On August 3, 2023 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in liver and viral diseases, reported recent business progress and financial results for the second quarter 2023 (Press release, Aligos Therapeutics, AUG 3, 2023, View Source [SID1234633733]).

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"We continue to make important progress in advancing our portfolio of drug candidates," said Lawrence Blatt, Ph.D., MBA, Chairman & CEO of Aligos Therapeutics. "Phase 2a enabling activities for our lead program in NASH, ALG-055009, are going well and we remain on track to file this important Phase 2a protocol to the US IND in Q4 2023. Additionally, our COVID-19 protease inhibitor, ALG-097558, is now dosing in the clinic in a first in human study and our best-in-class capsid assembly modulator, ALG-000184, continues to generate impressive DNA, RNA, and HBsAg lowering activity as dosing continues in CHB subjects. We look forward to sharing emerging data from these exciting programs at future scientific conferences."

Recent Business Progress

Aligos Portfolio of Drug Candidates

NASH Program (ALG-055009)

Dosing in the Phase 1 first-in-human study is now complete and the database is locked. Data at all dose levels continue to support a favorable risk-benefit profile for ALG-055009
Phase 2a enabling activities (e.g., drug manufacturing, non-clinical studies) are ongoing and on track for a Q4 2023 filing of the Phase 2a protocol
Key design elements/milestones of the Phase 2a study have been formulated and include:
Randomized, double-blind, placebo-controlled trial evaluating dosing for 12 weeks
Evaluation of multiple dose levels of ALG-055009 vs. placebo (gelcap formulation)
Primary endpoint based on change from baseline at 12 weeks in MRI-PDFF
Additional non-invasive biomarkers commonly evaluated in NASH trials will also be assessed
All sites will be in the US
Anticipated top line data: Q4 2024
Stephen Harrison, MD has signed on to be the Phase 2a study’s Principal Investigator
COVID-19 (ALG-097558)

The first-in-human study (ALG-097558-701) clinical trial application was approved in the UK
Dosing in Part 1, which is evaluating single ascending oral doses in healthy volunteers, of this multi-part study is ongoing
Dosing is expected to continue throughout 2023 and early 2024 with topline data anticipated in H1 2024
HBV Programs

Capsid-Assembly Modulator (ALG-000184)

Ongoing cohort data continue to show that 300 mg ALG-000184 + entecavir (ETV) is well tolerated and results in unprecedented HBsAg lowering activity for an oral CHB drug. Specifically, Hou et al., showed at EASL 2023 that a majority of HBeAg positive CHB subjects dosed with 300 mg ALG-000184 + ETV demonstrated declines of ≥0.4 and ≥1.0 log10 IU/mL at 12 and 24 weeks, respectively. The largest HBsAg reduction observed among subjects receiving this regimen was a 2 log10 IU/mL decline in a subject dosed for 36 weeks
Dosing with ALG-000184 + entecavir for up to 96 weeks in HBeAg positive and HBeAg negative CHB subjects is planned
Emerging data will continue to be presented at upcoming scientific conferences
ALG-125755

Dosing in Parts 1 and 2, which evaluated single ascending subcutaneous doses of ALG-125755 in healthy volunteers and virologically suppressed HBeAg negative CHB subjects, respectively, is now complete
Single doses of up to 320 mg ALG-125755:
Were found to be well tolerated with predicted PK
Lowered HBsAg levels across the dose range evaluated, but comparative efficacy data vs. competitor siRNAs are inconclusive
Further clinical evaluation of ALG-125755 is not prioritized with current funding. Further advancement will require partnership of the program.
Corporate

On July 31, 2023, Aligos Therapeutics, Inc. (the "Company") and Janssen Biopharma, LLC ("Janssen") filed a stipulation staying the case in their ongoing legal proceedings. The Company and Janssen have reached an agreement in principle to resolve their disputes and expect to finalize a settlement agreement promptly.
Financial Results for the Second Quarter 2023

Cash, cash equivalents and investments totaled $90.8 million as of June 30, 2023, compared with $125.8 million as of December 31, 2022. We continue to believe our cash balance provides sufficient cash to fund planned operations through the end of 2024.

Net losses for the three months ended June 30, 2023, were $18.8 million or basic and diluted net loss per common share of $(0.43), compared to net losses of $19.9 million or basic and diluted net loss per common share of $(0.47) for the three months ended June 30, 2022.

Research and development (R&D) expenses for the three months ended June 30, 2023, were $16.8 million compared with $16.5 million for the same period of 2022. The increase was primarily due to other costs including facility expenses due to the right-of-use asset impairment, largely offset by a decrease in third party expenses from the reduced manufacturing of drug supply in advance of our NASH program in 2022, and employee-related costs. Total R&D stock-based compensation expense incurred for the three months ended June 30, 2023, was $1.6 million compared with $2.2 million for the same period of 2022.

General and administrative (G&A) expenses for the three months ended June 30, 2023, were $9.2 million compared with $7.6 million for the same period of 2022. The increase in G&A expenses for this comparative period is primarily attributable to an increase in legal and related costs offset by a decrease in facility expenses. Total G&A stock-based compensation expense incurred for the three months ended June 30, 2023, was $1.6 million compared with $1.8 million for the same period of 2022.

On August 3, 2023 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported second quarter 2023 financial results and recent portfolio and business updates. As of June 30, 2023, Alector’s cash, cash equivalents and investments totaled $630.0 million (Press release, Alector, AUG 3, 2023, View Source [SID1234633732]).

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"We expect to complete enrollment in the INVOKE-2 Phase 2 clinical trial of AL002 in early Alzheimer’s disease in the third quarter," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "We have received positive feedback from the FDA and the EMA to conduct a primary analysis on approximately 90-100 symptomatic participants in our pivotal INFRONT-3 Phase 3 clinical trial of latozinemab in FTD-GRN. We anticipate completing enrollment in INFRONT-3 in the fourth quarter of this year."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector added, "While we continue to progress our late-stage clinical programs, we are also strategically advancing our innovative research portfolio to fuel our pipeline. I am particularly excited about the ongoing development of our blood brain barrier technology and the progress on our preclinical program targeting GPNMB for the treatment of Parkinson’s disease."

Recent Clinical Updates

Immuno-Neurology Portfolio
TREM2 Program (AL002) Being Developed in Collaboration with AbbVie

Alector closed screening and plans to complete enrollment in the INVOKE-2 clinical trial in the third quarter of 2023, with data expected in the fourth quarter of 2024. The INVOKE-2 Phase 2 trial is designed to evaluate the efficacy and safety of AL002 in slowing disease progression in individuals with early Alzheimer’s disease (AD).
Alector presented an update on INVOKE-2 at the Alzheimer’s Association International Conference (AAIC) in July 2023. The presentation highlighted that treatment-emergent MRI findings resembling amyloid-related imaging abnormalities (ARIA) in INVOKE-2 are similar to the ARIA reported following treatment with anti-amyloid beta antibodies.
Alector previously presented results from a Phase 1 trial of AL002 in healthy volunteers, which demonstrated both dose-dependent target engagement and activation of microglia.
Microglial activation is hypothesized to not only enhance clearance of misfolded proteins that accumulate and form amyloid plaques but also perform other supportive microglia functions, including maintenance of neuronal and synaptic health.
At AAIC, the company also presented a poster on mouse model data demonstrating that TREM2 activation improved AD biomarkers, including amyloid and tau.
Progranulin Programs (latozinemab (AL001) and AL101/ GSK4527226) Being Developed in Collaboration with GSK

Alector and GSK held a Type C meeting with the U.S. Food and Drug Administration (FDA) and received scientific advice from the European Medicines Agency (EMA) regarding the pivotal INFRONT-3 Phase 3 clinical trial of latozinemab in participants with frontotemporal dementia due to progranulin gene mutation (FTD-GRN).
Alector and GSK aligned with the FDA and EMA to conduct the primary analysis on symptomatic participants in INFRONT-3. Based on feedback from the regulatory agencies, the companies performed a sample size re-estimation that is anticipated to support a more focused enrollment of approximately 90-100 symptomatic participants for a treatment duration of 96 weeks.
Alector plans to complete enrollment in INFRONT-3 in the fourth quarter of 2023.
In a recent data cut from the FTD-C9orf72 cohort of the open label INFRONT-2 Phase 2 clinical trial of latozinemab, Alector confirmed a two- to threefold elevation in progranulin levels in CSF and plasma. The company conducted an analysis of disease progression rates for the 14 participants who were treated with latozinemab compared with baseline-matched controls from the ALLFTD registry. The small sample size and a high degree of variability in disease progression in both groups rendered the results uninformative regarding treatment effect.
In June 2023, Alector published a manuscript in the Journal of Translational Medicine titled "Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia." This publication details Alector’s development of latozinemab and highlights findings demonstrating that the sortilin receptor is a viable target for PGRN-elevating therapy, particularly in patients who have a PGRN deficiency leading to FTD.
Alector and GSK plan to initiate a global Phase 2 clinical trial with AL101/GSK4527226 in early AD. The companies also plan to present a poster on the pharmacokinetic and pharmacodynamic modeling of PGRN elevation in blood and CSF based on a Phase 1 trial of AL101 at the American College of Clinical Pharmacology (ACCP) being held in Bellevue, Washington from September 10-12, 2023.
Early Research Pipeline

Alector is developing its proprietary, versatile blood brain barrier technology to selectively support its next-generation product candidates.
The company is developing an investigational therapy, ADP027, which modulates GPNMB for the treatment of Parkinson’s disease.
Recent Corporate Updates

In the second quarter of 2023, the U.S. Patent and Trademark Office issued patents covering methods of treatment using AL002. The European Patent Office also issued a patent in the second quarter of 2023 covering AL002 compositions and methods of use.
Second Quarter 2023 Financial Results

Revenue. Collaboration revenue for the quarter ended June 30, 2023, was $56.2 million, compared to $79.9 million for the same period in 2022. The $23.6 million decrease was mainly due to $68.9 million of collaboration revenue recognized in the second quarter of 2022 due to changes in estimated costs to satisfy the performance obligations resulting from the termination of the AL003 program and a $16.7 million decrease in revenue recognized for the latozinemab programs. This was offset by a $33.5 million increase in revenue recognized for the AL101 programs, including a cumulative non-cash revenue adjustment due to contract modification to have GSK operationalize the AL101 Phase 2 trial, and a $28.5 million increase to collaboration revenue for the AL002 program due to changes in total expected costs and the addition of AL002 LTE and patient replacement revenue in 2023.

R&D Expenses. Total research and development expenses for the quarter ended June 30, 2023, were $46.2 million, compared to $54.5 million for the quarter ended June 30, 2022. The decrease of $8.4 million was mainly due to the Company’s strategy to prioritize late-stage programs and higher cost share amounts with GSK that are recorded as contra expense.

G&A Expenses. Total general and administrative expenses for the quarter ended June 30, 2023, were $13.6 million, compared to $15.8 million for the same period in 2022. The decrease of $2.2 million was primarily due to a decrease in consulting expenses related to accounting, recruiting, IT, and other general expenses.

Net Income. For the quarter ended June 30, 2023, Alector reported a net income of $1.4 million, or $0.02 net income per share, compared to a net income of $9.9 million, or $0.12 net income per share, for the same period in 2022.

Cash Position. Cash, cash equivalents, and investments were $630.0 million as of June 30, 2023. Management continues to expect that this will be sufficient to fund current operations through 2025.

2023 Guidance. Management is revising guidance for the year ending 2023, increasing collaboration revenue to now be between $90 million and $100 million, decreasing total research and development expenses to now be between $210 million and $220 million and tightening expectations for total general and administrative expenses to now be between $60 million and $65 million.

Second Quarter 2023 Conference Call

Alector’s management team will host a conference call discussing Alector’s results for the second quarter of 2023 and provide a business update. The conference call will be webcast and accessible via the investor relations section of Alector’s website at www.alector.com.

To access the call, please use the following information:

Date: Thursday, August 3, 2023
Time: 4:30 p.m. ET, 1:30 p.m. PT

The event will be webcast live under the investor relations section of Alector’s website at View Source and following the event a replay will be archived there for 30 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

On August 3, 2023 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported business highlights and financial results for the second quarter ended June 30, 2023 (Press release, Agios Pharmaceuticals, AUG 3, 2023, View Source [SID1234633731]).

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"Since our last quarterly update, Agios has made tremendous progress executing across our industry-leading pipeline of PK activators, and today we are further expanding our portfolio beyond PK activation through focused business development," said Brian Goff, chief executive officer at Agios. "We announced positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease, completed enrollment in three clinical studies, licensed a compelling preclinical program from Alnylam, and continued to strengthen our commercial capabilities to support future anticipated launches. We look forward to the readout of the Phase 2a study of AG-946 in lower-risk MDS by the end of this year and the readouts of the Phase 3 studies of mitapivat in thalassemia next year."

Second Quarter 2023 & Recent Highlights

PYRUKYND U.S. Launch: Generated $6.7 million in U.S. net revenue for the second quarter of 2023, a 20 percent increase over the first quarter of 2023. A total of 147 unique patients have completed prescription enrollment forms, representing an increase of 16 percent over the first quarter of 2023. A total of 99 patients are on PYRUKYND therapy, representing an 11 percent increase over the first quarter of 2023.
Sickle Cell Disease: Announced positive results from the Phase 2 portion of the RISE UP pivotal study of mitapivat in sickle cell disease.
Thalassemia: Completed enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in not regularly transfused and regularly transfused adults with thalassemia, respectively.
Lower-Risk Myelodysplastic Syndromes (LR-MDS): Completed enrollment of the Phase 2a study of AG-946 in LR-MDS.
Business Development: Announced an exclusive worldwide license agreement with Alnylam Pharmaceuticals for a novel siRNA for the potential treatment of polycythemia vera.
Leadership: Appointed Catherine Owens to the board of directors. Kaye Foster assumed the role of lead independent director.
Other: Data from Servier’s Phase 3 trial of vorasidenib in patients with residual or recurrent IDH mutant low-grade glioma were presented during the plenary session at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and published in the New England Journal of Medicine. As part of the divestiture of Agios’ oncology business to Servier, Agios retains rights to a potential $200 million milestone upon FDA approval of vorasidenib and 15% royalties on potential U.S. net sales.
Key Upcoming Milestones & Priorities

Agios expects to execute on the following additional key milestones and priorities by the end of 2023:

Pediatric PK Deficiency: Enroll more than half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of mitapivat.
Sickle Cell Disease: Enroll first patient in Phase 3 portion of RISE UP study of mitapivat, with the 100 mg dose selected from the successful Phase 2 portion.
Lower-risk Myelodysplastic Syndromes (LR-MDS): Announce data from the Phase 2a study of novel PK activator AG-946.
Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU).
Second Quarter 2023 Financial Results

Revenue: Net U.S. product revenue from sales of PYRUKYND for the second quarter of 2023 was $6.7 million, compared to $3.1 million for the second quarter of 2022. PYRUKYND received FDA approval on February 17, 2022.

Cost of Sales: Cost of sales for the second quarter of 2023 was $1.1 million.

Research and Development (R&D) Expenses: R&D expenses were $68.9 million for the second quarter of 2023 compared to $74.5 million for the second quarter of 2022. The year-over-year decrease was primarily driven by a decrease in workforce related expenses as a result of reduced headcount related to the evolution of our research organization.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $30.4 million for the second quarter of 2023 compared to $28.3 million for the second quarter of 2022. The year-over-year increase was primarily attributable to an increase in stock-based compensation expense.

Net Loss: Net loss was $83.8 million for the second quarter of 2023 compared to $91.8 million for the second quarter of 2022.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2023, were $946.9 million compared to $1.1 billion as of December 31, 2022. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue, interest income and vorasidenib milestone will enable the company to fund its operating expenses and capital expenditures at least into 2026. This does not include potential royalties from vorasidenib, commercializing mitapivat outside of the U.S. through one or more partnerships, or other potential strategic business or financial agreements.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2023 financial results and recent business activities. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On August 3, 2023 Imugene Limited (ASX:IMU), a clinical stage immunooncology company, reported that its CF33 Oncolytic Virus technology will be featured at the renowned Annual Meeting for the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), to be held in San Diego, USA on 1-5 November 2023 (Press release, Imugene, AUG 3, 2023, View Source [SID1234633684]).

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As one of the most prestigious immunotherapy events on the calendar, SITC (Free SITC Whitepaper) 2023 features cutting-edge research presentations by various experts, oral, and poster abstract presentations and various opportunities for networking and discussion with members of the oncology community.

The CF33 technology has been accepted for a Trial-in-Progress Poster titled: A Phase I Safety and Tolerability Study of VAXINIA (CF33-hNIS), a Novel Chimeric Oncolytic Poxvirus, Administered Intratumorally or Intravenously in Adults with Metastatic or Advanced Solid Tumors.
Abstract #: 730

Imugene will announce further details from this abstract closer to the time of the event. For more about SITC (Free SITC Whitepaper) 2023, please visit: View Source

On August 2, 2023 Eisai reported its Consolidated Financial Report for the Three-Month Period Ended June 30, 2023 (Presentation, Eisai, AUG 2, 2023, View Source [SID1234634859]).

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