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January 2020 corporate presentation

On January 13, 2020 Geron Corporation presented the corporate presentation (Presentation, Geron, JAN 13, 2020, View Source [SID1234553054]).

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Updated Corporate Slide Presentation

On January 13, 2020 Oncternal Therapeutics Presented the corporate Presentation (Presentation, Oncternal Therapeutics, JAN 13, 2020, View Source [SID1234553053]).

MorphoSys and Incyte Sign Global Collaboration and License Agreement for Tafasitamab

On January 13, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) and Incyte Corporation (NASDAQ: INCY) reported that the companies have entered into a collaboration and license agreement to further develop and commercialize MorphoSys’ proprietary anti-CD19 antibody tafasitamab (MOR208) globally (Press release, MorphoSys, JAN 13, 2020, View Source [SID1234553049]). Tafasitamab is an Fc-engineered antibody against CD19 currently in clinical development for the treatment of B cell malignancies. MorphoSys and Incyte will co-commercialize tafasitamab in the U.S., while Incyte has exclusive commercialization rights outside of the U.S.

"The global partnership with Incyte is an important step towards unlocking the full potential of tafasitamab and achieving our goal of rapidly bringing tafasitamab to patients inside and outside of the U.S.," said Jean-Paul Kress, M.D., Chief Executive Officer of MorphoSys. "The combination of our strong antibody and drug development expertise partnered with Incyte’s well-established hematology-oncology experience and their commercial operations in key territories has the potential to significantly broaden the tafasitamab opportunity. We are pleased to work with Incyte to jointly improve the lives of patients suffering from DLBCL and other devastating diseases."

"Bringing together Incyte’s expertise and MorphoSys’ commitment to innovation will allow us to make tafasitamab widely available to patients with cancer, upon approval," said Hervé Hoppenot, CEO of Incyte. "We look forward to collaborating closely with the team at MorphoSys and adding tafasitamab to our portfolio of oncology candidates as part of our committment to bringing new, advanced treatment options to patients and the clinical community around the world."

Under the terms of the agreement, MorphoSys will receive an upfront payment of $750 million and, in addition, Incyte will make an equity investment into MorphoSys of $150 million in new American Depositary Shares (ADS) of MorphoSys at a premium to the share price at signing of the agreement. Depending on the achievement of certain developmental, regulatory and commercial milestones, MorphoSys will be eligible to receive milestone payments amounting to up to $1.1 billion. MorphoSys will also receive tiered royalties on ex-U.S. net sales of tafasitamab in a mid-teens to mid-twenties percentage range of net sales.

In the U.S., MorphoSys and Incyte will co-commercialize tafasitamab, with MorphoSys leading the commercialization strategy and booking all revenues from sales of tafasitamab. Incyte and MorphoSys will be jointly responsible for commercialization activities in the U.S. and will share profits and losses on a 50:50 basis. Outside the U.S., Incyte will have exclusive commercialization rights, and will lead the commercialization strategy and book all revenues from sales of tafasitamab, paying MorphoSys royalties on ex-U.S. net sales.

Furthermore, the companies will share development costs associated with global and U.S.-specific trials at a rate of 55% (Incyte) to 45% (MorphoSys); Incyte will cover 100% of the future development costs for trials that are specific to ex-U.S. countries.

Both parties have agreed to co-develop tafasitamab broadly in relapsed/refractory diffuse large B cell lymphoma (r/r DLBCL), frontline DLBCL as well as additional indications beyond DLBCL, such as follicular lymphoma (FL), marginal zone lymphoma (MZL) and chronic lymphocytic leukemia (CLL). Incyte will be responsible for initiating a combination study of its investigational PI3K-delta inhibitor parsaclisib and tafasitamab in r/r B cell malignancies. Further, Incyte will be responsible for leading any potential registration-enabling studies in CLL and a phase 3 trial in r/r FL/MZL. MorphoSys will continue to be responsible for its currently ongoing clinical trials of tafasitamab in non-Hodgkin lymphoma (NHL), CLL, r/r DLBCL and frontline DLBCL. The parties will share responsibility in starting additional global trials, and Incyte intends to pursue development in additional territories including Japan and China.

MorphoSys recently submitted a Biologics License Application (BLA) for tafasitamab, in combination with lenalidomide, to the U.S. Food and Drug Administration (FDA) for the treatment of r/r DLBCL; the FDA decision regarding a potential approval is expected by mid-2020. The submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in r/r DLBCL is planned for mid-2020.

The agreement between MorphoSys and Incyte, including the equity investment, is subject to clearance by the U.S. antitrust authorities under the Hart-Scott-Rodino Act as well as by the German and Austrian antitrust authorities, and will become effective as soon as these conditions have been met.

MorphoSys and Incyte will host a joint conference call on January 13, 2020 at 7:00am PST/ 4:00pm CET.

Dial-in numbers for the conference call on Monday, January 13, 2020 at 7:00am PST; 3:00pm GMT; 10:00am EST; 04:00pm CET:

For Germany: +49 69 201 744 220
For the U.K.: +44 203 009 2470
For the U.S.: +1 877 423 0830

Participant PIN: 55656540#

Please dial in 10 minutes before the beginning of the conference.

A live webcast will be made available at www.morphosys.com and at investor.incyte.com.

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. MorphoSys is clinically investigating tafasitamab 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 relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). Based on interim data from L-MIND, in October 2017 the U.S. FDA granted Breakthrough Therapy Designation for tafasitamab plus lenalidomide in this patient population. Re-MIND, the real-world data lenalidomide alone matched control cohort met its primary endpoint in October 2019, demonstrating clinical superiority of the tafasitamab/lenalidomide combination compared to lenalidomide alone. 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 relapsed/refractory CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

Ambrx and Sino Biopharma Announce Collaboration to Develop Two Next-Generation Biologics

On January 12, 2020 Ambrx Inc., a clinical-stage biopharmaceutical company focused on the development of next generation biologics using an expanded genetic code and Sino Biopharmaceutical Limited (1177.HK), a leading Chinese pharmaceutical company with a large number of medical products covering multiple therapeutic areas, reported a joint collaboration to develop two products enabled by Ambrx’s unique non-natural amino acid incorporation technology platforms (Press release, Ambrx, JAN 12, 2020, View Source [SID1234553077]).

Under the agreement, Ambrx received an upfront payment to create two innovative drug candidates, with Sino Biopharma then leading IND enabling activities for both China and the US. The parties will jointly develop the programs to world-class standards with Sino Biopharma commercializing the products within the greater China area and Ambrx commercializing the products rest-of-world. Ambrx is eligible to receive additional milestone payments for development, regulatory, and sales-based events, as well as tiered royalties on future sales.

"We are delighted to join forces with Ambrx, a leader in precision engineered biologics and breakthrough protein therapeutics, to develop two next generation cancer drugs" commented Stephen Hsin Tse, Executive Director of Sino Biopharma. "This collaboration helps fulfill our strong commitment to develop and commercialize innovative biological drugs for the China market."

The collaboration combines Ambrx’s clinically validated ReCODE and EuCODE platforms with Sino Biopharma’s strong resources and commitment to bring nextgeneration biologic drugs to market.

"Sino Biopharma is a large, unique and fast-growing powerhouse in the Chinese pharmaceutical industry, combining strong drug development expertise with substantial commercial resources and infrastructure. We are delighted to collaborate with Sino Biopharma as we continue to push the boundaries of next generation biologics with our proprietary technologies." said Feng Tian, Ph.D., President and CEO of Ambrx. "We are very excited about the future of these two novel first-in-class and best-in-class drug candidates and look forward to working with Sino Biopharma to unleash the therapeutic potential of those drug candidates."

Ascendis Pharma A/S Provides Pipeline Update and Reviews Progress Towards Vision 3×3 at 38th Annual J.P. Morgan Healthcare Conference

On January 12, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address significant unmet medical needs, reported that it will provide an outlook for 2020 and review progress towards Vision 3×3, the company’s strategic roadmap through 2025 to achieve sustainable growth at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Ascendis Pharma, JAN 12, 2020, View Source [SID1234553048]).

"2019 was a transformative year for Ascendis Pharma. For our endocrinology rare disease portfolio, we were successful in our first phase 3 trial for a TransCon product candidate, TransCon hGH, and we advanced two other programs globally into phase 2. In addition, we established oncology as our second therapeutic area with a highly-differentiated pipeline leveraging the TransCon technology," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "As groundbreaking as 2019 was for Ascendis, it was only the beginning of our path to build a leading fully-integrated global biopharma company. We are on track with our Vision 3×3 goals to deliver multiple sources of sustainable growth as we seek to make a meaningful difference in patients’ lives."

Pipeline Updates and 2020 Outlook

TransCon hGH: TransCon hGH is an investigational long-acting prodrug of human growth hormone (hGH) in phase 3 development as a once-weekly treatment for growth hormone deficiency (GHD). TransCon hGH releases unmodified somatropin and has demonstrated a statistically significant increase in height velocity compared to a daily hGH in the phase 3 heiGHt Trial:

Ascendis Pharma recently held two pre-BLA meetings with the U.S. Food and Drug Adminstration (FDA) to review its Chemistry, Manufacturing and Controls (CMC), and clinical/non-clinical packages for TransCon hGH as a potential treatment for pediatric GHD. The company is on track to file a Biologics License Application with the FDA in the second quarter. A Marketing Authorisation Application to the European Medicines Agency is planned to follow in the fourth quarter.
Long-term data presented from the ongoing enliGHten Trial (long-term extension) continued to demonstrate statistically superior growth of subjects treated with once-weekly TransCon hGH in the heiGHt Trial who continued into enliGHten, compared to those who started treatment with daily Genotropin and switched to TransCon hGH after one year. The adverse event profile of TransCon hGH, which was comparable to Genotropin in the phase 3 heiGHt Trial, was consistent across the phase 3 clinical trials.
The company plans to submit regulatory filings to initiate a global, phase 3 clinical trial in adult GHD during the first quarter, and to initiate a trial in pediatric GHD in Japan during the fourth quarter.

TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours each day and fully address all aspects of the disease:

Following completion of screening of subjects in the recently expanded phase 2 PaTH Forward Trial, Ascendis intends to enroll approximately 55 subjects in the trial. The company expects to report top-line results from the trial around the end of March 2020, with six-month data from the open-label extension phase expected in the third quarter.
Preliminary data presented from the first eight subjects who completed four weeks of follow-up in the open-label extension portion of the phase 2 PaTH Forward Trial reinforce the company’s target product profile for TransCon PTH as a promising new potential therapy for HP in the absence of standard of care.
The company plans to submit regulatory filings to initiate a global, phase 3 clinical trial in adults with HP during the fourth quarter.

TransCon CNP: TransCon CNP is an investigational long-acting prodrug of CNP in development as a therapy for children with achondroplasia, the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose:

Ascendis is conducting the phase 2 ACcomplisH Trial of TransCon CNP in children (ages 2-10 years) with achondroplasia and plans to escalate sequential dose cohorts throughout 2020.
The company is expanding the TransCon CNP program in China through its strategic investment in VISEN Pharmaceuticals, with initiation of a second phase 2 trial in children with achondroplasia during the fourth quarter.

Oncology: Ascendis continues to advance a pipeline of multiple pre-clinical programs in oncology by applying both systemic and sustained localized TransCon technologies for clinically validated pathways:

Additional data from non-human primate studies demonstrated that a single dose of TransCon IL-2 b/g provided biased receptor binding and prolonged enhancement of lymphocyte counts, suggesting feasibility of every three week dosing and reduced risk of toxicity.
Ascendis Pharma’s innovative TransCon technology for sustained localized release intratumorally (IT) was accepted to participate in the FDA’s Emerging Technology Program. The program provides for enhanced interactions and dialogue with the FDA to discuss, identify and resolve potential technical and CMC regulatory questions related to the TransCon sustained IT programs prior to filing regulatory submissions.
The company plans to file an IND or equivalent for its first oncology program in 2020, furthering the goal to create best-in-class oncology therapeutics.
Presentation at J.P. Morgan Healthcare Conference on Monday, January 13

Live webcasts of the J.P. Morgan presentation and associated Question & Answer session will be available in the Investors and News section of the Ascendis Pharma website at:

View Source

The presentation will begin at 8:00 a.m. Pacific Time, followed by the Question & Answer session at 8:30 a.m. A webcast replay will also be available for 30 days.

The company’s corporate investor presentation and slides from the J.P. Morgan presentation are also available in the Investors and News section.