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Aprea Therapeutics Reports Third Quarter 2023 Financial Results and Provides a Business Update

On November 9, 2023 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company focused on precision oncology through synthetic lethality, reported financial results for the three and nine months ended September 30, 2023, and provided a business update (Press release, Aprea, NOV 9, 2023, View Source [SID1234637351]).

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"We are very pleased by the progress of our diversified programs this past quarter. Importantly, we presented initial clinical data from our Phase 1/2a study of our ATR inhibitor, ATRN-119, in solid tumors in a poster at the recent AACR (Free AACR Whitepaper)-NCI-EORTC International Conference. To date, ATRN-119 has demonstrated an ability to be a very compelling molecule, appearing to be well tolerated with no reports of dose-limiting toxicities and ongoing daily dosing may result in persistent tumor-reducing effect," said Oren Gilad, Ph.D., President and Chief Executive Officer of Aprea. "We are continuing with patients in the dose escalation portion of the study, and the dose expansion cohort is on track to be initiated in 2Q 2024. In our WEE1 inhibitor, ATRN-1051, program we are on track to file an IND with the FDA by the end of 2023, and plan to begin clinical testing in the first half of 2024. Our strong balance sheet continues to support our strategy and plans through our near-term milestones in both our ATR and WEE1 programs, with a cash runway through the end of the fourth quarter of 2024. We look forward to providing more updates as we make progress and reach important milestones in the coming weeks and months."

Key Business and Financial Updates

Hosted a Key Opinion Leader (KOL) event on October 31, 2023, highlighting the Company’s portfolio of small molecules focused on Synthetic Lethality (SL) by targeting the DNA Damage Response (DDR) Pathways. The event featured Key Opinion Leaders Dr. Fiona Simpkins, Professor in the Division of Gynecology Oncology and Department of OB-GYN at the University of Pennsylvania, Dr. Timothy Yap, medical oncology physician-scientist and Professor at the University of Texas MD Anderson Cancer Center, Dr. Eric Brown, a consultant to Aprea and a Professor at the University of Pennsylvania and a member of the Abramson Family Cancer Research Institute, and Aprea’s Dr. Nadeem Mirza, Senior Medical Advisor. The speakers, along with the management team, provided an overview of the Company’s lead ATR inhibitor candidate, ATRN-119, and its WEE1 inhibitor candidate, ATRN-1051, and highlighted the addressable unmet clinical need and potential combination therapies using these programs.
Presented initial clinical data on the Company’s ATR inhibitor, ATRN-119, and preclinical data on its WEE1 inhibitor, ATRN-1051, at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) on October 14, 2023. The first poster included initial data from the Company’s first-in-human Phase 1/2a dose escalation trial of ATRN-119 in solid tumors. The trial is being conducted to determine the recommended Phase 2 dose, with a daily dosing administration over a 56-day cycle. The Company is actively enrolling cohort 4 at 350mg, with subsequent 550mg cohort 5 and 800mg cohort 6 planned. The Company anticipates enrolling the first patient in the dose expansion portion of the study in Q2 2024.
The second poster presented preclinical data from the Company’s WEE1 inhibitor program that demonstrated the potential safety and efficacy of its highly differentiated WEE1 inhibitor, ATRN-1051, in the treatment of ovarian cancer. The data showed ATRN-1051 to be a highly potent and selective inhibitor of WEE1 that does not significantly affect the off-target PLK1, PLK2, and PLK3 family of kinases. ATRN-1051 shows potentially favorable PK properties and appears to cause lower inhibition of hERG, a potential indication of low cardiotoxicity. Importantly, at doses and scheduling that suppress tumor growth, ATRN-1051 causes little anemia. These findings have justified IND-enabling studies for clinical development of ATRN-1051. Evidence generated by Aprea suggests such off-targeting of the PLK family, which has been a challenge for other WEE1 inhibitors in the class, substantially limits the ability of WEE1 inhibitors to cause cell death.
Appointed Dr. Jean-Pierre Bizzari to its Board of Directors. Dr. Bizzari has been responsible for numerous global approvals of several billion-dollar therapies, has been involved in acquisition and licensing agreements with several major pharmaceutical companies, and is a member and leader on many scientific committees. The Company also named Dr. Richard Peters as Chairman of the Board; Dr. Peters has served as a member of the Board since June 2020 bringing over 2 decades of experience in developing new therapies for difficult-to-treat diseases.
Potential Upcoming Key Milestones

ATR Inhibitor Clinical Program (ATRN-119)

Phase 1/2a Monotherapy Dose Escalation study
1Q 2024 Complete dose escalation
Phase 1/2a Monotherapy Dose Expansion study
2Q 2024 First patient enrolled
WEE1 Inhibitor Program (ATRN-1051)

IND
4Q 2023 IND Submission
1Q 2024 IND Clearance

Phase 1/2a Monotherapy Dose Escalation Study

1H 2024 First patient enrolled
Select Financial Results for the Third Quarter ended September 30, 2023

As of September 30, 2023, the Company reported cash and cash equivalents of $25.4 million.
For the quarter ended September 30, 2023, the Company reported an operating loss of $3.5 million, compared to an operating loss of $4.2 million for the same period in 2022.
Research and Development (R&D) expenses were $2.1 million for the quarter ended September 30, 2023, compared to $1.1 million for the same period in 2022. The increase in R&D expense was related to IND enabling studies for ATRN-1051, the Company’s small molecule WEE1 inhibitor, offset in part by a decrease in personnel costs related to the former facility in Sweden.
General and Administrative (G&A) expenses were $1.7 million for the quarter ended September 30, 2023, compared to $3.1 million for the same period in 2022. The decrease in G&A expenses was due to a decrease in professional fees primarily associated with post-acquisition activities during 2022, a decrease in insurance premiums, and a decrease in personnel costs related to the former facility in Sweden.
The Company reported a net loss of $3.2 million ($0.86 per basic share) on approximately 3.7 million weighted-average common shares outstanding for the quarter ended September 30, 2023, compared to a net loss of $4.0 million ($2.32 per basic share) on approximately 1.7 million weighted average common shares outstanding for the same period in 2022.

APDN Ships First saRNA (self-amplifying mRNA) IVT Template Generated by Linea™ DNA Platform

On November 9, 2023 Applied DNA Sciences, Inc. (NASDAQ: APDN) ("Applied DNA" or the "Company"), a leader in PCR-based DNA technologies, reported that it has successfully manufactured and shipped its first research use only quantity of self-amplifying mRNA (saRNA) template generated by its Linea DNA platform (the "Platform") to an unidentified preclinical therapeutic mRNA developer (Press release, Applied DNA Sciences, NOV 9, 2023, View Source [SID1234637350]).

saRNA is a promising new form of mRNA therapy engineered to possess a self-replicating capability. Unlike conventional non-amplifying mRNA, saRNA carries the machinery necessary for its replication, resulting in a higher therapeutic index at a lower dose. Due to these advantages, interest in saRNA is rapidly growing, as evidenced by 10 active saRNA vaccine clinical trials as of Jan. 20231 and numerous companies engaged in preclinical studies to use saRNA to treat indications ranging from cancer to protein replacement therapies. Like mRNA, saRNA is also produced from a DNA template.

The manufacturing of saRNA, however, has proven to be challenging due to the large size and complexity of saRNA and its associated DNA IVT templates. The DNA templates for saRNA are often 3x-5x larger than the DNA templates for conventional non-amplifying mRNA. saRNA can also be challenging to produce efficiently in DNA plasmids, often leading to heterogeneous DNA sequences, low yields, and long manufacturing timelines.

In contrast, the Company’s Platform can rapidly and efficiently enzymatically produce the challenging DNA sequences necessary to produce saRNA with high levels of homogeneity, particularly in challenging sequences such as the poly(T) homopolymers required to produce saRNA. Due to these Platform advantages, the Company is working with additional customers to produce preclinical saRNA IVT templates.

"RNA technology is today central to the development of biologics for human health, and DNA is its manufacturing starting point," stated Dr. James A. Hayward, president and CEO of Applied DNA. "Development pipelines are beginning to see an uptake of saRNA for next-generation mRNA therapies. We believe our Platform’s ability to enzymatically produce the challenging and large DNA sequences needed to manufacture saRNA at scale puts us at the forefront of template manufacturing for this promising and growing mRNA modality."

Aeglea BioTherapeutics Reports Third Quarter 2023 Financial Results and Provides Corporate Update

On November 9, 2023 Aeglea BioTherapeutics, Inc. ("Aeglea" or the "Company") (NASDAQ:AGLE), a biotechnology company advancing a pipeline of antibody therapeutics with the potential to transform the treatment of inflammatory bowel disease ("IBD"), reported its third quarter 2023 financial results and provided program and corporate updates (Press release, Aeglea BioTherapeutics, NOV 9, 2023, View Source [SID1234637349]).

"We have made significant progress in our first quarter following the Spyre acquisition towards our goal of creating new, best-in-class medicines for patients with IBD. We believe that our combination strategy and pipeline, including SPY001, an extended half-life anti-α4β7 antibody, and SPY002 a dual monomer / trimer anti-TL1A antibody with picomolar affinity and extended half-life, offer the potential to meaningfully improve both efficacy and convenience relative to today’s standard of care," said Cameron Turtle, D.Phil., Chief Operating Officer of Aeglea. "In addition to progressing our portfolio towards first-in-human studies next year, we have built out our team with passionate and experienced biotechnology leaders."

Development Pipeline Overview and Update

With its acquisition of Spyre Therapeutics, Inc. ("Spyre") in June 2023, Aeglea shifted its disease focus to IBD. The Company’s approach combines best-in-class antibody engineering, rational therapeutic combinations, and precision immunology with the goal of maximizing efficacy, safety, and convenience of its IBD treatments under development. IBD is a chronic condition characterized by inflammation within the gastrointestinal tract,

including two main disorders: ulcerative colitis ("UC") and Crohn’s disease ("CD"). In the United States, it is estimated that approximately 2.4 million individuals are diagnosed with IBD.

The Company has four product candidates in preclinical development, three of which are validated targets in IBD. The fourth product candidate is a novel, undisclosed target.

SPY001 – a highly potent and selective anti-α4β7 monoclonal antibody engineered with half-life extension technology and formulated for high concentration, convenient dosing.

•SPY001 is currently progressing through IND-enabling studies and is expected to enter first-in-human ("FIH") studies in the first half of 2024.
•Interim data from a healthy volunteer study are expected by the end of 2024. The Company expects pharmacokinetic data to demonstrate proof of concept for its ability to potentially reach an every-eight-week or every-twelve-week dosing interval.

SPY002 – a highly potent and selective anti-TL1A monoclonal antibody engineered with half-life extension technology. The Company believes TL1A has emerged as one of the most promising targets in IBD and broader immunology indications.

•The Company’s extensive discovery campaign has identified lead clones which bind both TL1A monomers and trimers with picomolar affinity and have in vitro potency and pharmacokinetic half-lives that exceed all clinical-stage TL1A antibodies.
•The Company expects to begin FIH studies of the SPY002 program in the second half of 2024 with healthy volunteer interim data expected in the first half of 2025.

SPY003 – a highly potent and selective monoclonal antibody targeting the p19 subunit of IL-23 engineered with half-life extension technology.

•The Company continues preclinical development efforts on a potential best-in-class IL-23 monoclonal antibody. Recent data from the Phase 3 SEQUENCE study of risankizumab versus ustekinumab in Crohn’s disease validates the Company’s targeting of the p19 subunit as it demonstrated superiority to targeting the p40 subunit of IL-23.
•An IND/CTN is expected in 2025.
Corporate Updates

•In October 2023, the Company filed a definitive proxy statement regarding the Special Meeting of Stockholders to be held on November 21, 2023 (the "Special Meeting"). At the Special Meeting, stockholders are expected to vote on four proposals, including the conversion of the Company’s Series A Preferred Stock to common stock and an increase to the Company’s authorized shares.
•In September 2023, the Company strengthened its leadership team with the appointments of industry veterans Scott Burrows, as Chief Financial Officer, and Heidy Abreu King-Jones, as Chief Legal Officer and Corporate Secretary.
•In September 2023, the Company completed a reverse stock split of all outstanding shares of common stock at a ratio of 1-for-25, with trading on a split-adjusted basis on the Nasdaq Capital Market beginning on September 8, 2023.
•In July 2023, the Company sold the global rights to pegzilarginase to Immedica Pharma AB ("Immedica") for $15 million in upfront cash proceeds and up to $100 million in contingent milestone payments. In November 2023, holders of the Company’s contingent value rights (CVRs) are expected to receive a payment representing the Immedica sale proceeds net of expenses and adjustments pursuant to the CVR agreement entered into in connection with Aeglea’s acquisition of Spyre.

Third Quarter 2023 Financial Results
Cash Position: As of September 30, 2023, Aeglea had available cash and cash equivalents, marketable securities, and restricted cash of $204.9 million.
Research and Development (R&D) expenses: R&D expenses totaled $24.7 million for the third quarter of 2023 and $12.0 million for the third quarter of 2022. This increase was primarily related to increases in preclinical development and manufacturing expenses for the Company’s IBD pipeline, partially offset by a decrease in expenses associated with the legacy Aeglea rare disease pipeline.
General and Administrative (G&A) expenses: G&A expenses totaled $8.6 million for the third quarter of 2023 and $7.0 million for the third quarter of 2022. This increase was primarily due to an increase in legal and employee separation costs.
Gain on Sale of In-Process Research & Development Asset: During the third quarter of 2023, the Company recognized a $14.6 million gain on the sale of the global rights of pegzilarginase to Immedica.
Other Income and Expenses: Other Income and Expenses were a net $21.8 million expense in the third quarter of 2023, primarily driven by a $25.4 million non-cash forward contract liability expense related to an increase in fair value of the underlying Series A Preferred Stock between June 30, 2023 and the forward contract’s settlement on July 7, 2023.
Net Loss: Net loss totaled $40.1 million and $18.2 million for the third quarter of 2023 and 2022, respectively, which includes non-cash stock compensation expense of $4.8 million and $1.6 million for the third quarter of 2023 and 2022, respectively.

Adaptive Biotechnologies Reports Third Quarter 2023 Financial Results and Provides Business Update

On November 9, 2023 Adaptive Biotechnologies Corporation ("Adaptive Biotechnologies") (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, reported financial results for the quarter ended September 30, 2023 (Press release, Adaptive Biotechnologies, NOV 9, 2023, View Source [SID1234637348]).

"We have two compelling businesses in MRD and Immune Medicine, which continue to make significant progress in divergent ways," said Chad Robins, chief executive officer and co-founder of Adaptive Biotechnologies. "Given the advancements achieved to date by each business and their respective investment needs, we are conducting a review of strategic alternatives to maximize value for our patients and shareholders."

Recent Highlights

•
Strategic review underway with Goldman Sachs to maximize the value of the MRD and Immune Medicine businesses.
•
Revenue for the third quarter 2023 was $37.9 million, representing a 21% decrease from the third quarter 2022 driven primarily by a 61% reduction in GNE amortization and a 14% decrease in MRD pharma services and IM pharma services largely due to biopharmaceutical industry headwinds.
•
clonoSEQ test volume in the third quarter 2023 grew 56% to 15,072 tests delivered versus the third quarter of prior year.
•
Immune Medicine achieved a key milestone with the discovery of a novel target in multiple sclerosis, which validates our target discovery approach in autoimmune disorders.
•
Operating expenses, excluding cost of revenue, for the third quarter 2023 decreased 11% versus the same period last year as a result of continued operating efficiencies efforts.
Third Quarter 2023 Financial Results

Revenue was $37.9 million for the quarter ended September 30, 2023, representing a 21% decrease from the third quarter in the prior year. Immune Medicine revenue was $13.3 million for the quarter, representing a 52% decrease from the third quarter in the prior year. MRD revenue was $24.7 million for the quarter, representing a 24% increase from the third quarter in the prior year.

Operating expenses were $88.9 million for the third quarter of 2023, compared to $93.3 million in the third quarter of the prior year, representing a decrease of 5%. Interest expense from our revenue interest purchase agreement was $3.7 million for the third quarter of 2023, compared to $0.7 million in the third quarter of the prior year.

Net loss was $50.3 million for the third quarter of 2023, compared to $45.3 million for the same period in 2022.

Adjusted EBITDA (non-GAAP) was a loss of $29.8 million for the third quarter of 2023, compared to a loss of $25.9 million for the third quarter of the prior year.

Cash, cash equivalents and marketable securities was $371.1 million as of September 30, 2023.

2023 Financial Guidance Update

In light of the strategic review and evolving focus of Immune Medicine as a drug discovery business, Adaptive Biotechnologies is updating full year 2023 revenue guidance to exclude revenue from the Immune Medicine business.

MRD business full year 2023 revenue to be in the range of $100 million to $105 million.

Total company operating expenses, including cost of revenue, for full year 2023 to be around $375 million.

Webcast and Conference Call Information

Adaptive Biotechnologies will host a conference call to discuss its third quarter 2023 financial results after market close on Thursday, November 9, 2023 at 4:30 PM Eastern Time. The conference call can be accessed at View Source The webcast will be archived and available for replay at least 90 days after the event.

VANFLYTA® Approved in the EU as the First FLT3 Inhibitor Specifically for Patients with Newly Diagnosed FLT3-ITD Positive AML

On November 9, 2023 Daiichi Sankyo’s (TSE: 4568) reported the VANFLYTA (quizartinib) has been approved in the European Union (EU) for use in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by VANFLYTA single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive (Press release, Daiichi Sankyo, NOV 9, 2023, View Source [SID1234637289]).

VANFLYTA is the first FLT3 inhibitor approved in the EU specifically for the treatment of patients with newly diagnosed FLT3-ITD positive AML, which represents about 25 to 30% of all new AML cases.

The authorization by the European Commission (EC) follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on the results of the QuANTUM-First trial, which were published in The Lancet. In QuANTUM-First, VANFLYTA combined with standard cytarabine and anthracycline induction and standard cytarabine consolidation, and continued as maintenance monotherapy following consolidation, demonstrated a 22% reduction in the risk of death compared to standard chemotherapy alone (HR = 0.78 [95% CI: 0.62-0.98; p=0.032]) in patients with newly diagnosed FLT3-ITD positive AML. Median overall survival was 31.9 months for patients receiving VANFLYTA (n=268; 95% CI: 21.0-NE) compared to 15.1 months for patients in the control arm (n=271; 95% CI: 13.2-26.2) at a median follow-up of 39.2 months.

"This approval of VANFLYTA represents an important advancement for frontline treatment of patients with FLT3-ITD positive acute myeloid leukemia, an aggressive and historically difficult-to-treat subtype," said Richard F. Schlenk, MD, Professor and Head of the Trial Center of the National Center of Tumour Diseases, Heidelberg University Hospital and German Cancer Research Center, Germany. "VANFLYTA is a potent and selective FLT3 inhibitor that significantly improved overall survival when added to standard chemotherapy and it will be a valuable treatment option for newly diagnosed FLT3-ITD positive AML."

"This approval of VANFLYTA is very welcome news for eligible patients diagnosed with FLT3-ITD positive AML each year," said Samantha Nier, Network Director, Acute Leukemia Advocates Network (ALAN). "New medicines and treatment approaches are needed to help patients with this difficult type of leukemia live longer, and we look forward to VANFLYTA becoming available in countries throughout the EU."

The safety profile of VANFLYTA in QuANTUM-First was consistent with previous clinical trials with no new safety signals observed. The most common grade 3 or 4 treatment emergent adverse events (occurring in ≥ 10% of patients) were febrile neutropenia (43%), hypokalemia (19%), neutropenia (18%) and pneumonia (11%). QTcF > 500 ms occurred in 2.3% of patients receiving VANFLYTA and 0.8% of patients discontinued VANFLYTA due to QT prolongation. Ventricular arrhythmia events with VANFLYTA were uncommon. Two (0.8%) patients receiving VANFLYTA experienced cardiac arrest with recorded ventricular fibrillation on ECG (one with fatal outcome), both in the setting of severe hypokalemia.

"With the approval of VANFLYTA in the European Union, patients diagnosed with FLT3-ITD positive acute myeloid leukemia may for the first time receive a targeted therapy developed and approved specifically for their disease subtype," said Ken Keller, Global Head of Oncology Business, and President and CEO, Daiichi Sankyo, Inc. "VANFLYTA is the second innovative medicine from our oncology pipeline approved in the EU and its successful development reflects our commitment to creating new standards of care for patients with cancer."

About QuANTUM-First
QuANTUM-First is a randomized, double-blind, placebo-controlled, global phase 3 study evaluating VANFLYTA in combination with standard induction and consolidation therapy, including hematopoietic stem cell transplant (HSCT), and as maintenance monotherapy, in adult patients aged 18-75 with newly diagnosed FLT3-ITD positive AML. Patients were randomized 1:1 to receive VANFLYTA or placebo combined with cytarabine and anthracycline induction and cytarabine consolidation chemotherapy followed by up to three years of treatment with single-agent maintenance.

The primary study endpoint was overall survival. Secondary endpoints include event-free survival, postinduction rates of complete remission (CR) and composite complete remission (CRc), and the percentage of patients who achieve CR or CRc with FLT3-ITD measurable residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints including duration of CR also were evaluated.

QuANTUM-First enrolled 539 patients at 193 study sites in 26 countries across Asia, Europe, North America, Oceania and South America. For more information, visit ClinicalTrials.gov.

About FLT3-ITD Positive Acute Myeloid Leukemia
More than 474,500 new cases of leukemia were reported globally in 2020 with more than 311,500 deaths.3 AML accounts for 23.1% of total leukemia cases worldwide and is most common in adults.4,5 In Europe, approximately 18,000 people are diagnosed with AML each year and the five-year survival rate is reported at 17% for adult patients.6,7

A number of gene mutations have been identified in AML and FLT3 (FMS-like tyrosine kinase 3) mutations are the most common.8 Approximately 80% of FLT3 mutations are FLT3-ITD mutations, which drive cancer growth and contribute to particularly unfavorable prognosis including increased risk of relapse and shorter overall survival.1,2 FLT3-ITD mutations occur in about 25% of all AML cases, with frequency reported as high as 30%.1,2

About VANFLYTA
VANFLYTA is an oral, highly potent type II FLT3 inhibitor that selectively targets FLT3-ITD mutations and has been specifically developed for patients with FLT3-ITD positive AML.

VANFLYTA is approved in the EU in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by VANFLYTA single-agent maintenance therapy for adult patients with newly diagnosed AML that is FLT3-ITD positive.

VANFLYTA is approved in the U.S. in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive as detected by an FDAapproved test. VANFLYTA is not indicated as maintenance monotherapy following allogeneic hematopoietic stem cell transplantation (HSCT); improvement in overall survival with VANFLYTA in this setting has not been demonstrated.

VANFLYTA also is approved in Japan for the treatment of AML that is FLT3-ITD mutation positive, including for use in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy and as maintenance monotherapy for adult patients with newly diagnosed FLT3-ITD positive AML and as a monotherapy for relapsed/refractory AML that is FLT3-ITD positive as detected by an approved test. VANFLYTA is an investigational medicine in all countries outside of Europe, Japan and the U.S.

About the VANFLYTA Clinical Development Program
The VANFLYTA clinical development program includes a phase 1/2 trial in pediatric and young adult patients with relapsed/refractory FLT3-ITD positive AML in Europe and North America and several phase 1/2 combination studies as part of a strategic collaboration with The University of Texas MD Anderson Cancer Center.