On January 7, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update, which will be detailed as part of the company’s presentation at the 37th Annual J.P. Morgan Healthcare Conference on Tuesday, January 8th at 5:00 p.m. PT. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will highlight the company’s growth and diversification focused on bringing clinically differentiated, life-changing treatments to patients affected by rare disorders (Press release, PTC Therapeutics, JAN 7, 2019, View Source [SID1234532571]). Additionally, an overview of the company’s strategic vision will be provided as well as preliminary 2018 financial results and 2019 financial guidance. The presentation will be webcast live on the Events and Presentations page under the investors’ section of PTC Therapeutics’ website at www.ptcbio.com.

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Corporate Highlights

Advancing gene therapy portfolio

PTC plans to submit a BLA with the FDA followed by an MAA in Europe for the AADC deficiency gene therapy program in 2H 2019 with expected launch in 2020. Identification of patients with AADC deficiency has been a priority for the Company, with approximately 100 patients identified to date in the U.S. and Europe. PTC expects to screen about 100,000 patients who are at risk for AADC deficiency before the regulatory approval to maximize patient benefit at time of launch.

Friedreich’s ataxia program is advancing with an expected IND filing and entry into the clinic in 2019.

PTC is enhancing its internal research and in-house manufacturing capabilities with fully dedicated resources to maximize current and future programs.

Risdiplam regulatory filing progress. The SMA program is a collaboration between PTC, Roche and SMA Foundation.

Successfully completed enrollment of pivotal portion of FIREFISH trial in Type 1 SMA babies in 2018.

Regulatory submission targeted for the second half of 2019 based on recent feedback from the FDA & national health authorities in Europe that Part 1 of FIREFISH and SUNFISH may be sufficient to file NDA/MAA.

The goal of the clinical development program is to support the approval of a broad label so risdiplam can potentially benefit as many SMA patients as possible.

Short- and long-term cash milestones to PTC expected upon regulatory approval and commercial launches. PTC expects the peak annual royalties/milestones from risdiplam to exceed $200M.

Expanding commercial platform

TEGSEDI application filed with ANVISA- Brazilian regulatory authority. ANVISA granted priority review. PTC expects approval in Brazil by year end 2019. TEGSEDI has the potential to generate peak revenues of approximately $150M in LATAM.

Duchenne franchise expected to continue to grow over the next 5 years. Translarna ex-U.S. launch in patients 2 to 5 years of age now initiated. Non-ambulatory label expansion is currently under EMA regulatory review. Emflaza continues to accrue new patients in the U.S. with expected increased market share gained via differentiation and improved market access.

In recent interactions, the FDA invited PTC to submit a supplementary NDA (sNDA) for Emflaza for patients 2 to 5 years of age on the basis that existing data support its safety and efficacy in this population. PTC recently submitted the sNDA for potential approval in 2019. The previous written request from the FDA that a trial in patients 2 to 5 years of age be performed has been officially withdrawn and the trial will no longer be conducted. PTC now expects to launch Emflaza in this younger population before the end of 2019.

Growing pipeline and R&D capabilities

PTC’s alternative splicing platform has generated another development candidate. PTC258 was selected as a development candidate for Familial dysautonomia (FD), a rare genetic neurological disorder that effects the sensory and autonomic nervous systems, causing life-threatening medical complications from birth. PTC258 is advancing to IND enabling studies with the goal to enter the clinic in late 2019. This program is in collaboration with MGH and NYU.

Translarna’s dystrophin study was initiated in 4Q 2018 for potential U.S. regulatory submission in early 2020.

PTC’s oncology portfolio continues to advance with the initiation of a study in AML with PTC299 and a DIPG study for PTC596. PTC expects these studies to move to the expanded cohort stage in 2020. PTC596 is also being developed for the treatment of patients with Leiomyosarcoma (LMS) with the first patient in that trial expected to be dosed in Q1 2019.

Preliminary Unaudited 2018 Financial Results

PTC expects to report Translarna (ataluren) net product revenue for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $171 million for full year 2018, an increase of 18% over the prior year.

PTC expects to report EMFLAZA (deflazacort) net product revenue for the treatment of Duchenne muscular dystrophy (DMD) of approximately $91 million for full year 2018, an increase from $28.8 million in the prior year.

PTC expects to report year-end cash and cash equivalents of approximately $227 million.

PTC is currently in the process of finalizing its financial results for the 2018 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2018, subject to the completion of PTC’s financial closing procedures. In addition, the above information is subject to revision as PTC completes its financial closing procedures for fiscal 2018.

2019 Guidance

PTC anticipates full year net product revenues to be between $285 and $305 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2019 to be between $395 and $405 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2019 to be between $360 and $370 million, excluding estimated non-cash, stock-based compensation expense of approximately $35 million.

On January 7, 2019 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported its key 2019 initiatives in conjunction with its presentation at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Agios Pharmaceuticals, JAN 7, 2019, View Source [SID1234532570]). The company will webcast its presentation today at 9:30 a.m. PT (12:30 p.m. ET) at investor.agios.com.

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"During 2018, just ten years after the founding of Agios, we achieved approval of our second internally discovered oncology medicine, launched a robust registrational program in PK deficiency and successfully opened the company’s seventh IND," said David Schenkein, M.D., chief executive officer at Agios. "Our validated research platform and proven drug development strategy are poised to help drive future growth across our oncology and rare genetic disease portfolios. Our priorities for 2019 include expanding the reach of our IDH inhibitors into the frontline AML and solid tumor settings, completing enrollment in two pivotal studies of mitapivat and exploring the utility of PKR activators in other hemolytic anemias, and furthering clinical development for AG-270 in MTAP deleted tumors and AG-636 in lymphoma."

The company plans to achieve the following milestones in 2019.

Cancer:

Potential FDA approval of the supplemental new drug application (sNDA) for single agent TIBSOVO (ivosidenib) for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy.

Submit a sNDA to the FDA for TIBSOVO for second line or later IDH1m cholangiocarcinoma by year-end.

Initiate a registration-enabling Phase 3 study of vorasidenib (AG-881) in low-grade glioma with an IDH1 mutation by year-end.

Determine recommended dose of AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors and initiate expansion arms, including a single agent arm in a variety of MTAP deleted cancers and a combination arm in a solid tumor in the first half of 2019.

Initiate a Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in lymphoma in the first half of 2019.

Rare Genetic Diseases:

Complete enrollment in two global pivotal trials for mitapivat in adults with PK deficiency by year-end 2019:

ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients

ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions

Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.

The company highlighted select data presentations expected in 2019.

Updated data from the ongoing Phase 1 combination trial of TIBSOVO with azacitidine in patients with newly diagnosed AML with an IDH1 mutation in the first half of 2019.

Data from the perioperative ‘window’ trial with TIBSOVO and AG-881 in IDHm low-grade glioma in the first half of 2019.

Topline data from the Phase 3 ClarIDHy study of TIBSOVO in IDH1m advanced cholangiocarcinoma to be reported in the first half and full data to be presented in the second half of 2019.

Data from the dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with MTAP-deleted tumors in the second half of 2019.

The company also provided an update on the following 2018 milestones achieved in December:

Submitted an sNDA to the FDA for TIBSOVO for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy.

Submitted a Marketing Authorization Application to the European Medicines Agency for TIBSOVO for the treatment of adult patients with R/R AML with an IDH1 mutation.

Initiated a Phase 2 proof-of-concept trial of mitapivat in thalassemia.

2018 Year-End Cash and Guidance

Agios ended 2018 with approximately $805 million of cash, cash equivalents and marketable securities. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2018, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

Presentation at 37th Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 37th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 7, 2019 at 9:30 a.m. PT (12:30 p.m. ET). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation

On January 7, 2019 Moleculin Biotech, Inc is presented the corporate presentation (Presentation, Moleculin, JAN 7, 2019, View Source [SID1234532567]).

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On January 7, 2019 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical stage biotechnology company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported several executive promotions. The company also provided corporate updates, including selected fourth quarter 2018 financial results, financial guidance and corporate milestones for 2019 (Press release, Calithera Biosciences, JAN 7, 2019, View Source [SID1234532564]).

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"I am pleased to announce today’s promotions which highlight the talent, skill set and extensive achievements of our executive team," said Susan Molineaux, PhD, President and Chief Executive Officer of Calithera. "It is critical that we have the right leadership in place to reach key clinical milestones as we drive CB-839 towards commercialization with two randomized trials of the glutaminase inhibitor CB-839 for the treatment of patients with advanced renal cell carcinoma. We plan to report data from our Phase 2 ENTRATA trial in 2019, and the global CANTATA trial in 2020. In 2019, we look forward to a number of achievements including the initiation of additional clinical trials of CB-839 in collaboration with Pfizer, and the presentation of data on the arginase inhibitor INCB001158 at a medical meeting in the second half of 2019. Our goal is to have four unique therapeutics in the clinic including CB-280, an oral arginase inhibitor for the treatment of patients with cystic fibrosis, and CB-708, an oral small molecule CD73 inhibitor for the treatment of cancer by the end of 2019."

2019 Milestones

Calithera expects to reach the following milestones in 2019:

CB-839 data from Phase 2 renal cell carcinoma randomized ENTRATA trial 2H19. The ENTRATA trial is a randomized, double-blind trial designed to evaluate the safety and efficacy of CB-839 with everolimus versus everolimus alone in approximately 63 patients with metastatic, clear cell renal cell carcinoma patients who have been treated with at least two prior lines of therapy including a VEGFR-targeting tyrosine kinase inhibitor. The primary endpoint is progression free survival; overall survival will be assessed as a secondary endpoint.

CB-839 enrollment of the Phase 2 renal cell carcinoma CANTATA trial 2H19. The CANTATA trial is a randomized, global, double-blind trial comparing patients treated with CB-839 and cabozantinib to patients treated with cabozantinib alone. The trial will enroll approximately 400 patients with clear cell renal cell carcinoma who have previously received one or two prior lines of therapy. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for CB-839 in combination with cabozantinib for the treatment of this patient population.

CB-839 combination trial initiations in collaboration with Pfizer 1H19. Calithera and Pfizer have two clinical trial collaborations to evaluate Pfizer’s CDK4/6 inhibitor palbociclib, also known as IBRANCE, and the dual-mechanism poly (ADP-ribose) polymerase (PARP) inhibitor talazoparib also known as TALZENNA, each in combination with the glutaminase inhibitor CB-839.

INCB001158 arginase inhibitor data presentation at a medical meeting 2H19. INCB001158 is a small molecule immuno-oncology therapeutic being evaluated in multiple clinical trials as a single-agent and in combination with immunotherapies and chemotherapy for the treatment of patients with cancer. INCB001158 is being developed as part of a collaboration and license agreement with Incyte.

CB-280 IND acceptance and Phase 1 trial initiation in healthy volunteers 1H19. CB-280 is an arginase inhibitor for the treatment of cystic fibrosis. Arginase is believed to be critical in the pathology of cystic fibrosis. It impairs production of nitric oxide and generates metabolites of arginine that may impair lung function. CB-280 is an orally administered small molecule inhibitor of arginase. An investigational new drug (IND) application for CB-280 is planned for the first half of 2019.

CB-708 IND acceptance and Phase 1 trial initiation 2H19. The immuno-oncology target CD73 is an enzyme that plays a critical role in the process of ATP conversion to adenosine. An IND application for CB-708, an orally administered small molecule inhibitor of CD73, is planned for 2019.

Selected Fourth Quarter 2018 Financial Results and Financial Guidance for 2019

Based upon preliminary estimates, cash, cash equivalents and investments totaled $136.2 million at December 31, 2018. Calithera expects to utilize cash and investments between $75 and $85 million in 2019.

Key Executive Promotions

The promotions for its executive leadership team include Curtis C. Hecht, promoted to Chief Business Officer; Keith Orford M.D., Ph.D., promoted to Chief Medical Officer; and Sam Whiting, M.D., Ph.D., promoted to Senior Vice President of Clinical Development.

Mr. Hecht brings over 25 years of broad pharmaceutical experience including business development, strategic planning and commercialization. He has led business and corporate development efforts since joining Calithera in 2014. Key contributions include the Mars in-licensing agreement for arginase inhibitors, clinical collaborations with Bristol-Myers Squibb, Exelixis and Pfizer and the co-development and commercialization agreement with Incyte for INCB001158. He has built strategic portfolio management and lifecycle team functions, and leads commercial planning. Prior to Calithera, Mr. Hecht was Vice President of Business Development for inVentiv Heath Commercial Solutions. Until 2011, Mr. Hecht was at Roche in commercialization and business development roles of increasing responsibility, including the Global Alliance Director of the Roche-Genentech collaboration. Mr. Hecht has a B.S. in Chemistry from California State University, Sacramento, and an MBA from Carnegie Mellon University.

Dr. Orford has over 11 years of experience in pharmaceutical drug development in oncology and immuno-oncology. Since joining Calithera in 2015, he has overseen clinical development activities, including Clinical Operations and Medical Affairs. He has provided strategic development leadership for the company’s oncology and cystic fibrosis portfolio, including CB-839, a glutaminase inhibitor currently in late stage global clinical trials in renal cell carcinoma as well as multiple other clinical studies across a wide range of tumor types. He also serves as strategic development leader for the clinical collaboration between Calithera and Incyte. Prior to joining Calithera, Dr. Orford was the Clinical Development Lead in the Immuno-Oncology and Combinations Development Performance Unit at GlaxoSmithKline, where he oversaw the clinical activities on multiple early stage clinical trials with targeted agents and novel immune-based therapies. Prior to GlaxoSmithKline, Dr. Orford was at Merck, where he worked on early clinical development programs across oncology and other therapeutic areas. Previously, Dr. Orford was a Research Fellow and Instructor at Massachusetts General Hospital and Harvard Medical School, where he completed clinical training in Internal Medicine, as well as postdoctoral work studying the epigenetic regulation of hematopoietic and embryonic stem cell differentiation. Dr. Orford received his undergraduate, M.D., and Ph.D. degrees from Georgetown University.

Dr. Whiting joined Calithera in May 2016. Dr. Whiting’s deep expertise in clinical oncology gained as an academic medical oncologist and in pharmaceutical drug development have helped fuel the growth of the company’s pipeline. Prior to joining Calithera, Dr. Whiting served as Vice President of Research and Clinical Development at Gradalis. Dr. Whiting previously worked in development of small molecule

targeted and immune-oncology agents at VentiRx Pharmaceuticals and Oncothyreon. Previously, Dr. Whiting served as Assistant Professor of Medical Oncology at the University of Washington, Assistant Member of Clinical Research at the Fred Hutchinson Cancer Research Center, and Clinical Head of Gastrointestinal Oncology at the Seattle Cancer Care Alliance. Dr. Whiting completed fellowship training in medical oncology at the Fred Hutchinson Cancer Research Center. His training in internal medicine was at the ABIM Research Pathway at the University of Washington. Dr. Whiting received his B.S. with Honors in Chemistry from Lewis and Clark College, and his M.D. and Ph.D. as part of the Medical Scientist Training Program at the University of Washington Medical Center.

On January 7, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune disease, asthma and allergic disease and cancer, reported it will regain rights to develop and commercialize XmAb13676, a CD20 x CD3 bispecific antibody, from Novartis effective June 20, 2019, due to strategic pipeline reprioritization by Novartis (Press release, Xencor, JAN 7, 2019, View Source [SID1234532562]). Xencor granted Novartis co-development and ex-U.S. commercial rights in June 2016 through a collaboration and license agreement to develop and commercialize novel bispecific antibodies, including XmAb13676 and XmAb14045, and to access Xencor’s XmAb bispecific Fc and other Fc technologies. Currently XmAb13676 is being evaluated in an open-label Phase 1, multiple-dose, dose-escalation study to assess its safety, tolerability and preliminary anti-tumor activity in patients with B-cell malignancies, and initial data are expected in 2019.

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"We continue to work closely with Novartis across multiple programs in the collaboration, and both companies are eager to advance XmAb14045 in clinical development. Recently we presented encouraging early data from our Phase 1 study in patients with relapsed/refractory AML, observing multiple complete remissions on a weekly dosing schedule, and we continue to optimize dose in that study. Novartis also has internal XmAb preclinical bispecific programs progressing," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "Xencor will continue to develop XmAb13676 as planned, and we believe its tuned potency holds potential for the treatment of patients with B-cell malignancies."

Under the terms of the collaboration agreement, Xencor and Novartis continue to share costs for the worldwide development of XmAb14045 with Xencor maintaining U.S. commercialization rights and Novartis having commercialization rights in the rest of the world, and worldwide development costs for XmAb13676 will be shared until June 2020. Novartis received worldwide rights to Xencor’s bispecific technology to develop and commercialize four additional targets selected by Novartis. Xencor is eligible to receive clinical, regulatory and sales milestone payments for successful programs, as well as tiered, low double-digit royalties for sales of XmAb14045 outside of the U.S. and mid single-digit tiered royalties for worldwide sales of the four proprietary Novartis bispecific molecules.

About XmAb13676

XmAb13676 is a tumor-targeted antibody that contains both a CD20 binding domain and a cytotoxic T-cell binding domain (CD3) in a Phase 1 clinical trial for the treatment of B-cell malignancies. An XmAb bispecific Fc domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on XmAb13676. CD20 is highly expressed on B-cell tumors, including in chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma (NHL). Engagement of CD3 by XmAb13676 activates T cells for highly potent and targeted killing of CD20-expressing tumor cells.

About XmAb14045

XmAb14045 is a tumor-targeted antibody that contains both a CD123 binding domain and a cytotoxic T-cell binding domain (CD3) in a Phase 1 clinical trial for the treatment of acute myeloid leukemia (AML) and other CD123-expressing hematologic malignancies. An XmAb Bispecific Fc domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on XmAb14045. CD123 is highly expressed on AML cells and leukemic stem cells, and it is associated with poorer prognosis in AML patients. Engagement of CD3 by XmAb14045 activates T cells for highly potent and targeted killing of CD123-expressing tumor cells.