On February 28, 2023 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (Kiniksa), a biopharmaceutical company with a pipeline of immune-modulating assets designed to target a spectrum of cardiovascular and autoimmune diseases, reported fourth quarter and full-year 2022 financial results and recent portfolio execution (Press release, Kiniksa Pharmaceuticals, FEB 28, 2023, View Source [SID1234627850]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Kiniksa executed across its cardiovascular and emerging autoimmune franchises in 2022," said Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa. "Within the cardiovascular franchise, we are focused on increasing brand awareness of ARCALYST, the only FDA-approved treatment for recurrent pericarditis, and pursuing collaborative study agreements for mavrilimumab in rare cardiovascular diseases. In our emerging autoimmune franchise, we believe KPL-404, our CD40 antagonist, has significant potential for differentiation in targeting chronic autoimmune diseases. We are advancing the Phase 2 trial of KPL-404 in rheumatoid arthritis into the efficacy portion and expect data in the first half of 2024. Additionally, our profitable ARCALYST collaboration, non-dilutive capital from strategic out-licensing transactions, and continued financial discipline support these efforts while providing cash runway into at least 2025."

Portfolio Execution

ARCALYST (IL-1α and IL-1β cytokine trap)

· ARCALYST net product revenue was $39.9 million and $122.5 million for the fourth quarter and full-year 2022, respectively.

· Since launch, greater than 800 prescribers have written ARCALYST prescriptions for recurrent pericarditis, with 22% having written prescriptions for 2 or more patients.

· As of the end of the fourth quarter of 2022, there was a greater than 90% payer approval rate of completed patient cases for recurrent pericarditis.

· As of the end of the fourth quarter of 2022, ARCALYST average total duration of therapy was approximately 18 months after accounting for the approximately 45% of recurrent pericarditis patients who had discontinued therapy and restarted treatment.

- ARCALYST average initial duration of therapy remained approximately 12 months as of the end of the fourth quarter of 2022.

· As of the end of the fourth quarter of 2022, approximately 5% of the target 14,000 multiple-recurrence pericarditis patients were actively on ARCALYST treatment.

KPL-404 (monoclonal antibody inhibitor of CD40-CD154 interaction)

· Kiniksa has completed enrollment of the second and final cohort of the multiple ascending dose portion of the Phase 2 clinical trial of KPL-404 in rheumatoid arthritis. Following completion of this portion of the trial, the proof-of-concept portion will begin. The company expects data from the trial in the first half of 2024.

Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

· Kiniksa is pursuing collaborative study agreements to evaluate the potential of mavrilimumab in rare cardiovascular diseases where the granulocyte macrophage colony stimulating factor (GM-CSF) mechanism has been implicated.

Financial Results

· Total revenue for the fourth quarter of 2022 was $61.9 million, compared to $18.7 million for the fourth quarter of 2021. Total revenue for the full-year 2022 was $220.2 million, compared to $38.5 million for the full-year 2021.

- Total revenue for the fourth quarter of 2022 included $39.9 million in ARCALYST net product revenue and $21.9 million in license and collaboration revenue from Roche and Genentech, a member of the Roche Group (Genentech). Kiniksa did not report license and collaboration revenue in the fourth quarter of 2021.

- Total revenue for the full-year 2022 included $122.5 million in ARCALYST net product revenue and $97.7 million in license and collaboration revenue from Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd., a wholly-owned subsidiary of Huadong Medicine Co., Ltd and Genentech. Kiniksa did not report license and collaboration revenue in 2021.

· Total operating expenses for the fourth quarter of 2022 were $55.8 million, compared to $54.9 million for the fourth quarter of 2021. Total operating expenses for the full-year 2022 were $210.4 million, compared to $195.2 million for the full-year 2021.

- Total operating expenses for the fourth quarter of 2022 included $6.4 million in non-cash, share-based compensation expense, compared to $6.1 million for the fourth quarter of 2021.

- Total operating expenses for the full-year 2022 included $25.1 million in non-cash, share-based compensation expense, compared to $25.2 million for the full-year 2021.

· Net income for the fourth quarter of 2022 was $4.5 million, compared to a net loss of $36.3 million for the fourth quarter of 2021. Net income for the full-year 2022 was $183.4 million, compared to a net loss of $157.9 million for the full-year 2021.

- Net income for the full-year 2022 included a $172.3 million tax benefit primarily due to the release of a valuation allowance on non-cash deferred tax assets.

· As of December 31, 2022, Kiniksa had $190.6 million of cash, cash equivalents, and short-term investments and no debt.

Financial Guidance

· Kiniksa expects ARCALYST net product revenue for the full-year 2023 of between $190 million and $205 million.

· Kiniksa expects that its cash and cash equivalents will fund its current operating plan into at least 2025.

Conference Call Information

· Kiniksa will host a conference call and webcast at 8:30 a.m. Eastern Time on Tuesday, February 28, 2023, to discuss fourth quarter and full-year 2022 financial results and to provide a corporate update.

· Individuals interested in participating in the call via telephone may register here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. To access the webcast, please visit the Investors and Media section of Kiniksa’s website. A replay of the event will also be available on Kiniksa’s website within approximately 48 hours after the event.

On February 28, 2023 Isofol Medical AB (publ), (Nasdaq Stockholm: ISOFOL), reported that the board of Isofol has decided to appoint Thomas Andersson as CEO of the company from February 28, 2023 (Press release, Isofol Medical, FEB 28, 2023, View Source [SID1234627849]). As a consequence of this decision, the current CEO, Ulf Jungnelius, is resigning and is now being dismissed from his position.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Thomas Andersson (b. 1952) has long experience from leading positions in the pharmaceutical industry, including as CEO of Spectracure, Senior Advisor at Karolinska Institutet Innovations AB, Investment Manager at Karolinska Development AB, CEO of Lund University Bioscience AB, Head of Cell Therapy at Bavarian Nordic AS and CEO of Annexin Pharmaceuticals AB. He has been part of the founding of more than 15 companies, held over 20 board assignments and is currently Senior Advisor Business Development at Oslo Cancer Cluster Incubator as well as board member of Kongsberg Beam Technology AS, Thepler AS and Skogsmöllan AB. Thomas Andersson has a PhD in physical chemistry from Lund University.

"We are very happy to be able to welcome Thomas Andersson as Isofol’s new CEO. With his extensive experience in company management, drug development and business development, he has excellent background to create value for patients and shareholders based on the company’s drug candidate arfolitixorin," says Mats Franzén, chairman of the board of Isofol Medical AB (publ).

Thomas Andersson succeeds Ulf Jungnelius, who has been CEO of the company since 2019.

For more information, please contact
Isofol Medical AB (publ)
Mats Franzén, Chairman of the Board
E­-mail: [email protected]
Phone: +46 (0) 704 47 29 09

This information is information that Isofol Medical AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, at 17.45 CET on February 28, 2023.

On February 28, 2023 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor infiltrating lymphocyte, TIL, and peripheral-blood lymphocyte, PBL), reported fourth quarter and full year 2022 financial results and corporate updates (Press release, Iovance Biotherapeutics, FEB 28, 2023, View Source [SID1234627847]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "During 2022, we made considerable progress initiating our first BLA for lifileucel in advanced melanoma, preparing for launch, and advancing our immuno-oncology pipeline. As we prepare for commercialization and expand our pipeline, our planned acquisition of Proleukin will provide an immediate source of revenue, streamline our supply chain and logistics, reduce our future cost of goods and lower expenses for IL-2 used with TIL therapies in commercial and clinical settings. Our top priorities in 2023 are to complete the BLA submission in the first quarter, receive FDA approval, then successfully launch lifileucel. We also continue to develop our pipeline in multiple solid tumor indications and look forward to presenting new data."

Recent and Full Year 2022 Highlights and Corporate Updates

Acquisition of Proleukin

· Under a definitive agreement between Iovance and Clinigen Limited, Iovance will acquire worldwide rights to Proleukin (aldesleukin), an interleukin-2 (IL-2) product with uses that include administration following TIL infusion to promote T-cell activity. Iovance expects the benefits of this transaction to include immediate and future revenue, securing the IL-2 supply chain and logistics surrounding TIL therapy administration, and lower cost of goods and clinical trial expenses for Proleukin used with TIL therapies. The closing of this transaction is expected to occur by the end of the first quarter of 2023 or early in the second quarter of 2023, when all of the closing conditions and required regulatory approvals are achieved.

Iovance TIL Therapy (Lifileucel) in Advanced Melanoma

· Regulatory highlights:
o A rolling BLA submission for lifileucel in post-anti-PD-1 advanced (metastatic or unresectable) melanoma commenced in August 2022 and remains on track to complete in the first quarter of 2023.
o Iovance received positive feedback from the U.S. Food and Drug Administration (FDA) on both its potency assay matrix and its proprietary cell co-culture assay included in the potency assay matrix in April 2022.
o At a successful pre-BLA meeting in late July 2022, the FDA provided favorable feedback on the clinical efficacy data from Cohorts 2 and 4 of the C-144-01 clinical trial, including duration of follow up, and the potency assay matrix.
o Startup activities have begun for the randomized Phase 3 TILVANCE-301 trial after Iovance reached agreement with the FDA in the fourth quarter of 2022 regarding the registrational trial design for accelerated and full approvals of lifileucel in combination with pembrolizumab in frontline advanced melanoma. TILVANCE-301, which is also a confirmatory trial to support full approval of lifileucel in post-anti-PD-1 advanced melanoma, is expected to be well underway at the time of potential accelerated approval for lifileucel in this initial indication.

· C-144-01 trial presentations and publications in post-anti-PD-1 advanced melanoma:
o Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 37th Annual Meeting: Iovance presented positive clinical data from Cohorts 2 and 4 of the C-144-01 clinical trial in advanced melanoma during SITC (Free SITC Whitepaper) in November 2022 and in a subsequent companion publication in the Journal for ImmunoTherapy of Cancer (JITC).

· IOV-COM-202 (Cohort 1A) trial results in frontline advanced melanoma:
o In April 2022, Iovance announced updated positive Cohort 1A results for lifileucel in combination with pembrolizumab demonstrating a robust 67% ORR and durability of response in 12 patients. Safety was consistent with other studies of Iovance TIL therapies in combination with pembrolizumab.
o A January 2023 corporate update highlighted that results from nearly 20 patients treated in Cohort 1A remained consistent with previously reported efficacy and safety data and continue to support the frontline melanoma opportunity for lifileucel. Study enrollment remains ongoing.

Manufacturing and Commercial Preparations

· To date, more than 600 patients have been treated with Iovance TIL therapy manufactured using proprietary Iovance processes.

· The Iovance Cell Therapy Center (iCTC) is currently manufacturing TIL therapies for clinical trials while executing activities to support BLA submission and review, including pre-approval inspection readiness, in preparation for initiating commercial supply.

· The iCTC facility as currently built has annual capacity to supply TIL therapies for 2,000+ patients, with available shell space that can be built to supply TIL therapies for 5,000+ patients from this facility. Contract manufacturers provide additional flexibility and capacity for Iovance to meet potential commercial and clinical demand.

· Iovance is executing several initiatives ahead of potential commercialization, including on-boarding and personnel training at authorized treatment centers (ATCs), education and awareness, and other commercial launch readiness activities.

Clinical Pipeline

· Iovance TIL (LN-145) monotherapy in second or third line metastatic non-small-cell lung cancer (mNSCLC):
o Enrollment is ongoing at more than 40 active clinical sites in the U.S., Canada and Europe for the IOV-LUN-202 trial of LN-145 in patients with mNSCLC who have progressed on or after frontline chemo- and anti-PD1-therapy. A Trial in Progress (TIP) poster was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.
o Iovance is engaged in discussions with the FDA about the potential for IOV-LUN-202 to serve as a registrational trial for LN-145 in second/third line mNSCLC and intends to execute an updated regulatory strategy based on this dialogue and feedback.

· Iovance TIL (LN-145) in combination with anti-PD-1 in earlier line mNSCLC:
o Iovance reported positive initial results from Cohort 3A of the IOV-COM-202 clinical trial that explores the combination of TIL therapy (LN-145) and pembrolizumab as therapy for ICI naïve mNSCLC patients. A confirmed ORR by RECIST 1.1 of 47% (n=8/17) was observed, with responses observed across PD-L1 negative and positive patients.
o Cohort 3A enrollment remains ongoing and presentation of detailed results is expected at a medical meeting in 2023.
o A meeting with the FDA is planned in 2023 to discuss Cohort 3A results and a potential registrational trial of lifileucel in frontline advanced NSCLC.

· Iovance PD-1 inactivated TIL therapy (IOV-4001) in previously treated advanced melanoma or mNSCLC: The first patient was treated with IOV-4001 in the third quarter of 2022 in the IOV-GM1-201 trial of Iovance’s first genetically modified TIL therapy, IOV-4001. This is among the first clinical trials of a genetically modified TIL cell therapy for solid tumors. Study enrollment remains ongoing.

· Lifileucel in advanced cervical cancer: In 2022, Iovance updated the registrational strategy in advanced cervical cancer based on FDA feedback to reflect the emerging treatment landscape. Cohort 2 in the ongoing C-145-04 trial was expanded to be pivotal and began enrolling additional patients to support a BLA in cervical cancer following progression on or after chemotherapy and pembrolizumab.

Research Programs for Next-Generation TIL Therapies and Related Technologies

· A preclinical poster at the AACR (Free AACR Whitepaper) 2022 Annual Meeting in April highlighted the anti-tumor activity of IOV-4001 in a murine model of melanoma.

· Additional programs using the gene editing TALEN technology are expected to enter clinical development in 2024, including genetically modified TIL therapy with multiple inactivated checkpoint targets.

· Additional research and preclinical studies are exploring approaches to increase TIL potency using CD39/69 double negative TILs and stable gene incorporation enhancements such as tethered cytokines.

· A novel interleukin-2 (IL-2) analog (IOV-3001) is in IND-enabling studies supporting its use as part of the TIL treatment regimen following TIL infusion.

Corporate

· As of February 24, 2023, Iovance’s unaudited cash position is approximately $669.8 million, which includes net proceeds from an at-the market (ATM) equity financing facility of approximately $450.0 million raised during the fourth quarter of 2022 and first quarter of 2023. This cash position is expected to fund the previously disclosed acquisition of Proleukin and Iovance’s operating plan into the second half of 2024.

· Iovance currently owns more than 60 granted or allowed U.S. and international patents for TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity into 2038. More information on Iovance’s patent portfolio can be found on the Intellectual Property page on www.iovance.com.

Fourth Quarter and Full Year 2022 Financial Results

Iovance had $478.3 million in cash, cash equivalents, investments and restricted cash at December 31, 2022, compared to $602.1 million at December 31, 2021. With the net proceeds from the ATM equity financing facility of approximately $450.0 million raised during the fourth quarter of 2022 and first quarter of 2023 to date, the cash position is expected to be sufficient to fund current and planned operations into the second half of 2024.

Jean-Marc Bellemin, Chief Financial Officer of Iovance, said, "Our cash position, including proceeds from our ATM facility, is expected to support our planned acquisition of Proleukin as well as commercial launch preparations, internal manufacturing and clinical pipeline expansion into several milestones to create value for patients and shareholders."

Net loss for the fourth quarter ended December 31, 2022, was $105.3 million, or $0.64 per share, compared to a net loss of $99.3 million, or $0.63 per share, for the fourth quarter ended December 31, 2021. Net loss for the full year period ended December 31, 2022, was $395.9 million, or $2.49 per share, compared to a net loss of $342.3 million, or $2.23 per share, for the same period ended December 31, 2021.

Research and development expenses were $80.6 million for the fourth quarter ended December 31, 2022, an increase of $5.0 million compared to $75.6 million for the same period ended December 31, 2021. Research and development expenses were $294.8 million for the full year period ended December 31, 2022, an increase of $35.8 million compared to $259.0 million for the same period ended December 31, 2021.

The increases in research and development expenses in the fourth quarter and year-to-date 2022 over the prior year periods were primarily attributable to growth of the internal research and development team, including stock-based compensation expense, as well as facility-related and internal research program costs, which were partially offset by lower clinical and manufacturing costs driven by completion of enrollment of pivotal clinical trials.

General and administrative expenses were $26.5 million for the fourth quarter ended December 31, 2022, an increase of $2.7 million compared to $23.8 million for the same period ended December 31, 2021. General and administrative expenses were $104.1 million for the full year period ended December 31, 2022, an increase of $20.4 million compared to $83.7 million for the same period ended December 31, 2021.

The increase in general and administrative expenses in the fourth quarter and year-to-date 2022 compared to the prior year periods were primarily attributable to growth of the internal general and administrative and commercial teams, including stock-based compensation expense, and facility-related costs associated with the build-out of the new corporate headquarters, as well as costs associated with pre-commercial activities.

For additional information, please see the Company’s Selected Condensed Consolidated Balance Sheet and Statement of Operations below.

Webcast and Conference Call

To participate in the conference call, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com. The archived webcast will also be available for one year.

On February 28, 2023 Invitae (NYSE: NVTA), a leading medical genetics company, reported financial and operating results for the fourth quarter and year ended December 31, 2022 (Press release, Invitae, FEB 28, 2023, View Source [SID1234627846]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people.

"2022 was a transformative year for Invitae as we shifted our focus to drive more profitable growth, and we are pleased that the major initiatives under our strategic realignment are now largely completed," said Ken Knight, president and chief executive officer of Invitae. "Our talented teams made great progress, and continued their work in service of patients and customers while delivering on the financial objectives aligned to our new path. These continued improvements in our financial metrics reflect our new priorities and operating discipline. In light of that execution, we separately announced today significant developments in addressing approximately 96% of our near-term debt obligations. Our investors have agreed to extend the maturities of our 2024 convertible notes into 2028, along with equitizing a portion of the debt and adding capital, demonstrating their confidence in Invitae’s future. We have also repaid our term loan in full and further improved our balance sheet. Overall, we are looking forward to 2023, as we continue to drive operational excellence, along with setting the stage for our long-term growth opportunities for years to come."

Full Year and Fourth Quarter 2022 Financial Results

Generated revenue of $516.3 million in 2022, a 12.1% increase from $460.4 million in 2021. Fourth quarter 2022 revenue of $122.5 million, versus $126.1 million a year ago, reflecting the impact of exited businesses and geographies announced earlier in the year.
GAAP gross profit was $99.1 million in 2022, including $29.6 million in the fourth quarter. Non-GAAP gross profit was $219.7 million in 2022, including $58.5 million in the fourth quarter.
GAAP gross margin was 19.2% in 2022, as compared with 24.3% in 2021, and 24.2% in the fourth quarter of 2022, versus 23.8% in the fourth quarter of 2021. Non-GAAP gross margin was 42.5% in 2022, as compared with 36.6% in 2021. Non-GAAP gross margin was 47.8% in the fourth quarter of 2022, compared to 36.5% in the fourth quarter of 2021.
Cash, cash equivalents, restricted cash and marketable securities were $557.1 million as of December 31, 2022, compared to $596.0 million as of September 30, 2022 and $1.06 billion as of December 31, 2021.
Cash burn in 2022 was $509.6 million, compared to a cash burn of $849.2 million in 2021. 2022 cash burn included outflows of $53.3 million ($38.4 million related to realignment and $14.9 million acquisition-related payments), as well as an inflow of $44.5 million related to the selected assets sale of the RUO kitted solutions.
Cash burn in the fourth quarter was $41.8 million and included an outflow of $9.3 million related to realignment and $0.1 million of acquisition-related payments, as well as an inflow of $44.5 million related to the selected assets sale of the RUO kitted solutions. Excluding these items, cash burn would have been $77.0 million. This represents a continued improving trend since the fourth quarter of 2021 driven by our strategic realignment and cost reduction plans.
Total active healthcare provider accounts totaled 20,929 as of December 31, 2022, an increase of approximately 13% year-over-year.
Active pharmaceutical and commercial partnerships grew to 238, an increase of approximately 34% year-over-year, reflecting growing adoption of our product and services to pharmaceutical, health systems and other partners.
Revenue per patient was $475 in 2022, a decrease of approximately 3% from $491 in 2021. Revenue per patient was $511 in the fourth quarter of 2022, compared to $505 in the third quarter of 2022, and $476 in the fourth quarter of 2021, primarily as a result of our realignment efforts.
Total patient population as of December 31, 2022 is more than 3.6 million with over 62% available for data sharing.
Total GAAP operating expense, which excludes cost of revenue, for the fourth quarter of 2022 was $124.5 million, which includes items related to the strategic realignment. As a result, GAAP operating expense as a percentage of revenue was 102%, compared to 194% in the fourth quarter of 2021. Non-GAAP operating expense was $135.6 million for the fourth quarter of 2022. Non-GAAP operating expense as a percentage of revenue was 111%, compared to 171% in the fourth quarter of 2021.

Net loss for the fourth quarter of 2022 was $99.8 million, or a $0.41 net loss per share, compared to net loss of $205.1 million, or net loss per share of $0.90, for the fourth quarter of 2021. Non-GAAP net loss for the fourth quarter of 2022 was $82.0 million, or a $0.34 non-GAAP net loss per share, compared to a net loss of $184.7 million, or an $0.81 non-GAAP net loss per share, for the fourth quarter of 2021.

Financial Guidance

On a pro forma basis, the company exited fourth quarter 2022 at an annualized revenue of approximately $450 million for its remaining business. Management is expecting 2023 revenue to be over $500 million, representing low double-digit year-over-year growth compared to 2022 pro forma revenue.

Thanks to realignment initiatives, non-GAAP gross margin for 2023 is expected to be between 48-50%, compared to 42.5% in 2022. Cash burn is expected to be in the range of $250-275 million in 2023, a more than 45% reduction from 2022.

Webcast and Conference Call Details

Management will host a conference call and webcast today at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss financial results and recent developments. To access the conference call, please register at the link below:

View Source;confId=46549

Upon registering, each participant will be provided with call details and access codes.

The live webcast of the call and slide deck may be accessed here or by visiting the investors section of the company’s website at ir.invitae.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the company’s website.

On February 28, 2023 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported fourth quarter and full year 2022 financial results and recent portfolio and business updates (Press release, Alector, FEB 28, 2023, View Source [SID1234627845]). As of December 31, 2022, Alector’s cash, cash equivalents and investments totaled $712.9 million.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2022 was marked by significant momentum and focus on our programs and prioritization across our pipeline as we continued to cement our position as an innovator in advancing immuno-neurology," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "We shared positive data from our INFRONT-2 Phase 2 trial of latozinemab in patients with symptomatic FTD-C9orf72 and advanced our INVOKE-2 Phase 2 Alzheimer’s disease study with AL002, the most advanced TREM2 candidate in clinical development. We are encouraged by recent progress in the Alzheimer’s disease space and believe our first-in-class drug candidates that harness the power of microglia, the brain’s immune cells, have the potential to improve neurodegeneration outcomes either as stand-alone therapies or potentially in combination with anti-beta amyloid drugs."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector added, "Last year was transformational for Alector as we continued to strengthen our world-class and seasoned leadership team and bolster key talent across the organization. Looking ahead, we believe we are well-positioned both financially and organizationally to execute on our strategic priorities in 2023 and beyond."

Pipeline Prioritization and Cash Runway Extension Through 2025

Alector plans to prioritize its late-stage immuno-neurology programs, including its progranulin and TREM2 product candidates, to extend cash runway through 2025.

Recent Clinical Updates and 2023 Milestones

Immuno-Neurology Portfolio
Progranulin Programs (Latozinemab, AL101) Being Developed in Collaboration with GSK

Enrollment is ongoing globally in the INFRONT-3 randomized, placebo-controlled, pivotal Phase 3 clinical trial evaluating the efficacy and safety of latozinemab (AL001) in at-risk and symptomatic patients with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). In June 2022, the first patient from the trial was enrolled and dosed in the optional open-label extension (OLE) study, and enrollment in the OLE is ongoing. Alector is preparing to engage with regulatory authorities in mid-2023. The purpose of the meeting is to discuss statistical analysis plans based on emerging knowledge in the field that may enable Alector to complete the INFRONT-3 study with fewer patients and/or a shorter treatment duration. The company is targeting an INFRONT-3 data readout in early 2025 pending regulatory feedback.
In November 2022 at the 15th Clinical Trials on Alzheimer’s Disease (CTAD) conference, Alector presented positive data from the multiple-dose cohorts of the first-in-human Phase 1 study of AL101 in healthy volunteers.
Results demonstrated that AL101 was well tolerated and increased progranulin levels in plasma and CSF in a dose-dependent manner.
The pharmacokinetic (PK) and pharmacodynamic (PD) profile of AL101 supports future development in larger indications such as Alzheimer’s disease (AD) and Parkinson’s disease (PD).
Alector and GSK plan to advance the AL101 program in 2023, with the initiation of an Asian PK bridging study to be followed by a global Phase 2 clinical trial in early AD.
In March 2022 at the AD/PD 2022 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders, Alector presented positive 12-month data from the INFRONT-2 Phase 2 clinical trial of latozinemab in FTD patients with a C9orf72 genetic mutation (FTD-C9orf72). These data build upon the previously disclosed study results in FTD-GRN patients and support the company’s efforts to expand the progranulin franchise into additional neurodegenerative disease indications.
Clinical outcome assessments using the CDR plus NACC FTLD-SB scale found that treatment of FTD-C9orf72 patients with latozinemab resulted in a trend toward a delay of approximately 54% in annualized disease progression compared to a matched control cohort from the ALLFTD consortium.
Mean levels of glial fibrillary acidic protein (GFAP), a biomarker of astrogliosis that is an indicator of disease and/or injury to the central nervous system, decreased over 12 months in both plasma and cerebrospinal fluid (CSF) in latozinemab-treated FTD-C9orf72 patients.
Latozinemab was generally well tolerated when administered monthly for a year or more, consistent with other study cohorts.
The company expects to report additional data from the INFRONT-2 trial of latozinemab in FTD-C9orf72 patients during the second half of 2023.
TREM2 Program (AL002) Being Developed in Collaboration with AbbVie

In January 2023, the first patient was enrolled and dosed in a long-term extension (LTE) of the INVOKE-2 Phase 2 clinical trial for which Alector will receive a $17.8 million milestone payment from AbbVie. Alector plans to complete INVOKE-2 trial enrollment in the third quarter of 2023, with top-line data expected by the fourth quarter of 2024. The INVOKE-2 trial is designed to evaluate the efficacy and safety of AL002 in slowing disease progression in individuals with early AD.
AbbVie has an exclusive option to globally develop and commercialize AL002. AbbVie’s exercise of that option would prompt a $250 million payment to Alector.
AL002 targets Triggering Receptor Expressed on Myeloid cells 2 (TREM2) to increase TREM2 signaling and the functionality of microglia.
MS4A Program (AL044)

In September 2022, Alector commenced its first-in-human Phase 1 trial of AL044, with the intention of developing that candidate in Alzheimer’s disease and potential orphan indications. Based on initial PK and tolerability data, the company decided to close the trial. Alector is actively pursuing a back-up MS4A program to develop an IND-ready candidate with a potentially improved dosing and tolerability profile.
Recent Corporate Updates

In 2022, Alector expanded its management team with the appointments of Marc Grasso, M.D., as Chief Financial Officer in February, Gary Romano, M.D., Ph.D., as Chief Medical Officer in May and Peter Heutink, Ph.D., as Chief Scientific Officer in October.
Last year the U.S. Patent and Trademark Office issued patents covering the latozinemab composition of matter and the use of AL101 for treating AD, PD and other neurodegenerative diseases including FTD.
Fourth Quarter and Full Year 2022 Financial Results

Revenue. Collaboration revenue for the quarter ended December 31, 2022, was $14.4 million, compared to $14.0 million for the same period in 2021. Collaboration revenue for the year ended December 31, 2022, was $133.6 million, compared to $207.1 million for the same period in 2021. The decrease in year-over-year collaborative revenue was primarily due to $173.4 million collaboration revenue recognized from AL001 FTD-GRN license provided as part of the GSK Agreement in 2021, offset by revenue recognized in 2022 for GSK and AbbVie programs, including revenue recognized from the termination of the AL003 program.

R&D Expenses. Total research and development expenses for the quarter ended December 31, 2022, were $54.5 million, compared to $52.8 million for the same period in 2021. Total research and development expenses for the year ended December 31, 2022, were $210.4 million compared to $189.4 million for the same period in 2021. The increase in R&D expenses was mainly driven by increased spending to support advancement of several clinical and preclinical programs, including latozinemab and AL002, as well as increased personnel-related expenses.

G&A Expenses. Total general and administrative expenses for the quarter ended December 31, 2022, were $15.4 million, compared to $16.9 million for the same period in 2021. Total general and administrative expenses for the year ended December 31, 2022, were $61.0 million compared to $55.0 million for the same period in 2021. The increase in year-over-year G&A expenses is primarily due to personnel-related expenses and information technology expenses to support the company’s growth.

Net Loss. For the quarter ended December 31, 2022, Alector reported net loss of $52.4 million, or $0.63 net loss per share, compared to a net loss of $55.6 million, or $0.68 per share, for the same period in 2021. For the year ended December 31, 2022, Alector reported net loss of $133.3 million or $1.62 net loss per share, compared to a net loss of $36.3 million, or $0.45 net loss per share, for the same period in 2021.

Cash Position. Cash, cash equivalents and investments were $712.9 million as of December 31, 2022. Management anticipates that this will be sufficient to fund Alector’s operations through 2025.

2023 Guidance. Management anticipates, for the year ending 2023, collaboration revenue to be between $15 million and $25 million, total research and development expenses to be between $225 million and $245 million and total general and administrative expenses to be between $60 million and $70 million.