On October 10, 2018 Horizon Pharma plc (Nasdaq: HZNP) reported that its third-quarter financial results will be released on Wednesday, Nov. 7, 2018 (Press release, Horizon Pharma, OCT 10, 2018, View Source [SID1234529838]). Following the announcement, Horizon’s management will host a live webcast at 8 a.m. Eastern Time to review the Company’s financial and operating results.

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The live webcast and replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes before the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

On October 10, 2018 Vaccibody AS, a clinical stage immunotherapy company focused on developing personalized neoantigen cancer vaccines to target solid tumors, reported that the first 10 patients have been enrolled in its phase I/IIa cancer neoantigen vaccine trial and that vaccinations with VB10.NEO have started (Press release, Vaccibody, OCT 10, 2018, View Source [SID1234529872]). The trial is planned to enroll up to 40 patients with locally advanced or metastatic melanoma, non-small cell lung carcinoma, clear renal cell carcinoma as well as urothelial cancer or squamous cell carcinoma of the head and neck. The VB10.NEO vaccine is given in combination with standard of care checkpoint inhibitors.

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Mads Axelsen, MD, Chief Medical Officer in Vaccibody, said "We are very pleased with the enrolment in the neoantigen trial and with the interest we are experiencing from clinical investigator and from patients. To that end I would like to thank the experienced cancer experts and investigators in this trial namely Prof. Jürgen Krauss from Heidelberg, Prof. Angela Krackhardt from Munich and Prof. Elke Jäger from Frankfurt. Together with their dedicated teams they are doing an outstanding job with the neoantigen trial"

On October 10, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes (MDS), reported that the Company will be presenting at the BIO Investor Forum, October 17-18, 2018, at the Westin St. Francis Hotel in San Francisco (Press release, Onconova, OCT 10, 2018, View Source [SID1234529839]). Ramesh Kumar, CEO, will present at the conference and meet with investors.

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Presentation details
Date/Time: Thursday, October 18th at 11:00 AM
Venue: The Westin St. Francis, San Francisco
Presenter: Ramesh Kumar, CEO

On October 10, 2018 Constellation Pharmaceuticals, Inc. (Nasdaq: CNST), a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported several planned updates to the Phase 2 portion of its ongoing MANIFEST study, an open-label Phase 1/2 clinical trial of its BET inhibitor CPI-0610 in MF (Press release, Constellation Pharmaceuticals, OCT 10, 2018, View Source [SID1234530620]).

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MANIFEST is evaluating CPI-0610, either as a monotherapy or in combination with ruxolitinib, in patients with MF who are refractory or intolerant or have relapsed or lost response to the standard of care. Based on encouraging preliminary data in this trial, Constellation is amending the design of each second-line cohort to stratify all patients enrolled in the study based on transfusion dependence status. In addition, the Company is amending the design of MANIFEST to include a third cohort designed to evaluate treatment with CPI-0610 in combination with ruxolitinib as a first-line therapy in JAK 1/2-inhibitor-naïve MF patients.

The Company made these updates based on the unmet medical need for disease-modifying therapies in myelofibrosis, especially among patients requiring red-blood-cell transfusions. The changes are intended to enhance the clinical trial design by providing additional measures of potential clinical benefit and to expand the potential addressable patient population for CPI-0610.

"We are encouraged by the preliminary data from MANIFEST, which may indicate synergistic and disease-modifying effects of CPI-0610 in myelofibrosis," said Adrian Senderowicz, Senior Vice President and Chief Medical Officer of Constellation Pharmaceuticals. "We are enhancing the study’s design to better measure these potential effects. Among the key goals of MANIFEST are identifying the most appropriate endpoints and patient populations for future pivotal studies. We continue to look forward to determining proof of concept for CPI-0610 in MF by mid-2019."

MANIFEST Enhancements

The Phase 2 proof-of-concept portion of MANIFEST in a second-line setting is designed to study the anti-tumor activity and safety of the BET inhibitor CPI-0610 in treatment of patients with MF as a monotherapy and in combination with the JAK-1/2 inhibitor ruxolitinib. This portion of the study had been expected to enroll 70 patients (35 on monotherapy and 35 on combination therapy) to evaluate safety, pharmacokinetics, reduction in spleen volume, and patient-reported symptom improvement in all enrolled patients, as well as improvements in red-blood-cell transfusion independence rate in patients who were transfusion-dependent at baseline. Preliminary data from this trial demonstrated spleen volume reduction, symptom improvement, and positive hemoglobin effects, which Constellation believes may be the result of improved hematopoiesis. In addition, as Constellation reported previously, one of four patients treated with CPI-0610 was transfusion-dependent at baseline and later converted to transfusion independence.

As a result of this positive preliminary hematological data, all enrolled patients in both the combination and monotherapy arms will now be stratified by transfusion dependence status, with each cohort expected to enroll up to 16 transfusion-dependent patients and up to 25 non-transfusion-dependent patients. Proof of concept in these cohorts will be based on conversion to transfusion independence and other factors in transfusion-dependent patients; and by reduction in spleen volume, patient-reported symptom improvement, and other factors in non-transfusion-dependent patients.

In addition, Constellation is adding a third cohort to this trial, which will evaluate treatment with CPI-0610 in combination with ruxolitinib as first-line therapy in up to 43 JAK 1/2-inhibitor-naïve MF patients. This cohort will only enroll anemic patients. Constellation’s current plan for a potential pivotal trial of CPI-0610 as a first-line treatment would be to enroll all-comers. Proof of concept for this cohort will be measured by reduction in spleen volume, patient-reported symptom improvement, and other factors.

The Company expects to update this protocol change at View Source under study ID NCT02158858 in the near future.

Conference Call/Corporate Presentation

Constellation’s CEO Jigar Raythatha and CMO Adrian Senderowicz will host a conference call to discuss this protocol change in greater detail at 8:30 AM EDT on Thursday, October 11. To participate in the conference call, please dial (877) 473-2077 (US) or +1(661) 378-9662 (international) and refer to conference ID 4165317. A live webcast will be available in the investor section of the company’s website. The webcast will be archived for 60 days following the call. Constellation has posted an updated version of its corporate presentation in the investor section of the company’s website.

On October 10, 2018 Gotham Therapeutics, a biotechnology company developing a novel drug class targeting RNA-modifying proteins, reported with a $54 million Series A financing co-led by founding investor Versant Ventures, Forbion and S.R. One (Press release, Gotham Therapeutics, OCT 10, 2018, View Source [SID1234550889]). The syndicate also included Celgene Corporation and Alexandria Venture Investments. Gotham is part of New York’s rapidly growing biopharma community with a subsidiary at one of Europe’s leading life science clusters near Munich, Germany.

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Versant created and seeded Gotham based upon the seminal discoveries of co-founder Samie Jaffrey, M.D. Ph.D., a pioneer in an exciting new field within RNA metabolism called epitranscriptomics. Dr. Jaffrey is a professor of pharmacology at Weill Cornell Medicine and a member of the Scientific Advisory Board for Gotham Therapeutics. His work has shed light on the role of post-transcriptional mRNA modifications in health and disease. These modifications and their biological effects are driven by protein complexes commonly described and categorized as writers, erasers and readers of the epitranscriptomic code.

With its seed funding, Gotham built a platform to assess the impact of RNA-modifying proteins on disease biology and developed small molecules against priority targets. The Series A proceeds will allow Gotham to establish clinical proof of concept and to invest broadly in a pipeline of preclinical candidates that have potential to treat diseases intractable to classical approaches.

"As we pursue several important targets, the information we glean will help us further validate and build our platform for increasingly broad applications. Our goal is to become the leader in drugging key proteins that modulate mRNA functionality, thereby impacting disease onset and progression," said Lee Babiss, Ph.D., CEO of Gotham.

"While academic research and the pharmaceutical industry focused initially on modifications of DNA, a growing body of evidence indicates that mRNA modifications help determine to which degree genes are translated into proteins. RNA modifications and their associated protein complexes therefore represent an untapped frontier that could yield new therapeutic approaches," added Dr. Jaffrey.

Gotham has assembled an experienced founding team led by Dr. Babiss, former President of Pharma Research at Roche. Dr. Babiss is an early adopter of RNA drug discovery approaches who has a track record of translating discoveries into therapeutic candidates. He also has served as CSO of PPD, and as Head of Human Genetics and Personalized Healthcare at Glaxo Wellcome.

"After following the developments in the RNA drug discovery field for a number of years, we felt this was the right time to build a company that could capitalize on translating the scientific discoveries into a whole new class of drug candidates," said Carlo Rizzuto, Ph.D., Partner at Versant. "With our initial investment, the Gotham team constructed a platform able to validate critical links between specific types of RNA modifications and disease biology. We look forward to advancing a number of drug candidates with this new round of financing."

"We are excited to invest in Gotham, one of the pioneers in the fast-emerging field of RNA metabolism, which could create a paradigm shift in both cancer therapy and other major diseases," commented Holger Reithinger, Ph.D., General Partner at Forbion. "Gotham represents the first investment by our recently announced Forbion IV fund. Our new fund aims to help build leading companies around exciting new science, proven teams or in-licensed assets."

Dr. Reithinger will join Dr. Babiss, Dr. Rizzuto and Jill Carroll, Principal, SR One, on Gotham’s Board of Directors. Jorge DiMartino M.D., Ph.D., Vice President, Translational Development Oncology at Celgene and Head of Celgene’s Epigenetics Thematic Center of Excellence, has joined the board in an observer role.