On May 7, 2020 Bicycle Therapeutics plc (NASDAQ:BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycles) technology, reported financial results for the first quarter ended March 31, 2020 and discussed recent corporate updates (Press release, Bicycle Therapeutics, MAY 7, 2020, View Source [SID1234557305]).

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"We are very proud of the tremendous progress we’ve made executing against our 2020 goals in the first few months of this year," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "Since January, we’ve entered two new immuno-oncology collaborations, including a partnership with Genentech worth up to $1.7 billion, and reached key milestones in the advancement of our clinical programs, such as establishing a recommended Phase II dose for our most advanced Bicycle Toxin Conjugate (BTC) BT1718 and advancing BT5528, our first second-generation BTC, through the Phase I dose escalation quickly. We also welcomed to Bicycle our General Counsel, Zafar Qadir, who brings extensive legal expertise and experience that will be integral to the achievement of our near- and long-term objectives. Despite uncertainties associated with the COVID-19 pandemic, we remain focused on achieving our stated goals for the year as planned. We look forward to providing updates on our progress as we advance our novel therapeutic candidates into and through the clinic."

First Quarter 2020 and Recent Highlights

Appointed Zafar Qadir as General Counsel. Mr. Qadir joined Bicycle as head of the Company’s legal function in April 2020.
Provided Comprehensive Pipeline Progress Update. In April 2020, Bicycle announced updates across its wholly-owned and partnered programs in oncology and non-oncology indications:
The key objectives were met in the Phase I dose escalation portion of the Phase I/IIa study of BT1718, a BTC targeting MT1-MMP, in patients with solid tumors, sponsored by Cancer Research UK. A recommended Phase II dose was set at 20 mg/m2 administered once weekly. With once-weekly dosing, BT1718 appeared tolerable, with manageable adverse events. Additionally, preliminary signs of anti-tumor activity were observed, including one partial response of a 68% reduction in a target lesion. The Company continues to expect that Cancer Research UK will initiate the Phase IIa portion of the Phase I/IIa study of BT1718 in 2020, although timing may be dependent on the impact of the COVID-19 pandemic. At present, enrollment of new patients into the Phase IIa portion of the trial at Cancer Research UK clinical sites in the UK has been paused due to the COVID-19 pandemic.
To date, administered doses of BT5528, a second-generation BTC targeting EphA2, appear well-tolerated with manageable adverse events in the ongoing Phase I/II trial in patients with advanced solid tumors associated with EphA2 expression. Dosing in both the monotherapy and nivolumab combination arms is underway. The combination arm opened in the second quarter of 2020, and the monotherapy escalation continues toward clinically relevant doses.
In 2020, Bicycle expects to initiate a Phase I/II trial of BT8009, a second-generation BTC targeting Nectin-4, in patients with advanced solid tumors, subject to potential timing and other impacts of the ongoing COVID-19 pandemic.
IND-enabling activities for BT7480 are ongoing and on track to a potential initiation of clinical development in 2021, subject to potential timing and other impacts of the ongoing COVID-19 pandemic. BT7480 is a tumor-targeted immune cell agonist (TICA) that targets Nectin-4 and agonizes CD137.
The Company expanded its immuno-oncology pipeline, selecting BT7455 as a new TICA candidate. BT7455 targets EphA2 and agonizes CD137.
Cancer Research UK continues to advance preclinical development of BT7401, a systemic agonist of CD137.
Progress has also been made in the Company’s partnered programs beyond oncology: preparations for Oxurion’s Phase II trial of THR-149 are ongoing; three target programs in respiratory, cardiovascular, and metabolic diseases were transitioned to AstraZeneca for subsequent optimization towards potential candidate selection; and there has been early success in the collaboration with Dementia Discovery Fund (DDF) to develop Bicycles to modulate the activity of proteins implicated in the progression of dementia.
Entered into Strategic Collaboration with Genentech to Discover, Develop and Commercialize Novel Bicycle-based Immuno-oncology Therapies. In February 2020, Bicycle entered into a strategic collaboration agreement with Genentech. Under the terms of the agreement, Bicycle will be responsible for discovery research and early preclinical development up to candidate selection. Bicycle received a $30 million upfront payment. The upfront payment, an early milestone payment and potential future milestone payments could total up to $1.7 billion. Bicycle will also be eligible to receive tiered royalties. None of Bicycle’s wholly-owned oncology assets, including its immuno-oncology candidates, are included in the collaboration.
Upcoming Investor Presentation

Bicycle will present at the Bank of America 2020 Health Care Conference on Thursday, May 14, 2020 at 3:00 p.m. ET. The conference will be held in a virtual meeting format.

A live webcast of the presentation will be accessible in the Investors & Media section of Bicycle’s website at bicycletherapeutics.com. An archived replay of the webcast will be available for 60 days following the presentation date.

Financial Results

Cash and cash equivalents were $109.6 million as of March 31, 2020, compared with $92.1 million as of December 31, 2019. Cash at March 31, 2020 includes the $30 million upfront payment from Genentech.
Research and development expenses totaled $7.8 million for the three months ended March 31, 2020, compared to $6.3 million for the three months ended March 31, 2019. The increase of $1.5 million is primarily due to increased clinical and TICA program development expenses, partially offset by lower development expenses of other programs due to timing and an increase in personnel related costs, including $0.6 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $5.0 million for the three months ended March 31, 2020, compared to $3.4 million for the three months ended March 31, 2019. The increase of $1.6 million is primarily due to an increase in personnel related costs, including $1.3 million of incremental non-cash share-based compensation expense as well as professional fees and costs related to operations as a public company, partially offset by a favorable effect of foreign exchange rates.
Net loss was $11.3 million, or $(0.63) basic and diluted net loss per share, for the three months ended March 31, 2020, compared to net loss of $6.5 million, or $(7.80) basic and diluted net loss per share, for the quarter ended March 31, 2019.

On May 7, 2020 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported that Angelos Stergiou, M.D., ScD h.c., President and Chief Executive Officer of SELLAS, will present a corporate overview at the UBS Virtual Global Healthcare Conference on Monday, May 18, 2020 at 4:40 pm EDT (1:40 pm PDT) (Press release, Sellas Life Sciences, MAY 7, 2020, View Source [SID1234557321]).

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To access a live webcast of the presentation, please visit the Investors, Events and Presentations section of the Company’s website at www.sellaslifesciences.com. A replay of the webcast will be available on the Company’s website following the live presentation.

On May 7, 2020 Cryoport, Inc. (NASDAQ: CYRX) (NASDAQ: CYRXW) ("Cryoport"), a global leader in life sciences solutions, reported financial results for the three-month period ended March 31, 2020 (Press release, Cryoport, MAY 7, 2020, View Source [SID1234557363]).

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"We reported revenue of $9.8 million for the First Quarter of 2020, an increase of 47% from the First Quarter of 2019," said Jerrell Shelton, Chief Executive Officer of Cryoport. "This was driven largely by record revenue from our commercial agreements supporting Gilead’s YESCARTA and Novartis’ KYMRIAH which contributed $2.9 million to the First Quarter of 2020, representing an increase of 110% compared with the First Quarter of 2019. In the first quarter, we experienced minimal disruption of our commercial portfolio resulting from the COVID-19 pandemic and are working closely with our partners to ensure eligible patients continue to have access to these life-saving therapies.

"In April, Gilead’s Kite Pharma renewed its agreement with Cryoport, which cover its entire portfolio of therapies in development as well as YESCARTA. The ongoing rollouts of both YESCARTA and KYMRIAH to patients in the Americas, EMEA and APAC are expected to drive a continued ramp in activity related to our agreements supporting these commercial products. Revenue from our agreement with bluebird bio to support its commercial launch of ZYNTEGLO is expected to commence in the second half of 2020."

"Our revenue growth in the First Quarter was also attributable to Cryogene, our Houston based Bioservices operation, which contributed revenue of $1.3 million. Cryogene specializes in the long-term secure storage of biological specimens, materials and samples for research purposes and is expected to continue to operate with some, but relatively minimal, impact from the COVID-19 pandemic.

"We are very pleased to report strong clinical trial growth for the quarter as we added a net total of 29 clinical trials, bringing the total number of clinical trials supported by Cryoport to a record 465.

"As providers of mission-critical logistics solutions to the healthcare industry, we are actively monitoring the spread of COVID-19 and continue to operate our Global Logistics Center Network with minimal impact on our ability to conduct day-to-day operations for our life sciences clients. While a meaningful number of the clinical trials we support have been suspended temporarily, none of these trials, to our knowledge, have been terminated as a result of COVID-19. These suspensions did impact our clinical trial revenue in late First Quarter and that impact continues into the Second Quarter of 2020. For trials that are ongoing, we are supporting our clients’ needs effectively and with business continuity plans in place to limit disruption and minimize any potential risk to our employees. We believe the structural shift that is underway in the biopharma market toward large-molecule treatments plays to our strengths as these therapies require much more rigorous, specialized and temperature sensitive logistics and storage solutions that meet exacting requirements. We think these requirements and our ever-increasing competency in tailored information technology will ensure the continued medium- and long-term expansion of our business despite the near-term headwinds we are experiencing from COVID-19.

Two Cryoport supported therapies filed for commercial approval during the First Quarter of 2020, and a third filed subsequent to the quarter end. We continue to expect a further six Cryoport supported MAA’s and BLA’s to be filed in 2020, based on internal information and forecasts from the Alliance for Regenerative Medicine, although the timing of some of these may be impacted by COVID-19.

"We are the life sciences industry leader in temperature-controlled logistics and biostorage of life-saving advanced cell and gene therapies, vaccines and other fragile and high value commodities, providing essential and highly specialized solutions to the Biopharma market. With research into COVID-19 moving at a relatively rapid pace, we have been selected to support six clinical trials for potential COVID-19 treatments and vaccines. We are proud to use our expertise to contribute to the fight against the spread of COVID-19 and are working closely with our clients to assure the security of their products as they advance the trials of these much-needed therapies.

"The Reproductive Medicine market was impacted by COVID-19 imposed restrictions and, consequently, for the First Quarter of 2020 contributed revenue of $0.8 million. Upon the lifting of these restrictions, we will resume our services and believe we will be well-positioned to drive revenue growth in this market. One example that gives us confidence is our new multi-year agreement with Inception Fertility, LLC, a Houston-based company which operates The Prelude Network, the largest and fastest growing network of fertility centers in the United States.

Mr. Shelton concluded by saying, "Our strong balance sheet and liquidity position, along with long-term agreements with an exceptionally loyal client base and successfully implemented business mitigation plans, gives us confidence in our ability to navigate the uncertainties caused by the pandemic. We are confident in our future and continue to execute on our plans to support the anticipated expansion in demand for our cutting-edge supply chain solutions. This entails building out our infrastructure, including software, competencies, and further expanding our Global Supply Chain Network, with new Global Supply Chain Centers in Morris Plains, New Jersey and Houston, Texas. We also continue to evaluate potential M&A opportunities that are complementary, accretive and expand our solutions offerings and total addressable market."

Market Highlights:

Global Logistics Solutions

Biopharma

Biopharma revenue increased by 33% in the three-months ended March 31, 2020 compared to the same period in 2019.
Commercial revenue increased $1.5 million, or 110%, to $2.9 million for the three months ended March 31, 2020, as compared to $1.4 million for the same period in 2019.
Cryoport is now supporting a net total of 465 clinical trials as of March 31, 2020 compared with 383 as of March 31, 2019. The number of trials in Phase III grew to 62, compared with 49 as of March 31, 2019. Of the 465 total trials Cryoport supports, 384 are in the Americas, 65 in EMEA (Europe, the Middle East and Africa) and 16 in APAC (Asia Pacific). This compares to 338 in the Americas and 45 in EMEA as of March 31, 2019.
Gilead Sciences, Inc.’s subsidiary, Kite Pharma ("Kite"), renewed its agreement with Cryoport for an additional three years with evergreen provisions. The agreement covers all therapies in Kite’s portfolio, including the continued commercial rollout of its lead chimeric antigen receptor (CAR) T-cell therapy for the treatment of aggressive Non-Hodgkin Lymphoma, YESCARTA, all ongoing clinical programs as well as any new commercial product launches.
Selected by Cellular Biomedicine Group, Inc. (Nasdaq: CBMG), a developer of proprietary cell therapies for the treatment of cancer and degenerative diseases, to support two of its immuno-oncology and stem cell clinical trials in China.
Animal Health

Animal Health revenue remained flat at $0.2 million for both the three months ended March 31, 2020 and the three-months ended March 31, 2019; however, we are building a strong pipeline of potential clients and expect to grow Animal Health revenue in 2020.
Reproductive Medicine

As a result of the impact of COVID-19, Reproductive Medicine revenue remained relatively flat at $0.8 million for both the three months ended March 31, 2020 and the three months ended March 31, 2019.
Cryoport entered into a multi-year agreement with Inception Fertility, LLC, a Houston-based company which operates The Prelude Network, the largest and fastest growing network of fertility centers in the United States.
Global Bioservices

Bioservices revenue was $1.3 million for the three-month period ended March 31, 2020 resulting from the acquisition of Cryogene consummated in May 2019.
Financial Highlights:

Revenue increased 47% to $9.8 million for the three-month period ended March 31, 2020, compared with the same period in the prior year.
Excluding revenue from the Cryogene acquisition in May of 2019, revenue grew 28% for the three-month period ended March 31, 2020, compared with the same period in the prior year.
Gross margin for the three-months ended March 31, 2020 was 54%, compared to 52% for the same period in the prior year.
Operating costs and expenses increased by $3.2 million for the three-month period ended March 31, 2020, compared to the same period in the prior year, as a result of continued investments in software development, which will provide a platform for continuing the scaling of our business; engineering initiatives, which includes the development of revolutionary packaging and monitoring and communications resources and the build out competencies in support of advancing our infrastructure and the growing demand for Cryoport’s solutions.
Adjusted EBITDA for the three-month period ended March 31, 2020 was ($1.7 million), compared with ($0.4 million) in the same period in the prior year.
Net loss for the three-month period ended March 31, 2020 was $3.9 million, or $0.11 per share, compared to a net loss of $2.4 million, or $0.08 per share in the same period in 2019.
Cryoport reported $97.4 million in cash, cash equivalents and short-term investments as of March 31, 2020, compared with $94.3 million as of December 31, 2019.
Further information on Cryoport’s financial results is included on the attached condensed consolidated balance sheets and statements of operations, and additional explanations of Cryoport’s financial performance are provided in Cryoport’s annual report on Form 10-Q for the three months ended March 31, 2020, which will be filed with the Securities and Exchange Commission ("SEC") on or about May 8, 2020. The full report will be available on the SEC Filings section of the Investor Relations section of Cryoport’s website at www.cryoport.com.

Earnings Conference Call Information

IMPORTANT INFORMATION: A document titled "Cryoport First Quarter 2020 in Review", providing a review of Cryoport’s recent financial and operational performance and a general business update, will be issued at 4:05 pm ET on Thursday, May 7, 2020. The document is designed to be read by investors before the questions and answers conference call and can be accessed at http://ir.cryoport.com/events-and-presentations.

Cryoport management will host a conference call at 5:00 pm ET on May 7, 2020. The conference call will be in the format of a questions and answers session and will address any queries investors have regarding the Company’s reported results.

Conference Call Information

Date:

May 7, 2020

Time:

5:00 p.m. ET

Dial-in numbers:

+1 (855) 327-6837 (U.S.), +1 (631) 891-4304 (International)

Confirmation code:

Request the "Cryoport Call"

Live webcast:

‘Investor Relations’ section at www.cryoport.com or at this link. Please allow 10 minutes prior to the call to visit this site to download and install any necessary audio software.

Questions and answers will be recorded and available approximately three hours after completion of the live event on the Investor Relations section of the Company’s website at www.cryoport.comfor a limited time. To access the replay of the questions and answers, please follow this link. A dial-in replay of the call will also be available, to those interested, until May 14, 2020. To access the replay, dial +1 (844) 512-2921 (United States) or +1 (412) 317-6671 (International) and enter replay pin number: 10009027.

On May 7, 2020 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported financial results and a business update for the first quarter ended March 31, 2020 (Press release, VolitionRX, MAY 7, 2020, View Source [SID1234557391]). Volition management will host a conference call tomorrow, May 8 at 8:30 a.m. U.S. Eastern Time to discuss these results. Conference call details may be found below .

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Cameron Reynolds, President and Chief Executive Officer of Volition, upon releasing these results commented, "During the first quarter, we, together with our collaborators, have made very strong progress, particularly in assay and platform development, with our Nu.QTM Capture program and epigenetic toolkit, Nu.QTM Vet in collaboration with Texas A&M University and, at the start of the year with the acquisition of the epigenetics reagents company, Octamer GmbH. Our team has done an amazing job completing all these milestones in these difficult times."

View Source
An interview with Cameron Reynolds, President and Chief Executive Officer.

Mr. Reynolds added, "We have responded quickly to the COVID-19 pandemic and have a number of clinical trials underway to investigate how Nu.QTM might be clinically useful as either a diagnostic or prognostic tool for COVID-19. I am delighted to announce exciting preliminary results from our first study today and look forward to providing more details throughout this quarter. This work yet again shows the diverse potential of our Nu.QTM platform."

Company Highlights

Clinical – COVID-19

A proof of concept study involving 34 polymerase chain reaction (PCR) COVID-19 positive subjects and 50 control subjects revealed that nucleosomes were highly elevated in the PCR positive subjects.
Preliminary study results demonstrated an Area Under the Curve (AUC) for a single Nu.QTM assay of 98.7% for PCR positive COVID versus control subjects with a sensitivity of 100% at 94% specificity.
A second Nu.QTM assay also showed promising results with an AUC of 86.2%.
Volition plans to utilise results of this trial and other ongoing studies to further its aim of developing a clinically useful product to help in the battle against the COVID-19 global pandemic and potentially other diseases.
Clinical- Veterinary

In a proof of concept study conducted by Texas A&M University, a single Nu.QTM Vet assay detected almost 70% of both Canine Hemangiosarcoma and Canine Lymphoma with AUCs of 84.5% and 83.1% cancer versus healthy, respectively, at a specificity of 90%. These two cancers alone represent almost a third of all canine cancers.
Based on the results of this study, Volition plans to move forward with other Nu.QTM Vet assays in its pipeline, and with the larger range of cohorts and trials that it has collected and planned.
Abstract Publications

Three of Volition’s abstracts have been accepted for publication by ASCO (Free ASCO Whitepaper) (American Society of Clinical Oncology) and are expected to be publicly released on Wednesday, May 13.
Assay Development

Volition has completely re-engineered its Nu.QTM assays to now use a magnetic particle-based assay format leading to a step-change improvement in analytical performance which Volition expects to translate into improved clinical performance.
Volition reached its target of eight assays being finalized by the end of the first quarter with respect to its fully-automated magnetic bead-based chemiluminescent format. Studies are now ongoing for colorectal cancer, lung cancer and blood cancer with data read outs expected by the end of the second quarter.
Volition signed a contract with Shanghai Fosun Long March Medical Science Co., Ltd ("Fosun Long March"), China, to further develop our Nu.QTM magnetic particle-based assays for use on Fosun’s open-access platform LUMIART-II Automated Chemiluminescence Immunoassay System. The agreement also allows for the parties to negotiate an exclusive licensing agreement for Fosun Long March to distribute Volition’s Nu.QTM tests for the LUMIART System in China.
Epigenetic Toolbox

Volition has developed and is seeking patents on its novel Nu.QTM Capture-based epigenetic tools. Volition is using these tools to expand diagnostic developments that focus on circulating DNA fragment analysis, leading to a broader and potentially more powerful investigation of the epigenetic status of a patient’s circulating chromosome fragments, in addition to its ongoing work with its assay-based format in a range of cancers. Volition expects additional data on this in the coming months.
Organization

Volition completed the acquisition of its epigenetics reagents subsidiary, Octamer GmbH (renamed Volition Germany GmbH), in the first quarter of 2020.
In connection with the acquisition of Octamer, Volition expanded its Scientific Advisory Board to include Dr. Adrian Schomburg, one of the world’s leading experts on nucleosomes and founder and CEO of Octamer.
Financial

Cash and cash equivalents as of March 31, 2020 totalled approximately $12 million.
Upcoming Milestones

Volition expects to achieve the following milestones during 2020 and beyond:

Release a range of clinical data from COVID-19 studies currently underway.
Release a range of clinical data with its new optimised bead-based assays in colorectal, lung and other cancers.
Advance its previously announced large-scale colorectal and lung cancer trials in Europe, Asia and the U.S.
Advance the development of Nu.QTM Capture by determining the level of discrimination of tumor associated nucleosomes using mass spectrometry and/or sequencing.
Announce patient data demonstrating the wide utility of its epigenetic toolbox.
Complete further clinical studies for Nu.QTM Vet with the aim of launching its first product in 2020.
Publish several abstracts and peer reviewed scientific papers with clinical results as well as showing the robustness and utility of its Nu.QTM platform.
Mr. Reynolds concluded, "We are extremely proud of the accomplishments we have achieved thus far. I thank the dedicated Volition team for their tireless efforts especially given the challenging circumstances we all face during the COVID-19 pandemic. I, along with the rest of the Board and indeed the whole company, look forward to sharing the results of key studies over the coming year."

As a result of the COVID-19 pandemic during the first quarter of 2020, Volition implemented contingency planning to protect the health and well-being of its employees, with most employees working remotely where possible. Its laboratory in Belgium has remained open with the attendance of its dedicated laboratory technicians who have kept its research and development work on track with Volition’s expectations. Many of its small and medium sized studies have already been collected and their samples stored at its onsite biobank so the trial work underway and planned for the first half of 2020 is still tracking expectations. Regarding its large-scale studies, both the colorectal cancer and lung cancer studies underway in Taiwan are still ongoing with collection, however the study collection in the U.S. with the EDRN has been paused during the pandemic. The overall timing impact of the EDRN collection pause on the study is unknown at this stage, however, Volition will provide an update once the study re-commences.

For further details please contact [email protected]

VolitionRx Limited First Quarter 2020 Earnings
and Business Update Conference Call

Date: Friday, May 8, 2020
Time: 8:30 a.m. Eastern time
U.S. & Canada Dial-in: 1-877-407-9716 (toll free)
U.K. Dial-in: 0 800 756 3429 (toll free)
Toll/International: 1-201-493-6779
Conference ID: 13703451

Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with David Vanston, Chief Financial Officer and Scott Powell, Executive Vice President, Investor Relations.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until May 22, 2020. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13703451.

On May 7, 2020 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that the US Food and Drug Administration (FDA) has granted HQP1351, the Company’s core drug candidate, a Fast Track Designation (FTD) for the treatment of patients with chronic myeloid leukemia (CML) with certain genetic mutations who have failed to respond to treatments with existing tyrosine kinase inhibitors (TKIs) (Press release, Ascentage Pharma, MAY 7, 2020, View Source [SID1234557407]). This is the first FTD obtained by Ascentage Pharma, and it marks another milestone for HQP1351 following its recent Orphan Drug Designation by FDA.

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FTD is designed by FDA to expedite the development and review of drug candidates to treat serious disease/conditions that present urgent unmet clinical needs. This FTD for HQP1351 will lead to a series of benefits that could accelerate the clinical development and review for this drug candidate, including more frequent communications and meetings with FDA during the clinical development; and being allowed to enter Rolling Review, a process that allows the company to submit New Drug Applications (NDAs) by sections, rather than waiting until all required materials become available. This FTD also paves the way for HQP1351 to be qualified for Accelerated Approval and Priority Review designations in the future.

A key factor in determining FTD for a drug candidate is its ability to address an unmet medical need with additional clinical benefits to patients. To qualify for FTD, a drug candidate needs to demonstrate its potential to treat a condition with no existing therapies, offer significant clinical advantages over current treatment options, or bring clinical benefits to patients intolerant to existing therapies or with poor responses to them. This FTD represents FDA’s recognition of HQP1351’s potential in addressing some of these unmet medical needs.

CML is a rare hematologic malignancy with an annual incidence rate of approximately 1.9 cases/100,000. BCR-ABL tyrosine kinase inhibitors (TKIs) have significantly improved clinical management of CML. However, despite clinical benefits offered by the first-generation BCR-ABL inhibitor imatinib (Gleevec), and several second-generation TKIs, many patients develop drug resistance. Such acquired resistance to TKIs is a major challenge in the treatment of CML. BCR-ABL tyrosine kinase mutations represent a key mechanism of acquired drug resistance; T315I, which is the most common drug-resistant mutation, occurs in about 25% of patients with drug-resistant CML. Patients with the T315I mutation are resistant to both first- and second-generation BCR-ABL inhibitors, hence presenting an urgent unmet medical need for next-generation BCR-ABL inhibitors to more effectively target the T315I mutation. Although a third-generation TKI has already been approved in the United States, there are remaining concerns about its safety As a result, patients who failed to respond to existing TKI therapies continue to present an urgent unmet clinical need for safer and more effective therapies.

HQP1351 is a novel, orally active, potent third-generation BCR-ABL inhibitor designed to effectively target BCR-ABL mutants, including T315I, and it is being developed for the treatment of patients with CML resistant to first- and second-generation TKIs. HQP1351 is the first China-developed third-generation BCR-ABL inhibitor targeting drug-resistant CML. The drug candidate is currently being evaluated in pivotal Phase II studies in China, and Ascentage Pharma plans to submit an NDA for HQP1351 this year. In July 2019, HQP1351 was cleared by FDA to enter a Phase Ib study. Data from the Phase I clinical study of HQP1351 were selected for oral presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meetings two years in a row and was nominated as "Best of ASH (Free ASH Whitepaper)" research in 2019. Early data from studies of HQP1351 have demonstrated promising efficacy as well as favorable safety and tolerability profiles.

"Drug resistance to earlier-generation TKI represents an urgent unmet clinical need globally. HQP1351 is a novel, orally active, and potent third-generation BCR-ABL inhibitor being developed by an innovative biopharmaceutical company in China. In our Phase I study with a large sample size of more than 100 patients, HQP1351 demonstrated promising efficacy and a favorable safety profile, with clinical responses in many patients with relapsed or refractory CML who had no effective treatment option," Professor Xiaojun Huang, Director of the Institute of Hematology, Peking University, and the principal investigator of HQP1351 in China. "HQP1351 has the potential of becoming a new option in the clinical management of drug-resistant CML, symbolizing the great advances in biopharmaceutical R&D in China. This FTD by FDA signifies global recognition of the clinical data from China studies, and will hopefully soon benefit patients with CML worldwide. We look forward to further progress in the clinical development of HQP1351."

"HQP1351 is a China-developed third-generation BCR-ABL inhibitor. With this Fast Track Designation, received right after the recent Orphan Drug Designation by FDA, Ascentage Pharma has reached another major milestone in the global development of HQP1351," said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. "These two designations for HQP1351 indicate the urgency for addressing the unmet clinical need in the treatment of CML, and a recognition of HQP1351’s promising efficacy and safety profile by an ex-China health authority as supported by existing data. This FTD will help strengthen our communications and collaboration with FDA in future clinical development and expedite the development and review of HQP1351 in the US. Staying committed to the mission of addressing unmet clinical needs in China and around the world, we will further accelerate the clinical development of HQP1351 to hopefully soon provide a safer and more effective treatment option to patients with CML."