On May 14, 2020 Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported financial results for the quarter ended March 31, 2020 and provided an update on corporate progress (Press release, Exicure, MAY 14, 2020, View Source [SID1234558068]).

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"Exicure has continued to execute on its core clinical and preclinical objectives for 2020 thanks largely to the ongoing commitment of our team and their families, as well as the patients and caregivers involved in our clinical trials," said Dr. David Giljohann Exicure’s Chief Executive Officer. "We have continued to advance our preclinical program in Friedreich’s ataxia and have initiated our Phase 2 clinical trial of AST-008, our immuno-oncology drug candidate for Merkel cell carcinoma and cutaneous squamous cell carcinoma. While COVID-19 presents many ongoing uncertainties, we are confident that our financial resources will allow continued pursuit of our strategic objectives," concluded Dr. Giljohann.

AST-008 Phase 2 clinical trial is open and enrolling patients in the second quarter of 2020

We have dosed 20 patients and completed enrollment in the Phase 1b stage of our clinical trial. To date, we have not observed any treatment-related serious adverse events or any dose-limiting toxicity.
In the second quarter of 2020, we will begin enrolling patients in the Phase 2 dose expansion phase of the trial for intratumoral AST-008 in combination with pembrolizumab or cemiplimab, approved checkpoint inhibitors, to treat patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma.
Currently, seven clinical trial sites are open and we expect to open up to eight additional sites.
We are continuing to monitor the potential impact that COVID-19 may have on patient enrollment, site initiation, safety of patients and study integrity. We are putting in place a variety of measures to mitigate the effects of COVID-19 and maintain patient safety and trial continuity.
First Quarter Financial Results, Financial Guidance and Recent Developments

Cash Position: Cash, cash equivalents, and short-term investments were $98.8 million as of March 31, 2020 compared to $110.8 million as of December 31, 2019. During the quarter Exicure repaid, pursuant to its terms, the outstanding principal balance of $5.0 million on its loan with Hercules Technology Growth Capital and classified $1.2 million of cash to other noncurrent assets. On January 6, 2020 Exicure received gross proceeds of $3.0 million pursuant to the partial exercise of the underwriters’ option to purchase additional shares. This exercise represents additional proceeds in connection with Exicure’s underwritten public offering of Exicure common stock in December 2019.

Research and Development (R&D) Expenses: Research and development expenses were $6.1 million for the quarter ended March 31, 2020, compared to $3.4 million for the quarter ended March 31, 2019. We have increased full-time staffing in R&D from 19 at March 31, 2019 to 36 at March 31, 2020 and the associated increase in activity has driven our increase in costs. The increase in staffing and associated increases in platform and discovery related costs reflects increased preclinical R&D activities associated with our collaboration with Allergan plc, increased costs related to XCUR-FXN, our Friedreich’s ataxia program, as well as other preclinical discovery work in neurology and ophthalmology.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.6 million for the quarter ended March 31, 2020, compared to $2.2 million for the quarter ended March 31, 2019. This increase is primarily due to costs related to higher legal and accounting fees, higher D&O insurance expenses and certain other costs associated with being a public company.

Net Income (Loss): Exicure had net income of $1.1 million for the quarter ended March 31, 2020 compared to a net loss of $5.3 million for the quarter ended March 31, 2019, reflecting a difference of $6.4 million. The increase in net income was driven principally by the recognition of $9.2 million of revenue associated with Exicure’s collaboration with Allergan. In November of 2019, Exicure received an upfront payment of $25.0 million from Allergan, which was then deferred, and is now being recognized as revenue as we satisfy our obligations under the terms of the Allergan collaboration agreement. The $9.2 million increase in revenue was offset by the increases in R&D expenses and G&A expenses as discussed above.

Capital Resources Guidance: Exicure believes that, based on its current operating plans and estimates of expenses, as of the date of this press release, its existing cash, cash equivalents and short-term investments will be sufficient to fund our operations into early 2022.

Response to COVID-19: With the global spread of the ongoing COVID-19 pandemic in the first quarter of 2020, we have been closely monitoring developments and have taken active measures to protect the health of our employees and their families, our communities, as well as our clinical trial investigators, patients and caregivers. Under social distancing guidelines for COVID-19, we are operating with less than 50% of our research and development staff on-site at any one time while our general and administrative team has generally been working from home. We are carefully managing laboratory staffing and taking other appropriate managerial actions to maintain progress on our preclinical and collaboration programs. We are also working closely with our third-party manufacturers and other partners to manage our supply chain activities and in our clinical operations we are taking such action as we believe appropriate to maintain patient safety and trial continuity.

Pipeline Updates

Neurology

In December of 2019, Exicure announced the development of XCUR-FXN, an SNA–based therapeutic candidate for the treatment of Friedreich’s ataxia (FA). FA is driven by triplet repeats in the frataxin gene which compromises the patient’s ability to generate adequate levels of frataxin protein. Exicure believes its SNA technology has the potential to address this genetic challenge and that its therapeutic strategy may lead to increases in the frataxin protein. Exicure plans to design and develop XCUR-FXN with guidance from, and in collaboration with, the Friedreich’s Ataxia Research Alliance (FARA). Preclinical research is ongoing and IND-enabling studies for XCUR-FXN are expected to commence in 2020.
Exicure is continuing preclinical research on the application of its SNA technology in neurological conditions, building on its early proof-of-concept work with nusinersen and its new therapeutic candidate, XCUR-FXN. Exicure is currently exploring additional neurological conditions, including spinocerebellar ataxia, Batten disease, amyotrophic lateral sclerosis (ALS) and Huntington’s disease.
Immuno-oncology; AST-008

AST-008 is an investigational SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications. Exicure has now dosed 20 patients and completed enrollment in the Phase 1b stage of the Phase 1b/2 clinical trial. To date, Exicure has not observed any treatment related serious adverse events or any dose-limiting toxicity.
In the second quarter of 2020, we intend to enroll patients in a Phase 2 dose expansion phase of its Phase 1b/2 study for intratumoral AST-008 in combination with pembrolizumab or cemiplimab to treat two cohorts of patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma. Each cohort is expected to enroll up to 29 patients.
Collaborations

Exicure entered into a collaboration, option and license agreement with Allergan plc in late 2019 and is now actively engaged in preclinical research and discovery in two clinical programs related to the treatment of hair loss disorders. Under the terms of the collaboration, Exicure received a $25.0 million upfront payment and is eligible to receive up to $725 million in potential milestones. In early 2019, Exicure also entered into a collaboration agreement with Dermelix Biotherapeutics under which Dermelix has the option to develop a targeted therapy for the treatment of Netherton Syndrome (NS).

About FARA

The Friedreich’s Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich’s ataxia. www.CureFA.org.

On May 14, 2020 NanOlogy, LLC, a clinical-stage oncology company, reported that updated data from two of its ongoing clinical trials were presented as abstracts last week through the DDW ePosters and ePresentations site (Press release, NanOlogy, MAY 14, 2020, View Source [SID1234558067]). The clinical trials are evaluating endoscopic ultrasound guided fine needle injection (EUS-FNI) of NanoPac (submicron particle paclitaxel) suspension for treatment of locally advanced pancreatic cancer (LAPC) and mucinous cystic neoplasms (MCNs/IPMNs) of the pancreas.

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The lead author of the abstract on intratumoral NanoPac for LAPC is Neil Sharma, MD (Parkview Cancer Institute – Fort Wayne, IN). The lead author of the abstract on intracystic NanoPac for MCNs/IPMNs is Mohamed O. Othman, MD (Baylor College of Medicine – Houston, TX).

Pancreatic Cancer

The Phase 2a dose-rising and expansion trial is evaluating over 6 months the safety and preliminary efficacy of NanoPac delivered intratumorally by EUS-FNI in patients with LAPC, concurrent with or following SOC therapy for the disease. After completing the dose-rising cohort (n=11), a dose expansion cohort has now fully enrolled (n=22) in which patients receive 2 monthly intratumoral (IT) injections of NanoPac. FDA has also recently allowed expansion of up to 30 additional patients who will receive up to 4 monthly IT injections.

Dr. Sharma’s abstract submitted to DDW in December 2019 reports data from the first 7 evaluable subjects from the dose expansion cohort. No drug-related local or systemic SAEs were reported including no reports of acute pancreatitis in any subject, 4 subjects had a partial response, 2 had stable disease, and 1 had progressive disease. Encouraging data has continued to accrue from the fully enrolled dose expansion (2- injection) cohort, which are expected to be presented later in 2020.

Pancreatic Cyst

The Phase 2a dose rising and expansion trial is evaluating over 6 months the safety and preliminary efficacy of NanoPac delivered intracystically by EUS-FNI following aspiration in patients with MCNs/IPMNs. The study has now completed enrollment (n=19) with patients in the dose expansion phase (n=8) receiving two intracystic injections of NanoPac 12 weeks apart.

Dr. Othman’s abstract also submitted in December reports data from the first 9 subjects who had completed the trial. No confirmed drug-related local or systemic SAEs were reported including no reports of acute pancreatitis. Plasma paclitaxel levels have not exceeded 1 ng/mL indicating that NanoPac particles are retained in the cyst over time. Evaluation of cyst volume showed decreases ranging from 8% to 89% in 8 subjects, 6 of whom showed a volume decrease > 50%. Cyst volume increased in 1 subject.

Based on encouraging results from both trials, NanOlogy is designing follow-on clinical protocols for evaluation by FDA.

In 2020, about 57,600 new cases of pancreatic cancer will be diagnosed in the U.S. and 47,050 people will die from the disease. Pancreatic cancer is currently the third most frequent cause of cancer related death in western countries and is predicted to become the second leading cause of death from cancer within the next 10 years. It is one of the few cancers for which no meaningful improvement in survival has been achieved over recent time with only an 8% survival rate at 5 years.

MCNs/IPMNs are a subset of pancreatic cysts that risk progression to pancreatic cancer. While estimates vary widely, as many as 30,000 new cases of high risk MCNs/IMPNs are diagnosed annually in the U.S. These patients may ultimately be required to undergo surgical resection of the pancreas to remove the lesion, a complicated procedure associated with high morbidity. No approved drug therapy exists in the U.S to treat the condition.

In addition to pancreatic neoplasms, NanOlogy clinical programs are advancing in genitourinary, peritoneal, lung, and dermal cancers.

The NanOlogy submicron particle therapeutic platform is based on a proprietary production technology that converts taxane API crystals into stable submicron particles of pure drug with disproportionate size to surface area ratio. The particles are covered by two composition of matter patents (US 9,814,685) and (10,507,195) both valid until 2036 in the US and pending globally.

On May 14, 2020 The Janssen Pharmaceutical Companies of Johnson & Johnson reported updated results from the Phase 1b/2 CARTITUDE-1 study (NCT03548207) evaluating the efficacy and safety of JNJ-4528, an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy in the treatment of patients with relapsed or refractory multiple myeloma (Press release, Janssen Pharmaceuticals, MAY 14, 2020, View Source [SID1234558066]).1 Longer-term follow-up results from the Phase 1b portion of the study (n=29); to be shared in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program (Abstract #8505), show that all patients responded to treatment and the responses were deep and durable, with 86 percent of patients achieving stringent complete response at a median follow-up of 11.5 months and 86 percent of patients being alive and progression free at nine months.1

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The 100 percent overall response rate (ORR) included 97 percent of patients achieving a very good partial response or better and three percent achieving a partial response.1 Responses were observed among heavily pretreated patients (n=29) at a median administered dose 0.72×10 6 CAR+ viable T cells/kg. 1 Patients evaluated had received a median of five (range, 3-18) prior treatment regimens; 86 percent were triple-refractory and 28 percent were penta-refractory.1 The median time to first response was one month (range, 1-3) and 81 percent of evaluable patients (n=16) achieved minimal residual disease (MRD)-negative disease status at 10-5 or 10-6 at the time of first suspected complete response.1

"The longer-term results for JNJ-4528, as demonstrated through the latest findings from the CARTITUDE-1 study, show the continued treatment effect for heavily pretreated patients who faced a dismal prognosis," said Jesus G Berdeja, M.D., Director of Myeloma Research, Sarah Cannon Research Institute, and principal study investigator. "We’re encouraged by not only the relatively high rate of stringent complete responses, but also the progression-free survival seen in these patients."

"Janssen has a rich heritage in bringing transformational therapies to people living with blood cancers," adds Dr Patrick Laroche, M.D., Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag. "I am excited to see these latest results support early promising data previously presented and hope that one day JNJ-4528 can offer a viable treatment option for multiple myeloma patients."

The most common adverse events (AEs) observed in CARTITUDE-1 were neutropenia (100 percent) and cytokine release syndrome (CRS, 93 percent).1 In patients who experienced Grade 3 and above AEs, the most common were neutropenia (100 percent), thrombocytopenia (69 percent) and leukopenia (66 percent). The median time of onset of CRS was seven days (range, 2-12) post-infusion, with a majority of patients experiencing Grade 1-2 CRS and two patients (7 percent) experiencing Grade 3 or greater CRS.1 Neurotoxicity consistent with immune effector cell-associated neurotoxicity syndrome (ICANS) was observed in three patients (10 percent), including one patient (3 percent) with Grade 3 or greater toxicity.1 Three deaths were reported during the Phase 1b study: one due to CRS, one due to acute myeloid leukaemia (not treatment-related) and one due to progressive disease.1

"These recently updated data from the CARTITUDE-1 study suggest a durable response and tolerable safety profile for JNJ-4528," said Sen Zhuang, M.D., Ph.D., Vice President, Oncology Clinical Development, Janssen Research & Development, LLC. "We continue to advance the investigation of this novel CAR-T treatment with the goal of bringing a differentiated immunotherapy to patients with multiple myeloma, many of whom have exhausted all potential prior treatment options."

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicentre study evaluating the safety and efficacy of JNJ-4528 in adults with relapsed or refractory multiple myeloma, 97 percent of whom were refractory to the last line of treatment; 86 percent of whom were triple-class refractory meaning their cancer did not, or no longer responds to an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody.1,2

The primary objective of the Phase 1b portion of the study was to characterise the safety and confirm the dose of JNJ-4528, informed by the first-in-human study with LCAR-B38M* CAR-T cells (LEGEND-2).2 Based on the safety profile observed in this portion of the study, outpatient dosing will be evaluated in additional CARTITUDE studies.1 The Phase 2 portion of the study will evaluate the efficacy of JNJ-4528 with overall response as the primary endpoint.2

About JNJ-4528 (LCAR-B38M*)

JNJ-4528 (LCAR-B38M) is an investigational chimeric antigen receptor T cell (CAR-T) therapy for the treatment of patients with relapsed or refractory multiple myeloma. The design comprises a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies.1 CAR-T cells are an innovative approach to eradicating cancer cells by harnessing the power of a patient’s own immune system.3 BCMA is a protein that is highly expressed on myeloma cells.4

In December 2017, Janssen entered into an exclusive worldwide license and collaboration agreement with Legend Biotech to develop and commercialise JNJ-4528 (LCAR-B38M).5 In May 2018, Janssen initiated the Phase 1b/2 CARTITUDE-1 trial (NCT03548207) to evaluate the efficacy and safety of JNJ-4528 in adults with relapsed or refractory multiple myeloma, informed by the LEGEND-2 study results.2

In April 2019, JNJ-4528 was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA).6 PRIME offers enhanced interaction and early dialogue to optimise drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.7 In February 2020, the European Commission granted orphan designation for JNJ-4528.8

About Multiple Myeloma

Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.9 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.10 Around 50 percent of newly diagnosed patients do not reach five-year survival,11,12 and almost 29 percent of patients with multiple myeloma will die within one year of diagnosis.13

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.14 Refractory MM is when a patient’s disease progresses within 60 days of their last therapy.15 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.15 While some patients with MM have no symptoms at all, others are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.16 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and require new therapies for continued disease control.17

*LCAR-B38M identifies the investigational product in China, sponsored by Janssen’s development partner, Legend Biotech.

On May 14, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ:MOR) and Incyte (NASDAQ:INCY) reported updated results from the ongoing Phase 2 L-MIND study investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) (Press release, MorphoSys, MAY 14, 2020, View Source [SID1234558065]). The results, based on a November 30, 2019 data cut-off, corroborate previously reported primary analysis data.

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In this long-term analysis of the L-MIND data, 80 study patients receiving tafasitamab plus lenalidomide were included in the efficacy analysis. After a minimum of two years’ follow-up, outcomes from the L-MIND study are consistent with the primary analysis and confirm the durability of the response (DoR) and overall survival (OS) of tafasitamab in combination with lenalidomide followed by tafasitamab monotherapy in autologous stem cell transplantation (ASCT)-ineligible patients with r/r DLBCL. Assessment by an independent review committee (IRC) at data cut-off showed an objective response rate (ORR) of 58.8% (47 out of 80 patients) and a complete response (CR) rate of 41.3% (33 out of 80 patients). Median duration of response (mDOR) was 34.6 months, with median overall survival (mOS) of 31.6 months and median progression-free survival (mPFS) of 16.2 months. The safety profile was consistent with that observed in previously reported studies of tafasitamab in combination with lenalidomide. The full analysis will be presented virtually at the 25th EHA (Free EHA Whitepaper) Annual Congress to be held June 11-14, 2020.

"We are extremely encouraged by the long-term data from our L-MIND study which confirms the previously reported results from the primary analysis," commented Dr. Malte Peters, Chief Research and Development Officer, MorphoSys. "Tafasitamab in combination with lenalidomide has the potential to address the significant medical need in patients suffering from r/r DLBCL, and we are working diligently towards our key priority of making tafasitamab available to eligible patients."

"The updated data for L-MIND reinforce the potential of tafasitamab in combination with lenalidomide as treatment for patients with r/r DLBCL. We look forward to working with our partners at MorphoSys as we seek to bring this new therapeutic option to eligible patients globally," Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapeutics, Incyte.

A Biologics License Application (BLA) for tafasitamab in combination with lenalidomide for r/r DLBCL is currently under Priority Review by the U.S. Food and Drug Administration (FDA) (PDUFA action date August 30, 2020). The BLA is based on data including the primary analysis of L-MIND with a previous cut-off date as of November 30, 2018, and the primary analysis data from the retrospective observational matched control cohort Re-MIND evaluating efficacy outcomes of r/r DLBCL patients who received lenalidomide monotherapy.

In January 2020, MorphoSys and Incyte Corporation entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. If approved, MorphoSys and Incyte will co-commercialize tafasitamab in the United States while Incyte has exclusive commercialization rights outside the United States.

About L-MIND
L-MIND is a single arm, open-label Phase 2 study, investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) after up to two prior lines of therapy, including an anti-CD20 targeting therapy (e.g. rituximab), who are not eligible for high-dose chemotherapy and subsequent autologous stem cell transplantation. The study’s primary endpoint is objective response rate (ORR). Secondary outcome measures include duration of response (DoR), progression-free survival (PFS) and overall survival (OS). In May 2019, the study reached its primary completion. Primary analysis data with a cut-off date of November 30, 2018 included 80 patients enrolled into the trial who had received tafasitamab and lenalidomide and had been followed-up as per protocol for at least one year. Efficacy results in this update were based on response rates assessed by an independent review committee for all 80 patients.

About Re-MIND
Re-MIND, an observational retrospective study, was designed to isolate the contribution of tafasitamab in the combination with lenalidomide and to prove the combinatorial effect. The study compares real-world response data of patients with relapsed or refractory DLBCL who received lenalidomide monotherapy with the efficacy outcomes of the tafasitamab-lenalidomide combination, as investigated in MorphoSys’ L-MIND trial. Re-MIND collected the efficacy data from 490 r/r DLBCL patients in the U.S. and EU. Qualification criteria for matching patients of both studies were pre-specified. As a result, 76 eligible Re-MIND patients were identified and matched 1:1 to 76 of 80 L-MIND patients based on important baseline characteristics. Objective response rates (ORR) were validated based on this subset of 76 patients in Re-MIND and L-MIND, respectively. The primary endpoint of Re-MIND has been met and shows a statistically significant superior best ORR of the tafasitamab/lenalidomide combination compared to lenalidomide monotherapy.

About tafasitamab
Tafasitamab is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. In January 2020, MorphoSys and Incyte Corporation entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. If approved in the U.S., MorphoSys and Incyte will co-commercialize tafasitamab; Incyte will have exclusive commercialization rights outside the U.S. Tafasitamab is being clinically investigated as a therapeutic option in B-cell malignancies in a number of ongoing combination trials, including L-MIND and Re-MIND. Additionally, tafasitamab is being evaluated as part of the ongoing Phase 3 study B-MIND study assessing the combination of tafasitamab and bendamustine versus rituximab and bendamustine in r/r DLBCL. Tafasitamab is also currently being investigated in patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On May 14, 2020 CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that new data from two ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies have been accepted for an oral presentation at the EHA (Free EHA Whitepaper) Congress, which will take place virtually from June 11-14, 2020 (Press release, CRISPR Therapeutics, MAY 14, 2020, View Source [SID1234558064]).

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An abstract posted online today includes 12 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and 6 months of follow-up data for the first patient treated in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Updated data will be presented at EHA (Free EHA Whitepaper), including longer duration follow-up data for the first two patients treated in these trials and initial data for the second patient treated in the CLIMB-111 trial.

The accepted abstract is now available on the EHA (Free EHA Whitepaper) conference website: View Source

Abstract Title: Initial Safety and Efficacy Results With a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Hematopoietic Stem and Progenitor Cells in Transfusion-Dependent β-Thalassemia and Sickle Cell Disease
Session Title: Immunotherapy – Clinical
Abstract Code: S280

About the Phase 1/2 Study in Transfusion-Dependent Beta Thalassemia
The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study.

About the Phase 1/2 Study in Sickle Cell Disease
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with severe SCD. The study will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up study.

About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex Collaboration
CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.