On November 1, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported that 35 abstracts related to Genmab owned and partnered programs have been accepted for presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place December 1-4 in San Diego, California (Press release, Genmab, NOV 1, 2018, View Source [SID1234530513]). Abstracts accepted for presentation include updates on multiple daratumumab and ofatumumab trials, as well as pre-clinical data from Genmab’s DuoBody-CD3xCD20 and DuoHexaBody-CD37 programs. All abstracts are available on the ASH (Free ASH Whitepaper) website at www.hematology.org. Details regarding the key abstracts to be presented are included below.

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"We are elated that out of the over eighty-five ongoing daratumumab clinical studies, a record thirty abstracts containing daratumumab data in multiple myeloma and other indications were accepted for presentation at this year’s ASH (Free ASH Whitepaper) Annual Meeting. We are also thrilled that three abstracts related to pre-clinical data from wholly owned Genmab programs were accepted for inclusion at this prestigious event, including the first proprietary DuoBody program, DuoBody-CD3xCD20, and the first ever DuoHexaBody therapeutic program, DuoHexaBody-CD37," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Late breaking abstracts are not yet available.

Genmab Pre-Clinical Abstracts

DuoBody-CD3xCD20 Shows Unique and Potent Preclinical Anti-Tumor Activity In Vitro and In Vivo, and is Being Evaluated Clinically in Patients with B-Cell Malignancies – Poster presentation, Saturday, December 1

DuoHexaBody-CD37 a Novel Bispecific Antibody with a Hexamerization enhancing Mutation Targeting CD37, Demonstrates Superior CDC in Preclinical B-Cell Malignancy Models – Poster presentation, Monday, December 3

Targeting CD37 in B-Cell Malignancies Using the Novel Therapeutic Ab DuoHexaBody-CD37 Results in Efficient Killing of Tumor B-Cells Ex Vivo via CDC, Even in Relapsed and/or Refractory Patient Samples – Poster presentation, Monday, December 3

Daratumumab Abstracts Sponsored by Janssen Biotech, Inc.

Oral Presentations:
Efficacy and Updated Safety Analysis of a Safety Run-in Cohort from GRIFFIN, a Phase 2 Randomized Study of Daratumumab, Bortezomib, Lenalidomide, and Dexamethasone Versus Bortezomib, Lenalidomide, and Dexamethasone in Patients with Newly Diagnosed Multiple Myeloma Eligible for High-Dose Therapy and Autologous Stem Cell Transplantation – Oral presentation, Saturday, December 1

LYRA – A Phase 2 Study of Daratumumab Plus Cycolphosphamide, Bortezomib, and Dexamethasone in Newly Diagnosed and Relapsed Patients with Multiple Myeloma – Oral presentation, Saturday, December 1

One-Year Update of a Phase 3 Randomized Study of Daratumumab Plus Bortezomib, Melphalan, and Prednisone Versus Bortezomib, Melphalan, and Prednisone in Patients with Transplant-Ineligible Newly Diagnosed Multiple Myeloma: ALCYONE – Oral presentation, Saturday, December 1

Poster Presentations:
Three-Year Follow Up of the Phase 3 POLLUX Study of Daratumumab Plus Lenalidomide and Dexamethasone Versus Lenalidomide and Dexamethasone Alone in Relapsed or Refractory Multiple Myeloma – Poster presentation, Saturday, December 1

Subcutaneous Daratumumab in Patients with Relapsed or Refractory Multiple Myeloma: Part 2 Safety and Efficacy Update of the Open-label, Multicenter, Phase 1b Study (PAVO) – Poster presentation, Saturday, December 1

Pharmacokinetics of Subcutaneous Daratumumab in Patients with Relapsed or Refractory Multiple Myeloma: Primary Clinical Pharmacology Analysis of the Open-label, Multicenter, Phase 1b Study (PAVO) – Poster presentation, Saturday, December 1

Split First Dose Administration of Daratumumab for the Treatment of Patients with Multiple Myeloma: Clinical Pharmacology and Population Pharmacokinetic Analyses – Poster presentation, Saturday, December 1

Updated Results from the Phase 2 CENTAURUS Study of Daratumumab Monotherapy in Patients with Intermediate-risk or High-risk Smoldering Multiple Myeloma – Poster presentation, Saturday, December 1

Daratumumab Monotherapy for Patients with Relapsed or Refractory Natural Killer/T-cell Lymphoma (NKTCL), Nasal Type: An Open-label, Single-arm, Multicenter Phase 2 Study – Poster presentation, Saturday, December 1

Efficacy and Safety of Daratumumab, Bortezomib, and Dexamethasone Versus Bortezomib, and Dexamethasone in First Relapse Patients: Two-Year Update of CASTOR – Poster presentation, Sunday, December 2

Evaluation of Sustained Minimal Residual Disease Negativity in Relapsed / Refractory Multiple Myeloma Patients Treated with Daratumumab in Combination with Lenalidomide Plus Dexamethasone or Bortezomib Plus Dexamethasone: Analysis of POLLUX and CASTOR – Poster presentation, Sunday, December 2

Efficacy of Daratumumab in Combination with Standard of Care Regimens in Lenalidomide-Exposed or Refractory Patients with Relapsed / Refractory Multiple Myeloma: Analysis of CASTOR, POLLUX and MMY1001 Studies – Poster presentation, Sunday, December 2

Ofatumumab Abstracts Sponsored by Novartis

Oral Presentation:
Results of the Primary Analysis of COMPLEMENT A+B: A Phase III Study of Ofatumumab in Combination with Bendamustine Versus Bendamustine Alone in Patients with Indolent Non-Hodgkin’s Lymphoma That is Unresponsive or Relapsed Following Rituximab or Rituximab-containing Regimen – Oral presentation, Sunday, December 2

Poster Presentation:
Long-Term Evaluation of Efficacy and Safety of Ofatumumab Added to Fludarabine & Cyclophosphamide in Subjects with Relapsed Chronic Lymphocytic Leukemia: Final Analysis of COMPLEMENT 2 Trial – Poster presentation, Sunday, December 2

On November 1, 2018 Sierra Oncology, Inc. (Nasdaq: SRRA), a clinical stage drug development company focused on advancing targeted therapeutics for the treatment of patients with significant unmet needs in hematology and oncology, reported that clinical data from a translational biology study of momelotinib in 41 transfusion dependent patients with myelofibrosis (MF) will be reported in a poster at ASH (Free ASH Whitepaper) 2018 (Press release, Sierra Oncology, NOV 1, 2018, View Source [SID1234530529]). The impact of momelotinib on serum hepcidin, along with markers of iron storage and availability, erythropoiesis and inflammation were investigated to explore the biological mechanisms underlying the favorable effects of momelotinib on MF-associated anemia.

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"The results from this translational biology clinical study provide further evidence for momelotinib’s unique anemia benefit," said Dr. Christian Hassig, Chief Scientific Officer of Sierra Oncology. "Moreover, the findings provide clinical evidence that reinforce the differentiated profile of momelotinib as a potent inhibitor of ACVR1, a principal driver of hepcidin production in the liver. As with many inflammatory diseases associated with chronic anemia, myelofibrosis is characterized by high hepcidin, resulting in functional iron deficiency. In myelofibrosis, high levels of hepcidin are inversely correlated with survival. The observed reduction in hepcidin and restoration of iron homeostasis, coupled with net increases in various measures of erythropoiesis, provide important translational biomarker data accounting for the compelling transfusion-independence rates of 34-39% achieved in this transfusion dependent study population."

"As noted in our recent KOL call* featuring Dr. Srdan Verstovsek, one of the investigators in this clinical study, almost every myelofibrosis patient develops anemia and it typically becomes worse over time, often leading to transfusion dependency, yet there are no approved therapies to treat this facet of the disease," stated Dr. Nick Glover, President and Chief Executive Officer of Sierra Oncology. "Momelotinib inhibits JAK1, JAK2 and ACVR1, and is therefore uniquely positioned to address disease-related cytopenia in myelofibrosis, including anemia and transfusion dependency, while also improving splenomegaly and constitutional symptoms."

Sierra is currently preparing for discussions with regulators to determine the registration path for momelotinib and anticipates reporting next steps in the first half of 2019.

*KOL call featuring Dr. Srdan Verstovsek:
View Source

About the study (ClinicalTrials.gov Identifier NCT02515630):
In this Phase 2 open-label, translational biology study 41 transfusion-dependent (TD; ≥4 units red blood cells [RBC] transfusion in the 8 weeks prior to first dose of momelotinib) patients with primary or post-ET/PV MF (platelets ≥50 K) received 200 mg momelotinib once daily for 24 weeks.

By week 24, 14 (34.1%, 90% CI: 22.0–48.1%) patients had a ≥12-week transfusion-independent response (TI-R) and 39.0% had no RBC transfusion for ≥8 weeks at any time (90% CI: 26.2-53.1%).
At every study visit, median blood hepcidin decreased 6 hours after dosing with momelotinib. Serum iron, transferrin, hemoglobin, reticulocytes, and hematocrit increased at week 2 in patients with TI-R. Following this peak, serum iron decreased while hemoglobin, hematocrit and platelet count increased through week 24.
Adverse events (AEs) were consistent with previous studies of momelotinib in myelofibrosis, with cough, diarrhea, nausea, and fatigue as the most common. AEs ≥Gr 3 were experienced by 21 patients, most commonly anemia and neutropenia.
Title: Hepcidin Suppression by Momelotinib Is Associated with Increased Iron Availability and Erythropoiesis in Transfusion-Dependent Myelofibrosis Patient

Authors: Stephen T. Oh, Moshe Talpaz, Aaron T. Gerds, Vikas Gupta, Srdan Verstovsek, Ruben Mesa, Carole Miller, Candido Rivera, Angela Fleischman, Swati Goel, Mark Heaney, Casey O’Connell, Murat Arcasoy, Yafeng Zhang, Jun Kawashima, Tomas Ganz, Carrie Baker Brachmann

On November 1, 2018 Savara Inc. (NASDAQ: SVRA), an orphan lung disease company, reported it will release its third quarter 2018 financial results on Wednesday, November 7, 2018 (Press release, Savara, NOV 1, 2018, View Source [SID1234530545]). Savara management will also host a conference call for investors beginning at 5:30 p.m. ET on Wednesday, November 7, 2018 to discuss its third quarter 2018 financial results and provide a business update.

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Shareholders and other interested parties may access the conference call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from elsewhere outside the U.S. and request the "Savara Inc." call. A live webcast of the conference call will be available online in the Investors section of Savara’s website at View Source A replay of the webcast will be available on Savara’s website for 30 days, and a telephone replay will be available through November 12, 2018 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada and (412) 317-0088 from elsewhere outside the U.S. and entering the replay access code 10125683.

On November 1, 2018 Evelo Biosciences, Inc. (Nasdaq: EVLO) ("Evelo"), a clinical stage biotechnology company developing monoclonal microbials designed to act on the gut-body network for the treatment of a wide range of diseases, reported financial results and provided a business update for the third quarter ended September 30, 2018 (Press release, Evelo Biosciences, NOV 1, 2018, View Source [SID1234530573]).

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"Our continued progress reflects our commitment to advancing a broad portfolio of product candidates to treat multiple diseases," said Simba Gill, Ph.D., CEO of Evelo. "We are enrolling patients in our Phase 1b trial of EDP1066 for psoriasis and atopic dermatitis, and recently received approval from the MHRA to start a clinical trial of EDP1815, our second candidate for inflammatory diseases. Additionally, we expect the University of Chicago’s investigator-sponsored trial of EDP1503 for the treatment of metastatic melanoma to begin enrolling patients this quarter. These activities support our goal of ten potential clinical readouts across inflammation and oncology patient groups in 2019 and 2020."

Third Quarter and Recent Business Highlights:

Pipeline:
•
In September 2018, the UK Medicines and Healthcare Regulatory Agency (MHRA) accepted Evelo’s Clinical Trial Authorisation application for a Phase 1b study of EDP1815 for the treatment of psoriasis and atopic dermatitis patients.

Upcoming Anticipated Milestones:
•
Initiation of a Phase 1b clinical trial of EDP1815, an anti-inflammatory monoclonal microbial product candidate, in healthy volunteers and patients with psoriasis and atopic dermatitis in the fourth quarter of 2018.
•
Initiation of the University of Chicago’s investigator-sponsored, open-label Phase 2a clinical trial of EDP1503 in combination with a checkpoint inhibitor in patients with metastatic melanoma in the fourth quarter of 2018.
•
Initiation of a company-sponsored Phase 2a clinical trial of EDP1503 in combination with a checkpoint inhibitor in patients with multiple cancer types in the first half of 2019.
•
Clinical data from the ongoing Phase 1b clinical trial of EDP1066 in healthy volunteers and patients with psoriasis and atopic dermatitis in the first half of 2019.

Upcoming Events:
•
Stifel 2018 Healthcare Conference on November 14, 2018 in New York, New York.
•
Evercore ISI Healthcare Conference on November 27, 2018 in Boston, Massachusetts.
•
BMO Prescriptions for Success Healthcare Conference on December 12, 2018 in New York, New York.

Corporate:
•
In October 2018, Evelo appointed Daniel Char as general counsel. Daniel joins Evelo from Smith & Nephew, where he was the associate general counsel – commercial, responsible for advising and supporting the company’s global businesses on commercial, compliance, regulatory, clinical, licensing, manufacturing and international trade matters. Daniel earned a J.D. from Harvard Law School and a B.A. in Economics from Tufts University.

Third Quarter 2018 Financial Results:
•
Cash Position: As of September 30, 2018, cash, cash equivalents and investments were $164.3 million, as compared to cash and cash equivalents of $38.2 million as of December 31, 2017. This increase was due to net proceeds of $81.3 million from the issuance of Series C Preferred Stock, net proceeds of $75.8 million from Evelo’s initial public offering and $5.0 million of additional drawings from Evelo’s existing debt facility, partially offset by cash used to fund operating activities for the first nine months of 2018. Evelo

expects that its cash, cash equivalents and investments will enable it to fund its planned operating expenses and capital expenditure requirements into the second half of 2020.
•
Research and Development Expenses: R&D expenses were $11.2 million for the three months ended September 30, 2018, compared to $5.3 million for the three months ended September 30, 2017. The increase of $5.9 million was due primarily to increases in costs related to Evelo’s inflammation and oncology clinical development programs, gut-body network platform expenses as well as increased personnel costs.
•
General and Administrative Expenses: G&A expenses were $5.2 million for the three months ended September 30, 2018, compared to $2.7 million for the three months ended September 30, 2017. The increase of $2.5 million was due primarily to increased general and administrative personnel costs, professional and consulting fees, and facility expenses supporting Evelo’s growing organization and public company infrastructure.
•
Net Loss Attributable to Common Stockholders: Net loss attributable to common stockholders was $(15.9) million for the three months ended September 30, 2018, or $(0.50) per basic and diluted share, as compared to a net loss attributable to common stockholders of $(9.9) million for the three months ended September 30, 2017, or $(2.61) per basic and diluted share.

On November 1, 2018 Corcept Therapeutics Incorporated (NASDAQ: CORT), a company engaged in the discovery, development and commercialization of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended September 30, 2018 (Press release, Corcept Therapeutics, NOV 1, 2018, View Source [SID1234530622]).

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Financial Highlights

Revenue of $64.4 million, a 51 percent increase from third quarter 2017
GAAP net income of $0.14 per share, compared to $0.11 per share in third quarter 2017
Non-GAAP net income of $0.22 per share, compared to $0.14 per share in third quarter 2017
Cash and investments of $196.7 million, a $36.8 million increase from second quarter 2018
Reaffirmed 2018 revenue guidance of $250-270 million
Corcept reported revenue of $64.4 million for the third quarter, compared to $42.8 million in the same period last year. GAAP net income was $17.7 million, compared to $13.8 million in the third quarter of 2017. Excluding non-cash expenses related to stock-based compensation, use of deferred tax assets, interest on the company’s retired royalty financing obligation and related income tax effects, non-GAAP net income in the third quarter was $27.9 million, compared to $17.4 million in the third quarter of 2017. A reconciliation of GAAP to non-GAAP net income is set forth below.

Operating expenses for the third quarter were $41.5 million, compared to $29.1 million in the third quarter of 2017, primarily due to increased spending to advance relacorilant, CORT118335 and CORT125281 and costs arising from increased sales volume.

Cash and investments were $196.7 million at September 30, 2018, compared to $159.9 million at June 30, 2018. The company spent $8.9 million in the third quarter repurchasing 674,000 shares of its common stock. Under the terms of Corcept’s stock repurchase program as currently authorized, $91.1 million remains available for the repurchase of shares.

Relacorilant to Treat Patients with Cushing’s Syndrome

Enrollment open in relacorilant’s 130-patient Phase 3 trial
Relacorilant designated orphan drug for treatment of Cushing’s syndrome
The Phase 3 trial of Corcept’s proprietary, selective cortisol modulator, relacorilant, is expected to enroll 130 patients at sites in the United States and Europe. In the trial’s initial, open-label phase, patients will receive relacorilant for 22 weeks. Any patients who exhibit clinically meaningful improvements in hypertension or glucose tolerance will then enter a twelve-week, double-blind, placebo-controlled "withdrawal phase," during which half will continue to receive relacorilant and the rest, placebo. The rate and degree of relapse in patients receiving placebo will be measured against the rate and degree of relapse in those continuing relacorilant.

For more information about the trial, go to clinicaltrials.gov.

Because the FDA has designated relacorilant as an orphan drug for the treatment of Cushing’s syndrome, Corcept will receive tax credits for clinical trial costs, marketing application filing fee waivers if the drug is approved, as well as assistance from the FDA in the drug development process. Patents covering relacorilant’s composition of matter and use to treat Cushing’s syndrome expire in 2033.

"We’re excited to have started relacorilant’s Phase 3 trial," said Joseph K. Belanoff, MD. "Our Cushing’s syndrome franchise continues to add new prescribers of our first-generation cortisol modulator, Korlym, in every region of the country, and we expect it will continue to do so. Relacorilant promises to make the benefits of cortisol modulation even more widely available. Patients in relacorilant’s Phase 2 trial experienced significant clinical benefit, but not Korlym’s serious off-target effects – endometrial thickening, vaginal bleeding and hypokalemia. If these safety and efficacy results are confirmed in Phase 3, relacorilant will constitute a major clinical and commercial advance."

Oncologic & Metabolic Disorders

Selective cortisol modulator CORT118335 safe and well-tolerated in Phase 1; Phase 2 trials in antipsychotic-induced weight gain and non-alcoholic steatohepatitis (NASH) planned to start in first quarter 2019
Further results expected by year-end in Phase 1/2 trial of relacorilant plus Abraxane (nab-paclitaxel) to treat metastatic pancreatic cancer; FDA grants relacorilant orphan drug status for that indication
Controlled Phase 2 trial of relacorilant plus Abraxane to treat metastatic ovarian cancer planned to start by year-end
Dosing continues in Phase 1/2 trial of CORT125281 plus Xtandi (enzalutamide) in patients with metastatic castration-resistant prostate cancer
"Our clinical programs continue to make significant progress," said Robert S. Fishman, MD, Corcept’s Chief Medical Officer. "CORT118335 was well-tolerated in its Phase 1 trial. This compound is very potent in animal models of antipsychotic-induced weight gain and NASH – serious, widespread disorders for which patients have no good treatment options. We plan to open placebo-controlled, Phase 2 trials in both indications early next year.

"We are also excited to advance relacorilant as a treatment for solid tumors," said Dr. Fishman. "Our Phase 1/2 trial of relacorilant plus Abraxane has produced encouraging data. At ASCO (Free ASCO Whitepaper) earlier this year we reported that four of nine patients with metastatic pancreatic cancer who received the minimum therapeutic dose exhibited durable disease control, as did four of seven patients with metastatic ovarian cancer – notable results in patients with such dire disease, all of whom had progressed on prior taxane-based therapies. By year-end, we plan to open a controlled Phase 2 trial in patients with metastatic ovarian cancer and to have collected sufficient data in patients with metastatic pancreatic cancer to determine if a pivotal trial is warranted."

Conference Call

We will hold a conference call on November 1, 2018, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 877-260-1479 from the United States or 334-323-0522 internationally approximately ten minutes before the start of the call (passcode: 7489528). A replay will be available through November 15, 2018 at 888-203-1112 from the United States and 719-457-0820 internationally (passcode: 7489528).

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the stress hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients being diagnosed each year. Symptoms vary, but most people experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Cushing’s syndrome can affect every organ system in the body and can be lethal if not treated effectively. Our first approved product, Korlym, inhibits the effects of excess cortisol by modulating activity at the glucocorticoid receptor, one of the two receptors to which cortisol binds. Korlym was the first FDA-approved treatment for patients with Cushing’s syndrome.