On May 14, 2020 Vivoryon Therapeutics AG (Euronext Amsterdam: VVY, ISIN DE0007921835) reported its first quarter business update for the period ending March 31, 2020 (Press release, Vivoryon Therapeutics, MAY 14, 2020, View Source [SID1234557966]). The first quarter 2020 report is available for download on the Company website: View Source

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KEY HIGHLIGHTS

Vivoryon Therapeutics and Nordic Bioscience Entered into a Research and Development Collaboration
Vivoryon Therapeutics Announced Update on Phase 2b Alzheimer’s Clinical Trial, VIVIAD

POST PERIOD HIGHLIGHTS

Vivoryon Therapeutics Initiated Development Program for Meprin Protease Inhibitors with Intended Therapeutic Use in Fibrosis, Cancer and Alzheimer’s Disease
Vivoryon Therapeutics Announced Outcome of Exclusive Option Deal with MorphoSys

CORPORATE REVIEW

Financial Review (According to IFRS)

In the first quarter of 2020, research and development expenses rose to EUR 2,783k compared to EUR 447k in the first quarter of 2019. This increase was mainly driven by costs associated with the ramp-up phase of the clinical Phase 2b study in Alzheimer’s disease. General and administrative expenses in the first quarter 2020 resulted in EUR 580k, compared to EUR 488k in 2019. Vivoryon’s finance expenses amounted to EUR 300k in the reporting period. In line with corporate planning, revenues were not generated in the reporting period. The net loss in the first quarter resulted in EUR 3,328k compared to EUR 910k in the first quarter of 2019.

All results are within management expectations.

Vivoryon Therapeutics held EUR 20.2 million in cash and cash equivalents and EUR 19.7 million in other short-term securities as of March 31, 2020.

OPERATIONAL REVIEW

Vivoryon Therapeutics and Nordic Bioscience entered into a Research and Development Collaboration

Vivoryon announced that it entered into a research and development collaboration with Nordic Bioscience for the clinical development of PQ912 for Alzheimer’s Disease (AD). In addition to taking on the role as CRO (Clinical Research Organization) for Vivoryon Therapeutics’ Phase 2b trail, VIVIAD, Nordic Bioscience and Vivoryon will enter into a collaboration to benefit from Nordic Bioscience’s world leading expertise in the development of blood-based biomarkers for the identification of specific patients that may benefit most from treatment with PQ912, Vivoryon’s Phase 2 clinical-stage candidate in AD.

Vivoryon Therapeutics Announced an Update on Phase 2b Alzheimer’s Clinical Trial, VIVIAD

The clinical trial, VIVIAD, derived from "advancing disease modifying treatment and non-invasive diagnostics of Alzheimer’s disease," has been designed to test the efficacy and safety of various doses of PQ912 in 250 early-stage Alzheimer’s patients in a randomized, placebo-controlled study over the course of 48 to 96 weeks.

The primary endpoints of the study will assess the safety and tolerability of PQ912 in addition to its efficacy on working memory and attention. The secondary endpoints include long-term safety and tolerability of PQ912 and its efficacy on brain activity, cognition and daily activities.

Vivoryon has also added exploratory parameters selected with the rationale of advancing less invasive diagnostic technologies. This will include the Winterlight Labs speech assessment, the use of EEG to test neuronal network activity and connectivity, as well as a set of blood-based biomarkers run by Nordic Bioscience. The inclusion of these parameters will further strengthen PQ912’s data package and introduce more innovative and less demanding diagnostic tools to patients in the future.

POST PERIOD HIGHLIGHTS

Vivoryon Therapeutics Initiated Development Program for Meprin Protease Inhibitors with Intended Therapeutic Use in Fibrosis, Cancer and Alzheimer’s Disease

In March 2020, the Company entered into a research collaboration with the Fraunhofer Institute for Cell Therapy and Immunology (IZI), acquiring related patents for a meprin protease inhibitor and assay platform, to advance first-in-class small molecule meprin inhibitors.

The collaboration will combine Vivoryon’s expertise in translating basic research into marketable small molecule therapeutics with the department’s focus on discovery and development of new treatment options that target recognized pathologic post-translational modifications.

Vivoryon Therapeutics Announced Outcome of Exclusive Option Deal with MorphoSys

MorphoSys will not execute the option deal to license Vivoryon’s small molecule QPCTL inhibitors for oncology.

Vivoryon will continue to evaluate QPCTL inhibitors in oncology based on preclinical studies conducted in collaboration with the University of Kiel and will, based on existing proof-of-concept data, remain open for opportunities to collaborate with pharma partners in upcoming clinical development steps.

Vivoryon Therapeutics´ Ordinary General Meeting of Shareholders postponed until September

The Ordinary General Meeting of Vivoryon Therapeutics will be postponed until the 2nd half of September 2020 due to coronavirus restrictions.

On May 14, 2020 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that mitapivat and ivosidenib clinical data will be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress being held virtually June 11-14, 2020 (Press release, Agios Pharmaceuticals, MAY 14, 2020, View Source [SID1234557999]).

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The accepted abstracts are listed below and are available online on the EHA (Free EHA Whitepaper) meeting library website: View Source All presentations can be accessed on demand by registered meeting attendees on the EHA (Free EHA Whitepaper) Virtual Congress platform as of Friday, June 12 at 08:30 a.m. CEST / 2:30 a.m. ET and will be accessible until October 15, 2020.

Oral Presentation:

Title: Proof of concept for the oral pyruvate kinase activator mitapivat in adults with non-transfusion-dependent thalassemia: Interim results from an ongoing, phase 2, open-label, multicenter study
Date & Time: Friday, June 12, 2020 at 8:30 a.m. CEST / 2:30 a.m. ET
Oral Abstract Session: New therapeutic approaches for thalassemia
Abstract: S297
Presenter: Kevin H. M. Kuo, M.D., Toronto General Hospital

Poster Presentations:

Title: Mitapivat (AG-348) long-term safety and efficacy in pyruvate kinase deficiency: 3-year results of the Drive PK study
Poster Session: Enzymopathies, membranopathies and other anemias
Abstract: EP1561
Author: Rachael F. Grace, M.D., Boston Children’s Hospital

Title: Ivosidenib improves overall survival relative to standard therapies in relapsed or refractory mutant IDH1 AML: Results from matched comparisons to historical controls
Poster Session: Acute myeloid leukemia – Clinical
Abstract: EP540
Author: Peter Paschka, M.D., Universitätsklinikum Ulm

Title: Ivosidenib (IVO) in patients with IDH1-mutant relapsed/refractory myelodysplastic syndrome (R/R MDS): Updated enrollment of a phase 1 dose escalation and expansion study
Poster Session: Myelodysplastic syndromes – Clinical
Abstract: EP826
Author: Courtney D. DiNardo, M.D., University of Texas MD Anderson Cancer Center

Title: Pharmacokinetic/pharmacodynamic evaluation of ivosidenib or enasidenib combined with intensive induction and consolidation chemotherapy in patients with newly diagnosed IDH1/2-mutant AML
Poster Session: Acute myeloid leukemia – Clinical
Abstract: EP620
Author: Yue Chen, Agios Pharmaceuticals

Title: Ivosidenib (IVO) prior to hematopoietic cell transplant for patients with IDH1-mutant relapsed or refractory acute myeloid leukemia (R/R AML)
Poster Session: Acute myeloid leukemia – Clinical
Abstract: EP577
Author: Courtney D. DiNardo, M.D., University of Texas MD Anderson Cancer Center

Publication Only:

Title: Agile: Phase 3, double-blind, randomized, placebo-controlled study of ivosidenib in combination with azacitidine in adults with newly diagnosed acute myeloid leukemia and an IDH1 mutation
Publication Only (in abstract book): 04. Acute myeloid leukemia – Clinical
Abstract: PB1862
Author: Pau Montesinos, M.D., Ph.D., Hospital Universitari i Politècnic La Fe

Conference Call Information
Agios will host a virtual investor event on June 12, 2020 at 7:30 a.m. ET to review the mitapivat clinical data. The event will be webcast live and can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On May 14, 2020 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of cortisol, reported that it will present novel immuno-oncology data from the selective cortisol receptor modulator relacorilant at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Corcept Therapeutics, MAY 14, 2020, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-present-data-american-society-clinical [SID1234558028]). This year’s annual meeting will be held in a virtual format from Friday, May 29 through Sunday, May 31, 2020. Following its presentation, a copy of our poster will be available at the Research & Pipeline / Publications tab of our website.

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"Patients with adrenal cancer often have tumors that produce cortisol, which may limit the efficacy of immune checkpoint inhibitors," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "Our hypothesis is that administering a selective cortisol modulator in combination with an immunotherapeutic agent such as pembrolizumab will treat the symptoms of hypercortisolism and help pembrolizumab achieve its maximum effect.1 The data presented in this abstract have informed the design of our Phase 1b trial in which 20-patients with metastatic or unresectable adrenal tumors that produce cortisol will receive our selective cortisol modulator relacorilant in addition to pembrolizumab."

Impact of relacorilant, a selective glucocorticoid receptor antagonist,
on the immunosuppressive effects of endogenous cortisol (abstract no. 3091)

Session Title: Developmental Therapeutics – Immunotherapy
Session Type: Poster session
Location: Virtual meeting
Presentation Available Online: May 29 to November 20, 2020 (meeting registration required)
About Relacorilant

Relacorilant is a non-steroidal, selective modulator of the glucocorticoid receptor, the receptor for cortisol which is activated when cortisol levels are high. Relacorilant does not bind to the body’s other hormone receptors, including the progesterone receptor. Corcept is studying relacorilant as a potential treatment for a variety of serious disorders, including Cushing’s syndrome and advanced adrenal, ovarian and pancreatic cancer. Relacorilant is proprietary to Corcept and is protected by composition of matter and method of use patents through 2037. Relacorilant has received orphan designation in the United States for the treatment of both Cushing’s syndrome and pancreatic cancer.

On May 14, 2020 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company focused on developing targeted therapies to inhibit frontier cancer targets, reported its financial results for the first quarter ended March 31, 2020, and provided an update on its R&D pipeline and other corporate developments (Press release, Revolution Medicines, MAY 14, 2020, View Source [SID1234558044]).

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Highlights from the Quarter Ended March 31, 2020

"Revolution Medicines achieved important scientific, clinical and operational milestones during this quarter," said Mark A. Goldsmith, M.D., Ph.D., president, chief executive officer and chairman of Revolution Medicines. "In January, we presented initial data from our ongoing Phase 1 monotherapy trial evaluating our investigational new drug designed to inhibit SHP2, RMC-4630, in patients with KRAS mutant non-small cell lung cancer (NSCLC) at the AACR (Free AACR Whitepaper)-IASLC International Joint Conference. The findings represent the first reported evidence of clinical activity against KRAS mutant lung cancers by a SHP2 inhibitor, as well as initial evidence of the potential benefit of an intermittent dosing schedule. Our ongoing Phase 1/2 clinical program evaluating RMC-4630 in a range of tumor types continues to advance and enrollment has been in line with our expectations. We also continued to make progress across our broad preclinical pipeline that supports our strategy to target multiple nodes in the oncogenic RAS pathway and bring forward potential monotherapies and combination treatment regimens. During the period, the company also completed a successful IPO, raising gross proceeds of more than $273 million. Revolution Medicines’ strong balance sheet will support continued development of our promising pipeline on behalf of cancer patients."

"We acknowledge the severe health and economic impact of the COVID-19 pandemic we are all experiencing and the burden it has placed on our healthcare system and the clinical trial landscape. Early on, Revolution Medicines made appropriate adjustments to our operating approach, and we’ve continued to make progress on both our preclinical and clinical programs. At present, we do not expect material delays in our ongoing clinical trials, but it is reasonable to anticipate that for planned future studies some site initiations may be delayed, and enrollment may be slowed for some period of time. We are continuing to refine our approach as needed to minimize these impacts."

Scientific and Clinical Highlights

Revolution Medicines demonstrates first ever clinical activity against KRAS mutant lung cancers with SHP2 inhibitor – In January 2020, at the 6th AACR (Free AACR Whitepaper)-IASLC International Joint Conference, the company presented preliminary evidence demonstrating that RMC-4630, the company’s investigational SHP2 inhibitor, showed initial clinical activity in patients with NSCLC bearing KRAS mutations, particularly KRASG12C. Findings also demonstrated the potential benefit of an intermittent RMC-4630 dosing schedule.

RMC-4630 clinical program continues to advance – Revolution Medicines continues to explore optimal dosing and scheduling of RMC-4630 in both its ongoing Phase 1 monotherapy and Phase 1b/2 combination therapy trials. The company plans to expand its RMC-4630 combination therapy program and is prepared for the initiation of new studies evaluating the compound in combination with Amgen’s investigational KRASG12C(OFF) inhibitor, AMG 510, with the EGFR inhibitor osimertinib (Tagrisso), and with a PD-1 inhibitor. While the COVID-19 pandemic may indirectly cause some delays in the initiation of new clinical studies, the company currently expects enrollment in these studies to begin in 2020.

RMC-5552 – IND-enabling work continuing – RMC-5552, the company’s potent and selective inhibitor of mTORC1, continues to advance through investigational new drug (IND)-enabling development. The company remains on track to be IND-ready with this compound by the end of 2020.

Mutant-selective RAS(ON) inhibitor program advancing; development candidate to be nominated – Revolution Medicines is developing a portfolio of mutant-selective RAS(ON) inhibitors that it believes may be the first potent, selective, cell-active inhibitors of the active, GTP-bound form of RAS, or RAS(ON). The company continues to make significant progress toward optimizing key properties of such inhibitors. In line with previous projections, the company continues to anticipate nomination of its first development candidate from this portfolio in 2020.

SOS1 inhibitor program advances into lead optimization. Revolution Medicines’ program targeting SOS1, a protein that plays a central role in driving oncogenic signals through the RAS pathway, continues to advance. Our growing collection of potent and selective inhibitors exhibiting attractive preclinical profiles has enabled the program to progress into the lead optimization stage in pursuit of a development candidate.

Findings recently published in Cancer Research support combination of RMC-4630 with an anti-PD-1 antibody – In a paper published on April 29, 2020, Revolution Medicines researchers described ways in which a SHP2 inhibitor enhances the immune response to tumors, representing a second group of anti-tumor mechanisms beyond its direct effects within cancer cells themselves. The paper also reported deep and durable tumor growth inhibition following combination treatment with a SHP2 inhibitor and anti-PD-1 inhibitor in mouse cancer models, yielding complete tumor regressions and sustained immunological memory.

Multiple abstracts selected for presentation at upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II – Revolution Medicines has been notified that four of the company’s submissions have been selected for presentation at the upcoming virtual AACR (Free AACR Whitepaper) meeting scheduled to take place in June 2020. Titles and abstracts of these presentations will be disclosed by AACR (Free AACR Whitepaper) on May 15, 2020.

Anticipated Scientific and Clinical 2020 Milestones

Clinical data update from RMC-4630 program

Clinical trial initiations:

RMC-4630 combination with AMG 510

RMC-4630 combination with osimertinib

RMC-4630 combination with PD-1 inhibitor

Nomination of first development candidate for RAS(ON) inhibitor program

RMC-5552 IND-readiness

Corporate Highlights

Closed initial public offering – In February 2020, Revolution Medicines closed its initial public offering of 16,100,000 shares of common stock, including the exercise in full by the underwriters of their option to purchase an additional 2,100,000 shares of common stock, at a public offering price of $17.00 per share. The gross proceeds from the offering were $273.7 million, before deducting underwriting discounts, commissions and other offering expenses payable by Revolution Medicines.

The company expects to use the net proceeds from this offering to fund the development of its multiple RAS inhibitor programs, including the RAS(ON) portfolio, SOS1 inhibitor program, and 4EBP1/mTORC1 program, and other general corporate purposes, which may include the hiring of additional personnel, capital expenditures and the costs of operating as a public company.

USPTO grants key RMC-4630 patent – In March 2020, the United States Patent and Trademark Office issued U.S. Patent No. 10,590,090 to the company, providing, in part, composition of matter protection for its SHP2 inhibitors, including RMC-4630.

Facilities Expansion – To support Revolution Medicines’ expanding operations, the company completed a lease transaction in April 2020 that will provide an additional 19,483 square feet in Redwood City, CA. The company campus now includes two buildings that house office, laboratory and research and development space.

Q1 2020 Financial Highlights

Cash Position: Cash, cash equivalents and marketable securities were $347.9 million as of March 31, 2020, compared to $122.8 million as of December 31, 2019. The increase was primarily due to proceeds from the IPO in February 2020.

Revenue: Total revenue, consisting of revenue from our collaboration agreement with Sanofi, was $11.5 million for the quarter ended March 31, 2020, compared to $13.2 million for the quarter ended March 31, 2019. This decrease was primarily due to lower reimbursed research and development services in the quarter ended March 31, 2020 resulting from lower manufacturing costs. During the quarter ended March 31, 2019, we incurred upfront manufacturing costs related to the supply of RMC-4630 for our clinical trials.

R&D Expenses: Research and development expenses were $27.5 million for the quarter ended March 31, 2020, compared to $21.2 million for the quarter ended March 31, 2019. This increase was primarily due to an increase in research expenses associated with our RAS inhibitor programs.

G&A Expenses: General and administrative expenses were $5.2 million for the quarter ended March 31, 2020, compared to $2.4 million for the quarter ended March 31, 2019. This increase was primarily due to an increase in expenses associated with transitioning to and becoming a public company.

Net Loss: Net loss was $19.5 million for the quarter ended March 31, 2020, compared to net loss of $10.1 million for the quarter ended March 31, 2019.

On May 14, 2020 Omeros Corporation (Nasdaq: OMER) reported that the results of its pivotal trial of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy will be presented at the 25th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) to be held June 11-14, 2020 (Press release, Omeros, MAY 14, 2020, View Source [SID1234558061]). This year the congress will be held virtually .

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The presentation, entitled Narsoplimab (OMS721) for the Treatment of Adult Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy, will be delivered by Alessandro Rambaldi, M.D., Professor of Hematology at the University of Milan and Head of the Hematology and Bone Marrow Transplant Unit at ASST Papa Giovanni XXIII in Bergamo, Italy. Selected by EHA (Free EHA Whitepaper) for a podium presentation, it will include efficacy data not previously presented.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as "OMS721," is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in HSCT-TMA, in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.