On August 4, 2022 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in viral and liver diseases, reported recent business progress and financial results for the second quarter June 30, 2022 (Press release, Aligos Therapeutics, AUG 4, 2022, View Source [SID1234617544]).

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"In the second quarter, we continued to make strong progress in advancing our drug candidates and discovery programs," said Lawrence Blatt, PhD, MBA, CEO and Chairman of the Board at Aligos. "We completed enrollment in multiple cohorts across our Phase 1 CAM-2 and NASH studies and presented posters for these and other drug candidates and discovery programs at the recent International Liver Congress meeting hosted by the European Association for the Study of the Liver (EASL). We are similarly off to a strong start in Q3, where we have initiated enrollment in a 12-week cohort evaluating the safety and chronic suppressive activity of ALG-000184 in chronic hepatitis B (CHB) patients. We also remain on track to submit a clinical trial application (CTA) this quarter to evaluate our siRNA drug candidate, ALG-125755, in healthy volunteers and CHB patients. We believe our siRNA, which is designed to lower HBsAg levels, is an important addition to our clinical portfolio and has the potential to be a cornerstone of our functional cure strategy."

Recent Business Progress

Aligos Portfolio of Drug Candidates

HBV Programs

ALG-000184 (CAM-2): Enrollment of HBeAg positive subjects in 28-day and 12-week cohorts evaluating various dose levels is ongoing. We plan to share data from HBeAg positive cohorts at a scientific conference in Q4 2022.
ALG-125755 (siRNA): Phase 1 enabling nonclinical studies are nearly complete. We are on track to file a CTA in Q3 2022 to enable the evaluation of the safety, tolerability, pharmacokinetics, and antiviral activity of ALG-125755. Dosing is planned to begin in healthy volunteers in Q4 2022 and in CHB subjects
in Q1 2023.
NASH

ALG-055009: Enrollment of hyperlipidemic subjects in all 4 cohorts of the multiple ascending dose portion of our Phase 1 study is now complete. We are currently analyzing these data and remain on track to share top line data from the single and multiple ascending dose portions of this study in Q3 2022.

COVID-19 3CL Protease Inhibitor (PI) Program

ALG-097558: Evaluation of ALG-097558 in the hamster SARS-CoV-2 infection model indicates that when dosed prior to infection or up to 24 hours post-infection, the compound causes a significant reduction in the levels of infectious virus in the lungs.
Phase 1 enabling nonclinical studies are on track to be initiated in H2 2022. A subsequent CTA filing for a Phase 1 study evaluating the safety and pharmacokinetics of ALG-097558 in healthy volunteers is planned for Q1 2023.
COVID-19 3CL Protease Inhibitor Resistance Profiling

The potential for the emergence of viral resistance to 3CL protease inhibitors used to treat COVID-19 is of considerable concern. Together with our collaborators at KU Leuven, including its Centre for Drug Design and Discovery (CD3), a drug discovery unit and investment fund of KU Leuven, and the Rega Institute for Medical Research, we have begun to investigate the viral resistance profile of our 3CL protease inhibitors. A resistant mutant was derived by incubating SARS-CoV-2 infected cells with an early lead compound, ALG-097161. We identified a combination of 3 amino acid substitutions in the 3CL protease (L50F E166A L167F) that are associated with a greater than 50-fold increase in the EC50 values for ALG-097161 and PF-07321332 (nirmatrelvir) when assessed in a biochemical assay. In contrast, when ALG-097558 was profiled against the L50F E166A L167F resistant mutant, only a 3-fold shift in the EC50 value was observed. Experiments to further evaluate the viral resistance profile of ALG-097558 are currently ongoing.

Financial Results for the Second Quarter 2022

Cash, cash equivalents and investments totaled $159.3 million as of June 30, 2022, compared with $205.8 million as of December 31, 2021. We continue to believe our cash balance provides sufficient cash to fund planned operations into the first half of 2024.

Net losses for the three months ended June 30, 2022, were $19.9 million or basic and diluted net loss per common share of ($0.47), compared to net losses of $29.8 million or basic and diluted net loss per common share of $(0.79) for the three months ended June 30, 2021.

Research and development (R&D) expenses for the three months ended June 30, 2022, were $16.5 million compared with $24.6 million for the same period of 2021. The decrease in R&D expenses for this comparative period is primarily attributable to our continued wind-down related to the discontinuation of our STOPS and ASO programs offset by our expenditures related to the ongoing development and manufacturing activities associated with our CAM and NASH clinical program activities. Total R&D stock-based compensation expense incurred for the three months ended June 30, 2022, was $2.2 million compared with $2.0 million for the same period of 2021.

General and administrative (G&A) expenses for the three months ended June 30, 2022, were $7.6 million compared with $6.6 million for the same period of 2021. The increase in G&A expenses for this comparative period is primarily attributable to routine employee and facility related costs. Total G&A stock-based compensation expense incurred for the three months ended June 30, 2022, was $1.8 million compared with $1.5 million for the same period of 2021.

On August 4, 2022 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported financial results for the second quarter ended June 30, 2022 (Press release, NanoString Technologies, AUG 4, 2022, View Source [SID1234617543]).

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Second Quarter Financial Highlights

Product and service revenue of $32.0 million
GeoMx Digital Spatial Profiler (DSP) revenue of $10.9 million. GeoMx DSP revenue includes:
Instrument revenue of $5.2 million
Consumables revenue of $5.7 million, annualized pull-through of approximately $77,000 per installed system
nCounter revenue, inclusive of all service revenue, of $21.1 million. nCounter revenue includes:
Instrument revenue of $4.3 million
Consumables revenue of $12.2 million, annualized pull-through of approximately $45,000 per installed system
Service revenue of $4.6 million
Cash, cash equivalents and short-term investments balance of $272.3 million
"NanoString had an excellent second quarter, as our commercial execution continued to improve, and laboratories adopting our solutions drove a 65% year-on-year increase in orders for new spatial biology instruments. Orders for our CosMx Spatial Molecular Imager are ahead of our expectations, and are helping us build a large revenue backlog that we expect will set us up for significant growth in 2023," said Brad Gray, president & CEO of NanoString. "With complementary instrument platforms, our new AtoMx Spatial Informatics Platform, and blue-chip partnerships, we believe we are ideally positioned to win the spatial biology land-grab currently underway."

Operational Highlights

Spatial Biology

CosMx Orders: Secured customer orders for more than 25 CosMx Spatial Molecular Imager (SMI) systems, bringing total orders to date to more than 60 systems
Commercial Status: First CosMx beta instrument was shipped to a customer during the first week of August, with two additional beta instruments scheduled for shipments to customers later in August
Advances in Genome Biology and Technology (AGBT) Conference: Unveiled the AtoMx Spatial Informatics Platform and showcased data from 20 spatial biology studies, including 15 for CosMx SMI
Introduced Integrated Informatics Platform for GeoMx Using NGS: Began offering a streamlined end-to-end solution to fully integrate GeoMx with Illumina NextSeq sequencers
GeoMx Installed Base: Grew installed base to approximately 315 GeoMx DSP Systems at June 30, 2022, representing 66% growth over the prior year
Publications: Continued growth of peer-reviewed publications utilizing GeoMx DSP technology, with approximately 20 new publications in the second quarter, bringing the cumulative total to approximately 130 peer-reviewed publications as of June 30, 2022
nCounter

nCounter Installed Base: Grew installed base to approximately 1,085 nCounter Analysis Systems at June 30, 2022, representing 7% growth over the prior year
Publications: Surpassed 5,800 cumulative peer-reviewed publications utilizing nCounter technology at June 30, 2022
2022 Outlook

The company updated its 2022 outlook, with results expected as follows:

Orders for about 200 spatial biology systems, consistent with previous guidance, with an expected mix now comprised of approximately 50% GeoMx system orders and approximately 50% CosMx system orders
Cumulative orders for approximately 120 CosMx systems expected by the end of 2022, providing a revenue backlog valued at approximately $30 million
Total product and service revenue of $140 to $150 million, as compared to previous guidance of $150 to $160 million, reflecting an order mix that is weighted more heavily to CosMx, with material CosMx revenue recognition expected to begin in 2023
nCounter revenue, inclusive of all service revenue, of $90 to $95 million, consistent with previous guidance
Adjusted EBITDA loss of $75 to $85 million, as compared to previous guidance of $65 to $75 million
Financial Results

We have elected to present selected non-GAAP, or adjusted, financial measures, including Adjusted EBITDA. These adjusted financial measures are calculated excluding certain items that may make it more challenging to compare our GAAP operating results across periods. Such items may include collaboration revenue, stock-based compensation, depreciation and amortization, or one-time charges such as transaction related fees and expenses or restructuring charges and severance costs. A reconciliation of adjusted financial measures to the nearest comparable GAAP financial measure can be found in the tables at the end of this press release.

Supplemental Information

As a supplement to the table above, we have posted to the investor relations section of our website, at www.nanostring.com/Investor Relations/Financials/Quarterly Results, supplemental financial data that include our adjusted financial measures as compared to the nearest comparable GAAP financial measures, for the second quarter and the six months ended June 30, 2022 and for each quarter of and the full year of 2021.

Conference Call

Management will host a conference call today beginning at 1:30 pm PT / 4:30 pm ET to discuss these results and answer questions. Investors and other interested parties can register for the call in advance by visiting View Source After registering, an email confirmation will be sent including dial-in details and unique conference call codes for entry. Registration is open throughout the call, but to ensure connection for the full call, registration in advance is recommended. The link to the webcast and audio replay will be made available at the Investor Relations website: www.nanostring.com. A replay of the call will be available beginning August 4, 2022 at 7:30pm ET through midnight ET on August 11, 2022. To access the replay, dial (866) 813-9403 or (929) 458-6194 and reference Conference ID: 426771. The webcast will also be available on our website for one year following the completion of the call.

Non-GAAP, or Adjusted, Financial Information

We believe that the presentation of non-GAAP, or adjusted, financial information provides important supplemental information to management and investors regarding financial and business trends relating to our financial condition and results of operations. Reconciliation of adjusted financial measures to the most directly comparable financial result as determined in accordance with GAAP are included at the end of this press release following the accompanying financial data. A reconciliation of adjusted guidance measures to corresponding GAAP measures is not available on a forward-looking basis without unreasonable effort due to the uncertainty regarding certain expenses that may be incurred in the future. For further information regarding why we believe that these adjusted measures provide useful information to investors, the specific manner in which management uses these measures and some of the limitations associated with the use of these measures, please refer to "Notes Regarding Non-GAAP Financial Information" at the end of this press release.

On August 4, 2022 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the second quarter ended June 30, 2022, and shared recent operational progress (Press release, bluebird bio, AUG 4, 2022, View Source [SID1234617542]).

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"The second quarter marked significant progress for bluebird bio and a precedent-setting moment for the field of gene therapy," said Andrew Obenshain, chief executive officer, bluebird bio. "With the FDA advisory committee’s unanimous support for beti-cel and eli-cel for their target indications, we are now laser-focused on commercial readiness, and if approved, we anticipate launching both therapies in the fourth quarter of this year. Additionally, this quarter we advanced the remaining CMC steps ahead of our lovo-cel BLA submission, and we remain on track to submit the BLA in the first quarter of next year."

RECENT HIGHLIGHTS

BETI-CEL

UNANIMOUS POSITIVE VOTE AT FDA ADVISORY COMMITTEE MEETING – On June 10, the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted (13-0) that the benefits of betibeglogene autotemcel (beti-cel) gene therapy outweigh the risks for people with beta-thalassemia who require regular red blood cell transfusions. If approved, beti-cel will be the first ex-vivo LVV gene therapy available in the U.S.
ICER REVIEW – The Institute for Clinical and Economic Review (ICER) completed its review of beti-cel for people with beta-thalassemia and determined in its final report that beti-cel will be cost effective at a price up to $3.0 million. bluebird anticipates setting a price for beti-cel upon potential FDA approval.
ELI-CEL

UNANIMOUS POSITIVE VOTE AT FDA ADVISORY COMMITTEE MEETING – On June 9, the FDA CTGTAC voted (15-0) that the benefits of elivaldogene autotemcel (eli-cel) gene therapy outweigh the risks for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD). If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death.
LOVO-CEL

CONTINUED PROGRESS TOWARD BLA SUBMISSION – bluebird bio remains on track to submit a biologics licensing application (BLA) to the FDA for lovotibeglogene autotemcel (lovo-cel) for sickle cell disease in the first quarter of 2023. As previously communicated, the Company has treated all patients in HGB-206 Group C who will form the primary basis of efficacy for BLA submission. This quarter, the Company completed manufacturing of commercial drug product validation lots, marking significant progress on CMC requirements and final steps to BLA submission. Additionally, bluebird completed enrollment of all patients in the HGB-210 study necessary to support manufacturing data requirements for the BLA submission. The remaining step prior to BLA submission is completion of vector and drug product analytical comparability, which the Company expects to complete in the fourth quarter of 2022.

The Company remains in active dialogue with the FDA about the resolution of the partial clinical hold for patients under 18. The Company is continuing to enroll and treat patients 18 and older in the HGB-210 study.
COMPANY

TOM KLIMA APPOINTED CHIEF COMMERCIAL & OPERATING OFFICER – Effective August 8, Tom Klima will serve as Chief Commercial & Operating Officer. Klima joined bluebird in May 2021 as Chief Commercial Officer to hone the Company’s commercial strategy and oversee launch execution plans for its gene therapy portfolio. His new role reflects expanded responsibilities for program management and patient supply chain in addition to sales, marketing and market access.
UPCOMING INVESTOR EVENT

Members of the management team will participate in the 2022 Wedbush PacGrow Virtual Healthcare Conference, Wednesday, August 10, at 9:10 a.m. ET as part of the panel titled Miss Con-GENE-iality – Updates in Gene Tx.

UPCOMING ANTICIPATED MILESTONES

BETI-CEL

The FDA has set a PDUFA goal date for August 19, 2022, and if approved, the Company anticipates first apheresis in the fourth quarter of 2022.
beti-cel is being reviewed under Priority Review for the treatment of beta-thalassemia in patients requiring regular red blood cell transfusions. bluebird bio anticipates receiving a Priority Review Voucher (PRV) upon potential approval of beti-cel.
ELI-CEL

The FDA has set a PDUFA goal date of September 16, 2022, and if approved, the Company anticipates therapy availability in the fourth quarter of 2022.
eli-cel is being reviewed under Priority Review for the treatment of cerebral adrenoleukodytrophy in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor. bluebird bio anticipates receiving a PRV upon potential approval of eli-cel.
bluebird bio is in active communication with the FDA to resolve the eli-cel clinical hold and anticipates the FDA’s questions may be resolved concurrent with the agency’s ongoing review of the Company’s BLA submission.
LOVO-CEL

The Company is in active communication with the FDA to resolve the lovo-cel partial clinical hold and resume enrollment and treatment of patients under the age of 18.
The Company expects to complete vector and drug product analytical comparability in the fourth quarter of 2022.
The Company plans to submit its BLA for lovo-cel in Q1 2023.
SECOND QUARTER 2022 FINANCIAL RESULTS

Cash Position: The Company’s restricted cash, cash and cash equivalents and marketable securities balance was approximately $218 million, including restricted cash of approximately $45 million, as of June 30, 2022. The full-year 2022 cash burn is expected to be less than $340 million.

As of today, the Company has raised approximately $24.7 million in gross proceeds through its At-the-Market (ATM) equity facility. Of this $24.7 million, $8.0 million in net proceeds were realized in the second quarter and are reflected in the restricted cash, cash and cash equivalents and marketable securities balanced as of June 30, 2022. The Company is exploring additional financing opportunities, including public or private equity financings and monetizing any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.
Revenues: Total revenue was $1.5 million for the three months ended June 30, 2022, compared to $0.1 million for the three months ended June 30, 2021.
R&D Expenses: Research and development expenses from continuing operations were $63.8 million for the three months ended June 30, 2022, compared to $84.6 million for the three months ended June 30, 2021. The decrease of $20.8 million was primarily due to decreased employee compensation, benefits, other head-count related expenses, information technology and facility-related costs, lab expenses and clinical trial costs. These decreased costs were partially offset by increased manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses from continuing operations were $36.7 million for the three months ended June 30, 2022, compared to $55.0 million for the three months ended June 30, 2021. The decrease of $18.3 million was primarily due to decreased employee compensation, benefit, and other head-count related expenses and decreased commercial readiness activities due to the Company’s decision to focus its efforts on the U.S. market for beti-cel, eli-cel, and lovo-cel. These decreased costs were partially offset by increased information technology and facility-related costs due to the addition of the Company’s office lease in Somerville, Massachusetts.
Net Loss: Net loss from continuing operations was $100.1 million for the three months ended June 30, 2022, compared to $155.8 million for the three months ended June 30, 2021.

On August 4, 2022 Lyell Immunopharma, Inc. (Lyell) (Nasdaq: LYEL), a clinical‑stage T-cell reprogramming company dedicated to developing curative cell therapies for patients with solid tumors, reported financial results for the second quarter of 2022 (Press release, Lyell Immunopharma, AUG 4, 2022, View Source [SID1234617541]).

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"As a clinical stage company, we continue to make progress advancing our pipeline and growing our capabilities," said Liz Homans, CEO of Lyell Immunopharma. "Our strong financial position enables us to see our current pipeline through important milestones in evaluating T-cell exhaustion and lack of durable stemness as key barriers to successful cell therapy in patients with solid tumor cancers while also advancing next generation technologies such as T-cell rejuvenation. We have recently presented new preclinical data that further elucidate the potential of our reprogramming technologies to endow T cells with the ability to resist exhaustion and maintain properties of durable stemness, and we remain on track to announce initial clinical data in 2023 from our lead program, LYL797."

Recent Business Highlights

Presented preclinical data for LYL797, Lyell’s CAR T-cell therapy targeting ROR1+ solid tumors that incorporates Gen-R and Epi-R, at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) demonstrating that Gen-R and Epi‑R reprogramming technologies can enhance and prolong anti-tumor functions of ROR1-targeting CAR T-cell therapy in solid tumor model systems.
Presented preclinical data for LYL132, a TCR therapy targeting NY-ESO-1+ solid tumors that incorporates Epi-R and is being developed in collaboration with GSK, at ASGCT (Free ASGCT Whitepaper) demonstrating that Epi-R reprogramming technology creates populations of stemlike NY-ESO-1-targeting TCR T cells that lead to products with increased proliferative capacity and prolonged functional activity in the presence of persistent antigen exposure.
Presented preclinical data at the International Society for Stem Cell Research 2022 Annual Meeting (ISSCR) demonstrating the application of Lyell’s rejuvenation technology yielded improvements in antitumor properties of engineered adoptive T-cell products as compared to non-rejuvenated T-cell controls.
Second Quarter 2022 Financial Results

Revenue

Revenue was $35.7 million and $2.6 million for the three months ended June 30, 2022 and 2021, respectively, primarily related to the recognized portion of the upfront license fee of Lyell’s Collaboration and License Agreement entered into in 2019 and amended in June 2020 and December 2021 (GSK Agreement) with GlaxoSmithKline Intellectual Property (No. 5) Limited and Glaxo Group Limited (together, GSK). The increase of $33.1 million was primarily related to recognizing $35.3 million in revenue due to a mutual agreement with GSK to conclude research activities on an undisclosed target for hematological cancers.
GAAP and Non-GAAP Operating Expenses

Lyell reported a net loss of $36.3 million for the second quarter ended June 30, 2022, compared to a net loss of $62.6 million for the same period in 2021. Non-GAAP net loss, which excludes non-cash stock-based compensation and non-cash expenses related to the change in the estimated fair value of success payment liabilities, was $10.3 million for the second quarter ended June 30, 2022 compared to $38.1 million for the same period in 2021.
Research and development (R&D) expenses were $43.7 million for the second quarter ended June 30, 2022, compared to $46.4 million for the same period in 2021. The decrease in R&D expense was primarily driven by a reduction in the success payment liability balance, which offset increases in facilities and technology costs to support the expansion of our R&D and manufacturing capabilities. Non‑GAAP R&D expenses, which exclude non-cash stock-based compensation and non-cash expenses related to the change in the estimated fair value of success payment liabilities for the second quarter ended June 30, 2022, were $35.9 million, compared to $32.1 million for the same period in 2021.
General and administrative (G&A) expenses were $30.5 million for the second quarter ended June 30, 2022, compared to $19.1 million for the same period in 2021. The increase in G&A expense was primarily due to an $8.1 million increase in stock-based compensation expense, primarily related to award modifications and new awards granted. Non‑GAAP G&A expenses, which exclude non-cash stock-based compensation, for the second quarter ended June 30, 2022 were $12.2 million, compared to $9.0 million for the same period in 2021. The increase in non-GAAP G&A expenses was driven by litigation-related expenses and public company operating costs.
A discussion of these non-GAAP financial measures, including reconciliations of the most comparable GAAP measures to non-GAAP financial measures, is presented below under "Non-GAAP Financial Measures."

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of June 30, 2022 were $787.0 million, compared to $898.3 million as of December 31, 2021. Lyell believes that its cash, cash equivalents and marketable securities balances will be sufficient to meet working capital and capital expenditure needs into 2025.

On August 4, 2022 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported second quarter 2022 financial results and recent portfolio and business updates (Press release, Alector, AUG 4, 2022, View Source [SID1234617540]). As of June 30, 2022, Alector’s cash, cash equivalents and investments totaled $808.9 million.

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Commentary on the Quarter:

"We continue to make significant progress in building our pipeline of novel immuno-neurology and immuno-oncology drug candidates which engage the innate immune system to treat neurodegeneration and cancer," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector.

"With the recent submission of an Investigational New Drug Application (IND) for our next first-in-class clinical drug candidate, AL044, Alector continues to build its position as an innovator in developing new approaches to tackle Alzheimer’s disease (AD) and other serious neurodegenerative disorders. The company is planning its first AL044 study in healthy volunteers. AL044 modulates MS4A, which is a genetic risk locus for AD and an immune checkpoint expressed on microglia cells. MS4A activity is associated with multiple AD pathologies, including: Abeta and Tau accumulation, earlier age of onset, and increased rate of cognitive decline. We believe that AL044 has significant potential to be effective in the treatment of AD."

"Patient enrollment is progressing in INVOKE-2, a Phase 2 study of AL002, which is our first-in-class anti-TREM2 antibody for early AD. Following a voluntary exclusion of APOE e4/e4 homozygous patients, we have not observed additional serious adverse events related to amyloid-related imaging abnormalities (ARIA). We are implementing earlier patient monitoring to the trial that is consistent with recently published guidelines for ARIA monitoring and management as we continue to prioritize patient safety. TREM2 is an important risk gene for AD and is believed to control the survival, function, proliferation, and migration of microglia, the brain’s immune cells."

"In addition to our robust AD portfolio, we are opening additional sites and expanding recruitment efforts to enroll our Phase 3 INFRONT-3 pivotal study of latozinemab for frontotemporal dementia (FTD) with progranulin mutations (FTD-GRN). Later this year, we also expect to share new data from the Phase 1 study of AL101, the second product candidate from the company’s progranulin portfolio."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector, added, "We are pleased to announce that the company has completed IND enabling studies for our immuno-oncology program, AL009, and anticipates submitting an IND in 2022. AL009 is a first-in-class multi Siglec inhibitor that works to enhance both the innate and adaptive immune system response to tumors by blocking a critical glycan checkpoint pathway that drives immune inhibition. As a leader in targeting myeloid biology, this brings us closer to achieving our goal of building a portfolio of innate immuno-oncology therapies by utilizing the company’s expertise in the central and peripheral innate immune system."

"Alongside these encouraging clinical updates, Alector continues to attract world class talent and we are delighted to announce that Peter Heutink, Ph.D., will be joining us as Chief Scientific Officer. Peter brings considerable experience in human genetics of neurodegenerative diseases, functional genomics, and induced pluripotent stem cell (iPSC) technology that will help drive science and discovery at Alector."

Clinical Programs

Immuno-Neurology Portfolio
Progranulin Assets (Latozinemab, AL101)

Alector continues to progress the INFRONT-3 randomized, placebo-controlled, pivotal Phase 3 trial evaluating the efficacy and safety of latozinemab in at-risk and symptomatic patients with FTD-GRN. The company continues to build awareness of FTD, support genetic testing and educate the FTD patient community about the INFRONT-3 pivotal study to support clinical trial enrollment.

The company expects to report data at a medical conference later this year from the Phase 1 trial of AL101 in healthy volunteers. AL101 is being developed to elevate progranulin levels in a manner similar to latozinemab (AL001) with plans to investigate AL101 for the treatment of AD and Parkinson’s disease.
TREM2 and SIGLEC 3 Assets (AL002, AL003)

Enrollment in the INVOKE-2 Phase 2 study of AL002 continues with no additional serious adverse events related to ARIA following the exclusion of APOE e4/e4 homozygous patients. The company is implementing MRI monitoring prior to dose escalation for all clinical trial participants, consistent with recently published guidelines for ARIA monitoring and management. The INVOKE-2 Phase 2 clinical trial is designed to evaluate the efficacy and safety of AL002 in slowing disease progression in individuals with early AD. AL002 is being developed in collaboration with AbbVie and targets Triggering Receptor Expressed on Myeloid cells 2 (TREM2) to increase TREM2 signaling and the functionality of microglia brain specific immune cells.

As previously announced, AbbVie decided to terminate the CD33 collaboration program after Alector and AbbVie collaboratively reviewed next steps for AL003, which targets CD33. Both companies concluded the program did not warrant further development based on insufficient evidence of an effect on pharmacodynamic biomarkers from a Phase 1 study. This decision allows Alector to focus its resources on other more promising programs.
Novel MS4A Asset (AL044)

Alector has submitted a U.S. IND for AL044, which targets MS4A, a major genetic risk locus for AD and an immune checkpoint expressed on microglia immune system cells. Preclinical data suggest that AL044 may mimic or surpass the effects of a genetic variant of MS4A that is protective against AD.
Immuno-Oncology Portfolio
SIRPα and Multi-Siglec Assets (AL008, AL009)

The company intends to re-acquire the rights that were granted to Innovent Biologics for the development and commercialization of AL008. Innovent submitted an IND to the Chinese regulatory authorities, and Alector plans to utilize data and documentation from this regulatory filing to support a potential IND submission in the U.S. AL008 is a novel innate immuno-oncology candidate with a dual mechanism of action combining the inhibition of the CD47-SIRP-alpha (SIRPα) pathway with the activation of Fc receptors to promote immuno-stimulatory pathways that drive anti-tumor immunity.

The company has completed IND enabling studies for its first-in-class AL009 immuno-oncology program and anticipates submitting an IND later this year. AL009 is a dual function biologic that inhibits multiple Siglec receptors on myeloid cells and simultaneously activates a stimulating receptor on the same cells. The company plans to prioritize tumor types that have immunosuppressive phenotypes and expects to study AL009 as a monotherapy and in combination with standard of care.
Recent Corporate Updates

Dr. Peter Heutink, Ph.D., will join Alector as its soon-to-be new Chief Scientific Officer (CSO) in the fall of 2022. Dr. Heutink is a full professor, a group leader and the site speaker of the German Center for Neurodegenerative Diseases (DZNE) – Tübingen. In addition, he is a full professor of Genome Biology for Neurodegenerative Diseases at the Eberhard Karls University Tübingen, Germany and an honorary professor of Functional Genomics of Ageing and Neurodegeneration at the University Medical Center Groningen (UMCG), the Netherlands. Dr. Heutink has led teams that identified mutation in genes for Parkinson’s disease, FTD, chorea, ataxia, hemochromatosis, porencephaly and various development disorders. He has also made important contributions to genome wide association studies for major depression, Tourette Syndrome, obsessive compulsive disorders, FTD, and Parkinson’s Disease. He has made contributions to a series of genome wide association, exome and whole genome sequencing studies as a member of the International Parkinson’s Disease Genomics Consortium (IPDGC), the International Frontotemporal Dementia Genomics Consortium (IFGC) and the International FTLD-TDP WGS consortium and is a member of the steering committee of the Global Parkinson’s Genetics Program. Dr. Heutink has been involved in many industry collaborations to bring genetic findings towards the clinic and is an author/co-author of over 400 peer-reviewed publications.

John Maraganore has joined Alector as a strategic advisor. Mr. Maraganore served as the founding CEO and Director at Alnylam Pharmaceuticals where he led the company’s efforts to form significant partnerships with leading pharmaceutical and biotech companies and built an integrated biotech company with a market capitalization exceeding $25 billion.

The U.S. Patent and Trademark Office has issued additional patents covering the latozinemab composition of matter and the use of AL101 for treating AD, Parkinson’s disease and other neurodegenerative diseases including FTD.
Second Quarter 2022 Financial Results

Revenue. Collaboration revenue for the quarter ended June 30, 2022, was $79.9 million, compared to $6.6 million for the same period in 2021. The increase of $73.3 million was due to a $52.0 million net increase to collaboration revenue under the AbbVie Agreement due to changes in estimated costs to satisfy the performance obligations resulting from the termination of the AL003 program, partially offset by increases in total expected costs for the AL002 program. The increase was also due to a $21.3 million increase in revenue recognized from the GSK Agreement.

R&D Expenses. Total research and development expenses for the quarter ended June 30, 2022, were $54.5 million, compared to $47.8 million for the quarter ended June 30, 2021. The increase in R&D expenses was mainly driven by increased personnel-related expenses, including stock-based compensation, as well as an increase in AL101 expenses.

G&A Expenses. Total general and administrative expenses for the quarter ended June 30, 2022, were $15.8 million, compared to $14.1 million for the same period in 2021. The increase was primarily due to an increase in personnel-related expenses, including stock-based compensation, offset by reduced legal expenses mainly to support the GSK collaboration deal in 2021.

Net Income (Loss). For the quarter ended June 30, 2022, Alector reported net income of $9.9 million, or $0.12 net income per share, compared to a net loss of $55.1 million, or $0.69 net loss per share, for the same period in 2021.

Cash Position. Cash, cash equivalents, and investments were $808.9 million as of June 30, 2022. Management anticipates that this will be sufficient to fund current operations into the second half of 2024.