On February 23, 2023 Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, reported financial results for the fourth quarter and full year ended December 31, 2022 and provided recent business highlights (Press release, Shattuck Labs, FEB 23, 2023, View Source [SID1234627630]).

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"During the fourth quarter of 2022, we continued to focus on clinical execution to position ourselves for key clinical data readouts for SL-172154 in 2023. Enrollment in our AML/HR-MDS clinical trial of SL-172154 is moving along nicely, and, in the fourth quarter, we advanced into the first combination cohort with azacitidine. This keeps us on track to share initial data from the dose-escalation portion of this trial from relapsed/refractory AML/HR-MDS patients both as monotherapy and in combination with azacitidine in the first half of 2023. As it relates to our trials in platinum-resistant ovarian cancer, in early 2022 we established a collaboration with ImmunoGen to combine SL-172154 with mirvetuximab soravtansine and, in the fourth quarter, we initiated enrollment in this Phase 1B clinical trial. We expect initial data from the combination trial with SL-172154 and liposomal doxorubicin midyear 2023, and we expect initial data from the combination trial of SL-172154 and mirvetuximab soravtansine in the second half of 2023," said Taylor Schreiber, M.D., Ph.D., and Chief Executive Officer of Shattuck. "I am also very pleased to announce the promotion of Dr. George Fromm to CSO of the ARC platform and Dr. Suresh de Silva to CSO of the GADLEN platform. We look forward to benefiting from the expertise of these scientific program leaders as we continue our momentum in 2023. Drs. Fromm and de Silva have been invaluable to Shattuck from the time they each joined the company in 2017, and they will continue to play a critical role as we build-out our pipeline over the coming years. 2023 is an important year for Shattuck, with multiple data readouts, which we believe will establish SL-172154 as both a differentiated CD47 inhibitor and a CD40 agonist capable of activating the CD40 pathway in human cancer patients. We expect that these data will position Shattuck well for continued growth in the years ahead."
"After enrolling patients in the final dose levels of 12 and 24 mg/kg with the SL-279252 clinical program, and not meeting a stringent efficacy threshold required to justify further development, we have decided to discontinue development of SL-279252. The development program for SL-279252 has yielded tremendously important preclinical, translational, and clinical data for the ARC platform, and we thank the patients and their families for participating in our clinical trial. We remain focused on delivering novel therapeutics that benefit patients with cancer with high unmet need and look forward to the combination data in both of our ongoing clinical trials with SL-172154 in the coming quarters," continued Dr. Schreiber.
Clinical Milestones Expected in 2023
ARC Platform
SL-172154 (SIRPα-Fc-CD40L)
•Complete data from Phase 1A dose-escalation clinical trial of SL-172154 as monotherapy in platinum-resistant ovarian cancer expected midyear 2023

•Initial data from Phase 1B clinical trial of SL-172154 in combination with liposomal doxorubicin in platinum-resistant ovarian cancer expected midyear 2023
•Initial dose-escalation data, as monotherapy and in combination with azacitidine, for Phase 1A/B clinical trial of SL-172154 in relapsed/refractory AML and HR-MDS expected in the first half of 2023
•Complete dose-escalation data, as monotherapy and in combination with azacitidine, for Phase 1A/B clinical trial of SL-172154 in AML and HR-MDS and initial dose-expansion cohort data with SL-172154 in combination with azacitidine in frontline TP53 mutant AML and HR-MDS expected in the second half of 2023
•Initial data from Phase 1B clinical trial of SL-172154 in combination with mirvetuximab soravtansine in platinum-resistant ovarian cancer expected in the second half of 2023
GADLEN Platform
GADLEN Preclinical Compounds
•Additional detail and further program guidance regarding the advancement of potential product candidates from the GADLEN platform expected in 2023
Fourth Quarter 2022 Recent Business Highlights and Other Recent Developments
ARC Clinical-Stage Pipeline
SL-172154 (SIRPα-Fc-CD40L)
•Completed Phase 1A Monotherapy Dose-Escalation Clinical Trial of SL-172154 in Platinum-Resistant Ovarian Cancer: This open-label, multi-center, dose-escalation clinical trial evaluated the safety, tolerability, pharmacokinetics, anti-tumor activity, and pharmacodynamic effects of SL-172154 administered intravenously in patients with advanced platinum-resistant ovarian cancer. We reached a maximum administered dose of 10.0 mg/kg and expect to present complete dose-escalation data from the trial midyear 2023.
•Dosed First Patients in First Combination Cohort with Azacitidine in Ongoing Phase 1A/B Clinical Trial in AML and HR-MDS: This trial is evaluating the safety, tolerability, pharmacokinetics, anti-tumor activity, and pharmacodynamic effects of SL-172154 as both monotherapy and in combination. In AML, SL-172154 may be evaluated in combination with azacitidine and venetoclax. In both HR-MDS and TP53 mutant AML, SL-172154 may be evaluated in combination with azacitidine. Patients have been dosed in both the monotherapy and combination dose-escalation cohorts of this trial and enrollment is ongoing. Dose escalation will continue in a parallel staggered manner, and initial dose-escalation data, as monotherapy and in combination with azacitidine, are expected in the first half of 2023.
•Enrollment Progressing in Phase 1B Clinical Trial of SL-172154 in Combination with Liposomal Doxorubicin in Advanced Platinum-Resistant Ovarian Cancer: Enrollment is continuing in this trial, which is evaluating the safety, tolerability, pharmacokinetics, anti-tumor activity, and pharmacodynamic effects of SL-172154, using the selected dose of 3.0 mg/kg, in combination with liposomal doxorubicin in patients with advanced platinum-resistant ovarian cancer. We expect to present initial data from this trial in combination with liposomal doxorubicin midyear 2023.
•Initiated Enrollment in Phase 1B Clinical Trial of SL-172154 in Combination with Mirvetuximab Soravtansine in Advanced Platinum-Resistant Ovarian Cancer. This trial is evaluating the safety, pharmacokinetics, pharmacodynamic effects, and preliminary anti-tumor activity of SL-172154 administered in combination with mirvetuximab soravtansine in subjects with platinum-resistant ovarian, primary peritoneal, or fallopian tube cancers. Mirvetuximab soravtansine is an antibody-drug conjugate targeting folate receptor alpha, or FRα, which provides for both direct tumor cell killing as well as enhanced macrophage phagocytosis through binding with Fc gamma receptors and has received accelerated approval for platinum-resistant ovarian cancer patients whose tumors are shown to be FRα positive, defined as ≥75%, as determined by the VENTANA FOLR1 (FOLR1-2.1) Assay, using the PS2+ scoring method. Pre-clinical studies have shown that both of these killing mechanisms are complementary to the mechanism

of SL-172154 by enhancing the activity of macrophages to phagocytose FRα- expressing ovarian cancer cells, and that SL-172154 may broaden the activity of mirvetuximab soravtansine, particularly in patients with tumors that express lower levels of FRα. We intend to enroll patients with broader FRα expression, including those with "high" (greater than ≥75% of tumor cells staining with 2+ intensity), "medium" (≥50% to <75% of tumor cells staining with 2+ intensity) , and "low" (≥25% to <50% of tumor cells staining with 2+ intensity) expression of FRα, as determined by the VENTANA FOLR1 (FOLR1-2.1) Assay, using the PS2+ scoring method. We expect to present initial data from this trial in combination with mirvetuximab soravtansine in the second half of 2023.
SL-279252 (PD1-Fc-OX40L)
•Completed Phase 1 Dose Escalation Clinical Trial of SL-279252 in Advanced Solid Tumors and Announces Discontinuation of Clinical Development of the SL-279252 Program: In the fourth quarter of 2021, we reported initial data from this trial demonstrating evidence of initial anti-tumor activity and dose-dependent pharmacodynamic activity. As previously stated, our internal benchmark for continued program development of SL-279252 was response rates equal or exceeding 20% in the 12 and 24 mg/kg cohorts. We did not observe an overall response rate necessary to justify continued development in a very difficult PD-1 relapsed/refractory patient population, and we are announcing the discontinuation of clinical development of SL-279252.
Gamma Delta T Cell Engager (GADLEN) Preclinical Pipeline
Preclinical Pipeline Development
•Announced two potential product candidates, currently in preclinical development, from the GADLEN platform—one targeting the CD20 antigen intended for development in autoimmune disease, and a second targeting the B7-H3 antigen for development in oncology: As Shattuck advances its preclinical pipeline, we anticipate further program guidance regarding the advancement of potential product candidates from the GADLEN platform in 2023.
Corporate Update
•Appointed CSOs for ARC and GADLEN Platforms: In January 2023, Dr. George Fromm was promoted to CSO of the ARC platform, and Dr. Suresh de Silva was promoted to CSO of the GADLEN platform. Dr. Fromm joined Shattuck Labs in 2017 and is one of its scientific co-founders. He previously served as Shattuck’s Vice President of R&D and co-led the preclinical development of SL-279252 and SL-172154 into clinical trials. Earlier, Dr. Fromm served as the Senior Director of Research and Development at Heat Biologics, Inc., where he directed the discovery and clinical-based research efforts for their phase I/II trials and co-invented a "next-generation" vaccine platform that combines a cell-based immunotherapy vaccine and a T cell costimulatory fusion protein in a single treatment. Dr. de Silva previously served as Shattuck’s Vice President of Product Development from 2018-2022 and as Shattuck’s Executive Director of Research and Development from 2017 to 2018. Dr. de Silva joined Shattuck in 2017 and is one of its scientific co-founders and co-led the preclinical development of SL-279252 and SL-172154. Prior to joining Shattuck, Dr. de Silva served as the Director of Research and Development at Heat Biologics, Inc. in Durham, NC, where he led external research collaborations and co-developed the ComPact cell-based vaccine platform.
Upcoming Events
•American Association for Cancer Research Annual Meeting (AACR) (Free AACR Whitepaper), April 14-19, 2023
▪Poster presentation on the preclinical development of GADLEN compounds
•Shattuck plans to attend the following investor conferences. Details of the presentations and webcasts will be announced prior to the events.
▪Cowen 43rd Annual Healthcare Conference, March 6-8, 2023
▪Oppenheimer 33rd Annual Healthcare Conference, March 13-15, 2023
▪Needham 22nd Annual Healthcare Conference, April 17-20, 2023

Fourth-Quarter and Full-Year 2022 Financial Results
•Cash Position: As of December 31, 2022, cash and cash equivalents and investments were $161.3 million, as compared to $268.8 million as of December 31, 2021.
•Research and Development (R&D) Expenses: R&D expenses for the quarter ended December 31, 2022 were $21.9 million, as compared to $16.2 million for the quarter ended December 31, 2021. R&D expenses for the year ended December 31, 2022 were $82.9 million, as compared to $56.6 million for the year ended December 31, 2021. This increase was primarily driven by increases in manufacturing of trial materials to support clinical development of our ongoing clinical trials, personnel-related costs, and lab supplies.
•General and Administrative (G&A) Expenses: G&A expenses for the quarter ended December 31, 2022 were $4.8 million, as compared to $4.6 million for the quarter ended December 31, 2021. General and administrative expenses for the year ended December 31, 2022 were $21.1 million, as compared to $18.7 million for the year ended December 31, 2021. This increase was primarily driven by a litigation settlement of $1.4 million and increases in personnel-related and other operating costs.
•Net Loss: Net loss was $25.4 million for the quarter ended December 31, 2022, or $0.60 per basic and diluted share, as compared to a net income of $7.8 million for the quarter ended December 31, 2021, or $0.19 per basic share and $0.18 per diluted share. Net loss for the year ended December 31, 2022 was $101.9 million, or $2.41 per basic and diluted share, as compared to $45.0 million, or $1.07 per basic and diluted share, for the year ended December 31, 2021.
2023 Financial Guidance
Shattuck believes its cash and cash equivalents and investments will be sufficient to fund its operations into the second half of 2024, beyond results from its Phase 1 clinical trials of SL-172154. This cash runway guidance is based on the Company’s current operational plans and excludes any additional capital that may be received, proceeds from business development transactions, and/or additional costs associated with clinical development activities that may be undertaken.
About SL-172154
SL-172154 (SIRPα-Fc-CD40L) is an investigational ARC fusion protein designed to simultaneously inhibit the CD47/SIRPα checkpoint interaction and activate the CD40 costimulatory receptor to bolster an anti-tumor immune response in patients with advanced cancer. Multiple Phase 1 clinical trials are ongoing for patients with advanced platinum-resistant ovarian cancer (NCT05483933) and patients with AML and HR-MDS (NCT05275439).

On February 23, 2023 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases and gene therapies, reported that it plans to host a conference call on Thursday, March 2, 2023, at 8:30 a.m. ET to discuss its financial results for the quarter and full-year ended December 31, 2022 and provide a business update (Press release, Selecta Biosciences, FEB 23, 2023, https://selectabio.gcs-web.com/news-releases/news-release-details/selecta-biosciences-host-conference-call-and-webcast-discuss-11 [SID1234627628]).

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Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 8768458. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On February 23, 2023 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported fourth quarter and full year 2022 financial results and corporate highlights (Press release, Relay Therapeutics, FEB 23, 2023, View Source [SID1234627627]).

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"We successfully executed against our strategy in 2022, advancing multiple clinical and pre-clinical programs and continuing to demonstrate the power of our Dynamo platform," said Sanjiv Patel, M.D., president and chief executive officer of Relay Therapeutics. "We reported additional RLY-4008 interim data showing an overall response rate of 63% across all doses and 88% at the pivotal dose, building on the previously reported early data and supporting our belief that limiting off-target effects and toxicity can allow us to improve efficacy. We also expanded our breast cancer portfolio, continuing clinical development of RLY-2608 and announcing three new programs. With a robust pipeline and cash in hand to fund us into 2025, we are excited to continue to execute on our plans and deliver against our milestones this year, as we work toward our goal of bringing life-changing therapies to patients."

2022 Corporate Highlights

RLY-4008 (FGFR2 inhibitor)

•
Presented additional interim data from patients with FGFRi-naïve FGFR2-fusion cholangiocarcinoma (CCA) at European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2022. Key highlights included:
o
All doses – 63% interim overall response rate (ORR): across all dose levels and schedules, 24 of 38 efficacy-evaluable patients experienced a partial response (22 confirmed, 2 unconfirmed)
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Pivotal dose (70 mg once daily) – 88% ORR: 15 of 17 efficacy-evaluable patients experienced a partial response (14 confirmed, 1 unconfirmed in an ongoing patient)
•
13 out of these 15 responders remain on treatment; 1 responder came off study to be resected with curative intent

Most treatment emergent adverse events were expected FGFR2 on-target, low-grade, monitorable, manageable and largely reversible
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There were no observed Grade 4 or 5 adverse events, and off-target toxicities of hyperphosphatemia and diarrhea continued to be clinically insignificant
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Based on discussions with the U.S. Food and Drug Administration, moved forward with a single-arm pivotal trial design for patients with pan-FGFR (FGFRi) treatment-naïve FGFR2-fusion CCA at 70 mg once daily to potentially support accelerated approval
o
Anticipate completing enrollment in pivotal cohort in the second half of 2023

Breast Cancer Portfolio

•
RLY-2608 (pan-mutant and isoform-selective PI3Kα inhibitor)
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Monotherapy: Following December 2021 initiation of the first-in-human trial, continued to enroll dose escalation portion of trial assessing RLY-2608 as a single agent in patients with unresectable or metastatic solid tumors with PI3Kα mutation
o
Combination: In April 2022, initiated dose escalation portion of a fulvestrant combination arm in patients with HR+, HER2–, PI3Kα-mutated, locally advanced or metastatic breast cancer
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Anticipate disclosing initial clinical data from both dose escalation portions in the first half of 2023
•
In June 2022, disclosed three new programs:
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Selective CDK2 inhibitor, anticipated clinical start in early 2024
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ERα degrader, development candidate nomination in 2023
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RLY-5836 (chemically distinct pan mutant-PI3Kα inhibitor), expected to enter the clinic in the second quarter of 2023

Corporate Highlights

•
Appointed Sekar Kathiresan, M.D., CEO of Verve Therapeutics, to Board of Directors
•
Raised $300.0 million of gross proceeds in an underwritten follow-on public offering

2023 Anticipated Milestones

•
RLY-4008
o
Full dose escalation data in the first half of 2023
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Complete enrollment of pivotal cohort in the second half of 2023
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Data from non-CCA expansion cohorts in the second half of 2023
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Breast Cancer
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RLY-2608: initial clinical data from dose escalation portions of monotherapy and combination arms of trial in the first half of 2023
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RLY-5836: clinical start in the second quarter of 2023
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ERα degrader: development candidate nomination in 2023
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Selective CDK2 inhibitor: clinical start in early 2024

Fourth Quarter and Full Year 2022 Financial Results

Cash, Cash Equivalents and Investments: As of December 31, 2022, cash, cash equivalents and investments totaled approximately $1 billion compared to $958.1 million as of December 31, 2021.

Relay Therapeutics expects its current cash, cash equivalents and investments will be sufficient to fund its current operating plan into 2025.

R&D Expenses: Research and development expenses were $67.3 million for the fourth quarter of 2022, as compared to $51.9 million for the fourth quarter of 2021. The increase was primarily due to $6.8 million of additional clinical trial expenses and $6.3 million of additional employee related costs, which includes $3.1 million of additional stock-based compensation expense. Research and development expenses were $246.4 million for the full year 2022, as compared to $172.7 million for the full year 2021. The increase was primarily due to $32.7 million of additional clinical trial expenses, $24.7 million of additional employee related costs, which includes $5.7 million of additional stock-based compensation expense, and $10.8 million of additional preclinical programs and platform technologies.

G&A Expenses: General and administrative expenses were $16.4 million for the fourth quarter of 2022, as compared to $15.5 million for the fourth quarter of 2021. The increase was primarily due to additional employee related costs, which includes $0.5 million of additional stock-based compensation expense. General and administrative expenses were $66.0 million for the full year 2022, as compared to $57.4 million for the full year 2021. The increase was primarily due to additional employee related costs, which includes $1.9 million of additional stock-based compensation expense.

Net Loss: Net loss was $67.5 million for the fourth quarter of 2022, or a net loss per share of $0.56, as compared to a net loss of $67.5 million for the fourth quarter of 2021, or a net loss per share of $0.64. Net loss was $290.5 million for the full year 2022, or a net loss per share of $2.59, as compared to a net loss of $363.9 million for the full year 2021, or a net loss per share of $3.82. Net loss for the full year 2021 included one-time expenses of $134.9 million associated with the acquisition of ZebiAI Therapeutics, Inc.

On February 23, 2023 Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, reported financial results for the fourth quarter and full year 2022 (Press release, Prothena, FEB 23, 2023, View Source [SID1234627626]). In addition, the Company provided 2023 financial guidance and business highlights.

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"In 2022 we meaningfully advanced our protein dysregulation portfolio as we continue our transition to a fully integrated commercial company. The foundation we established in 2022 has positioned the next 24 months as a transformational period for Prothena, with many significant clinical milestones expected across six different programs in areas of high unmet patient need," said Gene Kinney, Ph.D., President and Chief Executive Officer Prothena. "For PRX012, we received clearance from the FDA for our IND application and Fast Track designation was granted. We also initiated the Phase 1 SAD and MAD study for PRX012 and completed the Phase 1 SAD and initiated the MAD study for PRX005. Additionally, we presented key data on birtamimab and PRX123 at ASH (Free ASH Whitepaper) and AD/PD, respectively. As we grew our organization’s capabilities to prepare for the future, we also added significant strategic depth to our board of directors through the appointment of an established biotech leader, Helen S. Kim, MBA."

2022 Business Highlights and Upcoming Milestones

Neurodegenerative Diseases Portfolio

Alzheimer’s Disease (AD)

PRX012, a potential best-in-class, next-generation subcutaneous antibody for the treatment of AD, that targets a key epitope at the N-terminus of amyloid beta (Aβ) with high binding potency
•Received clearance from the U.S. Food and Drug Administration (FDA) in March 2022 for the investigational new drug (IND) application
•FDA granted Fast Track designation in April 2022
•Initiated Phase 1 single ascending dose (SAD) and multi ascending dose (MAD) studies in healthy volunteers and patients with AD
•Significant presence at upcoming International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) in March/April 2023 highlighted by oral presentation of preclinical data showing superior binding characteristics of PRX012 and a symposium featuring key thought leaders
•Ongoing Phase 1 SAD and MAD studies; topline data expected year end 2023

PRX005, a potential best-in-class antibody, for the treatment of AD that specifically targets a key epitope within the microtubule binding region (MTBR) of tau, a protein implicated in diseases including AD, frontotemporal dementia (FTD), progressive supranuclear palsy (PSP), chronic traumatic encephalopathy (CTE), and other tauopathies. PRX005 is part of a global neuroscience research and development collaboration with Bristol Myers Squibb
•Topline data from Phase 1 SAD study announced January 2023 showing single doses of PRX005 across three dose cohorts were generally safe and well tolerated, meeting the primary objective of the study; results expected from the Phase 1 SAD study at an upcoming medical conference
•Ongoing Phase 1 MAD study; topline data expected year end 2023

PRX123, a potential first-in-class dual Aβ/tau vaccine, for the treatment and prevention of AD, that is a dual-target vaccine targeting key epitopes within the N-terminus of Aβ and MTBR-tau to promote amyloid clearance and blockade of pathogenic tau
•Oral presentation on preclinical data at AD/PD 2022 demonstrated that Prothena’s dual Aβ/tau vaccine generated anti-Aβ and anti-MTBR-tau antibodies to enable clearance of Aβ and to inhibit cell-to-cell transmission of pathogenic tau species
•Investigational new drug (IND) application filing expected by year end 2023

Parkinson’s Disease (PD)

Prasinezumab, a potential first-in-class antibody, for the treatment of PD, that is designed to target key epitopes within the C-terminus of alpha-synuclein and is the focus of a worldwide collaboration with Roche
•Oral presentation by partner Roche at AD/PD 2022 of the Phase 2 PASADENA study further supports a potential effect on delaying motor progression in patients with early PD
•Ongoing Phase 2b PADOVA trial in patients with early PD is being conducted by Roche (NCT04777331); topline data expected in 2024

Rare Peripheral Amyloid Diseases Portfolio

AL Amyloidosis

Birtamimab, a potential best-in-class amyloid depleter antibody, for the treatment of AL amyloidosis, that is designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure
•Poster presented at the XVIII International Symposium on Amyloidosis (ISA) in September 2022 titled: Birtamimab in Patients with Mayo Stage IV AL Amyloidosis: Rationale for Confirmatory AFFIRM-AL Phase 3 trial
•Oral presentation at the 2022 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December 2022: Consistent Survival Benefit Observed with Birtamimab in Mayo Stage IV AL Amyloidosis Patients in Phase 3 VITAL Study
•Published data on in-hospital mortality in amyloid light chain amyloidosis in Journal of Comparative Effectiveness Research
•Confirmatory Phase 3 AFFIRM-AL trial ongoing (NCT04973137); topline data expected in 2024

ATTR Amyloidosis

NNC6019 (formerly PRX004), a potential first-in-class antibody, for the treatment of ATTR cardiomyopathy, that is designed to deplete the pathogenic, non-native forms of the transthyretin (TTR) protein and is being developed by Novo Nordisk as part of their up to $1.2 billion acquisition of Prothena’s ATTR amyloidosis business and pipeline
•Received $40 million milestone payment from Novo Nordisk in December 2022 related to the continued advancement of NNC6019 in a Phase 2 study
•Ongoing Phase 2 study in patients with ATTR cardiomyopathy is being conducted by Novo Nordisk (NCT05442047); topline data expected in 2024

2022 Organizational and Corporate Highlights

•Announced the appointment of Helen S. Kim, MBA, to its Board of Directors. With the appointment of Ms. Kim, Prothena expanded its Board to 10 directors
•Raised net proceeds of $172.4 million through an underwritten public follow-on offering of 3,250,000 ordinary shares in December 2022

Upcoming Investor Conferences

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

•Oppenheimer 33rd Annual Healthcare Conference on Monday, March 13, 2023; a fireside chat will be held at 10:00 AM ET
•Jefferies Biotech on the Bay Summit on Thursday, March 16, 2023; 1 on 1 investor meetings will be held

Fourth Quarter and Full Year of 2022 Financial Results
For the fourth quarter and full year of 2022, Prothena reported a net income of $6.3 million and a net loss of $116.9 million, respectively, as compared to a net loss of $33.2 million and a net income of $67.0 million for the fourth quarter and full year of 2021, respectively. Net income per share on a diluted basis was $0.12 for the fourth quarter of 2022 and net loss per share of for the full year of 2022 was $2.47, as

compared to net loss per share of $0.71 and net income per share on a diluted basis of $1.38 for the fourth quarter and full year of 2021, respectively.
Prothena reported total revenue of $49.9 million and $53.9 million for the fourth quarter and full year of 2022, respectively, as compared to total revenue of $1.2 million and $200.6 million for the fourth quarter and full year of 2021, respectively. Total revenue for the fourth quarter and full year of 2022 included a $40.0 million milestone payment from Novo Nordisk and revenue for fourth quarter and full year of 2022 included BMS collaboration revenue of $9.9 million and $13.9 million, respectively. This compares to total revenue of $1.2 million for the fourth quarter of 2021 from BMS collaboration revenue, and total revenue of $200.6 million for the full year of 2021, which included $79.7 million in collaboration revenue from BMS, a $60.0 million clinical milestone payment from Roche and $60.7 million from the sale of intellectual property and related rights to Novo Nordisk.
Research and development (R&D) expenses totaled $36.9 million and $135.6 million for the fourth quarter and full year of 2022, respectively, as compared to $22.1 million and $82.3 million for the fourth quarter and full year of 2021, respectively. The increase in R&D expense for the fourth quarter and full year of 2022 compared to the same periods in the prior year was primarily due to higher manufacturing costs, higher personnel related expenses, higher clinical trial expenses, higher consulting and other R&D expenses. For the full year of 2022, the higher costs were offset in part by lower collaboration expenses with Roche and lower manufacturing expenses related to the NNC6019 (formerly PRX004) program. R&D expenses included non-cash share-based compensation expense of $3.5 million and $14.8 million for the fourth quarter and full year of 2022, respectively, as compared to $2.9 million and $9.5 million for the fourth quarter and full year of 2021, respectively.
General and administrative (G&A) expenses totaled $13.1 million and $49.9 million for the fourth quarter and full year of 2022, respectively, as compared to $12.2 million and $46.3 million for the fourth quarter and full year of 2021, respectively. The increase in G&A expenses for the fourth quarter and full year of 2022 compared to the same periods in the prior year was primarily related to higher personnel related and consulting expenses offset in part by lower legal expenses. G&A expenses included non-cash share-based compensation expense of $3.9 million and $16.5 million for the fourth quarter and full year of 2022, respectively, as compared to $4.0 million and $15.1 million for the fourth quarter and full year of 2021, respectively.
Total non-cash share-based compensation expense was $7.4 million and $31.3 million for the fourth quarter and full year of 2022, respectively, as compared to $6.9 million and $24.7 million for the fourth quarter and full year of 2021, respectively.

As of December 31, 2022, Prothena had $712.6 million in cash, cash equivalents and restricted cash, and no debt.
As of February 17, 2023, Prothena had approximately 52.6 million ordinary shares outstanding.

2023 Financial Guidance

The Company expects the full year 2023 net cash used in operating and investing activities to be $213 to $229 million and expects to end the year with approximately $512 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2023 net cash used in operating and investing activities is primarily driven by an estimated net loss of $250 to $275 million, which includes an estimated $46 million of non-cash share-based compensation expense

Conference Call Details

Prothena management will discuss these results and its 2023 financial guidance during a live audio conference call today, Thursday, February 23, 2023, at 4:30 PM ET. The conference call will be made available on the Company’s website at www.prothena.com under the Investors tab in the Events and Presentations section. Following the live audio webcast, a replay will be available on the Company’s website for at least 90 days.

To access the call via dial-in, please dial +1 (888) 350-3870 (U.S. and Canada toll free) or +1 (646) 960-0308 (international) five minutes prior to the start time and refer to conference ID number 92750. A replay of the call will be available until March 2, 2023, via dial-in at (800) 770-2030 (U.S. toll free) or +00 1 647 362-9199 (international), Conference ID Number 92750.

On February 23, 2023 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported financial results for the fourth quarter and full year ended December 31, 2022, and provided recent business highlights (Press release, Personalis, FEB 23, 2023, View Source [SID1234627625]).

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Recent Business Highlights

Announced a continuing partnership with Moderna to provide genomic testing for their upcoming clinical studies evaluating mRNA-4157/V940, an investigational personalized cancer vaccine, jointly developed by Moderna and Merck

Partnered with Criterium and the Academic Breast Cancer Consortium (ABRCC) to conduct a prospective clinical trial to validate the clinical performance of the NeXT Personal assay to evaluate minimal residual disease (MRD) and subsequent recurrence in patients with early-stage, resectable triple negative breast cancer (TNBC)

Initiated a research collaboration with University Medical Center Hamburg-Eppendorf (UKE) and its new Fleur-Hiege Center for Skin Cancer Research, where Dr. Klaus Pantel, Dr. Christoffer Gebhardt, and team are using NeXT Personal to track tumor response to immunotherapy (IO) in patients with melanoma, with the aim of gathering evidence to advance the use of ultra-sensitive MRD detection in routine clinical practice for IO therapy monitoring

"We are excited to have completed our strategic review of the business, which has resulted in a new threefold focus on winning in MRD, leveraging our technology to power companies working on personalized cancer vaccines, and supporting pharmaceutical customers with clinical trials," said Aaron Tachibana, Interim Chief Executive Officer and Chief Financial Officer. "Our partnership with Moderna using our NeXT Platform to provide genomic testing is an example of how we enable customers. With our sharpened focus in both our clinical and biopharma businesses, we believe we are well-positioned to advance the standard of care by transforming cancer recurrence detection and ongoing therapy monitoring."

Fourth Quarter Highlights

Reported total company revenue of $16.7 million for the fourth quarter of 2022 and $65.0 million for the full year of 2022.

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Revenue from pharma tests, enterprise, and other customers of $15.8 million in the fourth quarter of 2022 compared with $15.4 million in the fourth quarter of 2021; revenue from enterprise customers includes revenue from Natera of $8.2 million in the fourth quarter of 2022

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Revenue from population sequencing for the U.S. Department of Veterans Affairs Million Veterans Program (VA MVP) of $0.9 million in the fourth quarter of 2022, which is initial revenue from the new contract awarded in September 2022, compared with $5.3 million in the fourth quarter of 2021

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Cash, cash equivalents, and short-term investments of $167.7 million as of December 31, 2022

Fourth Quarter and Full Year 2022 Financial Results

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Revenue of $16.7 million in the fourth quarter 2022; revenue of $65.0 million in the full year 2022
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Net loss of $31.1 million, and net loss per share of $0.67 based on a weighted-average basic and diluted share count of 46.3 million in the fourth quarter 2022; net loss of $113.3 million, and net loss per share of $2.48 based on a weighted-average basic and diluted share count of 45.7 million in the full year 2022

First Quarter and Full Year 2023 Outlook

Personalis expects the following for the first quarter of 2023:

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Total company revenue of approximately $17.5 million
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Revenue from pharma tests, enterprise sales, and other customers of approximately $14.5 million
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Revenue from population sequencing of approximately $3.0 million

Personalis expects the following for the full year of 2023:

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Total company revenue in the range of $68 million to $72 million, based on its strategic review of accounts and pruning of unprofitable business
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Revenue from pharma tests, enterprise sales, and all other customers in the range of $59 million to $63 million
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Revenue from population sequencing of approximately $9.0 million
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Net loss of approximately $103 million, down from $113 million in 2022 due to realization of headcount reduction savings, partially offset by investments in clinical evidence generation and non-cash depreciation expense for the new facility
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Cash usage of approximately $75 million, down from $119 million in 2022

Webcast and Conference Call Information

Personalis will host a conference call to discuss the fourth quarter and full year 2022 financial results after market close on Thursday, February 23, 2023 at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. To access the live call via telephone, please register in advance using the link here. Upon registering, each participant will receive an email confirmation with dial-in numbers and a unique personal PIN that can be used to join the call. The live webinar can be accessed at View Source A replay of the webinar will be available shortly after the conclusion of the call and will be archived on the company’s website.