On May 12, 2021 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company on a quest to deliver targeted therapies that treat rare forms of cancer, reported abstracts on new analyses from the previously completed SIMPLIFY-1 and SIMPLIFY-2 studies have been accepted into the program for the 2021 Annual Meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) (Press release, Sierra Oncology, MAY 12, 2021, View Source [SID1234579805]). An abstract highlighting the association between transfusion independence and improved overall survival in myelofibrosis patients treated with momelotinib has been selected for oral presentation.

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"Overall survival remains the gold standard benchmark for oncology therapies. We are excited to showcase these new analyses in an oral presentation demonstrating a strong correlation between achieving or maintaining transfusion independence at Week 24 and overall survival for momelotinib patients in SIMPLIFY-1. A similar trend was seen in SIMPLIFY-2," said Barbara Klencke, MD, Chief Development Officer of Sierra. "Higher rates of transfusion independence at Week 24 were observed in anemic patients receiving momelotinib, regardless of the degree of baseline anemia, and in all subgroups of patients when analyzed either by baseline platelet count or baseline transfusion status as we describe in a separate accepted poster. Together, these data support the potential long-term benefit of achieving or maintaining transfusion independence by inhibiting ACVR1 / ALK2 in addition to JAK1 and JAK2 with momelotinib in myelofibrosis patients, regardless of baseline platelet count, transfusion status or degree of anemia. We look forward to the presentation of the full data sets at the EHA (Free EHA Whitepaper) annual meeting next month."

Transfusion Independence is Associated with Improved Overall Survival in Myelofibrosis Patients Receiving Momelotinib

Efficacy data examining the association between transfusion independence and overall survival for momelotinib patients from SIMPLIFY-1 (JAKi-naïve) and SIMPLIFY-2 (JAKi-exposed) will be presented in an oral presentation by Ruben Mesa, MD, Director of the Mays Cancer Center, UT Health San Antonio, MD Anderson Cancer Center.

Previously published data from both SIMPLIFY studies demonstrate robust overall survival for momelotinib-treated patients (median not yet reached in SIMPLIFY-1 and 34.3 months in SIMPLIFY-2). Additionally, previously reported week 24 transfusion independence rates were higher in the momelotinib arms of SIMPLIFY-1 (67% vs. 49%) and SIMPLIFY-2 (43% vs. 21%). The new analyses suggest JAKi-naïve patients receiving momelotinib who maintain or achieve transfusion independence at week 24 have favorable overall survival compared to non-responders, with a similar trend observed in SIMPLIFY-2.

Presentation Details

Abstract: S202
Title: Transfusion Independence is Associated with Improved Overall Survival in Myelofibrosis Patients Receiving Momelotinib
Presenter: Ruben Mesa, MD, Director of the Mays Cancer Center, UT Health San Antonio, MD Anderson Cancer Center
Session Title: Novel Therapies and Targets in MPN
Virtual Presentation Available: Friday, June 11, 2021 beginning at 9:00 am CEST
Live Q&A Discussion: Wednesday, June 16, 2021, 4:00 – 4:45 pm CEST

Improved Transfusion Independence Rates for Momelotinib vs. Ruxolitinib in Anemic JAKi Naïve Myelofibrosis Patients Independent of Baseline Platelet or Transfusion Status

Progressive anemia is a common occurrence in myelofibrosis, with nearly all patients requiring transfusions as their disease advances. As described above, the analyses in Abstract S202 show that JAKi-naïve patients receiving momelotinib who maintain or achieve transfusion independence at week 24 have favorable overall survival compared to non-responders, with a similar trend observed in SIMPLIFY-2. Therefore, it is important to understand which patients are most likely to achieve transfusion independence at week 24.

Abstract EP1081 will highlight new analyses from SIMPLIFY-1 to demonstrate that the prognostically important week 24 transfusion independence rates in JAK inhibitor-naïve myelofibrosis patients were consistently higher in anemic patients receiving momelotinib compared to ruxolitinib, regardless of the platelet count, transfusion status or degree of anemia at baseline. Data to be featured further support the potential benefits of inhibiting ACVR1 / ALK2 in addition to JAK1 and JAK2 in myelofibrosis patients, and will be presented by Jean-Jacques Kiladjian, MD, PhD, Professor of Clinical Pharmacology, Paris Diderot University; Consultant Hematologist, Head, Clinical Investigation Center, Saint Louis Hospital, Paris, France.

Presentation Details

Abstract: EP1081
Title: Improved Transfusion Independence Rates for Momelotinib vs. Ruxolitinib in Anemic JAKi Naïve Myelofibrosis Patients Independent of Baseline Platelet or Transfusion Status
Presenter: Jean-Jacques Kiladjian, MD, PhD
Session Name: Myeloproliferative Neoplasms – Clinical
Virtual Presentation Available: Friday, June 11, 2021 beginning at 9:00 am CEST

On May 12, 2021 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies and cytokines for the treatment of cancer and autoimmune diseases, reported that Bassil Dahiyat, Ph.D., president and chief executive officer, will participate in a virtual fireside chat at the 2021 RBC Capital Markets Global Healthcare Virtual Conference on Wednesday, May 19, 2021 at 1:20 p.m. ET / 10:20 a.m. PT (Press release, Xencor, MAY 12, 2021, View Source [SID1234579823]).

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A live webcast will be available under "Events & Presentations" in the Investors section of the Company’s website located at www.xencor.com. Following the webcast, a replay will be archived on the website for at least 30 days.

On May 12, 2021 Kuraray hereby reported that due to the impact of the cold wave that struck the United States in the first quarter of the fiscal year ending December 31, 2021, it has posted an extraordinary loss as part of consolidated quarterly operating results for said period (Press release, Kuraray, MAY 12, 2021, https://pdf.irpocket.com/C3405/eq9A/Kj61/qLHN.pdf [SID1234579899]). In addition, Kuraray has revised its consolidated operating results forecasts for the first six months of said fiscal year, as detailed below, from the previous operating results forecasts announced on February 10, 2021.

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1. Posting of Extraordinary Loss and Its Details With regard to the impact of the severe cold wave that struck many parts of the United States in mid-February 2021, in its March 10, 2021 press release "Notice Regarding the Impact of a Cold Wave in the United States," Kuraray announced that a number of Group manufacturing sites located in a Houston, Texas, suburb and run by local subsidiary Kuraray America, Inc., suspended operations from Mid-February onward. Full production, except for certain lines, resumed as of April 2, 2021 as announced in a subsequent press release issued on the same date. Having assessed the impact of the cold wave on its consolidated operating results, Kuraray recorded an extraordinary loss of ¥3,016 million as a disaster loss for the first quarter of the fiscal year ending December 31, 2021.

2. Revisions to consolidated earnings forecast for the second quarter of fiscal 2021 (January 1-June 30, 2021)

(2) Reason for the revisions
In the consolidated first quarter, shipments in Kuraray’s businesses mainly in the Vinyl acetate and Isoprene segments increased due to growth in demand, including for mainstay applications for automobiles, displays, and electronic and electric devices. We assume that demand will remain firm in the second quarter as well. Furthermore, in the first quarter, we recorded a loss on litigation of ¥3,054 million related to a fire that occurred at our U.S. subsidiary in May 2018. As stated above, we also recorded a disaster loss of ¥3,016 million due mainly to the suspension of production for some equipment at our U.S. subsidiary caused by a severe cold wave in the southern United States in February. Based on these circumstances, the forecast of consolidated operating results for the second quarter of fiscal 2021 (January 1, 2021 to June 30, 2021) is as shown above.

On May 12, 2021 Knight Therapeutics Inc. (TSX: GUD) ("Knight"), a leading pan-American (ex-US) specialty pharmaceutical company, reported that Samira Sakhia, President and Chief Operating Officer, is scheduled to participate in a fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference on Tuesday, May 18, 2021 at 8:35 am ET (Press release, Knight Therapeutics, MAY 12, 2021, View Source [SID1234579947]). A live webcast of the event will be available at the following link: Event. A copy of the presentation will be available at www.gud-knight.com.

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On May 12, 2021 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer reported a business update and reports financial results for the quarter ended March 31, 2021 (Press release, Panbela Therapeutics, MAY 12, 2021, View Source [SID1234583759]). Management is hosting an earnings call today at 4:30 p.m. ET.

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The first quarter 2021 was marked by meaningful clinical progress.

Q1 and Recent Highlights

Abstract accepted with poster presentation at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting June 4-8, 2021.
Partial clinical hold lifted from the company’s Phase 1 first-line study of SBP-101.
Research agreement entered into with Johns Hopkins University School of Medicine; Preclinical studies underway.
As previously announced, in April the U.S. Food and Drug Administration (FDA) lifted the partial clinical hold on the company’s Phase 1 first-line study of SBP-101 when used in combination with standard of care agents gemcitabine and nab-paclitaxel for treatment of patients with metastatic pancreatic ductal adenocarcinoma. The company has agreed to include in the design of future studies the exclusion of patients with a history of retinopathy or at risk of retinal detachment and scheduled periodic ophthalmologic monitoring for all patients, and in future dose-finding studies screening for retinal toxicity will be included.

"Year to date, we have focused on advancing SBP-101 in its first indication and exploring the broader potential of polyamine metabolic inhibition," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer. "With the hold lifted, we are excited to move the pancreatic cancer program forward. Additionally, the research initiated with Johns Hopkins will help to inform development outside of pancreatic cancer as well as potentially in combination with a checkpoint inhibitor."

Based on interim data from our Phase I trial, SBP-101 demonstrated a 62% objective response rate in combination with gemcitabine & abraxane (G&A); more than double the historical standard of care for metastatic pancreatic cancer with G&A.

We believe SBP-101 has the potential to expand into other cancers with known elevated levels of polyamine metabolism.

Upcoming Milestones

Public release of additional data from phase 1 trial – ASCO (Free ASCO Whitepaper) Annual Meeting June 4-8, 2021
Initiation of randomized phase 2 study mid-year
Public release of preclinical data across tumors outside of pancreatic cancer 2H’21
First Quarter ended March 31, 2021 Financial Results

General and administrative expenses were $1.1 million in the first quarter of 2021, compared to $0.5 million in the first quarter of 2020. The change in the quarter is primarily associated with increased headcount and other increased costs associated with maintaining our common stock on the Nasdaq Capital Market.

Research and development expenses were $1.1 million in the first quarter of 2021, compared to $0.6 million in the first quarter of 2020. The change in quarter is due primarily to higher manufacturing costs in preparation for future clinical trials.

Net loss was $2.3 million, or $0.23 per diluted share, compared to a net loss of $1.8 million, or $0.27 per diluted share, in the first quarter of 2020.

Total cash was $8.1 million as of March 31, 2021. Total current assets were $8.8 million and current liabilities were $1.3 million as of the same date. The company had no debt as of March 31, 2021.

Conference Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

About SBP-101

SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, suggesting potential complementary activity with an existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Recently observed serious visual adverse events are being evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .