On March 3, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) and Incyte Corporation (NASDAQ: INCY) reported that their joint collaboration and license agreement for the further development and global commercialization of MorphoSys’ investigational compound tafasitamab (MOR208) has received antitrust clearance and becomes effective today (Press release, MorphoSys, MAR 3, 2020, View Source [SID1234555125]).

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The agreement becoming effective triggers the $750 million upfront payment by Incyte to MorphoSys, as well as Incyte’s equity investment into MorphoSys of $150 million in new American Depositary Shares (ADS) within the defined timelines.

Additional information about the collaboration can be found in MorphoSys’ and Incyte’s press releases dated January 13, 2020, as well as in MorphoSys’ Form 6-K filed with the Securities and Exchange Commission (SEC) on January 14, 2020 and in Incyte’s Form 8-K filed with the SEC on January 15, 2020.

The U.S. Food and Drug Administration (FDA) recently accepted filing of MorphoSys’ Biologics License Application (BLA) for tafasitamab in combination with lenalidomide for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date is August 30, 2020.

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. In the U.S., MorphoSys and Incyte will co-commercialize tafasitamab, outside the U.S. Incyte will have exclusive commercialization rights.

Tafasitamab is being clinically investigated as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label Phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of tafasitamab in combination with lenalidomide in patients with r/r DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). The ongoing Phase 3 study B-MIND assesses the combination of tafasitamab and bendamustine versus rituximab and bendamustine in r/r DLBCL. In addition, tafasitamab is currently being investigated in patients with r/r CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g., ibrutinib) in combination with idelalisib or venetoclax.

On March 3, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX) reported its financial results for the fourth quarter and year ended December 31, 2019 and recent company developments, along with a business outlook for 2020 (Press release, TG Therapeutics, MAR 3, 2020, View Source [SID1234555124]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "2019 was a transformational year for TG as we were able to report positive outcomes for umbralisib in both previously treated marginal zone lymphoma and follicular lymphoma from the UNITY-NHL trial. We also confirmed a submission pathway with the FDA and early this year commenced a single rolling submission based on these data, which we hope to complete in the first half of this year." Mr. Weiss continued, "Looking forward, we expect 2020 to be yet another impactful year as we await the topline results from our Phase 3 programs in CLL and MS and potentially our first FDA approval around year-end."

2019 Highlights & Recent Developments

Marginal Zone Lymphoma & Follicular Lymphoma:

oReceived breakthrough therapy designation (BTD) for patients with marginal zone lymphoma (MZL) who have received at least one prior therapy including an anti-CD20 regimen, and orphan drug designation for umbralisib for the treatment of patients with MZL.

oAnnounced positive outcome from the MZL cohort of the UNITY-NHL Phase 2b trial, which met the primary endpoint of Overall Response Rate (ORR), as determined by Independent Review Committee (IRC).

oPresented interim safety and efficacy data from the MZL cohort of UNITY-NHL during oral presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, the 55th American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting and the 2019 International Conference on Malignant Lymphoma (ICML).

oAnnounced positive outcome from the follicular lymphoma (FL) cohort of the UNITY-NHL Phase 2b trial, with ORR results meeting the Company’s prespecified 40 – 50% target, as determined by IRC. The Company plans to present the data at a future medical conference.

oReceived guidance from the FDA allowing submission of a single New Drug Application (NDA) for MZL and FL indications. In January 2020, a rolling NDA submission for umbralisib to treat adult patients with previously treated MZL and FL was initiated, with completion of submission targeted for first half of 2020.

Chronic Lymphocytic Leukemia:

oAwaiting topline progression free survival (PFS) results from the Company’s Phase 3 UNITY-CLL trial evaluating "U2" (the combination of umbralisib and ublituximab) in patients with frontline and previously treated chronic lymphocytic leukemia (CLL).

oFinal long-term results from the Phase 3 GENUINE study demonstrated that ublituximab in combination with ibrutinib improved PFS, as determined by IRC.

oPresented triple therapy data at the 61stAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition from the Phase 1/2 study of ublituximab in combination with umbralisib and venetoclax, in patients with relapsed/refractory CLL, during an oral session.

Multiple Sclerosis:

oUpdated Phase 2 extension trial data for ublituximab in relapsing forms of multiple sclerosis (RMS), as well as the ULTIMATE I & II Phase 3 RMS program study design and demographic data, were presented at the 35th Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

oAwaiting topline data from the Company’s Phase 3 ULTIMATE I & II trials evaluating ublituximab in patients with RMS.

Early Pipeline:

oTG-1801: Commenced a Phase 1 first-in-human, dose-escalation study of TG-1801, the Company’s anti-CD47/CD19 bispecific antibody, in patients with relapsed or refractory B-cell lymphoma and presented the first preclinical data of TG-1801 at the 24th European Hematology Association (EHA) (Free EHA Whitepaper) annual congress.

oTG-1701: Presented the first clinical data from TG-1701, the Company’s once daily, oral, BTK inhibitor, as a single agent and as a triple therapy in combination with U2 at ASH (Free ASH Whitepaper) 2019.

Key Objectives for 2020

Report topline PFS results from the Phase 3 UNITY-CLL trial evaluating U2 in patients with frontline and previously treated CLL, and if successful, target a potential New Drug Application (NDA)/Biologics Licensing Application (BLA) submission by year-end.

Complete rolling NDA submission for umbralisib in patients with previously treated MZL and FL, in the first half of 2020.

Report topline results from the Phase 3 ULTIMATE I & II trials in RMS, in the second half of 2020.

Continue to advance our early pipeline candidates including TG-1501 (cosibelimab), TG-1701 and TG-1801.

Financial Results for the Fourth Quarter and Full Year 2019

R&D Expenses: Other research and development (R&D) expense (not including non-cash compensation and non-cash in-licensing expense) was $29.5 million and $148.3 million for the three and twelve months ended December 31, 2019, respectively, compared to $51.1 million and $149.8 million for the three and twelve months ended December 31, 2018, respectively. The decrease in R&D expense is primarily attributable to the winding down of our late-stage clinical development programs during the year ended December 31, 2019.

G&A Expenses: Other general and administrative (G&A) expense (not including non-cash compensation) was $2.9 million and $9.5 million for the three and twelve months ended December 31, 2019, respectively, as compared to $1.7 million and $7.9 million for the three and twelve months ended December 31, 2018, respectively.

Net Loss: Net loss was $39.6 million and $172.9 million for the three and twelve months ended December 31, 2019, respectively, compared to a net loss of $53.9 million and $173.5 million for the three and twelve months ended December 31, 2018, respectively. Excluding non-cash items, the net loss for the three and twelve months ended December 31, 2019 was approximately $34.0 million and $161.4 million, respectively, compared to a net loss of $52.4 million and $156.6 million for the three and twelve months ended December 31, 2018, respectively.

Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $140.4 million as of December 31, 2019. The Company believes its cash, cash equivalents and investment securities on hand as of December 31, 2019, will be sufficient to fund the Company’s planned operations well into 2021.

Conference Call Information

The Company will host a conference call today, March 3, 2020, at 8:00 AM ET, to discuss the Company’s fourth quarter and year-end 2019 financial results and provide a business outlook for 2020.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Year-End 2019 Earnings Call. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

On March 3, 2020 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported financial results for the fourth quarter and full year ended December 31, 2019 and recent business highlights (Press release, Magenta Therapeutics, MAR 3, 2020, View Source [SID1234555123]).

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"2019 was a year marked by crucial progress towards our vision of immune reset, including the advancement of our two lead conditioning programs and our two clinical programs. We generated unprecedented data from our ADC-based targeted conditioning platform, and we are particularly pleased with our new MGTA-117 clinical candidate for targeted conditioning for stem cell transplant or gene therapy. Results presented last month at the TCT conference highlighted the potency, safety and broad therapeutic index of MGTA-117, well above that of currently approved ADCs at this stage of development. We look forward to moving this program into the clinic with initial clinical data expected in 2021," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta. "We also presented updated clinical data for our first-line stem cell mobilization program, MGTA-145. We have completed dosing in the Phase 1 trial and are moving forward with multiple Phase 2 studies this year. We are developing MGTA-145 as the new standard of care for first line stem cell mobilization and immune system rebuild with the potential to benefit all of the patients eligible for transplant each year."

Recent Business Highlights:

New MGTA-117 ADC clinical candidate for conditioning demonstrates broad therapeutic index; advancing MGTA-117 to generate patient clinical data in 2021: Magenta presented new data at the TCT conference in February 2020 demonstrating that MGTA-117’s chemically modified linker-toxin between antibody and payload resulted in potent depletion of stem and progenitor cells with an improved therapeutic index over prior molecules: potency ratio of 30 fold (therapeutic index; typical range for approved ADCs at this stage of development is two to six fold). MGTA-117 was developed under a partnership with Heidelberg Pharma that grants Magenta exclusive worldwide development and commercialization rights for ADCs using an amanitin payload and targeting CD117. The antibody and payload are advancing in GMP manufacture. Magenta is scaling up manufacturing of MGTA-117 and completing IND-enabling studies in 2020. The Company intends to move this new product candidate into the clinic with initial clinical data expected in 2021.

Reported first-ever successful gene therapy transplant of non-human primates with targeted single-agent CD117-ADC with no chemotherapy: Data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2019, showed the first-ever successful transplant of gene-modified cells in non-human primates using a tool molecule CD117-targeted, single-agent ADC, without the use of chemotherapy or radiation. These landmark results validate and advance Magenta’s conditioning platform.

Completed dosing in Phase 1 MGTA-145 trial, demonstrating rapid, single-day first line stem cell mobilization and collection; met all primary and secondary endpoints: At TCT, Magenta presented data from the Phase 1 trial of MGTA-145 in healthy volunteers. Data showed that MGTA-145 was safe and well tolerated as a single agent and in combination with plerixafor and demonstrated rapid, single-day mobilization and collection of sufficient numbers of stem cells. The Company has completed dosing in the Phase 1 trial and intends to move this program into multiple Phase 2 trials in patients in 2020. The Phase 2 trials will include both allogeneic and autologous transplant settings and will evaluate mobilization and collection of functional cells and engraftment of the cells after transplant to rebuild the immune system.

Presented first preclinical immune reset data with CD45-ADC at ACR: In November 2019, Magenta presented the first data on the use of targeted ADCs to reset the immune system and halt progression of autoimmune disease. Results showed that a single dose of CD45-ADC removed disease-causing cells, enabled successful reset and rebuild of the immune system and was well tolerated in models of multiple sclerosis, systemic sclerosis and inflammatory arthritis. Further, a single dose of CD45-ADC significantly delayed disease onset in a model of multiple sclerosis that has successfully provided preclinical proof of concept for clinically validated standard of care therapies. Magenta has identified a lead antibody and has progressed this program into IND-enabling studies, which the Company plans to further advance in 2020. On November 11, 2019, Magenta announced that it had exercised its option with Heidelberg Pharma for exclusive worldwide development and marketing rights for ADCs using an amanitin payload and targeting CD45.

Presented additional data from Phase 2 study of MGTA-456 showing clinically meaningful durable benefits for patients with inherited metabolic disorders: In updated results presented at TCT, two patients with cerebral adrenoleukodystrophy treated with MGTA-456 in the Phase 2 study in inherited metabolic disorders showed early and durable resolution of disease at one year of follow-up, as measured by resolution of brain inflammation on MRI. The two patients also had stable Loes and neurological function scores, consistent with a halt in disease progression. Patients with Hurler syndrome showed normalized levels of blood a-L-iduronidase and had decreased levels of Hurler-specific urine glycosaminoglycans, the toxic metabolites implicated in disease. Magenta intends to complete enrollment in the Phase 2 trial in 2020 and continue dialogue with the FDA under the RMAT designation on design of a registration-enabling study, and to have discussions with the European Medicines Agency for development in Europe.

Appointed Chief People Officer and SVP of Manufacturing: In February, Magenta announced that it had expanded its senior leadership with two new strategic hires, Kristen Stants as Chief People Officer and Li Malmberg, Ph.D., as Senior Vice President, Head of Manufacturing. Ms. Stants is a seasoned human resources professional who joined Magenta from Alexion Pharmaceuticals, where she served as Head of Talent Strategy, responsible for organizational development and talent acquisition to expand the company’s therapeutic pipeline. Dr. Malmberg is an accomplished technical leader with more than 25 years of manufacturing experience, coming to Magenta from Celgene Corporation, where she served as Vice President, Head of Biologics Development and Manufacturing, responsible for the company’s manufacturing development and biologics manufacturing organization and advanced more than 20 biologics molecule and launched one commercial product.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019, were $145.7 million, compared to $142.6 million on December 31, 2018. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the fourth quarter of 2021.

Research and Development Expenses: Research and development expenses were $18.7 million in the fourth quarter of 2019, compared to $12.4 million in the fourth quarter of 2018. The increase was driven primarily by investments in manufacturing related to our conditioning programs and MGTA-456, increases in personnel to support a clinical-stage company, as well as clinical activities for MGTA-145.

General and Administrative Expenses: General and administrative expenses were $5.9 million for the fourth quarter of 2019, compared to $5.5 million for the fourth quarter of 2018. The increase was primarily due to an increase in personnel and facilities associated with the growth of the Company.

Net Loss: Net loss was $23.2 million for the fourth quarter of 2019, compared to net loss of $16.7 million for the fourth quarter of 2018.

On March 3, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the Company’s lead drug candidate, tipifarnib, for the treatment of adult patients with relapsed or refractory angioimmunoblastic T-cell lymphoma (AITL), follicular T-cell lymphoma (FTCL) and nodal peripheral T-cell lymphoma with T follicular helper (TFH) phenotype (Press release, Kura Oncology, MAR 3, 2020, View Source [SID1234555122]).

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"This important designation from the FDA comes just two months after tipifarnib was awarded Fast Track for the treatment of patients with HRAS mutant head and neck squamous cell carcinomas (HNSCC)," said Bridget Martell, M.A., M.D., Acting Chief Medical Officer of Kura Oncology. "We believe that this designation reflects tipifarnib’s significant potential in these devastating disease settings, and we are now actively preparing to initiate a second registration-directed trial of tipifarnib in advanced nodal lymphomas of TFH phenotype, including AITL."

Fast Track designation is granted by the FDA for products that are intended for the treatment of serious or life-threatening disease or conditions, which demonstrate the potential to address an unmet medical need. The designation offers the opportunity for frequent interactions with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, as well as eligibility for rolling submission of a New Drug Application.

In December 2019, Kura reported updated clinical data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting showing robust and durable activity from tipifarnib as a monotherapy in relapsed or refractory AITL. The data demonstrated an objective response rate (ORR) of approximately 50% in a heavily pre-treated patient population, with a median of three prior regimens. Additionally, enhanced anti-tumor activity was observed in patients who carried mutations in the killer-cell immunoglobulin-like receptor, or KIR, a CXCL pathway-associated biomarker. These patients had an ORR of 70% and a complete response rate of 40%.

About T-Cell Lymphomas

Peripheral T-cell lymphomas compromise up to 20% of all aggressive non-Hodgkin lymphomas and consist of many different subtypes of fast-growing lymphomas, representing approximately 20,000 incident cases per year worldwide. Outcomes for these patients are poor, with 5-year survival of approximately 30%. Although several drugs have been approved in the relapsed and/or refractory setting, none has led to a survival benefit. In addition, the National Comprehensive Cancer Network guidelines currently recommend clinical trials for these patients. Significant advances in the genetic landscape of T-cell lymphomas have led to revisions to the World Health Organization classification and the introduction of new entities. As a result, cases of AITL, an aggressive form of T-cell lymphoma frequently characterized by high levels of CXCL12 expression, are now unified with FTCL under the classification of nodal lymphomas of TFH phenotype.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent, selective farnesyl transferase inhibitor in-licensed from Janssen in December 2014. Previously, tipifarnib was studied in more than 5,000 cancer patients, showing compelling and durable anti-cancer activity in certain patient subsets. However, no molecular mechanism of action was determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next-generation sequencing and emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib. The Company has received multiple issued patents for tipifarnib in the U.S. and foreign countries, including one issued by the U.S. Patent and Trademark Office in September 2019 that further extends Kura’s exclusivity to the use of any farnesyl transferase inhibitor for the treatment of CXCL12-expressing peripheral T-cell lymphoma and acute myeloid leukemia.

On March 3, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported the pricing of its previously announced registered underwritten public offering of 6,250,000 shares of its common stock at a price to the public of $24.00 per share (Press release, Karyopharm, MAR 3, 2020, View Source [SID1234555121]). The gross proceeds to Karyopharm from the offering, before deducting the underwriting discounts and commissions and other estimated offering expenses, are expected to be $150.0 million. The offering is expected to close on or about March 6, 2020, subject to customary closing conditions. In addition, Karyopharm has granted the underwriters a 30-day option to purchase up to 937,500 additional shares of its common stock.

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J.P. Morgan, Morgan Stanley and Jefferies are acting as joint book-running managers for the offering. RBC Capital Markets, Baird and H.C. Wainwright & Co. are acting as co-managers for the offering.

Karyopharm intends to use the net proceeds of the offering (i) to maintain and grow the infrastructure to support the continued commercialization of selinexor in the United States, including further developing our sales, marketing and market access functions along with related general and administrative capabilities; (ii) to support continued clinical development of selinexor in hematologic malignancies and solid tumors; (iii) to conduct activities to support regulatory submissions for oral selinexor as a potential second line therapy for patients with relapsed or refractory multiple myeloma and as a potential new treatment for patients with relapsed/refractory diffuse large B-cell lymphoma; (iv) for conducting clinical trials of two of our pipeline drug candidates in oncology, eltanexor, a second-generation SINE compound, and KPT-9274, a dual acting p21-activated kinase 4 (PAK4) allosteric modulator and nicotinamide phosphoribosyltransferase (NAMPT) inhibitor; and (v) for working capital and other general corporate purposes.

An automatically effective shelf registration statement relating to the shares of common stock offered in the public offering described above was filed with the Securities and Exchange Commission (SEC) on February 26, 2020. The securities may be offered only by means of a written prospectus, including a prospectus supplement, forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from J.P. Morgan Securities LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204, or by email at [email protected]; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attention: Prospectus Department; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by email at [email protected], or by phone at (877) 821-7388.