 as a monotherapy and in combination with either Jounce’s internal anti-PD-1 inhibitor, pimivalimab (formerly JTX-4014), or an approved anti-PD-1 inhibitor, is currently enrolling patients with advanced solid tumors.

About Pimivalimab

Pimivalimab (formerly JTX-4014) is a well-characterized fully human IgG4 monoclonal antibody designed to block binding to PD-L1 and PD-L2. Pimivalimab demonstrated a 17% durable overall response rate in a Phase 1 trial of 18 heavily pre-treated PD-(L)1 inhibitor naïve patients, which excluded all tumor types for which PD-(L)1 inhibitors were approved. In this Phase 1 trial, pimivalimab was shown to have an acceptable safety profile. Pimivalimab is currently being assessed in the INNATE Phase 1 trial (NCT04669899) in combination with JTX-8064, a LILRB2 (ILT4) inhibitor. Pimivalimab is also being assessed in the SELECT Phase 2 clinical trial (NCT04549025) in combination with vopratelimab, a clinical-stage monoclonal antibody that binds to and activates ICOS, the Inducible T cell CO-Stimulator, a protein on the surface of certain T cells commonly found in many solid tumors. The SELECT trial compares vopratelimab plus pimivalimab to pimivalimab alone in immunotherapy naïve NSCLC patients who have been pre-selected with the TISvopra predictive biomarker, an 18 gene RNA tumor inflammation signature which predicted the emergence of ICOS hi CD4 T cells and clinical benefit in the ICONIC trial of vopratelimab alone and in combination with a PD-1 inhibitor.

About Vopratelimab

Vopratelimab is a clinical-stage monoclonal antibody that binds to and activates ICOS, the Inducible T cell CO-Stimulator, a protein on the surface of certain T cells commonly found in many solid tumors. Vopratelimab is currently being assessed in the SELECT Phase 2 clinical trial (NCT04549025) in combination with Jounce’s internal investigational PD-1 inhibitor, pimivalimab (formerly JTX-4014), compared to pimivalimab alone. The SELECT trial is currently enrolling approximately 75 immunotherapy naïve NSCLC patients who have been pre-selected with the TISvopra predictive biomarker, an 18 gene RNA tumor inflammation signature which predicted the emergence of ICOS hi CD4 T cells and clinical benefit in the ICONIC trial of vopratelimab alone and in combination with a PD-1 inhibitor. SELECT is powered to demonstrate the statistical superiority of the combination of vopratelimab plus pimivalimab compared to pimivalimab.

On April 28, 2021 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported that it was awarded by the International Society for Pharmaceutical Engineering (ISPE) for the 2021 Facility Of the Year Awards (FOYA) in two categories (Press release, Takeda, APR 28, 2021, View Source [SID1234578683]). Takeda’s new solid pharmaceutical packaging building in Hikari, Japan, was recognized with the 2021 "Process Intelligence and Innovation" category award. Additionally, the end-to-end high potent drug facility in Grange Castle, Ireland, was selected as "Facility Integration" category winner.

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"I am honored that Takeda receives two awards for the production and packaging of small molecule solid dosage form products," said Thomas Wozniewski, global manufacturing & supply officer of Takeda. "In 2018, the Los Angeles plasma facility received two FOYA awards, now, two of our sites in Japan and in Europe got awarded in the categories ‘Process intelligence and innovation’ as well as ‘Facility integration’. This illustrates that Takeda is constantly investing into state of the art facilities applying best in class process as well as digital standards. Both projects in Hikari and in Grange Castle also demonstrate that significant technology improvements for small molecule modalities are still achievable, both in a cost as well as in a time efficient manner."

Takeda’s Hikari plant is located in the south of Japan in the Yamaguchi prefecture. One of Takeda`s largest plants, it features advanced production systems for active pharmaceutical ingredients (API), drug formulation, biological products, and others offering a stable supply of high-quality pharmaceutical products throughout the world. The project at the Hikari site is a four-story building designed to elevate pharmaceutical packaging operations to a new industrial standard. The facility features highly automated end-to-end packaging equipment, including "end of line" case packers, automated guided vehicles (AGVs) and robots to feed the automated storage and retrieval system (ASRS). Additionally, the site developed and introduced an automatic line clearance system (ALC) with 360° cameras and laser sensors utilizing artificial intelligence (AI) to help significantly increase efficiency in the pharmaceutical packaging process. Takeda’s Hikari plant exemplifies how novel application of commercially available and custom developed process manufacturing tools leads to superior results and advanced process understanding.

The Grange Castle site in Ireland is located in the Dublin area, and it includes three manufacturing facilities. The production facility which now got awarded by the ISPE is a standalone, high containment, cutting-edge production facility dedicated to manufacturing Takeda’s treatment for multiple myeloma. The application of good design practices and superior conceptual planning led to the excellent integration of facility and process. The innovative design as an ‘all-in-one’ facility incorporates the entire end-to-end production process from active pharmaceutical ingredients to drug product and packaging under one roof. This significantly simplifies the supply chain for one of Takeda´s global oncology products to ensure unconstrained availability to patients worldwide.

"The design teams have implemented best-in-class digital technologies to guarantee the facilities use the latest developments in paperless automation, robotics and augmented reality. This strategic use of digital and automated systems has led to a state-of-the-art facility design that positions Takeda as a frontrunner in our industry," added Gunter Baumgartner, head of Global Engineering at Takeda.

An official ceremony of all winning projects and awards is planned at this year’s ISPE Annual Meeting & Expo in Boston, MA, in November.

About the ISPE Facility of the Year Awards Program
Established in 2004, the Facility of the Year Awards (FOYA) recognizes state-of-the-art projects utilizing new, innovative technologies to improve the quality of products, reduce the cost of producing high-quality medicines, and demonstrate advances in project delivery. The FOYA program provides a platform for the pharmaceutical science and manufacturing industry to showcase its accomplishments in facility design, construction, and operations, while sharing the development of new applications of technology and cutting-edge approaches. For more information, visit View Source

On April 28, 2021 Shanghai BDgene Technology Co., Ltd. ("BDgene"), a leading global gene therapy development company with original delivery technology, reported the company has successfully completed a round A financing of 60 million yuan (Press release, BDgene Therapeutics, APR 28, 2021, View Source [SID1234641044]). This financing was led by Tsinghua Holdings Capital , Ennovation Ventures, Longmen Capital, ETP Ventures, Innovation Angel Fund and other institutions followed in the investment. Probe Capital served as the exclusive financial advisor. The funds will be used for preclinical and clinical development of multiple products, laboratory and team building etc. BDgene had previously received a strategic investment from Serve Accurate Faithful Evaluation in June 2019 , and a pre-A round of investment from Keytone Ventures in March 2020.

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According to the data, BDgene was established in 2018 and is an innovative company focusing on the development of in vivo gene editing and in vitro transgene therapy therapies. Since its establishment, the company has attracted much attention from investment institutions and has received investment from many institutions. Why is it sought after by capital? What is the core technology? What are the competitive advantages of leading products?

Gene therapy has become the third revolutionary therapy, but delivery technology is still the most difficult point

For difficult to treat genetic, infectious, and degenerative diseases, gene therapy has the characteristic of "can be cured once". In recent years, it has become the third revolutionary therapy after chemical drugs and antibody drugs.

From the first appearance of the third-generation gene editing technology CRISPR in 2012, to the first in-vivo clinical trials of CRISPR in March 2020, to October 2020 after CRISPR won the Nobel Prize in Chemistry and other major events, the gene editing technology gradually matured , But delivery technology has always been the biggest obstacle in the field of gene editing therapy.

In January 2020, Nobel Prize winner Jennifer Doudna wrote an article in "Nature" magazine that "Delivery remains perhaps the biggest bottleneck to somatic-cell genome editing".

In the past, gene therapy delivery technologies that have been clinically verified by humans in the world mainly include three platforms: Adeno-associated virus, lentivirus vector, and lipid nanoparticles. These platforms have certain drawbacks for gene editing delivery. The ideal gene editing delivery tool needs to have both instantaneous and high-efficiency characteristics to ensure the safety and effectiveness of the treatment.

Create China’s original delivery technology to help gene therapy achieve a leap from "1 to 100"

Dr. Cai Yujia, the founder of BDgene, studied under the professors of Karolinska Institutet in Sweden, the Secretary-General of the Nobel Committee, Professor Thomas Perlmann, and Professor Jacob G. Mikkelsen, Aarhus University, Denmark, and he received a doctorate degree in gene therapy. He has mastered the core technology of gene therapy vectors and has rich experience in the production design and transformation of gene therapy virus vectors. After returning to China, he served as a researcher at the Institute of Systems Biomedicine, Shanghai Jiaotong University. In recent years, BDgene has successfully developed VLP-mRNA, a gene therapy delivery platform that has been clinically validated by humans, to deliver CRISPR/Cas9 mRNA to achieve safe and controllable gene editing therapy in vivo.

BDgene successfully completed the world’s second clinical study of in vivo CRISPR gene editing therapy in early November 2020 (in March 2020, Editas’ Zhang Feng team completed the world’s first case), which is also the world’s first CRISPR antiviral therapy Clinical research. This delivery technology delivers Cas9 mRNA through the lentiviral shell, which combines the high efficiency of viral vectors in infecting cells (in vitro infection rate of 99.8%, in vivo infection rate> 50%) and transient mRNA expression (degraded within 72 hours). Therapeutic effects have been achieved on animal models of different diseases, with technical advantages such as transient expression in vivo, no long-term safety risks associated with AAV expression of CRISPR, no obvious off-target, and low immunogenicity. Currently, with the permission of the Ethics Committee of the Eye, Ear, Nose and Throat Hospital Affiliated to Fudan University, the team of Professor Hong Jiaxu is presiding over the initial safe and controllable clinical application research based on this technology. In the future, this technology will be extended to the treatment of other viral infections, eye diseases and neurological diseases.

Mr. Zhang Yang, Chairman of Tsinghua Holdings Capital said, "The gene therapy industry has the characteristics of huge market space and extremely high technical threshold. In the past, gene editing technology has gradually broken through, but delivery technology is still the biggest bottleneck. In the past 10 years, Dr. Cai Yujia has been devoted to the development of gene therapy Delivery technology, It has successively successfully developed multiple original delivery technology platforms such as virion delivery CRISPR/Cas9 mRNA technology and next-generation lentivirus delivery technology. BDgene’s original viral keratitis treatment product has perfect preclinical animal experiment data. It is currently conducting non-registered human clinical trials in top domestic eye hospitals. BDgene also deployed gene editing technology to treat hereditary, infectious and degenerative diseases, viral vectors to treat blood diseases, and mRNA vaccines. "

Speaking of this Series A financing being exclusively led by Tsinghua Holdings Capital, Mr. Zhang Yang said, "Thank you very much for the trust of BDgene founder Professor Cai Yujia and old shareholders. In the future, he will support the development of the company in the long-term and jointly promote BDgene’s original delivery technology and The product helps gene therapy to achieve a leap from "1 to 100", so that more patients who are refractory to and incurable can really benefit in the future."