Researchersarepoised to makeunprecedentedbreakthroughsinhuman health thanks toadvancesin biomedical and computational sciencesthathave drivencritical tools and technologiessuch as genetic engineering,synthetic biology, andartificial intelligence.

Thats the messageDr. VictorDzau, president oftheU.S.National Academy ofMedicine, delivered to Medicine by Designs fifthannual symposium on Dec. 7 and 8.

Thevirtual event, whichattracted more than 500 registrants from across North America, focused on the theme of better science throughconvergence theintegration of approaches from engineering, science, medicine and other fields to expand knowledge and spark innovation.

I think for younger people, there is really not a more exciting time, in my opinion, to do research than now, because we can really see that some of the initial concepts that people have about health and medicinecan be realizedand truly transform the way we do health andmedicine.

In his talk, Dzau focused on the National Academy of MedicinesHealthy Longevity Global Challenge,an international competition that aims to catalyze transformative ideas and breakthroughs that will extend human healthand lifespan.

That program is one of the inspirations for Medicine by DesignsGrand Questions Program, which seeks to fund bold research that promises dramatically better health outcomes by changing the future of regenerative medicine.

Through our Grand Questions Program, we are thinking about what comes next and how to overcome fundamental problems in regenerative medicine,saidMichael Sefton, executive director of Medicine by Design andUniversity Professorin the department of chemical engineering and applied chemistry and theInstitute of Biomedical Engineeringat the University of Toronto.

We have a broad definition of regenerativemedicine, andpreventing degeneration can be as important as the next cell therapy.

Sefton pointed out that the symposium theme of better science through convergencefocusedon a key aspect of Medicine by Design:That we combine campus and hospital investigators, transformative science and translational elements, junior and senior investigators, and local and international collaborators, to address fundamental problems in regenerative medicine.

Thesymposium also featured a talk byRobert Langer, David H. KochInstitute Professorin the department of chemical engineeringat the Massachusetts Institute of Technology. The most highly cited engineer in history, he spoke about lessons helearnedfromhisscientific and business successes and how he decidedto be his own champion after facing criticism for his novel ideas early in his career.

If you try to do things whether its convergence, or things that a lot of people disagree with you have tohang in there, Langer said.Having good intellectual property has been key toraising the funds to do these things, and medicine is an incredibly expensive thing.

And finally, you really need teams that are super driven, and I think these startup companies have been a wonderful way to do this.

The symposium was organized around four sessions: translation, inflammation, biomaterials andimmunoengineering.Invited speakers from across North AmericaincludedKim Warren(AVROBIO),Kenneth Walsh(University of Virginia),Sarah Heilshorn(Stanford University)andMegan Levings(University of British Columbia).

All speakers fromU of T and its partnerhospitals were lead investigators on Medicine by Designs multi-disciplinary, multi-institution team projects. They included:John Dick,Clinton RobbinsandShaf Keshavjee(University Health Network (UHN));Molly Shoichet(department of chemical engineering and applied chemistry and Institute of Biomedical Engineering);Juan CarlosZiga-Pflcker(Sunnybrook Health Sciences Centre);andAndras Nagy(Sinai Health System).

Ted Sargent, vice-president of research and innovation, and strategic initiatives,and a University Professor in the Edward S. Rogers Sr. department of electrical and computer engineering,opened the symposium by congratulatingMedicine by Design on its successful mid-term review, which was conducted in early 2020 by a panel of international experts and theCanada First Research Excellence Fund(CFREF), which funds Medicine by Design.

Medicine by Design has amplified existing areas ofexcellenceatU of Tandour partner hospitals (Toronto Academic Health Sciences Network),and pushed the boundaries of regenerative medicine to tackle cell-based therapies, strategies for endogenous repair and the use of a stem cell lens to target the triggers of disease,Sargent said. In fact, Medicine by Design is such a compelling collaborative, cross-disciplinary initiative that itis a template fora new class of initiatives at the University ofToronto theInstitutional Strategic Initiativesportfolio whosepurpose is to mobilize ambitious,groundbreaking, collaborative, multi-institutional research networks that tackleimportantresearch problems, buildmajorexternal partnershipsboth with industry and emerging companies as well as with global academic peers;and foster societal impact.

They support the pursuit of grand challenges and bold ideas across disciplinary boundaries,further elevate U of Ts profile in high priority research areas of strategic importance,and enable us to realize transformational impacts on issues of major societal import.

The symposium also offered an opportunity for almost 45trainees to present their research during a poster session.KerstinKaufmann, a post-doctoral fellow in the laboratory ofJohn Dick(Princess Margaret Cancer Centre,UHN), won first place.JonathanLabriola, apost-doctoral fellowinSachdev Sidhuslab(Donnelly Centre for Cellular and Biomolecular Research, U of T), placed second, whileSabihaHacibekiroglu, a post-doctoral fellow in the lab ofAndras Nagy(Lunenfeld-Tanenbaum Research Institute, UHN)placed third.The awards were sponsored by STEMCELL Technologies.

YasamanAghazadeh,a post-doctoral fellow in the labsofCristina Nostro(McEwen Stem Cell Institute, UHN)andSara Nunes Vasconcelos(Toronto General Hospital Research Institute,UHN),won theCCRMTranslation Awardfor the poster with the greatest translational potential.AndAi Tian, a post-doctoral fellow fromJulien Muffatslab (The Hospital for Sick Children), won thePeoples Choice Award, a new award this year that wasdetermined byvotingby symposium attendeesand sponsored byBlueRockTherapeutics.

Funded by a $114-million grant from CFREF, Medicine by Design brings together more than 145principal investigators at the University of Toronto and its affiliated hospitals to work at the convergence of engineering,medicineand science. It builds on decades of made-in-Canada excellence in regenerative medicine dating back to the discovery of stem cells in the early 1960s by Toronto researchers James Till andErnest McCulloch.

Regenerative medicine uses stem cells to replace diseased tissues and organs, creating therapies in which cells are the biological product. Regenerative medicine can also mean triggering stem cells that are already present in the human body to repair damaged tissues or to modulate immune responses. Increasingly, regenerative medicine researchers are using a stem cell lens to identify critical interactions or defects that prepare the ground for disease, paving the way for new approaches to preventing disease before it starts.

(Photo of Robert Langer by Jason Alden)

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Medicine by Design symposium highlights importance of convergence in regenerative medicine and human health - News@UofT

Dec. 28, 2020 13:04 UTC

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced its New Drug Submission for TRIKAFTA, Vertexs investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review process will enable access for patients as early as possible, said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

With Priority Review, the conventional review timeline of 300 days is reduced to 180 days. The expected approval target by Health Canada is in the first half of 2021.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple approved medicines that treat cystic fibrosis (CF) a rare, life- threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertexs history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic in this press release, including expectations for patient access to our medicine, and statements regarding the anticipated timing of the expected approval target by Health Canada. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the New Drug Submission to Health Canada may not be approved in the expected timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at http://www.sec.gov and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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Vertex Announces New Drug Submission for Investigational Triple Combination Medicine for the Treatment of Cystic Fibrosis Has Been Accepted for...

The treatment landscape of lymphoid malignancies space is rich with available immunotherapy agents, said Joshua Brody, MD. However, the field continues to push toward improving response rates by refining established modalities, such as CAR T-cell therapy and checkpoint inhibitors, as well as introducing novel modalities.

During the5th AnnualInternational Congress on Immunotherapies in Cancer, a program run by Physicians Education Resource (PER), Brody, an assistant professor of medicine, hematology, and medical oncology and director of the Lymphoma Immunotherapy Program at The Tisch Cancer Institute at Mount Sinai, gave a presentation on established and upcoming immunotherapies in the lymphoid malignancy pipeline.

We are somewhat lucky, and maybe a bit spoiled in lymphoid malignancies, such as lymphomas and some B-cell malignancies, to have [seen] a huge amount of progress [with immunotherapy], said Brody.

However, lymphoid malignancies are highly heterogeneous. As such, not all patients are able to derive responses from the same therapy, Brody explained.

CAR T-cell therapy represents a significant advance in the treatment paradigms of several hematologic malignancies, including lymphoid malignancies, said Brody.

The results [of CAR T-cell therapy treatment] for patients with aggressive B-cell lymphomas have been amazing, Brody said. [CAR T-cell therapy] has shown higher response rates [in this space] than any other immunotherapy for any type of cancer; response rates are above 80%. Many of those [responses] are long lasting, complete remissions [CRs].

Notably, it is thought that patients who remain in CR for at least 6 to 12 months after CAR T-cell therapy infusion are likely cured of their lymphoma, Brody said. For example, it is likely that around 35% to 40% of patients with diffuse large B-cell lymphoma (DLBCL) who receive CAR T-cell therapy are effectively cured.

Third-line [DLBCL], the most common type of lymphoma, [was thought to be] an incurable disease setting, said Brody. We [thought we could] cure people in the first- or second-line setting, but not beyond. Im not sure how we define a miracle, but it seems miraculous to me to change the incurable to partly curable.

Now, ongoing research efforts are attempting to refine CAR T-cell therapies to enhance responses and expand the utility of the modality to other hematologic malignancies beyond DLBCL, acute leukemia, and mantle cell lymphoma, explained Brody. For example, developing armored CAR T cells and fourth-generation CAR T cellsT cellsredirected for antigenunrestricted cytokineinitiated killingmay allow for improved delivery of cytotoxic payloads to liquid as well as solid tumors.

Additionally, novel CAR T-cell therapies may overcome some of the ongoing challenges observed with current, autologous products, such as toxicity, accessibility, and feasibility. For example, utilizing an allogeneic approach to CAR T-cell therapy may allow for an off-the-shelf option that could alleviate the risk of cytokine release syndrome and neurotoxicity.

Beyond CAR T-cell therapy, bispecific monoclonal antibodies, such as the investigational CD20/CD3-directed REGN1979 agent, could potentially be used in patients who progress after CAR T-cell therapy. Findings from a phase 1 trial (NCT02290951) demonstrated high objective response rates and CR rates among patients with heavily pretreated, relapsed/refractory NHL who were treated with REGN1979.1

Although the toxicity challenges of CAR T-cell therapies are likely not going to be solved with bispecific antibodies, the novel agents may offer an alternative option that can decrease the risk of adverse effects.

At least with [bispecific antibodies], we can tweak and optimize [dosing] a bit, explained Brody. CAR T-cell therapy is usually a one-shot therapy, [whereas] bispecifics can be given in low, medium, or high doses. We can up-titrate the therapy to try to minimize or avoid some of these toxicities.

Additionally, bispecific antibodies are potentially able to avoid antigen escape, which is a common concern with CD19/CD20-directed CAR T-cell therapies, Brody explained.

PD-1/PD-L1 inhibitors have transformed the treatment landscape in melanoma, lung cancer, renal cell carcinoma, and bladder cancer. However, Hodgkin lymphoma is the most responsive disease to antiPD-1 agents because chromosomal amplifications or translocations are hardwired to overexpress PD-1, Brody said.

As such, responses with checkpoint inhibitors, such as nivolumab (Opdivo), are seen in around 70% of patients with Hodgkin lymphoma who progress following autologous stem cell transplant.2

The introduction of checkpoint inhibitors to this space has been particularly encouraging for younger patients who fail curative-intent chemotherapy and who, historically, had dismal prognoses.

Currently, ongoing efforts are attempting to bring PD-1 inhibitors to the second-line setting and, potentially, up-front settings in combination with chemotherapy.

Although it is true that hot tumors are more likely to respond to immunotherapy compared with cold tumors, it is not the only factor that contributes to whether a tumor will respond. As such, alternative methods of improving immune response in patients with lymphoid malignancies are being evaluated.

For example, one investigational method is to utilize radiotherapy or other means to mobilize dendritic cells loaded with tumor antigens to antigen-presenting cells. Ultimately, this compound could serve as a therapeutic anti-cancer vaccine to deliver the antigens, as well as costimulatory signals, to the cancer cells.

As such, an ongoing trial (NCT03789097) led by Brody is currently testing an in situ vaccine combining radiation therapy, the immune cell growth factor Flt3L/CDX-301, the immune-cell activating factor Poly-ICLC, and pembrolizumab (Keytruda).3 Initial findings from the study have yielded encouraging results, according to Brody.

In situ vaccines such as this may be able to cross prime T cells and increase the effectiveness of immunotherapy for patients, Brody concluded.

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Immunotherapy Continues on Positive Trajectory in Lymphoid Malignancies - OncLive

According to Penn State College of Medicine researchers, the absence of a protein that works to prevent tumour formation may explain why some patients are resistant to common cancer therapy.

They said that testing cancers for the presence of this protein may help clinicians identify patients who may be resistant to or relapse when treated with the therapy.

Epidermal growth factor receptor (EGFR) is a protein that plays a role in cell division and survival signalling and is active in skin, bladder, oesophagal, lung, liver, pancreatic, colon, and head and neck cancers.

Patients with high amounts of this protein in their tumours tend to have a poor prognosis. EGFR-targeting therapies are used in clinical practice and are often initially effective in many patients. However, some patients are resistant to the therapy and many who were initially responsive to treatment relapse within a year.

Douglas Stairs, assistant professor of pathology and laboratory medicine and pharmacology, investigated why these patients may be resistant to EGFR therapies.

He said mutations in the gene that contains the instructions for building EGFR or other genetic and cellular factors account for about 70 per cent of resistance causes.

There are still some reasons for resistance that are alluding scientists, said Stairs, a Penn State Cancer Institute researcher. Our previous work showed that too much EGFR and reduced amounts of a protein called p120 catenin (p120ctn) can cause cancer to develop. We hypothesized that reduced amounts of p120ctn might also cause resistance to EGFR therapies.

In healthy cells, p120ctn strengthens cell-to-cell contact by cooperating with other proteins to strengthen connections between epithelial cells, which serve as the barrier between the bodys external and internal surfaces.

According to Stairs, scientists know that the cancer cells often have reduced amounts of p120ctn, but are unsure why.

To test their hypothesis, Stairs and colleagues cultured genetically-engineered esophageal cancer cells -- one set with normal amounts of EGFR and p120ctn, one set with higher amounts of EGFR, one set with lower amounts of p120ctn and another set with high amounts of EGFR and low amounts of p120ctn. They then treated each cell line with a series of EGFR-targeting therapies.

Cells with high amounts of EGFR died when treated with the therapies, while those with normal amounts of EGFR were not affected by the therapies.

The cell lines that had high amounts of EGFR and reduced amounts of p120ctn were resistant -- demonstrating that loss of p120ctn is a critical component to the cells resistance to EGFR-targeted therapies.

The results were published in PLOS ONE.

Stairs said that while these results are promising, his lab will continue to explore the role of p120ctn loss in EGFR therapy resistance by testing the effect in cancer cells sampled from patients with colon, lung, oral or other cancers.

They will also explore whether the cells with increased EGFR and decreased p120ctn are resistant to other EGFR therapies approved by the U.S. Food and Drug Administration.

We also need to investigate further how the loss of p120ctn causes this resistance, Stairs said. For now, we know that if patients who have high levels of EGFR in their samples were also tested for their levels of p120ctn, it may provide a clue to clinicians as to which patients are at risk for resistance to EGFR-targeting therapies or relapse.

(This story has been published from a wire agency feed without modifications to the text.)

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Resistance to certain cancer therapies may be caused by loss of anti-tumour protein : Study - Hindustan Times

Multiple chimeric antigen receptor (CAR) T-cell therapies for the treatment of lymphomas and multiple myeloma have moved forward in the regulatory process, with 1 new FDA approval in 2020 and others anticipated in the near future.

In July, brexucabtagene autoleucel (Tecartus; KTEX19) received accelerated approval for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) based on the results of the phase 2 ZUMA-2 trial (NCT02601313), bringing the treatment landscape of this hematologic malignancy into a new era.1

This approval is only the very beginning, and we are walking into a sophisticated CAR T-cell therapy era with many constructs being designed with [different mechanisms of action], Michael Wang, MD, said in an interview with Targeted Therapies in Oncology (TTO).

Additional actions by the FDA this year included granting priority review designations to lisocabtagene maraleucel (liso-cel) for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma, after at least 2 prior therapies,2 as well as to idecabtagene vicleucel (ide-cel; bb2121)as treatment of adult patients with multiple myeloma who have received at least 3 prior therapies, including an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibody.3

The approval of brexucabtagene autoleucel, an antiCD19 CAR T-cell product, in MCL was based on objective response rate (ORR) data from patients treated on a single-arm trial who had previously received anthracycline- or bendamustine-containing chemotherapy, an anti-CD20 antibody, and a Bruton tyrosine kinase inhibitor (n = 74).2,4 Eligible patients received leukapheresis and optional bridging therapy, followed by conditioning chemotherapy and a single infusion of brexucabtagene autoleucel 2 106CAR T cells/kg.

The results of ZUMA-2 were published in the New England Journal of Medicine in April and demonstrated a 93% (95% CI, 84%-98%) ORR in 60 response-evaluable patients, 67% (95% CI, 53%-78%) of whom had a complete response (CR). ORRs were consistent across key patient subgroups. Two patients (3%) each had stable and progressive disease.

Progression-free and overall survival (OS) rates at 12 months were 61% and 83%, respectively, and 57% of patients remained in remission at the 12.3-month median follow-up.4 Cytokine release syndrome (CRS) was the most concerning adverse event, occurring in 91% of patients; grade 3 or higher CRS occurred in 15%.

Notably, the patient cohort comprised patients with a median of 3 prior lines of therapy (range, 1-5) and more than half (56%) were considered to have intermediateor high-risk features by the simplified Mantle Cell Lymphoma International Prognostic Index at baseline.

Before CAR T-cell therapy, we did not have any effective means [of getting patients with high-risk MCL into remission]. We used allogeneic transplantation [and] were able to put some of the patients into a long-term remission, but at a heavy price of mortality, said Wang, a professor in the Department of Lymphoma & Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center in Houston. Overall, this brings hope to the high-risk patient population. It looks as though fewer patients are relapsing.

Lisocabtagene Maraleucel In February, the FDA granted liso-cel a priority review designation, an action supported by the safety and efficacy findings of the phase 1 TRANSCEND-NHL-001 trial (NCT02631044).2

Histologic subtypes eligible for treatment included diffuse large B-cell lymphoma (DLBCL); high-grade double- or triple-hit B-cell lymphoma; transformed DLBCL from indolent lymphoma; primary mediastinal B-cell lymphoma; and grade 3B follicular lymphoma. Patients were administered 2 sequential infusions of CD8+ and CD4+ CAR T cells following optional bridging therapy and lymphodepleting chemotherapy and were assigned to 1 of 3 target dose levels: 50 106 (1 or 2 doses), 100 106 , or 150 106 CAR-positive T cells. Investigators determined that the recommended target dose was 100 106 CAR-positive T cells.

In the 256 patients who received at least 1 dose of liso-cel and were included in the efficacy-evaluable group, the ORR was 73% (95% CI, 67%-78%), with 53% (95% CI, 47%-59%) achieving a CR. Investigators observed all-grade CRS (42%) and neurological events (30%), but most cases were grade 1 or 2 in severity.

Due to relatively low rates of CRS and neurological events, the administration of liso-cel has been explored in both the inpatient and outpatient settings. One that included a cohort of patients treated in the outpatient setting with proper monitoring versus the traditional inpatient setting demonstrated consistent safety.6

Based on these results, the indication is that you can deliver [liso-cel] in the outpatient setting and the outcomes are good compared with those treated in the inpatient setting, explained study author Carlos R. Bachier, MD, the director of cellular research at Sarah Cannon in Nashville, Tennessee, in an interview with TTO. Aside from that, they also showed that liso-cel could be safely administered outside of university programs and in more community-based programs, most of them being aligned [with] or part of stem cell and bone marrow transplant programs.

The target action date for a decision on the biologics license application (BLA) for liso-cel was extended twice in 2020 and remains under review. In May, the FDA moved the Prescription Drug User Fee Act (PDUFA) goal date out 3 months from its original August deadline.2,7 Bristol Myers Squibb, the company responsible for developing the product, submitted additional information to the agency following the initial BLA submission, which resulted in more review time. Once again, the target action date was pushed in November, this time due to incomplete manufacturing facility inspections resulting from ongoing travel restrictions due to COVID-19. The FDA provided no new action date.8

For patients with multiple myeloma, the B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy idecel is currently under review for approval in patients who have received at least 3 prior therapiesincluding an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibodybased on results of the phase 2 KarMMa trial (NCT03361748).9

Updated trial results were presented at the American Society of Clinical Oncology 2020 Virtual Scientific Program, and showed that both the primary and key secondary end points of ORR and CR rate were 75% and 33%, respectively. The median duration of response was 10.7 months, and the median progression-free survival was 8.8 months in all patients receiving ide-cel. Corresponding medians were 19.0 and 20.2 months among those achieving a CR or stringent CR. The median OS was 19.4 months in all treated patients.

The 128 patients treated received 1 of 3 target dose levels: 150, 300, or 450 106 CAR-positive T cells. The investigators noted that the highest efficacy outcomes were seen in patients in the 450 106 CAR-positive T-cell group, with an ORR of 82% and a 39% CR rate.

The incidence of CRS was 84% across the treatment cohort and increased with higher target doses. Overall, less than 6% of patients have grade 3 or higher CRS and only 1 patient in the highest target dose cohort had a grade 5 event. Neurological toxicity was low across target doses, with no grade 4 or 5 events reported.

At baseline, the majority of patients (51%) had high tumor burden, 39% had extramedullary disease, and 35% had high-risk cytogenetics including deletion 17p or translocations in t(4;14) or t(14;16).

In May, the FDA issued a refusal letter regarding the BLA for ide-cel because the Chemistry, Manufacturing, and Control (CMC) module required more information before they could complete the review.10 In September, the resubmitted application received a priority review and the agency assigned a PDUFA action date of March 27, 2021.11

If approved, ide-cel would be the first CAR T-cell therapy available for the treatment of patients with multiple myeloma.

References:

1. FDA approves brexucabtagene autoleucel for relapsed or refractory mantle cell lymphoma. FDA. Updated July 27, 2020. Accessed November 18, 2020. https://bit. ly/3pEDQV5

2. US Food and Drug Administration (FDA) accepts for priority review Bristol-Myers Squibbs biologics license application (BLA) for lisocabtagene maraleucel (liso-cel) for adult patients with relapsed or refractory large B-cell lymphoma. Press release. Bristol Myers Squibb. February 13, 2020. Accessed November 18, 2020. https:// bit.ly/37ruQbs

3. US Food and Drug Administration (FDA) accepts for priority review Bristol Myers Squibb and bluebird bio application for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121). Press release. Bristol Myers Squibb. September 22, 2020. Accessed November 18, 2020. https://bit.ly/3kDhakH

4. Wang M, Munoz J, Goy A, et al. KTE-X19 CAR T-cell therapy in relapsed or refractory mantle-cell lymphoma. N Engl J Med. 2020;382(14):1331-1342. doi:10.1056/ NEJMoa1914347

5. Abramson JS, Palomba ML, Gordon LI, et al. Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study. Lancet. 2020;396(10254):839-852. doi:10.1016/ S0140-6736(20)31366-0

6. Bachier CR, Palomba ML, Abramson JA, et al. Outpatient treatment with lisocabtagene maraleucel (liso-cel) in 3 ongoing clinical studies in relapsed/refractory (R/R) large B cell non-Hodgkin lymphoma (NHL), including second-line transplant noneligible (TNE) patients: Transcend NHL 001, Outreach, and PILOT. Paper presented at: 2020 Transplantation & Cellular Therapy Meetings; February 19-23, 2020; Orlando, FL. Abstract 29. Accessed November 18, 2020. bit.ly/37I7DC9

7. Bristol Myers Squibb provides update on biologics license application (BLA) for lisocabtagene maraleucel (liso-cel). Press release. Bristol Myers Squibb. May 6, 2020. Accessed November 18, 2020.https://bit.ly/2YFWAs8

8. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). News release. Business Wire. November 16, 2020. Accessed November 18, 2020. https://bwnews.pr/3pKQMZI

9. Bristol Myers Squibb and bluebird bio announce submission of biologics license application (BLA) for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121) to FDA. Press release. Bristol Myers Squibb. March 31, 2020. Accessed November 18, 2020. https://bit.ly/2JwKbxO

10. Bristol Myers Squibb and bluebird bio provide regulatory update on idecabtagene vicleucel (ide-cel, bb2121) for the treatment of patients with multiple myeloma. News release. Business Wire. May 13, 2020.Accessed November 18, 2020. https:// bwnews.pr/3cpgJa1

11. US Food and Drug Administration (FDA) accepts for priority review Bristol Myers Squibb and bluebird bio application for anti-BCMA CAR T cell therapy idecabtagene vicleucel (ide-cel, bb2121). Press release. Bristol Myers Squibb. September 22, 2020. Accessed November 18, 2020. https://bit.ly/3kDhakH

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CAR T-Cell Therapies Are Set to Expand Into More Hematologic Malignancy Indications - Targeted Oncology

A team of researchers at the University School of Medicine recently published an article in the Journal of Biological Chemistry about their study of HIV fusion to cell membranes using cryo-electron microscopy. The study determined the initial steps of HIV infection in cells information that can be applied to understanding the infection process of other viruses including SARS-CoV-2, the virus that leads to COVID-19 infection.

Previous studies revealed that the HIV infection process includes the fusion of viral membranes to cell membranes. This is achieved by binding viral surface proteins, or proteins located on the surface of a virus, to receptor proteins, located on cell membranes.

According to Amanda Ward, medical student in the Tamm Lab, the binding of the membranes of viruses to cells is a process that is largely the same even when considering a diverse group of viruses.

Specifically for HIV, spike proteins found on the surface of the virus for binding are similar to the spike proteins found on influenza, Ebola and SARS-CoV-2 viruses, said Lukas Tamm, director of the University's Center for Membrane and Cell Physiology.

According to Ward, while the exact proteins used in the fusion of HIV versus viruses such as influenza vary, the steps for fusion are similar. Therefore, having a technique that can observe the slight variations in the initial steps of viral infection can help attain an understanding of the overall initial infection process and enable further research.

To identify the specific intermediate steps of HIV fusion and understand how they are inhibited, researchers utilized techniques known as cryo-electron tomography and cryo-electron microscopy.

Cryo-electron tomography allows researchers to get snapshots as an enveloped virus undergoes membrane fusion [which depict the] first steps of how [HIV] gets into a cell and can establish infection, Ward said.

Cryo-electron microscopy is a specific form of electron microscopy, Ward said.

Electron microscopy works the same way as light microscopy but instead of shining a light at the sample and observing its interaction with the light, it introduces electrons to the sample and is performed in a vacuum so that as soon as an electron contacts anything solid it shatters, Ward said.

The cryo-electron microscopy technique won the 2017 Nobel Prize in Chemistry and is useful for sample preparation and data processing. In the Universitys study, this technique was used to collect and freeze very thin samples of HIV and blebs, or mini cells so rapidly that the water within the sample does not form a crystalline structure as seen in ice, said Ganser-Pornillos, assistant professor of molecular physiology and biological physics at the School of Medicine.

The benefit behind using blebs is that [whole] cells are too large to be frozen that quickly, [so] we pinched off small pieces of membranes from those cells [to form blebs] that are small enough ... to be subjected to rapid freezing, Tamm said.

With Cryo-electron microscopy you can see exactly what is going on when the virus interacts with the cell without any stain or artifacts to disrupt the image, Ganser-Pornillos said. This contrasts traditional electron microscopy, which requires staining samples and therefore disrupts the structure of the samples themselves.

According to Ward, the studys findings revealed the normal process of HIV fusion and how it is disrupted by the newly-discovered proteins Serinc3 and Serinc5. These proteins are able to block HIV infection and may provide a new avenue for designing therapeutics that could inhibit HIV fusion to cells.

Additionally, experimental results suggest that Serinc3 and Serinc5 inhibit the overall fusion of HIV particles without targeting a specific step in the fusion process.

Because previous research indicates that energy is required for each step of the fusion pathway, the studys paper suggests it is plausible that Serinc3 and Serinc5 increase the energy needed to advance through different intermediate fusion steps. This additional energy requirement would inhibit HIV infection.

Ward said that in response to this studys findings, she would like to conduct further research on how the viral membrane itself could be important to the fusion process.

The end goal here is to have a detailed mechanism of how viruses infect cells, so that means understanding how every molecule rearranges at every step of this process ... This is one step on the way to that, Ward said.

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U.Va. research discovers the initial phases of HIV entry into cells - University of Virginia The Cavalier Daily

Dr. Lars Kneller, family physicain and medical director of Beacon Health, La Porte, and Dr. Jennifer A. Alderink, DO, family physician and co-site director of Beacon Medical Group, La Porte, pose with their vaccine certificates after they received the Pfizer-BioNTechs COVID-19 mRNA vaccine at Franciscan Health Michigan City on Dec. 19. (Photo provided)

Story by WNLPs Bob Wellinski

Its not often people are excited about getting a vaccine to the point they couldnt sleep the night before. But for Dr. Jennifer A. Alderink, DO, and other healthcare workers, this isnt just any vaccine. Its a vaccine of hope.

Alderink, a family medicine physician and co-site director of Beacon Medical Group, La Porte, was one of several hundred healthcare workers to receive the first round of Pfizer-BioNTechs COVID-19 mRNA vaccine at Franciscan Health Michigan City. Administration of the vaccines started on Dec. 18.

It went great. I was really excited to know it was starting and were finally taking a step forward I couldnt sleep the night before, said Alderink.

With the vaccine comes hope of life getting back to normal.

We definitely have more hope than we did three months ago. Then we really didnt know a date on vaccinations, and COVID cases were surging, said Alderink.

She described development of the COVID-19 vaccine as remarkable, noting that everyone involved in creating and approving the vaccine managed to shrink a 3-to-4-year process into nine months, with safety and efficacy a priority.

Its not something weve experienced before, said Alderink. Innovation often comes out of necessity, and it was an emergency. Shrinking that time down was out of necessity. A lot of things had to fall into place, a lot of funding had to fall in place, for this to come about quickly.

But the biggest component of the equation was the technology used to create the vaccine.

You have to go back to high school biology and look at a model of a cell, said Alderink.

The vaccine utilizes strands of genetic material called messenger RNA (mRNA). Alderink explained that scientists replicated the genetic code for the unique spike protein on the COVID-19 virus. The code is placed in a lipid fat molecule, which protects the mRNA from enzymes in the body. It also allows the mRNA to enter muscle cells near the vaccination site. Once inside the cytoplasm, the cells recognize it and then produce the spike protein from the message on the mRNA.

After the spike protein is made within the muscle cells, the body very quickly recognizes it as foreign and starts producing antibodies to destroy it. From there, the information travels through the lymphatic system to the lymph nodes, where the body makes immune cells.

Its pretty cool. So that if were exposed to the actual coronavirus, our immune system is already primed against it, said Alderink.

She said another cool thing about the mRNA is that it cannot get into the cells nucleus. Nor can it survive very long, and it is quickly broken down.

mRNA technology has been used in cancer treatments and, according to Alderink, has been underdeveloped for more than 10 years. The technology has been sitting there waiting for us to do something great with it.

She said mRNA was going to be used for the SARS outbreak in 2003, but that event fortunately didnt turn out to be an epidemic or pandemic.

The Pfizer-BioNTechs two-dose vaccine will provide 95 percent protection. A week after the first vaccine, the patient has 50 percent protection. The second vaccine, three weeks later, provides 95 percent protection after a week.

Its pretty quick, said Alderink.

She said the medical community is uncertain right now if the vaccine is effective against the new virus turning up in Europe and Asian countries. And its not clear if patients will have to be revaccinated in subsequent years after the initial vaccination.

Alderink understands the hesitations of some who are nervous about the new vaccine.

Im a physician and Im very trustful of my colleagues, physicians, scientists. (But) I can understand the other side, the people being nervous about something that appears so new and that went through the studies so quickly. None of us have ever been through this before so thats a normal response.

Regarding that hesitation, she explained the twofold importance of having frontline healthcare workers go first. One: so they can continue to work and take care of people without the fear of getting sick. Two: helping put the public at ease about the new vaccine.

My hope is that with health workers and essential workers going first, we can show that it is safe and that it is working so that people will start trusting this new technology and the vaccine, Alderink said.

Since the mRNA vaccine doesnt introduce live or inactive viruses into the patient, theres no chance of getting sick from it. Alderink said some side effects could include a mild headache, enlarged lymph nodes a day or two after being vaccinated, low-grade fever and/or body aches. These are more common after the second dose, but less common in people 65 and older, according to the study.

As for Alderink herself, It wasnt really any more sore than the flu shot.

In a pandemic like this, were asking people to take that leap of faith. Thats the hard part about a large-scale vaccination program like this. Were asking people to do something to protect themselves, but were also asking people to participate in public health in doing something for all of us, including close family members, the community and people they come in contact with. Thats a tough thing when people are dealing with a lot of stress, said Alderink.

Were beyond the point where weve got to find a way to move forward and I think the vaccine definitely provides us the best opportunity at this point to do that.

Alderink believes that with the vaccine, it will be another six to nine months before life starts to return to normal. Were seeing the light at the end of the tunnel.

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Local physician on vaccine: We're seeing the light at the end of the tunnel - What's New LaPorte?

Deepu Madduri, MD, discusses how chimeric antigen receptor T-cell therapy is used for patients with multiple myeloma.

Deepu Madduri, MD, assistant professor of medicine, hematology and medical oncology and assistant professor of urology at Mount Sinai, discusses how chimeric antigen receptor (CAR) T-cell therapy is used for patients with multiple myeloma.

CAR T-cell therapy, bispecific agents, and antibody-drug conjugates are the current treatment options available for the relapsed/refractory multiple myeloma population. However, CAR T-cell therapy is typically reserved for patients who are younger and more fit, who do not have rapidly progressing disease. The bispecific agents are used mostly in elderly patients with many comorbidities and cannot handle cytokine release syndrome. Patients with rapid progression cannot wait the 4 to 5 weeks it takes to manufacture their cells for CAR-T cell therapy, says Madduri.

There are different roles for CAR T-cell therapy compared with bispecific therapy. Madduri feels it is important to keep in mind that multiple myeloma is an incurable disease for these patients, so the field does need all these therapies to treat at different points. The question is how to sequence them so patients achieve the best progression-free survival they can.

Read more here:
Addressing the Role of CAR T-Cell Therapy in Multiple Myeloma - Targeted Oncology

News Release

Monday, December 28, 2020

NIH- and BARDA-funded trial will enroll up to 30,000 volunteers.

The Phase 3 trial of another investigational coronavirus disease 2019 (COVID-19) vaccine has begun enrolling adult volunteers. The randomized, placebo-controlled trial will enroll approximately 30,000 people at approximately 115 sites in the United States and Mexico. It will evaluate the safety and efficacy of NVX-CoV2373, a vaccine candidate developed by Novavax, Inc., of Gaithersburg, Maryland. Novavax is leading the trial as the regulatory sponsor. The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, and the Biomedical Advanced Research and Development Authority (BARDA), part of the U.S. Department of Health and Human Services Office of the Assistant Secretary for Preparedness and Response, are funding the trial.

Addressing the unprecedented health crisis of COVID-19 has required extraordinary efforts on the part of government, academia, industry and the community, said NIAID Director Anthony S. Fauci, M.D. The launch of this study the fifth investigational COVID-19 vaccine candidate to be tested in a Phase 3 trial in the United States demonstrates our resolve to end the pandemic through development of multiple safe and effective vaccines.

The trial is being conducted in collaboration with Operation Warp Speed (OWS), a multi-agency collaboration overseen by HHS and the Department of Defense that aims to accelerate development, manufacture and distribution of medical countermeasures for COVID-19. Some of the U.S. trial sites participating are part of the NIAID-supported COVID-19 Prevention Network (CoVPN). The CoVPN includes existing NIAID-supported clinical research networks with infectious disease expertise and was designed for rapid and thorough evaluation of vaccine candidates and monoclonal antibodiesfor preventing COVID-19.

Volunteers will be asked to give informed consent prior to their participation in the trial. They will be grouped into two cohorts: individuals 18 through 64 years old and those aged 65 and older, with a goal of enrolling at least 25% of all volunteers who are 65 years old or older. Trial organizers also are emphasizing recruitment of people who are at higher risk of severe COVID-19 disease, including those who are Black (including African Americans), Native American, or of Latino or Hispanic ethnicity, and people who have underlying health conditions such as obesity, chronic kidney disease or diabetes.

Weve come this far, this fast, but we need to get to the finish line, said NIH Director Francis S. Collins, M.D., Ph.D. That will require multiple vaccines using different approaches to ensure everyone is protected safely and effectively from this deadly disease.

After providing a baseline nasopharyngeal and blood sample, participants will be assigned at random to receive an intramuscular injection of either the investigational vaccine or a saline placebo. Randomization will be in a 2:1 ratio with two volunteers receiving the investigational vaccine for each one who receives placebo. Because the trial is blinded, neither investigators nor participants will know who is receiving the candidate vaccine. A second injection will be administered 21 days after the first.

Participants will be followed closely for potential vaccine side effects and will be asked to provide blood samples at specified time points after each injection and during the following two years. Scientists will analyze the blood samples to detect and quantify immune responses to SARS-CoV-2, the virus that causes COVID-19. Of note, specialized assays will be used to distinguish between immunity as a result of natural infection and vaccine-induced immunity. The trials primary endpoint is to determine whether NVX-CoV2373 can prevent symptomatic COVID-19 disease seven or more days after the second injection relative to placebo.

Novavaxs investigational vaccine, NVX-CoV2373, is made from a stabilized form of the coronavirus spike protein using the companys recombinant protein nanoparticle technology. The purified protein antigens in the vaccine cannot replicate and cannot cause COVID-19. The vaccine also contains a proprietary adjuvant, MatrixM. Adjuvants are additives that enhance desired immune system responses to vaccine. NVX-CoV2373 is administered in liquid form and can be stored, handled and distributed at above-freezing temperatures (35 to 46F). A single vaccine dose contains 5 micrograms (mcg) of protein and 50 mcg of adjuvant.

In animal tests, NVX-CoV2373 vaccination produced antibodies that blocked the coronavirus spike protein from binding to the cell surface receptors targeted by the virus, preventing viral infection. In results of a Phase 1 clinical trial published in the New England Journal of Medicine, NVX-CoV2373 was generally well-tolerated and elicited higher levels of antibodies than those seen in blood samples drawn from people who had recovered from clinically significant COVID-19. NVX-CoV2373 also is being evaluated in a Phase 2b trial in South Africa, now fully enrolled with 4,422volunteers, and data from a Phase 1/2 continuation trial in the United States and Australia is expected as early as first quarter 2021. Novavax also recently completed enrollment of more than 15,000 volunteers in a Phase 3 trial of the candidate vaccine in the United Kingdom, which is also testing two injections of 5 mcg of protein and 50 mcg of Matrix-M adjuvant administered 21 days apart.

An independent Data and Safety Monitoring Board (DSMB) will provide oversight to ensure the safe and ethical conduct of the study. All Phase 3 clinical trials of candidate vaccines supported through OWS are overseen by a common DSMB developed in consultation with the NIH Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) initiative.

Adults who are interested in joining this study can visit Coronaviruspreventionnetwork.org, Novavax.com/PREVENT-19 or ClinicalTrials.gov and search identifier NCT04611802.

About the COVID-19 Prevention Network: The COVID-19 Prevention Network (CoVPN) was formed by the National Institute of Allergy and Infectious Diseases (NIAID) at the U.S. National Institutes of Health to respond to the global pandemic. Through the CoVPN, NIAID is leveraging the infectious disease expertise of its existing research networks and global partners to address the pressing need for vaccines and antibodies against SARS-CoV-2. CoVPN will work to develop and conduct studies to ensure rapid and thorough evaluation of vaccines and antibodies for the prevention of COVID-19. The CoVPN is headquartered at the Fred Hutchinson Cancer Research Center. For more information about the CoVPN, visit: coronaviruspreventionnetwork.org.

About HHS, ASPR, and BARDA: HHS works to enhance and protect the health and well-being of all Americans, providing for effective health and human services and fostering advances in medicine, public health, and social services. The mission of ASPR is to save lives and protect Americans from 21st century health security threats. Within ASPR, BARDA invests in the innovation, advanced research and development, acquisition, and manufacturing of medical countermeasures vaccines, drugs, therapeutics, diagnostic tools, and non-pharmaceutical products needed to combat health security threats. To date, BARDA-supported products have achieved 55 FDA approvals, licensures or clearances. To learn more about federal support for the nationwide COVID-19 response, visit http://www.coronavirus.gov.

About Operation Warp Speed:OWS is a partnership among components of the Department of Health and Human Services and the Department of Defense, engaging with private firms and other federal agencies, and coordinating among existing HHS-wide efforts to accelerate the development, manufacturing, and distribution of COVID-19 vaccines, therapeutics, and diagnostics.

About the National Institute of Allergy and Infectious Diseases:NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on theNIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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Phase 3 trial of Novavax investigational COVID-19 vaccine opens - National Institutes of Health

BEIJING--(BUSINESS WIRE)--InnoCare Pharma (HKEX: 09969), a leading biopharmaceutical company, announced today that its BTK inhibitor orelabrutinib received approval from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (CLL) /small lymphocytic lymphoma (SLL), and the treatment of patients with relapsed/refractory mantle cell lymphoma (MCL). Both new drug applications (NDAs) were previously granted priority review by the Center for Drug Evaluation (CDE) of the NMPA.

I would like to thank our entire team at InnoCare for their hard work and contributions, said Dr. Jasmine Cui, the co-founder, chairwoman and CEO of InnoCare. This approval marks the maturation of InnoCare from a clinical to a commercial stage company.

The approval of orelabrutinib would not have been possible without the strong support from our partners, physicians, experts and patients who participated in a series of clinical trials, said Dr. Cui. Science drives innovation for the benefit of patients. From the initial R&D to patients, orelabrutinib has gone through an extraordinary journey. Treatment with orelabrutinib has demonstrated clear benefit for patients. We remain highly focused on the development of additional high-quality innovative drugs that can address unmet needs.

Orelabrutinib is a BTK inhibitor developed by InnoCare for the treatment of cancer and autoimmune diseases.

Compared with other BTK inhibitors, orelabrutinib shows a higher complete response (CR) rate in the treatment of relapsed/refractory (R/R) CLL/SLL patients, and we expect even deeper responses with a longer treatment of patients with orelabrutinib, said Jianyong Li, M.D., Professor and Director of the Department of Hematology and Director of the Pukou CLL Center at the First Affiliated Hospital of Nanjing Medical University. We believe that the approval of orelabrutinib will provide a safe and effective new treatment solution for lymphoma patients in China.

Data from trials with orelabrutinib have shown sustained efficacy in the treatment of R/R MCL patients, said Jun Zhu, M.D., Ph.D., Professor and Director of the Department of Internal Medicine and Lymphoma, Peking University Cancer Hospital. We believe the improved safety profile resulting from high selectivity and the convenience of daily oral administration will help make orelabrutinib a favorable treatment option for patients of B-cell malignancies.

China represents a large pharmaceutical market. The development of novel drugs requires focused drug discovery efforts, world-class research and development capabilities, state-of-the-art clinical research and significant capital investment.

"Continuous breakthroughs in life science technology are the foundation for the sustainable development of China's healthcare industry in the future, said Professor Yigong Shi, the co-founder and President of the Science Advisory Board (SAB) of InnoCare. To promote the research and development of innovative drugs such as orelabrutinib, cutting-edge science is essential, and it is equally critical to integrate collaborations among industry, academia, research institutions and hospitals. By doing so, R&D can keep up with rapidly evolving market needs. At present, although science and technology continue to evolve, we are still far from defeating malignant tumors and autoimmune diseases. I sincerely believe that more innovative companies will emerge in China and make greater contributions to the development of China's healthcare system."

Lymphoma is a malignant tumor that originates from the lymphoid hematopoietic system. Relevant data show that lymphoma is one of the malignant tumors with the fastest growth of incidence rate and one of the top ten malignant tumors with the highest mortality rate in China. Every year, approximately 93,000 people are newly diagnosed with lymphoma, and more than 50,000 people die from it1. BTK is a key kinase of the B cell receptor (BCR) signaling pathway and an important regulator of B cell proliferation and survival mainly in non-Hodgkin's lymphoma (NHL). BTK inhibitors can block BCR-induced BTK activation and downstream signaling pathways, thereby inhibiting the growth of B-cell tumors and promoting cell apoptosis.

Orelabrutinib is a highly selective and novel BTK inhibitor developed by InnoCare that can avoid off-target-related adverse events with improved safety and efficacy.

About the latest clinical date of orelabrutinib, please refer to: https://cn.innocarepharma.com/en/media/press-release/20201208/

About InnoCare

InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancer and autoimmune diseases. We strategically focus on lymphoma, solid tumors, and autoimmune diseases with high unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, New Jersey, and Boston.

1 Source: https://baijiahao.baidu.com/s?id=1677968646475994848&wfr=spider&for=pc

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InnoCare Announces the Approval of Orelabrutinib in China for Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic...