VolitionRx Limited Announces First Quarter 2021 Financial Results and Business Update

On May 11, 2021 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported financial results and a business update for the first quarter ended March 31, 2021 (Press release, VolitionRX, MAY 11, 2021, View Source [SID1234579670]). Volition management will host a conference call tomorrow, May 12 at 8:00 a.m. U.S. Eastern Time to discuss these results. Conference call details may be found below.

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"We have made significant progress on many fronts during 2021 on all four of the Company’s key pillars: Nu.Q, Nu.Q Vet, Nu.Q NETs and Nu.Q Capture," commented Cameron Reynolds, President and Chief Executive Officer of Volition. "We have also significantly strengthened our balance sheet, our intellectual property portfolio, and our executive team to drive commercial focus."

View Source

An interview with Cameron Reynolds, President and Chief Executive Officer and Terig Hughes, Chief Financial Officer.

Mr. Reynolds continued, "Silver One, our manufacturing facility and service lab, is proving to have been an excellent addition to Volition’s commercialization efforts and is now producing finished product and components at commercial scale for our anticipated product launches. I am delighted that the Nu.Q Vet beta launch has not only provided revenue but also invaluable real-world learnings allowing us to move ahead soon with our expected national U.S. and international launches. We are also seeing a high level of interest in both the licensing and distribution of our products and are actively negotiating multiple agreements."

Company Highlights

Financial

Cash and cash equivalents as of March 31, 2021 totaled approximately $33.1 million compared with $19.4 million as of December 31, 2020.
During the first quarter of 2021, added an aggregate of approximately $20.3 million in cash mostly through an underwritten public offering of our common stock that closed in February, as well as through periodic sales of common stock under Volition’s at-the-market equity distribution program.
Secured a further $4 million in non-dilutive funding consisting of a cash grant of $1.3 million and loans totalling $2.7 million from the Walloon Regional Government and associated agencies.
Continued to manage expenditures carefully with a cash burn rate of approximately $2 million per month.
Personnel

As Volition transitions from a research and development company to a commercial company, it strengthened the leadership team with the appointment of a new Chief Financial Officer, Mr. Terig Hughes, as well as the appointment of Mr. Gael Forterre as Chief Commercial Officer. In addition, Volition promoted Dr. Gaetan Michel to Chief Operating Officer and Dr. Mark Eccleston to Chief Technology Officer.
Volition announced the appointment of two Independent Directors to its main Board – Kim Nguyen, an HR executive with global expertise at Google, and Richard Brudnick, a pharmaceutical business executive with extensive commercial know-how.
As of May 1, Dr. Tom Butera, a seasoned veterinary executive with an extensive commercial track record, joined the team as Chief Executive Officer of Volition’s Veterinary subsidiary to drive its product launches.
Intellectual Property

28 patent families covering both human and animal use of Volition’s Nucleosomics platform.
64 granted patents (ten in the U.S., 14 in Europe and 42 rest of world).
93 patents pending.
Continued focus on filings during the first quarter and expect portfolio to grow in the quarters and years ahead.
Publications

Volition’s list of publications and abstracts is growing.
Year to date data for Nu.Q has been presented at two international conferences and Volition has collaborated on three clinical paper publications.
These publications are another very important step forward for the Company.
Nu.Q Vet Cancer Screening Test Beta Launch

Beta launch of the Nu.Q Vet Cancer Screening Test in late November 2020 in Texas. This beta launch is facilitating real-world learnings to help shape the marketing mix before Volition’s planned launch nationally across the U.S. expected in the next few months.
Volition has received strong indications of interest in the whole Nu.Q Vet platform, from a range of small and very large companies, and it is progressing potential licensing discussions with several well-known major players in the veterinary space around the world.
In addition to the Texas beta launch, Volition is finalizing beta launch planning in both Asia and Europe.
Nu.Q Capture

The clinical paper published in Nature’s Scientific Reports in March highlighted for the first time that histone H2A1R3 citrulline is, in plasma, upregulated in colorectal cancer patients and so could be a biomarker Volition targets for future Nu.Q immunoassay development.
It showed that the use of Nu.Q Capture may open up the possibility of using mass spectrometry not only for biomarker discovery but also as a high throughput platform for screening and/or diagnostics.
Volition aims for Nu.Q Capture to be an important enabling technology in the liquid biopsy space and expects to launch its first product with this technology in 2022.
Clinical – NETosis including COVID-19

Volition believes that the versatility of the Nu.Q platform and the range of applications for which these assays can be leveraged may help increase diagnostic power and monitor disease progression and potentially treatment response across a broad range of diseases that involve the over production of NETs, such as COVID-19, pneumonia, influenza and sepsis.
From a sepsis product development perspective, to date Volition has completed animal studies in the monitoring of disease progress and treatment efficacy which have demonstrated the effective use of Nu.Q NETs.
Human studies in the monitoring of disease progress and treatment efficacy in sepsis are now underway at a leading UK hospital.
From a COVID-19 product development perspective, Volition has conducted studies of serial testing of COVID-19 patients at leading UK hospitals to determine the predictive value of its test, with further data due to be presented at upcoming conferences and potentially additional studies and collaborations as well.
Clinical – Cancer

As previously reported, due to the pandemic collection for the U.S. EDRN study for colorectal cancer has been paused, and collection for the blood cancer studies in the U.S. have been delayed with an updated timeline expected in the near future.
In Asia, Volition has reached its target patient cohorts for both colorectal cancer studies and the lung cancer study, all collected in conjunction with the National Taiwan University, notwithstanding the pandemic. Volition expects to run the assays and finalize the analysis over the coming months with data due to be reported at future conferences.
Volition has installed a Service Lab in Silver One, which will undertake sample processing for external parties such as pharmaceutical companies, biotech companies and academic researchers. This service, branded as Nu.Q Discover, has already generated interest, and Volition expects to see initial revenue from this during 2021.
Volition is producing recombinant nucleosomes in-house and at large-scale, building a library for sale through Nu.Q Discover and also for internal R&D use.
Volition is still in active discussions on its first potential licensing deal with a large healthcare company.
Upcoming Milestones

Volition expects to achieve the following milestones during 2021 and beyond, pandemic permitting:

National launch of the Nu.Q Vet Cancer Screening Test in the U.S.
Beta launches of the Nu.Q Vet Cancer Screening Test in Asia and Europe.
Continue to focus on driving revenue in the coming quarters in four key areas:
– Nu.Q Vet products,
– Disease monitoring tests (e.g. Nu.Q NETs for COVID-19, sepsis),
– Nu.Q Discover, and
– Licensing of its technology for others to commercialize in both humans and animals.

Continue to progress the research program for the use of Nu.Q in NETosis, in monitoring disease progression of COVID-19, sepsis and potentially other diseases and as a possible companion diagnostic for a treatment for sepsis.
Continue to advance its previously announced large-scale blood, lung and colorectal cancer trials in Europe, Asia and the U.S.
Publish several abstracts and peer-reviewed scientific papers with clinical results showing the robustness and utility of its Nu.Q platform.
Advance the development of Nu.Q Capture.
Continue to file patents to expand and extend its intellectual property portfolio.
VolitionRx Limited First Quarter 2021 Financial Results and Business Update

Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with Terig Hughes, 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 26, 2021. 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 13719633.

Prothena Reports First Quarter 2021 Financial Results, and Provides Updated Financial Guidance and R&D Update

On May 11, 2021 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical company with a robust pipeline of novel investigational therapeutics built on protein dysregulation expertise, reported financial results for the first quarter of 2021. In addition, the Company provided an update on its R&D programs and 2021 financial guidance (Press release, Prothena, MAY 11, 2021, View Source [SID1234579669]).

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"We recently welcomed Hideki Garren to Prothena as Chief Medical Officer and look forward to his expertise contributing to our transition to a fully integrated research, development and commercial biotechnology company," said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "The oral presentations at AD/PD and AAN highlight the continued translation of our differentiated protein dysregulation scientific platform from preclinical findings to clinical benefit for patients across multiple programs in our portfolio. We remain on track for significant news flow this year with the planned initiation of three late-stage clinical studies for birtamimab, prasinezumab and PRX004. We will advance two programs in our Alzheimer’s portfolio towards the clinic over the next 12 months, with IND filings for our anti-tau PRX005 and anti-abeta PRX012 programs, designed to be best-in-class treatments for Alzheimer’s disease. Additionally, our cash position was strengthened by our public offering in March and will be further bolstered when we receive the $60 million milestone earned from Roche. Together this enhances our ability to fund the company through multiple key clinical milestones."

First Quarter and Recent Highlights:

Birtamimab, a potential treatment for AL amyloidosis, is a humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure

Based on the significant survival benefit favoring birtamimab, from the previous VITAL study in a subset of patients categorized as Mayo Stage IV at baseline (HR=0.413, p=0.025, over 9 months), and multiple in-depth discussions with the U.S. Food and Drug Administration (FDA), Prothena announced plans in February 2021 to advance birtamimab into the confirmatory Phase 3 AFFIRM-AL study in Mayo Stage IV patients with AL amyloidosis. AFFIRM-AL is a global, registration-enabling Phase 3 study that will be conducted under a Special Protocol Assessment (SPA) agreement with FDA to enable registration at p≤0.10 for the primary endpoint of all-cause mortality.
Prasinezumab, a potential treatment for Parkinson’s disease, is a humanized monoclonal antibody designed to target a key epitope within the C-terminus of alpha-synuclein and is the focus of the worldwide collaboration with Roche

$60 million milestone earned with first patient dosed in PADOVA study in the second quarter
Two oral presentations by Roche at the 15th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD 2021) released new data from Part 1 of the Phase 2 PASADENA study. The first presentation highlighted newly presented data from a pre-specified exploratory subgroup analysis showing slowing of clinical decline with prasinezumab was more evident in subgroups with more rapid disease progression. Separately, new digital biomarker data presented from Roche’s remote monitoring technology used in the study was consistent with a potential disease modifying effect of prasinezumab in slowing Parkinson’s disease progression.
PRX004, a potential treatment for ATTR amyloidosis, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the TTR protein

Oral presentation at the American Academy of Neurology (AAN) 2021 Virtual Meeting featuring results from the Phase 1 study of PRX004 in ATTR amyloidosis. PRX004 showed favorable results as demonstrated by slowing of neuropathy progression for all 7 evaluable patients at 9 months, including improvement in neuropathy in 3 of the 7 patients, and improved cardiac systolic function for all 7 patients. In this Phase 1 study, PRX004 was found to be generally safe and well tolerated across all dose levels.
PRX005, a potential treatment for Alzheimer’s disease (AD), is an investigational antibody that targets tau, a protein implicated in diseases including AD, frontotemporal dementia (FTD), progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE) and other tauopathies.

Oral presentation at AD/PD 2021 highlighting new preclinical data demonstrating that targeting an epitope within the microtubule binding region (MTBR) of tau with PRX005 resulted in superior attributes for the potential treatment of AD. PRX005 demonstrated significant inhibition of cell-to-cell transmission and neuronal internalization in vitro and in vivo, and slowed pathological progression in a tau transgenic mouse model.
Corporate

Appointed Hideki Garren, M.D., Ph.D., as Chief Medical Officer, responsible for leading the clinical and medical organizations to advance Prothena’s clinical pipeline. Dr. Garren joined Prothena after serving as Vice President, Global Head of Neuroimmunology at F. Hoffman-La Roche Ltd. (Roche) & Genentech Inc.
Executed a public offering in March that raised net proceeds of approximately $78 million through the issuance of 4,025,000 ordinary shares
Upcoming Milestones:

Birtamimab

Phase 3 AFFIRM-AL study initiation expected mid-2021
VITAL study 9-month results expected to be presented at a medical conference in 2021
Prasinezumab

Results from Part 2 of the PASADENA study expected to be presented at an upcoming medical conference
PRX004

Phase 2/3 study in patients with ATTR-cardiomyopathy expected to initiate 4Q 2021
PRX005

IND expected by 3Q 2021
$80 million potential payment from Bristol Myers Squibb upon exercising their US license option in 2021
PRX012, a potential treatment for Alzheimer’s disease, is a high affinity monoclonal antibody targeting a key epitope within the N-terminus of Aβ

IND expected by 1Q 2022
Upcoming Investor Conferences

Members of the senior management team will present and participate in investor meetings at the following upcoming investor conferences:

BofA Securities 2021 Virtual Health Care Conference, May 13, 2021, at 8:45 AM ET
RBC Capital Markets Global Healthcare Conference, May 19, 2021 at 8:35 AM ET
Jefferies Virtual Healthcare Conference, June 1, 2021 at 9:00 AM ET
First Quarter 2021 Financial Results and Updated 2021 Financial Guidance

For the first quarter of 2021, Prothena reported a net loss of $36.7 million, as compared to a net loss of $23.6 million for the first quarter of 2020. Net loss per share for the first quarter of 2021 was $0.91, as compared to a net loss per share of $0.59 for the first quarter of 2020.

Prothena reported total revenue of $0.2 million for the first quarter of 2021, from collaboration and license revenue from Roche, as compared to total revenue of $0.1 million for the first quarter of 2020, from collaboration revenue from Roche.

Research and development (R&D) expenses totaled $21.1 million for the first quarter of 2021, as compared to $15.2 million for the first quarter of 2020. The increase in R&D expense for the first quarter of 2021 compared to the same period in the prior year was primarily due to higher R&D consulting expense, higher personnel expense, higher manufacturing expense primarily related to our PRX012 and birtamimab programs, higher clinical trial expense primarily related to birtamimab partially offset by lower PRX004 clinical trial expenses and higher collaboration expense with Roche related to the prasinezumab program. R&D expenses included non-cash share-based compensation expense of $2.0 million for the first quarter of 2021, as compared to $2.0 million for the first quarter of 2020.

General and administrative (G&A) expenses totaled $11.1 million for the first quarter of 2021, as compared to $9.7 million for the first quarter of 2020. The increase in G&A expenses for the first quarter of 2021 compared to the same period in the prior year was primarily related to higher personnel expense and higher expense for our directors and officers insurance premium. G&A expenses included non-cash share-based compensation expense of $4.2 million for the first quarter of 2021, as compared to $3.5 million for the first quarter of 2020.

Total non-cash share-based compensation expense was $6.2 million for the first quarter of 2021, as compared to $5.5 million for the first quarter of 2020.

As of March 31, 2021, Prothena had $345.7 million in cash, cash equivalents and restricted cash (does not include the $60 million milestone earned in the second quarter from Roche) and no debt.

As of May 4, 2021, Prothena had approximately 44.2 million ordinary shares outstanding.

The Company continues to expect the full year 2021 net cash used in operating and investing activities to be $51 to $74 million, which includes receiving the $60 million milestone earned in the second quarter from Roche. The Company is updating its projected year end cash balance to approximately $316 million in cash, cash equivalents and restricted cash (midpoint) (versus prior guidance of $235 million) to include an additional $81 million in net proceeds primarily from the public offering in March. The estimated full year 2021 net cash used in operating and investing activities is primarily driven by an estimated net loss of $79 to $111 million, which includes an estimated $20 million of non-cash share-based compensation expense.

Kronos Bio Reports Recent Business Progress and First Quarter Financial Results and Announces Virtual R&D Day

On May 11, 2021 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, reported recent business progress and first quarter financial results (Press release, Kronos Bio, MAY 11, 2021, View Source [SID1234579668]).

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"With continued progress in our preclinical and clinical programs and significant milestones expected this year, we look forward to hosting a research and development day to present our pipeline and unveil our development strategy for both of our clinical-stage SYK inhibitors entospletinib and lanraplenib," said Norbert Bischofberger, Ph.D., president and CEO. "We recently showcased progress with our CDK9 program at the AACR (Free AACR Whitepaper) annual meeting in April, where we presented preclinical data indicating that KB-0742 could have utility in the treatment of MYC-amplified cancers. We look forward to sharing more about our development plans for this compound at our research and development day."

Dr. Bischofberger added: "I would also like to take this opportunity to reflect on the recent passing of our Board Member, Dr. John C. Martin, a dear friend and mentor, and to convey our deepest sympathies to John’s family and everyone who was fortunate enough to have known him."

Recent Highlights

Presented preclinical data for KB-0742, the company’s potent oral, highly selective cyclin dependent kinase 9 (CDK9) inhibitor, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data showed that CDK9 inhibition on an intermittent dosing schedule with KB-0742 resulted in sustained inhibition of tumor growth in multiple types of solid tumors, and suggested that genomic amplification of MYC, a well-characterized transcription factor and a long-recognized driver of cancer, is a key feature in defining sensitivity to CDK9 inhibition.

Announced the appointment of Taiyin Yang, Ph.D., to its board of directors. Dr. Yang currently serves as the executive vice president of Pharmaceutical Development and Manufacturing at Gilead Sciences, Inc., and has more than four decades of experience developing and manufacturing medicines in a variety of therapeutic categories.

Announced a positive End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for spleen tyrosine kinase (SYK) inhibitor entospletinib. Kronos Bio will proceed with its plan to assess measurable residual disease (MRD) negative complete response (CR) as the primary endpoint in a registrational Phase 3 trial to support potential accelerated approval of entospletinib in patients newly diagnosed with NPM1-mutated acute myeloid leukemia (AML). The company plans to initiate the Phase 3 trial in mid-2021, with MRD negative CR data expected in the second half of 2023.

Announced the first patient was dosed in the Phase 1/2 clinical trial of KB-0742, which is being developed to treat MYC-amplified solid tumors.
Pipeline updates planned at upcoming Virtual R&D Day on May 25, 2021

Unveil the development strategy for the company’s SYK inhibitors entospletinib and lanraplenib in AML. Kronos Bio executives will be joined by Eytan M. Stein, M.D., assistant attending physician and director, Program for Drug Development in Leukemia, Leukemia Service, Department of Medicine at Memorial Sloan Kettering Cancer Center, who will provide an overview of AML and the current treatment landscape.

Highlight the opportunity to target MYC through CDK9 inhibition with KB-0742, including a review of preclinical data, expectations for initial safety, pharmacokinetic and pharmacodynamic data anticipated in the fourth quarter of 2021 and potential populations for the expansion cohorts in the second stage of the company’s Phase 1/2 clinical trial.

Provide an overview of the company’s differentiated drug discovery platform and potential future pipeline programs.
The live webinar will begin at 1 p.m. ET on Tuesday, May 25, 2021, and will conclude at approximately 4:00 p.m. ET. Registration is accessible on the Investors & Media section of the company’s website at www.kronosbio.com. A replay of the webcast will be archived and available following the event.

First Quarter Financial Highlights

Cash, Cash Equivalents and Investments: As of March 31, 2021, cash, cash equivalents and investments totaled $440.6 million.

R&D Expenses: Research and development expenses were $17.6 million for the first quarter of 2021, which includes $2.5 million in non-cash stock-based compensation expense. R&D expenses for the quarter were primarily driven by costs associated with initiating and conducting the company’s clinical trials.

G&A Expenses: General and administrative expenses were $8.6 million for the first quarter of 2021, which includes $2.7 million in non-cash stock-based compensation expense.

Net Loss: Net loss for the first quarter of 2021 was $26.1 million, or $0.48 per share, including non-cash stock-based compensation expense of $5.2 million.

Neurocrine Biosciences to Present at the 2021 RBC Global Healthcare Conference

On May 11, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the 2021 RBC Capital Markets Healthcare Conference at 10:55 a.m. Eastern Time on Tuesday May 18, 2021 (Press release, Neurocrine Biosciences, MAY 11, 2021, View Source [SID1234579667]). Matt Abernethy, Chief Financial Officer, and Eiry Roberts, Chief Medical Officer, will present at the conference.

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The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

Syndax Pharmaceuticals Reports First Quarter 2021 Financial Results and Provides Clinical and Business Update

On May 11, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the first quarter ended March 31, 2021. In addition, the Company provided a clinical and business update (Press release, Syndax, MAY 11, 2021, View Source [SID1234579666]).

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"We are excited to provide an update on the positive interim Phase 1 data from the ongoing AUGMENT-101 trial of SNDX-5613 in patients with mixed lineage leukemia rearranged (MLLr) and nucleophosmin (NPM1c) mutant acute leukemias," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "As Dr. Eytan Stein indicated in his presentation, complete eradication of leukemia need not occur simultaneously with complete recovery of blood cell counts, and today we are able to share that two prior responders have advanced from complete response with incomplete platelet recovery (CRp) to a complete response (CR). The benefit we observe in AUGMENT-101 support our strong conviction that SNDX-5613 has the potential to meaningfully shape the treatment paradigm for these patients who are in desperate need of improved therapeutic options. As previously announced, we have identified a candidate recommended Phase 2 dose (RP2D) and look forward to commencing the pivotal Phase 2 portion of the trial."

"In addition, we continue to make progress with axatilimab in patients with chronic graft versus host disease (cGVHD). We are pleased to announce today that we have completed enrollment of 23 patients in the Phase 2 expansion portion of the Phase 1/2 trial. We look forward to sharing full updated results from the Phase 1/2 trial later this year, with top-line data from the ongoing pivotal AGAVE-201 trial expected in 2023. Supported by emerging data, we firmly believe that axatilimab has the potential to benefit many of the more than 10,000 patients diagnosed with cGVHD in the U.S. each year."

Recent Progress and Anticipated Milestones

SNDX-5613

The Company reported new data from the ongoing Phase 1 dose escalation portion of the Phase 1/2 AUGMENT-101 trial of SNDX-5613, a highly selective oral menin inhibitor, in patients with MLLr and NPM1c mutant relapsed/refractory (R/R) acute leukemias. The new data reported today, showed two prior responders have improved from CRp to CR with no evidence of minimal residual disease (MRD-). With the addition of these two patients, a total of 7/31 patients (23%) have achieved CR/CRh.

Syndax previously announced positive data from the Phase 1 portion of the AUGMENT-101 trial in April 2021 and hosted a conference call featuring Eytan M. Stein, M.D., Assistant Attending Physician and Director, Program for Drug Development in Leukemia, Department of Medicine at Memorial Sloan Kettering Cancer Center. As of the data cutoff date, the overall response rate (ORR) among evaluable patients was 48% (n=15), with 67% (n=10) of these responders achieving MRD- status, and four proceeding to receive stem cell transplant. The ORR in evaluable patients harboring an MLL-rearrangement (n=24), was 54% (n=13), and in evaluable patients harboring an NPM1c mutation (n=7), was 29% (n=2). Across all patients enrolled in the trial as of the data cutoff date (n=43), SNDX-5613 was generally well-tolerated, with no discontinuations due to treatment-related adverse events observed in heavily pretreated patients.

Syndax remains on track to initiate the pivotal Phase 2 portion of the trial this quarter.

Axatilimab

Syndax reported that enrollment is now complete in the Phase 2 expansion portion of the Phase 1/2 trial of axatilimab, its anti-CSF-1R monoclonal antibody, in patients with cGVHD. The Company anticipates reporting updated results later this year for 40 patients, including the 17 in the Phase 1 portion and 23 from the Phase 2 expansion portion, which evaluated 1 mg/kg of axatilimab every two weeks. Syndax reported preliminary data from the Phase 1 portion of the trial during an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2020 which highlighted the tolerability and high response rate of axatilimab in cGVHD patients refractory to multiple therapeutic agents.

The Company’s pivotal Phase 2 AGAVE-201 trial of axatilimab in patients with cGVHD is ongoing, with topline data expected in 2023. The trial will evaluate the safety and efficacy of three doses and schedules of axatilimab. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for cGVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score.

Earlier this year, the Company announced that the U.S. Food and Drug Administration granted Orphan Drug Designation to axatilimab for the treatment of patients with cGVHD and idiopathic pulmonary fibrosis.

First Quarter 2021 Financial Results

As of March 31, 2021, Syndax had cash, cash equivalents and short-term investments of $271.3 million and 51.6 million shares and share equivalents issued and outstanding. This includes 3.3 million pre-funded warrants.

First quarter 2021 research and development expenses increased to $21.9 million from $9.6 million. The increase was primarily due to increased clinical trial activities and increased CMC activities.

General and administrative expenses for the first quarter 2021 decreased to $5.7 million from $5.9 million. The decrease is primarily due to decreased pre-commercialization expenses for entinostat.

For the three months ended March 31, 2021, Syndax reported a net loss attributable to common stockholders of $27.7 million or $0.54 per share compared to $19.1 million or $0.56 per share for the prior year period.

Financial Update and Guidance

For the second quarter of 2021, research and development expenses are expected to be $30 to $35 million, and total operating expenses are expected to be $35 to $40 million. For the full year of 2021, research and development expenses are expected to be $90 to $100 million, and total operating expenses are expected to be $110 to $120 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Tuesday, May 11, 2021.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors section of the Company’s website, www.syndax.com.