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.

On August 4, 2022 OPKO Health, Inc. (NASDAQ: OPK) reported that business highlights and financial results for the three and six months ended June 30, 2022 (Press release, Opko Health, AUG 4, 2022, View Source [SID1234617539]).

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Business highlights for the second quarter of 2022 and subsequent weeks include the following:

Acquired ModeX Therapeutics, Inc. (ModeX), gained proprietary immunotherapy technology, seasoned scientific team, new executives and Directors. ModeX is focused on developing uniquely designed multi-specific immune therapies for cancer and infectious diseases. In connection with the acquisition, Dr. Elias Zerhouni joined OPKO as President and Vice Chairman of the Board of Directors, Dr. Gary Nabel joined as Chief Innovation Officer and a member of OPKO’s Board of Directors, and Alexis Borisy joined OPKO’s Board. The company acquired ModeX for $300 million in OPKO common stock.

Pfizer launched NGENLA (somatrogon) in Germany, Japan and additional global markets. NGENLA treats pediatric patients with decreased growth due to insufficient secretion of growth hormone and it reduces the frequency of required injections from once daily to once weekly. In the second quarter, OPKO received $85 million in milestone payments from Pfizer, OPKO’s global commercial partner, related to the launching of NGENLA in Europe and Japan.

Completed sale of GeneDx LLC (formerly GeneDx, Inc.) to Sema4 Holdings Corp. (Sema4). Sema4 acquired GeneDx for an upfront cash payment of $150 million, subject to adjustments, plus 80 million shares of Sema4 Class A common stock, with up to an additional $150 million in revenue-based milestones over the next two years (payable in cash or Sema4 shares at Sema4’s discretion). Based on the closing price of Sema4’s Class A common stock on April 29, 2022, the date the transaction closed, the total upfront consideration was approximately $322 million and the total aggregate consideration including potential milestones was approximately $472 million.

BioReference Laboratories’ (BRL) Scarlet Health announced a collaboration with Teladoc Health. Under the collaboration with Teladoc Health, a global leader in whole-person virtual care, BRL will offer in-home, on-demand phlebotomy service from Scarlet Health. The collaboration removes barriers to access and increases convenience through a seamless, comprehensive experience that meets people at their location. With Scarlet’s at-home, on-demand phlebotomy service, Teladoc Health saw laboratory order completion rates increase by 22.5% in an initial trial.
Second Quarter Financial Results

Pharmaceuticals: Revenue in the second quarter of 2022 increased to $35.9 million from $35.7 million in the second quarter of 2021, primarily attributable to higher RAYALDEE sales, partially offset by a decrease in OPKO’s pharmaceutical business in Chile, due to foreign exchange fluctuations. Revenue from sales of RAYALDEE in the second quarter of 2022 was $6.2 million compared with $5.0 million in the prior-year period. Revenue from the transfer of intellectual property was $87.2 million in the second quarter of 2022 compared with $9.5 million in the 2021 period. During the second quarter of 2022, OPKO received $85.0 million in milestone payments from Pfizer related to the commencement of NGENLA sales in Europe and Japan. The second quarter of 2021 included a $5.0 million non-refundable upfront payment under the license agreement with Nicoya Therapeutics. Total costs and expenses were $67.7 million in the second quarter of 2022 compared with $58.9 million in the prior-year period. This increase was primarily attributable to higher amortization expense related to the reclassification of NGENLA’s in-process research and development upon its approval in Europe and Japan during the first quarter of 2022 and spending related to the ModeX acquisition, partially offset by lower RAYALDEE cost per unit and foreign exchange fluctuations at OPKO’s international operating companies. Operating income was $55.4 million in the second quarter of 2022 compared with an operating loss of $13.7 million in the second quarter of 2021.

Diagnostics: Revenue from services in the second quarter of 2022 was $186.8 million compared with $397.2 million in the prior-year period, the decrease primarily due to lower COVID-19 testing volume. BRL processed approximately 0.9 million COVID-19 PCR tests in the second quarter of 2022 versus 2.8 million in the second quarter of 2021. Total costs and expenses were $244.3 million in the second quarter of 2022 compared with $367.2 million in the second quarter of 2021, resulting in an operating loss of $57.5 million compared with operating income of $30.0 million in the 2021 period. Significant investments in growth initiatives, principally our digital health platforms and non-traditional COVID lines of business negatively impacted operating margins at BRL. Operating loss for the second quarter of 2022 includes a $15.4 million gain on the sale of GeneDx, partially offset by $5.8 million of operating losses prior to closing in April 2022.

Consolidated: Consolidated total revenues for the second quarter of 2022 were $309.9 million compared with $442.4 million for the comparable period of 2021. Operating loss for the second quarter of 2022 was $10.7 million compared with operating income of $5.6 million for the 2021 quarter. Net loss for the second quarter of 2022 includes non-cash expense of $71.2 million related to a decrease in Sema4’s stock price at June 30, 2022. After giving effect to this non-cash expense, net loss for the three months ended June 30, 2022 was $101.7 million, or $0.14 per share, compared with net loss of $16.2 million, or $0.03 per diluted share, for the 2021 quarter.

Cash and equivalents: Cash and cash equivalents were $210.5 million as of June 30, 2022. In addition, OPKO has $53.1 million available under its line of credit with JP Morgan.
Conference Call and Webcast Information

OPKO’s senior management will provide a business update, discuss second quarter financial results and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call using this link. Callers who pre-register will be given a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing (833) 630-0584 (U.S.) or (412) 317-1815 (International). A webcast of the call may also be accessed at OPKO’s Investor Relations page and here.

A telephone replay will be available until August 11, 2022 by dialing (877) 344-7529 (U.S.) or (412) 317-0088 (International) and providing the passcode 5775821. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here.

On August 4, 2022 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune and inflammatory diseases, reported the company will participate in a fireside chat at the 2022 Wedbush PacGrow Healthcare Virtual Conference on Wednesday, August 10th, 2022, at 4:05 p.m. ET/1:05 p.m. PT (Press release, Alpine Immune Sciences, AUG 4, 2022, View Source [SID1234617538]).

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A live webcast of the fireside chat will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

On August 4, 2022 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that it will host a conference call and live audio webcast on Thursday, August 11, 2022 at 4:30 p.m. Eastern Time to discuss financial results for the second quarter ended June 30, 2022 and to provide a business update (Press release, CymaBay Therapeutics, AUG 4, 2022, View Source [SID1234617537]).

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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

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Conference Call Details
To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID#13730902. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source