On October 29, 2019 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported the presentation of a poster at the AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference today in Boston, MA (Press release, Moleculin, OCT 29, 2019, View Source [SID1234549978]). The poster, entitled "Dose and Schedule-Dependent Efficacy of Liposomal Annamycin in Pre-clinical Models of Acute Myeloid Leukemia," presents data documenting the high activity of Annamycin against AML, including in vitro studies in a panel of human AML cell lines, as well as in vivo studies in both human and murine AML models developed under the Company’s sponsored research agreement with MD Anderson Cancer Center.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors.

"This study highlights an important new finding," commented Walter Klemp, Moleculin’s Chairman and CEO. "We’ve known for some time that Annamycin is effective in AML animal models and the activity that we believe is coming from our current AML clinical trials seems to correlate with this. But, what’s new here is the observation that Annamycin may also be more effective than other drugs due to its high uptake and effectiveness in eliminating AML cells localized in different organs. Additional important observations made with these studies indicates that the long-term exposure of healthy mice (at least 12 doses so far) to a highly efficacious dose of 4 mg/kg administered weekly is not toxic and that even two weekly doses of 4 mg/kg are producing a significant increase in survival. And, because Annamycin is designed to be non-cardiotoxic, this extended dosing regimen may prove to be feasible and beneficial in humans. This potentially opens the door for expanded and improved dosing regimens in future clinical trials."

Quoting from the accepted abstract: "In vivo studies confirmed anti-tumor efficacy of Annamycin in both human and murine AML models. Based on bioluminescence imaging, the liposomal formulation of the drug significantly delayed AML progression in the human OCL-AML3/NSG model at 4 mg/kg with once weekly dosing. Similarly, significant dose-dependent reduction of peripheral blood AML blasts was observed in the murine AML-Turq2 model, and this reduction was strongly correlated with prolongation of animal survival. The median survival of mice receiving four doses of L-Ann once a week at 4 mg/ml was 37 days while mice receiving vehicle lived only 14 days (p=0.0002). Different doses and administration schedules of [Annamycin] were tested in an effort to maximize survival benefits. In summary [Annamycin] is effective in AML, demonstrating significant activity in both in vitro and in vivo mouse models with a distinct pattern of intracellular uptake and organ distribution using a once a week schedule. This suggests that [Annamycin] with this profile, including a lack of cardiotoxicity and activity against [doxorubicin] resistant tumors, may be an advantageous approach in the treatment of AML."

On October 29, 2019 Verastem, Inc. (Nasdaq: VSTM), operating as Verastem Oncology (or "the Company"), focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, reported financial results for the three months ended September 30, 2019, and provided an overview of recent accomplishments and clinical development progress for duvelisib (COPIKTRA) (Press release, Verastem, OCT 29, 2019, View Source [SID1234549994]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the third quarter, we achieved $9.0 million in revenue, including $4.0 million in net product revenue from COPIKTRA, a 33% increase over the prior quarter, and we remain on track to achieve the revenue that we have guided for this year. We continue to make progress with COPIKTRA sales as the intent to prescribe and new prescriber base grows week over week due to solid progress across our commercial efforts, including physician education and contracting. We also believe in the long-term potential for our current COPIKTRA indications," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We are deeply committed to our long-term strategy to achieve sustainable growth and progress our mission on behalf of patients. In order to achieve these ambitious goals and provide us with greater financial flexibility going forward, we are streamlining our organization and reducing operating expenses, which will result in approximately $25 million in annualized cost savings next year."

Key Third Quarter 2019 and Recent Accomplishments

Corporate and Financial

Implementing a Corporate Restructuring as Part of the Long-Term "6-2-5" Strategy – Verastem Oncology continues to deliver on its "6-2-5" strategic plan in which we aim to narrow the gap between revenue and commercial spend by year end 2019, achieve cash flow break-even for both the commercial and clinical COPIKTRA program by mid-2021, and the indications for COPIKTRA are broadened with at least one additional marketed product, along with a pipeline of assets in development by mid-2024. In order to support this strategy, the company will be reducing overall operating expenses, including the elimination of approximately 40 current positions across all functions. The workforce reduction is designed to streamline operations, speed execution, and reflect the focused, account-based approach in the field. The Company expects minimal impact on top-line revenue results with these changes. The overall reduction in operating expenses is expected to result in approximately $25 million in annualized cost savings in 2020. Verastem Oncology estimates that it will incur approximately $1.0 million in pre-tax charges for severance and other costs related to the workforce reduction, the majority of which will be incurred in 2019.
Signed Exclusive License Agreement with Sanofi for the Development and Commercialization of Duvelisib in Select Eurasian Territories – In July 2019, the Company announced its entry into an exclusive license agreement with Sanofi, under which Verastem Oncology granted exclusive rights to Sanofi to develop and commercialize products containing COPIKTRA in Russia and CIS, Turkey, the Middle East and Africa. Under the terms of the agreement, Verastem Oncology received an upfront payment of $5 million (USD) and is eligible to receive aggregate payments of up to $42 million if certain development and sales milestones are successfully achieved, plus double-digit percentage royalties based on future net sales of COPIKTRA in the licensed territories. In exchange, Sanofi received exclusive rights to develop and commercialize COPIKTRA and hold the marketing authorization and product license for COPIKTRA in the licensed territories. Additionally, Sanofi will have the right to collaborate with Verastem Oncology on certain global development and clinical trial activities.
COPIKTRA (duvelisib)

Ongoing Commercial Rollout of COPIKTRA in the United States (U.S.) – COPIKTRA, the Company’s oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first FDA-approved dual inhibitor of PI3K-delta and PI3K-gamma continues to gain momentum in the U.S. generating revenues of $4.0 million during the third quarter of 2019, a 33% increase over the prior quarter. As of the end of the third quarter 2019, the number of prescribing physicians had increased by over 30%, compared to the end of the prior quarter.
Yakult Doses First Patient in Japanese Bridging Study Evaluating COPIKTRA in Relapsed or Refractory CLL/SLL – In early October, Verastem Oncology’s partner Yakult Honsha Co., Ltd. (Yakult) dosed the first patient in a Phase 1b Japanese bridging study evaluating COPIKTRA in patients with relapsed or refractory CLL/SLL following at least one prior therapy. Yakult’s multicenter, open-label Phase 1b study is expected to enroll approximately 10 patients and the primary endpoint of the study is objective response rate. Secondary endpoints of the study include overall survival, progression free survival and safety. This Phase 1b study is expected to serve as a bridging study based on the efficacy and safety observed in Verastem Oncology’s Phase 3 DUO study. The results of the Phase 1b bridging study are expected to form the basis of a regulatory submission for COPIKTRA for the treatment of relapsed or refractory CLL/SLL in Japan.
Duvelisib Receives Orphan Drug Designation from FDA for the Treatment of T-Cell Lymphoma – In early October, duvelisib (COPIKTRA) received orphan drug designation from the FDA for use in the treatment of T-Cell lymphoma. The designation was created to encourage the development of drugs that may provide significant benefit to patients suffering from rare diseases. Duvelisib is not currently approved for the treatment of T-cell lymphoma. The Company recently completed the dose optimization/dose selection phase of the PRIMO study in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) and has submitted the data for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2019 Annual Meeting in December. The registration-directed portion of the PRIMO study is currently on going and is being conducted in the U.S., Europe and Japan.
Presented New Preclinical Duvelisib Data at the 5th International Conference on New Concepts in Lymphoid Malignancies– In early October, an abstract was presented at the meeting that showed superior anti-cancer activity of the dual PI3K-delta/gamma inhibitor duvelisib compared to the PI3K-delta inhibitor idelalisib in preclinical models of mantle cell lymphoma (MCL). Verastem Oncology’s goal is to expand into additional lymphoid malignancy indications and these data provide additional support for the future study of duvelisib through clinical trials in patients with MCL. Duvelisib is not approved for use in MCL.
Presented Seven COPIKTRA Abstracts at Two Prestigious Medical Meetings – Throughout September, Verastem Oncology continued to have a strong scientific presence for COPIKTRA at important medical congresses. The Company presented a total of seven COPIKTRA abstracts at two prestigious medical oncology meetings; the 18th Annual International Workshop on Chronic Lymphocytic Leukemia (iwCLL) and the Society of Hematologic Oncology 2019 Annual Meeting. Collectively, the presented abstracts highlighted a wide range of duvelisib clinical data, including data from the Phase 3 DUO study in patients with relapsed or refractory CLL/SLL, dose modification data from the Phase 3 DUO study, data from a post-hoc analysis evaluating the effect of COPIKTRA on lymphocytosis, including in patients with high-risk factors, and data from the Phase 2 DYNAMO in patients with refractory marginal zone lymphoma. These presented data continue to support the ongoing commercialization of COPIKTRA.
Third Quarter 2019 Financial Results

Total revenue for the three months ended September 30, 2019 (2019 Quarter) was $9.0 million. Net product revenue for the 2019 Quarter was $4.0 million, compared to $0.5 million for the three months ended September 30, 2018 (2018 Quarter), following the FDA’s approval of COPIKTRA on September 24, 2018. License and collaboration revenue for the 2019 Quarter was $5.0 million, compared to $15.0 million for the 2018 Quarter. The 2018 Quarter included license revenue of $15.0 million related to an upfront payment pursuant to a license and collaboration agreement executed between Verastem Oncology and CSPC Pharmaceutical Group Limited in September 2018. The 2019 Quarter includes a $5.0 million upfront payment received pursuant to a license and collaboration agreement executed between Verastem Oncology and Sanofi in July 2019.

Total operating expenses for the 2019 Quarter were $35.1 million, compared to $41.4 million for the second quarter of 2019 and compared to $37.1 million for the 2018 Quarter.

Research and development (R&D) expense for the 2019 Quarter was $12.2 million, compared to $11.6 million for the 2018 Quarter. The increase of $0.6 million, or 5.2%, was primarily related to an increase of $0.4 million in contract research organizations (CRO) costs and an increase of $0.3 million related to personnel costs, including non-cash stock-based compensation, partially offset by a decrease of $0.1 million in consulting costs. The $0.4 million increase in CRO costs is primarily related to an increase of $1.5 million for the Company’s planned DUETTO and TEMPO studies, partially offset by a decrease of $1.0 million resulting from site closures for the Phase 3 DUO and Phase 2 DYNAMO studies as patients continue to complete treatment.

Selling, general and administrative expense for the 2019 Quarter was $22.2 million, compared to $25.4 million for the 2018 Quarter. The decrease of $3.2 million, or 12.6%, was primarily due to a decrease of $2.3 million in consulting and professional fees, primarily related to the support of commercial launch preparation activities in the 2018 Quarter and a decrease of $0.9 million in personnel related costs, including non-cash stock-based compensation.

Net loss for the 2019 Quarter was $30.1 million, or $0.41 per share (basic and diluted), compared to $21.7 million, or $0.29 per share (basic and diluted), for the 2018 Quarter.

For the 2019 Quarter, non-GAAP adjusted net loss was $26.2 million, or $0.35 per share, compared to non-GAAP adjusted net loss of $19.4 million, or $0.26 per share, for the 2018 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

As of September 30, 2019, Verastem Oncology had cash, cash equivalents and short-term investments of $160.2 million.

Financial Guidance for Fiscal 2019

Verastem Oncology is reiterating its full-year 2019 guidance and expects net product revenue for COPIKTRA to be in the range of $12-14 million. This guidance is based on product revenue to date, current run rates and near-term expectations.

Conference Call and Webcast Information

The Verastem Oncology management team will host a conference call and webcast today, Tuesday, October 29, 2019, at 4:30 PM (ET). The call can be accessed by dialing (877) 341-5660 (U.S. and Canada) or (315) 625-3226 (international), five minutes prior to the start of the call and providing the passcode 5785818.

The live, listen-only webcast of the conference call can be accessed by visiting the investors section of the Company’s website at www.verastem.com. A replay of the webcast will be archived on the Company’s website for 90 days following the call.

About Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are cancers that affect lymphocytes and are essentially the same disease, with the only difference being the location where the cancer primarily occurs. When most of the cancer cells are located in the bloodstream and the bone marrow, the disease is referred to as CLL, although the lymph nodes and spleen are often involved. When the cancer cells are located mostly in the lymph nodes, the disease is called SLL. The symptoms of CLL/SLL include a tender, swollen abdomen and feeling full even after eating only a small amount. Other symptoms can include fatigue, shortness of breath, anemia, bruising easily, night sweats, weight loss, and frequent infections. However, many patients with CLL/SLL will live for years without symptoms. In 2018, there were approximately 200,000 patients in the United States affected by CLL/SLL with nearly 20,000 new diagnoses. While there are therapies currently available, real-world data reveals that a significant number of patients either relapse following treatment, become refractory to current agents, or are unable to tolerate treatment, representing a significant medical need. The potential of additional oral agents, particularly as a monotherapy that can be used in the general community physician’s armamentarium, may hold significant value in the treatment of patients with CLL/SLL.

About Follicular Lymphoma

Follicular lymphoma (FL) is typically a slow-growing or indolent form of non-Hodgkin lymphoma (NHL) that arises from B-lymphocytes, making it a B-cell lymphoma. In 2018, this lymphoma subtype accounted for 20 to 30 percent of all NHL cases, with more than 140,000 people in the United States with FL and more than 13,000 newly diagnosed patients. Common symptoms of FL include enlargement of the lymph nodes in the neck, underarms, abdomen, or groin, as well as fatigue, shortness of breath, night sweats, and weight loss. Often, patients with FL have no obvious symptoms of the disease at diagnosis. Follicular lymphoma is usually not considered to be curable, but more of a chronic disease, with patients living for many years with this form of lymphoma. The potential of additional oral agents, particularly as a monotherapy that can be used in the general community physician’s armamentarium, may hold significant value in the treatment of patients with FL.

About Peripheral T-Cell Lymphoma

Peripheral T-cell lymphoma (PTCL) is an aggressive type of non-Hodgkin lymphoma (NHL) that develops in mature white blood cells called "T cells" and "natural killer (NK) cells"1 which circulate with the lymphatic system.2 PTCL accounts for between 10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects people aged 60 years and older.1 Although there are many different subtypes of peripheral T-cell lymphoma, they often present in a similar way, with widespread, enlarged, painless lymph nodes in the neck, armpit or groin.2 There is currently no established standard of care for patients with relapsed or refractory disease.1

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.3,4,5 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status, and is being investigated in combination with other agents through investigator-sponsored studies.6 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

On October 29, 2019, Intrexon Corporation ("Intrexon") and TS AquaCulture LLC ("TS AquaCulture"), a Virginia limited liability company that is managed by Third Security, LLC ("Third Security"), reported that it entered into a stock purchase agreement (the "Purchase Agreement"), pursuant to which, upon the terms set forth therein, TS AquaCulture purchased from Intrexon 8,239,199 shares of common stock, par value $0.001 per share, of AquaBounty Technologies, Inc., a Delaware corporation ("AquaBounty"), for an aggregate purchase price of $21,586,701.38 and Intrexon assigned to TS AquaCulture all of Intrexon’s rights, and TS AquaCulture accepted and assumed all of such rights and obligations, under the Relationship Agreement, dated as of December 5, 2012, by and between Intrexon and AquaBounty (Filing, 8-K, Intrexon, OCT 29, 2019, View Source [SID1234550093]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Randal J. Kirk and shareholders affiliated with him beneficially own approximately 46.2% of Intrexon’s voting stock. Mr. Kirk is Intrexon’s Chief Executive Officer and Chairman of Intrexon’s board of directors (the "Board") and currently serves as the Senior Managing Director and Chief Executive Officer of Third Security and owns 100% of the equity interests of Third Security. Third Security directly owns shares of Intrexon common stock and is also the manager of certain entities that directly own shares of Intrexon common stock, and therefore may be deemed to beneficially own approximately 34.7% of Intrexon’s common stock.

The Purchase Agreement was unanimously approved by the independent members of Intrexon’s Board of Directors, with the recommendation of the Audit Committee and an independent special committee of the Board.

The foregoing is a summary description of certain terms of the Purchase Agreement and, by its nature, is incomplete. A copy of the Purchase Agreement is filed herewith as Exhibit 10.1 to this Current Report on Form 8-K and is incorporated herein by reference. Intrexon encourages all readers to read the entire text of the Purchase Agreement.

On October 29, 2019 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited off-the-shelf CAR T-cells (UCART), reported the Company has dosed the first patient in its UCARTCS1 clinical trial, MELANI-01, the first allogeneic off-the-shelf CAR-T product candidate the U.S. Food and Drug Administration (FDA) has cleared to enter into clinical development for relapsed/refractory multiple myeloma (R/R MM) (Press release, Cellectis, OCT 29, 2019, View Source [SID1234549963]). The UCARTCS1 clinical trial is a Phase 1 dose-escalation study to evaluate the safety, expansion, persistence and clinical activity of UCARTCS1 cells in R/R MM patients.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This first patient dosing for our MELANI-01 clinical trial is an important advancement, as our team has worked tirelessly to develop and take the CS1 target from the lab to the clinic," said Dr. André Choulika, Chairman and CEO, Cellectis. "In taking this next clinical step, we look forward to deepening our understanding of UCARTCS1 as a potential new treatment option for relapsed/refractory multiple myeloma patients in the future."

The MELANI-01 clinical trial is currently open at MD Anderson Cancer Center in Houston, Texas, under the supervision of Dr. Krina Patel, Principal Investigator, Study Coordinating Investigator, Assistant Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine at MD Anderson Cancer Center, as well as Hackensack Meridian in New Jersey under the supervision of Dr. David Siegel, Director of the Multiple Myeloma Institute at John Theurer Cancer Center (JTCC) at Hackensack University Medical Center. Another site is planned to open at Weill Cornell Medicine in New York under the leadership of Dr. Adriana Rossi, Associate Clinical Director, Myeloma Center and Assistant Professor of Medicine, Division of Hematology and Medical Oncology.

About Multiple Myeloma (MM)
Multiple myeloma is a cancer that affects a type of white blood cells called plasma cells that are specialized mature B cells, which secrete antibodies to combat infections. Multiple myeloma is characterized by the uncontrolled proliferation of neoplastic plasma cells in the bone marrow, where they overcrowd healthy blood cells. Although MM is a chronic disease and an exact cause has not yet been identified, researchers have made significant progress over the years in managing the disease through better understanding MM’s pathophysiology. The progress in finding a cure needs to be continued as The American Cancer Society estimates that 32,110 new cases of MM will be diagnosed, and 12,960 deaths are expected to occur in 2019 in the U.S. alone.

About UCARTCS1
UCARTCS1 is an allogeneic, off-the-shelf, gene-edited T-cell product candidate designed for the treatment of multiple myeloma. CS1 (SLAMF7) is highly expressed on MM tumor cells and is an attractive target. The limitation so far has been the presence of the CS1 target on the surface of T-cells, which has hindered the access to CAR-Ts. For example, the introduction of a CAR construct in T-cells induces cross T-cell reactivity and leads to destruction of the CS1+ T-cell population during manufacturing. Cellectis solved this issue by using TALEN gene editing to knock out the CS1 gene from T-cells before introducing the CS1 CAR construct.

On October 29, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; Nasdaq: MOR) reported topline results from the primary analysis of the retrospective observational matched control cohort (Re-MIND) (Press release, MorphoSys, OCT 29, 2019, View Source [SID1234549979]). This study was designed to compare the effectiveness of lenalidomide monotherapy based on real-world patient data with the efficacy outcomes of the tafasitamab/lenalidomide combination, as investigated in MorphoSys’s L-MIND trial.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Re-MIND collected outcome data from 490 non-transplant eligible patients with relapsed/ refractory diffuse large B cell lymphoma (r/r DLBCL) who had received lenalidomide monotherapy in the U.S. and the EU in a real-world setting. 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. ORR was 67.1% (95% confidence interval (CI): 55.4-77.5) for the tafasitamab/ lenalidomide combination, compared to 34.2% (CI: 23.7-46.0) for the lenalidomide monotherapy (p<0.0001). Superiority was consistently observed across all secondary endpoints, including complete response (CR) rate (tafasitamab/lenalidomide combination 39.5%; CI: 28.4-51.4 versus lenalidomide monotherapy 11.8%; CI: 5.6-21.3; p<0.0001), as well as in pre-specified statistical sensitivity analyses. In addition, there was a significant difference observed in overall survival, which was not reached in the tafasitamab/lenalidomide combination as compared to 9.3 months in the lenalidomide monotherapy (hazard ratio 0.47; CI: 0.30-0.73; p<0.0008).

"Encouraged by the results we achieved with the real-world data approach, we re-affirm our plans to pursue advancement of tafasitamab to market in combination with lenalidomide as a potential, chemo-free treatment option for patients with r/r DLBCL, subject to FDA approval. The data announced today complement the previously published data of the single-arm
L-MIND study and MorphoSys has started the rolling submission of our BLA to the FDA, which we plan to complete by end of this year," commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

"I’m very excited about this real-world data approach of the Re-MIND trial to isolate a single-agent contribution of tafasitamab in combination with lenalidomide in a matched patient population in r/r DLBCL. This study further strengthens the synergistic effect of tafasitamab and lenalidomide, as already observed in the L-MIND trial," said Pier Luigi Zinzani, M.D., Ph.D., Professor of Hematology, Head of Lymphoma Group, Institute of Hematology,
"L. e A. Seràgnoli", University of Bologna, Bologna, Italy, and one of the lead investigators in MorphoSys’s Re-MIND study.

Details of the L-MIND primary analysis were published on June 22, 2019, and can be found here.

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. Based on earlier reported interim data from L-MIND, in October 2017 the U.S. FDA granted Breakthrough Therapy Designation for tafasitamab plus lenalidomide in this patient population. MorphoSys is working towards completion of a BLA submission to the U.S. FDA based on L-MIND by end of 2019.

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’s L-MIND trial. Re-MIND collected the efficacy data from 490 r/r DLBCL patients in the U.S. and EU. Eligible patients were matched 1:1 to the L-MIND study population based on important baseline characteristics, such as relevant prognostic factors, laboratory characteristics and patient demographics.