On August 8, 2022 (GLOBE NEWSWIRE) — Merus N.V. (Nasdaq: MRUS) ("Merus", the "Company," "we", or "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported financial results for the second quarter that ended June 30, 2022 and provided a business update (Press release, Merus, AUG 8, 2022, View Source [SID1234617953]).

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"At the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting, we provided an update on our lead bispecific antibody, Zeno, which demonstrated strong efficacy across multiple tumor types, clinically meaningful duration of response and a very well tolerated safety profile. We continue to believe Zeno has the potential to be both first in class and best in class for patients with NRG1 fusion cancer," said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. "Additionally, we continue to make progress with our pipeline and look forward to providing an update on MCLA-129 in the second half of 2022, and Peto in the first half of 2023."

Clinical Programs

Zenocutuzumab (Zeno or MCLA-128: HER3 x HER2 Biclonics): NRG1+ cancer and other solid tumors

We shared updated interim clinical data on our Zeno program (eNRGy trial and Early Access Program) in patients with NRG1 fusion (NRG1+) cancer at the ASCO (Free ASCO Whitepaper) 2022 Annual Meeting. Highlights from the presentation included:

As of the April 12, 2022 data cutoff date, 110 patients with NRG1+ cancer were treated with Zeno, efficacy was assessed in 79 patients with measurable disease having the opportunity for 6 months or more follow-up and who met the criteria for the primary analysis population
Overall Response Rate (ORR) per RECIST criteria as assessed by investigator was 34% (27/79) (95% Cl; 24%-46%) across multiple tumor types
Pancreatic ductal adenocarcinoma ORR 42% (8/19) (95% CI: 20-67%)
Non-small cell lung cancer (NSCLC) ORR 35% (16/46) (95% CI: 21-50%)
Tumor shrinkage was observed in 70% of patients (55/79)
Median time to response was 1.8 months, and median duration of exposure was 6.3 months
Median duration of response was 9.1 months, and 20/83 patients were continuing treatment as of the data cutoff date
Zeno has demonstrated a consistent and well tolerated safety profile, with few grade 3 or 4 treatment-related adverse events
As announced in 2021, based on feedback received from the U.S. Food and Drug Administration (FDA), Merus believes that the eNRGy trial design and planned enrollment has the potential to support a Biologics License Application submission for Zeno for a tumor agnostic indication for the treatment of patients with NRG1+ cancer. To date, we have enrolled a cohort of patients that we believe may constitute a registrational data set, and continue to enroll patients to gather further safety and efficacy data on Zeno in NRG1+ cancer. We believe Zeno has the potential to be first and best in class and a new standard of care for patients with NRG1+ cancer.

We believe the favorable safety profile of Zeno may also allow for future, potential benefit in combination with other cancer therapies. Accordingly, we are initiating a clinical trial evaluating Zeno in combination with afatinib for NRG1+ NSCLC. In addition, beyond NRG1+ cancer, we are initiating a clinical trial evaluating Zeno as a treatment for castration resistant prostate cancer, and are actively exploring ways in which targeting both HER2 and HER3 with Zeno has potential for the treatment of other cancers.

Details of the eNRGy trial can be found at www.ClinicalTrials.gov and Merus’ trial website at www.nrg1.com, or by calling 1-833-NRG-1234.

Petosemtamab (Peto or MCLA-158: Lgr5 x EGFR Biclonics): Solid Tumors
Dose expansion continues in the phase 1 trial: clinical update planned for 1H2023

Peto is currently enrolling patients with advanced solid tumors in the expansion phase of a phase 1 open-label, multicenter study.

We plan to provide a clinical update for Peto at a medical conference in the first half of 2023. The planned presentation will provide the opportunity to present a robust update across the program, including approximately 40 patients with head and neck squamous cell carcinoma with meaningful clinical follow up, and an update on the gastro-esophageal cohort, to inform clinical development strategy and planned regulatory interactions.

MCLA-145 (CD137 x PD-L1 Biclonics): Solid Tumors
Phase 1 trial continues

MCLA-145 is currently enrolling a global, phase 1, open-label, single-agent clinical trial evaluating MCLA-145 in patients with solid tumors. The trial consists of a dose escalation phase, followed by a planned dose expansion phase. Merus is also planning to evaluate the combination of MCLA-145 with a PD-1 blocking antibody.

MCLA-129 (EGFR x c-MET Biclonics): Solid Tumors
Phase 1 trial continues: clinical update planned for 2H2022

MCLA-129 is currently enrolling patients in a phase 1/2, open-label clinical trial consisting of dose escalation followed by dose expansion. MCLA-129 is subject to a collaboration and license agreement with Betta Pharmaceuticals Co. Ltd. (Betta), which permits Betta to exclusively develop MCLA-129 in China, while Merus retains global rights outside of China. A clinical update is planned for the second half of 2022.

In July, Merus entered into a clinical supply agreement with AstraZeneca for Tagrisso (osimertinib), a third-generation EGFR-TKI, for a planned investigation of the combination of Tagrisso and MCLA-129 in patients with NSCLC in the dose expansion phase of the trial. Under the terms of the non-exclusive agreement, AstraZeneca will supply Tagrisso for use by Merus in the combination study.

Corporate Activities

Incyte
In the second quarter of 2022, Merus achieved a milestone payment for a pre-clinical candidate nomination of a novel bispecific antibody (target pair program) under the global collaboration and license agreement ("Agreement") with Incyte Corporation. This marks the third program to reach candidate nomination under the Agreement. Candidate nomination triggers a program advancing to the next phase of development for IND-enabling studies by Incyte. Incyte also recently announced its plan to initiate a clinical program later this year with INCA32459, a novel LAG3xPD-1 bispecific antibody developed under the collaboration agreement with Merus, that achieved candidate nomination in 2021.

Merus receives reimbursement for research activities related to the collaboration and is eligible to receive potential development, regulatory and commercial milestones and sales royalties for any products, if approved.

Cash Runway, Merus expects to be funded beyond 2024

As of June 30, 2022, Merus had $396.8 million cash and cash equivalents sufficient to fund company operations beyond 2024.

Second Quarter 2022 Financial Results

We ended the second quarter with cash, cash equivalents and marketable securities of $396.8 million compared to $430.7 million at December 31, 2021.

Collaboration revenue for the three months ended June 30, 2022 increased by $0.3 million as compared to the three months ended June 30, 2021, primarily as a result of an earned milestone in 2022 partially offset by decrease in amortization of upfront payment. The change in exchange rates did not significantly impact collaboration revenue.

Research and development expense for the three months ended June 30, 2022 increased by $6.5 million as compared to the three months ended June 30, 2021, primarily as a result of a personnel related expenses including stock-based compensation of $2.9 million due to an increase in employee headcount and an increase in external clinical services and drug manufacturing costs, including costs to fulfill our obligations under our collaboration agreements, related to our programs of $1.4 million.

General and administrative expense for the three months ended June 30, 2022 increased by $2.1 million as compared to the three months ended June 30, 2021, primarily as a result of an increase in stock-based compensation expense of $1.1 million, consulting costs of $0.7 million and personnel related expenses of $0.5 million due to an increase in employee headcount.

Collaboration revenue for the six months ended June 30, 2022 increased by $3.6 million as compared to the six months ended June 30, 2021, primarily as a result of an increase from Lilly upfront payment amortization and reimbursement revenues of $4.2 million partially offset by a decrease of Incyte revenue recognized of $0.2 million and a decrease in other upfront payment amortization and reimbursement revenues of $0.4 million. The Incyte decrease is primarily driven by a decrease in cost reimbursements of $0.9 million and amortization of upfront payments of $0.3 million, offset by the achievement and recognition of a $1.0 million development milestone in June 2022. The change in exchange rates did not significantly impact collaboration revenue.

Research and development expense for the six months ended June 30, 2022 increased by $12.7 million as compared to the six months ended June 30, 2021, primarily as a result of an increase in external clinical services and drug manufacturing costs, including costs to fulfill our obligations under our collaboration agreements, related to our programs of $5.2 million and an increase personnel related expenses including stock-based compensation of $5.0 million due to an increase in employee headcount.

General and administrative expense for the six months ended June 30, 2022 increased by $4.5 million as compared to the six months ended June 30, 2021, primarily as a result of an increase in stock-based compensation expense of $2.2 million, personnel related expenses of $1.1 million due to an increase in employee headcount, and finance and human resources costs of $0.9 million.

Other income (loss), net consists of interest earned and fees paid on our cash and cash equivalents held on account, accretion of investment earnings and net foreign exchange (losses) gains on our foreign denominated cash, cash equivalents and marketable securities. Other gains or losses relate to the issuance and settlement of financial instruments.

On August 8, 2022 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the second quarter of 2022 (Press release, Y-mAbs Therapeutics, AUG 8, 2022, View Source [SID1234617850]).

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"The highlight of the second quarter was FDA acceptance of our BLA filing for omburtamab," said Thomas Gad, President, and Interim Chief Executive Officer. "We are excited about the upcoming PDUFA date, which we believe brings us closer to the potential approval and achieving our goal of delivering omburtamab to children suffering from high-risk neuroblastoma brain tumors. We were also thrilled to announce that the Phase 2 naxitamab chemo-immunotherapy trial met primary endpoints achieving an impressive response rate further underscoring DANYELZA potential in high-risk neuroblastoma. Our strong balance sheet with $133.7 million in cash is expected to support us through multiple potentially value-creating catalysts and into mid-2024."

Second Quarter 2022 and Recent Corporate Developments

On July 12, 2022, Y-mAbs announced clearance of the IND for GD2-SADA
On May 31, 2022, Y-mAbs announced FDA acceptance of the Biologics License Application for OMBLASTYS (omburtamab) for the treatment of neuroblastoma for priority review
On May 26, 2022, Y-mAbs announced that the naxitamab chemoimmunotherapy investigational trial for High-Risk Neuroblastoma met its primary endpoint. The data was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).
On April 27, 2022, Y-mAbs announced a management change. Dr. Claus Moller stepped down as Chief Executive Officer and Board Member, and the role of Interim Chief Executive Officer was assumed by Thomas Gad, the Company’s Founder and President, Interim Chief Executive Officer and Head of Business Development and Strategy, and Board Member. The Board, under the newly appointed Chairman, Dr. Jim Healy, has begun a search for Dr. Moller’s successor.
On April 8, 2022, Y-mAbs presented pre-clinical data from the GD2-SADA construct at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting.
Financial Results

Revenues

Y-mAbs reported net revenues of $10.8 million and $21.3 million for the quarter and six months ended June 30, 2022, which represented a decrease of 1% and an increase 30%, respectively, over $11.0 million and $16.3 million in the comparable periods of 2021. Net revenues in the quarter and six months ended June 30, 2022 included $1.0 million of license revenue, compared to $2.0 million of license revenue in the corresponding periods in 2021. DANYELZA product revenue for the quarter and six months ended June 30, 2022 was $9.8 million and $20.3 million, respectively, which represented increases of 9% and 42%, respectively, over the corresponding periods in 2021. DANYELZA product revenue of $9.8 million in the second quarter 2022 deceased 7% compared to the first quarter of 2022 DANYELZA product revenues of $10.5 million. The decline included a slight decrease in new US patients earlier in the second quarter 2022, followed by a rebound in June, while international revenues benefitted from increased royalty income from partner’s sales, offset by a decrease in volume due to the timing of partner orders.

We have now delivered DANYELZA to 36 centers across the U.S., corresponding to an increase of 6% in the number of centers since the end of the first quarter of 2022. During the second quarter of 2022, approximately 40% of the vials sold in the U.S. were sold outside Memorial Sloan Kettering ("MSK"), a decrease from the prior quarter as a result of a decreased number of new patients at institutions outside MSK earlier in the second quarter followed by a rebound in June, as noted above, all while new patients at MSK continued to grow.

Operating Expenses

Research and Development

Research and development expenses were $26.4 million for the three months ended June 30, 2022, compared to $19.8 million for the three months ended June 30, 2021. The $6.6 million increase reflects an increase in outsourced manufacturing, inclusive of $2.9 million of naxitamab inventory vials that were designated for use in clinical trials during the three months ended June 30, 2022, our increased clinical trial activity and employee-related costs. Having completed the resubmission of the BLA for omburtamab in the first quarter 2022, we are focusing on pipeline development programs for potential DANYELZA label expansion, omburtamab and advancing the SADA constructs into the clinic.

Research and development expenses increased by $8.0 million to $49.3 million during the six months ended June 30, 2022 compared to the prior year period. The $8.0 million increase reflects an increase in outsourced manufacturing, inclusive of $2.9 million of naxitamab inventory vials that were designated for clinical use during the six months ended June 30, 2022, and our increased clinical trial activity, with particular focus on the DANYELZA, omburtamab and SADA technologies.

Selling, General, and Administration

Selling, General, and Administrative expenses increased by $9.6 million and $11.1 million, to $23.1 million and $36.5 million, for the quarter and six months ended June 30, 2022, respectively, compared to the prior year periods. The increase in selling, general and administrative expenses in both periods was primarily the result of a $10.7 million charge related to contractual severance related benefits for our former Chief Executive Officer, which was inclusive of $9.3 million of non-cash share-based compensation expense, and, to a lesser extent, the launch and commercialization of DANYELZA, which include employee-related costs and commercial expenses.

Net Loss

We reported a net loss for the quarter ended June 30, 2022, of $41.1 million, or $0.94 per basic and diluted share, compared to net loss of $22.9 million, or $0.53 per basic and diluted share for the quarter ended June 30, 2021. The decrease in earnings was primarily driven by the $10.7 million charge for contractual severance related benefits for our former Chief Executive Officer and increased research and development expenses focused on pipeline development programs for DANYELZA label expansion, omburtamab, and advancing the SADA constructs into the clinic.

We reported a net loss for the six months ended June 30, 2022, of $69.2 million, or $1.58 per basic and diluted share, compared to a net income of $10.5 million, or $0.25 per basic share and $0.23 per diluted share, for the six months ended June 30, 2021. Net income in the six months ended June 30, 2021, included a $62.0 million net gain from the sale of our DANYELZA Priority Review Voucher, after sharing 40% of the net proceeds from the sale with MSK, pursuant to the terms of our license agreement with MSK. The decrease in earnings in the six months ended June 30, 2022 also reflects the unfavorable impact of a $10.7 million charge for contractual severance related benefits for our former Chief Executive Officer, and increased research and development expenses, both as noted above, partially offset by the favorable impact of DANYELZA’s growing revenues.

Cash and Cash Equivalents

We had approximately $133.7 million in cash and cash equivalents as of June 30, 2022, and we continue to expect full year 2022 cash burn of $78-83 million. Our cash and cash equivalents balance of approximately $133.7 million as of June 30, 2022, which, when combined with forecasted DANYELZA revenue growth, is expected to be sufficient to fund our operations as currently planned into mid-2024. This estimate is based on our current business plan. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect.

This estimate does not include any assumption for net proceeds received on the anticipated priority review voucher, which we could sell upon the potential approval of omburtamab. In addition, no new partnerships or other new business development-related sources of income are included in the assumptions, potential omburtamab revenues upon approval are also excluded, and the DANYELZA revenues are only assumed to increase modestly each year for the purpose of this analysis of runway.

Financial Guidance

Management reiterates all elements of its financial guidance including:

DANYELZA 2022 revenues of $45-$50 million;
Operating expenses of $162-167 million;
Total cash burn of $78-83 million for 2022; and
Cash position sufficient to fund current operations into mid-2024.
The revenue guidance includes an incremental benefit from international revenues.

Webcast and Conference Call

Y-mAbs will host a conference call on Tuesday, August 9, 2022, at 9 a.m. Eastern Time. To participate in the call, please dial 877-407-0792 (domestic) or 201-689-8263 (international) and reference the conference ID 13730680.

A webcast will be available at: View Source;tp_key=db740a8735

On August 8, 2022 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a clinical-stage precision oncology company designing and developing novel targeted therapies for cancer treatment, reported financial results for the quarter ended June 30, 2022 and provided operational updates (Press release, Turning Point Therapeutics, AUG 8, 2022, View Source [SID1234617849]).

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"We are pleased with our continued pipeline advancement and expansion," said Athena Countouriotis, M.D., President and CEO. "We look forward to a productive second half of the year, with multiple data readouts and continued regulatory progress."

Second quarter and recent operational highlights include:

REPOTRECTINIB:

Announced receipt of positive feedback from the FDA at a pre-NDA meeting completed during the second quarter. The feedback focused on the proposed patient follow-up within the ROS1-positive advanced NSCLC patient cohorts of the ongoing TRIDENT-1 registrational study. The purpose of the pre-NDA meeting was to discuss the company’s planned NDA for repotrectinib for the treatment of ROS1+ advanced NSCLC. The FDA agreed with the company’s plan to provide data for ROS1+ TKI-naïve and TKI-pretreated advanced NSCLC patients with at least six months of follow-up from the first post-baseline scan at the time of NDA submission.
Received Breakthrough Therapy designation (BTD) from the FDA for repotrectinib for the treatment of patients with ROS1-positive metastatic NSCLC who have been previously treated with one ROS1 tyrosine kinase inhibitor and who have not received prior platinum-based chemotherapy. This represents the eighth regulatory designation granted by the FDA for repotrectinib.
ELZOVANTINIB:

Initiated the Phase 1b/2 SHIELD-2 combination study of elzovantinib and aumolertinib in EGFR mutant MET-amplified advanced non-small cell lung cancer. The combination of elzovantinib and aumolertinib is being studied in patients with EGFR mutant MET-amplified advanced NSCLC who have progressed following treatment with osimertinib. The study will evaluate the safety, tolerability and preliminary efficacy of the combination regimen.
TPX-4589

Initiated patient dosing in the third dosing level cohort in the Phase 1 study of TPX-4589. TPX-4589 is a potentially first-in-class anti-Claudin18.2 antibody drug conjugate (ADC) that suppresses cell proliferation of gastric and pancreatic cell lines with nanomolar potency in preclinical models. It is currently being studied in two ongoing Phase 1 studies in patients with advanced solid tumors.
BUSINESS DEVELOPMENT:

Announced a strategic research and development alliance with The University of Texas MD Anderson Cancer Center to expand the evaluation of repotrectinib and elzovantinib. The planned focus of the alliance includes monotherapy and potential combinations with other agents – including chemotherapy, immunotherapies and other targeted agents.
Entered into an exclusive license agreement with LaNova Medicines Limited to develop and commercialize LM-302, now known as TPX-4589, a novel antibody drug conjugate targeting Claudin18.2, in the United States and rest of the world excluding Greater China and South Korea. Claudin18.2 is a protein expressed in many gastrointestinal cancers, including gastric, gastroesophageal junction and pancreatic cancer. TPX-4589 is currently in Phase 1 clinical trials in both the United States and China.
Announced a definitive merger agreement with Bristol Myers Squibb to acquire Turning Point Therapeutics for $76.00 per share. The transaction was unanimously approved by both the Bristol Myers Squibb and Turning Point Therapeutics Boards of Directors and is anticipated to close during the third quarter of 2022.
Upcoming Milestones

REPOTRECTINIB

Present detailed study results, including intracranial activity, from the ROS1-positive advanced NSCLC cohorts of the TRIDENT-1 study at a medical conference in the second half of 2022.
Provide a clinical data update from the NTRK+ advanced solid tumor cohorts from TRIDENT-1 in the second half of 2022.
ELZOVANTINIB

Initiate the Phase 2 portion of the SHIELD-1 study in the second half of 2022, pending FDA feedback on data from the intermediate dose level.
Provide a clinical data update from the Phase 1 SHIELD-1 study in the second half of 2022.
TPX-0131

Provide early interim data from initial patients treated in the dose-finding portion of the FORGE-1 study in the fourth quarter of 2022 or early 2023.
TPX-4589

Present preclinical data at a medical conference by early 2023.
Provide additional guidance on clinical development plan by early 2023.
DISCOVERY

Nominate 2 development candidates in the second half of 2022.
Provide details on the other 2 GTPase signaling discovery programs in the second half of 2022.
Second Quarter 2022 Financial Results

Revenue: Revenue recognized during the second quarter of 2022 was $0.1 million from the sale of clinical supply to Zai Lab (Shanghai) Co. Ltd. (Zai), compared to $5.2 million during the second quarter of 2021, consisting of $5.0 million earned upon the achievement of development milestones under the license agreement with Zai regarding repotrectinib and $0.2 million from the sale of clinical supply to Zai.

R&D Expenses: Research and development expenses were $86.8 million for the second quarter of 2022 compared to $44.7 million for the second quarter of 2021. Primary drivers of the year-over-year increase were investments made to develop repotrectinib, discovery efforts and personnel expenses. In addition, R&D expenses for the second quarter of 2022 included an upfront payment of $25.0 million to LaNova for the in-licensing of its intellectual property that has not yet achieved regulatory approval.

G&A Expenses: General and administrative expenses were $37.7 million for the second quarter of 2022 compared to $17.2 million for the second quarter of 2021. G&A expenses for the second quarter of 2022 included approximately $17.7 million of transaction costs incurred in connection with the pending acquisition by Bristol Myers Squibb.

Net Loss: Net loss was $123.1 million for the second quarter of 2022 compared to net loss of $56.3 million for the second quarter of 2021.

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2022 totaled $818.3 million, reflecting a net decrease of approximately $99.9 million from March 31, 2022.

On August 8, 2022 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported its financial results for the quarter ended June 30, 2022, its recent business highlights, and a preview of anticipated select milestones (Press release, Sutro Biopharma, AUG 8, 2022, View Source [SID1234617848]).

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"This quarter, Sutro continued to execute on the promise of our platform, as marked by the recently announced collaboration with Astellas covering research and development of immunostimulatory ADCs, or iADCs—a novel modality with the potential to turn cold tumors hot," said Bill Newell, Sutro’s Chief Executive Officer. "Additionally, we are pleased to see Merck dosing patients in its Phase 1 study as part of our cytokine derivative collaboration. This represents the sixth clinical-stage product candidate enabled by Sutro’s platform. Looking ahead, we are optimistic about the potential of our pipeline of ADCs; this includes STRO-002 for patients with platinum-resistant ovarian cancer, as well as our newly unveiled STRO-003, an optimized ROR1 ADC, which we anticipate will be our next proprietary product candidate to move into clinical studies."

Recent Business Highlights and Anticipated Select Milestones

STRO-002, FolRα-Targeting ADC: STRO-002 is being studied in the clinic, in both the U.S. and Europe, for patients with ovarian and endometrial cancers.

The Phase 1 dose-expansion cohort for patients with advanced ovarian cancer has completed enrollment and is ongoing. Sutro expects to report additional data on efficacy, safety, and durability from the dose-expansion cohort, together with the design of a potential registrational study, in the second half of 2022.
Discussions with the FDA on a potential registrational study for patients with advanced ovarian cancer were held mid-year 2022, in which the agency signaled that an accelerated approval pathway could be available for STRO-002 in a platinum-resistant ovarian cancer patient population.
Additional ongoing clinical studies for STRO-002 include a combination study with bevacizumab for patients with advanced ovarian cancer and a dose-expansion study for patients with endometrial cancer.
STRO-001, CD74-Targeting ADC: The Phase 1 study for patients with B-cell malignancies, including patients with non-Hodgkin’s lymphoma (NHL) and multiple myeloma (MM), continues in dose escalation.

Dose escalation is ongoing to achieve a recommended Phase 2 dose (RP2D), with the last reported doses of 5.0 mg/kg in the MM cohort and 5.0 mg/kg in the NHL cohort.
Sutro is opening additional sites for STRO-001 outside of the U.S. and Greater China, to increase the rate of enrollment; and Sutro’s partner BioNova Pharma (BioNova) is advancing clinical development of BN301 (STRO-001) for patients with hematological malignancies in Greater China.
Additional Pipeline Programs: A Sutro Research Forum highlighted STRO-003 and its emerging research portfolio.

STRO-003 is an optimally designed ADC targeting ROR1, with precisely positioned β-Glucuronidase-cleavable linkers, attached to eight next-generation exatecan warheads, which inhibit topoisomerase-1 resulting in DNA disruption.
Patient-derived xenograft models (PDX) have shown potent cell killing by STRO-003 in low antigen expressing tumors; and STRO-003 has shown encouraging tolerability in preclinical rodent and non-human primate studies.
Sutro provided details on its product and process design, which enables its emerging portfolio including novel therapeutic modalities—for example, a single antibody which was conjugated to be site-specific, with two different payloads with synergistic mechanisms.
Collaboration Updates: Sutro continues to seek to maximize the value of its proprietary cell-free platform by working with partners on programs in multiple disease spaces and geographies and has generated from collaborators an aggregate of approximately $583 million, which includes payments and equity investments through June 30, 2022, in addition to the $90 million upfront payment from Astellas received in July 2022.

Sutro entered into a collaboration with Astellas on the discovery and development of iADCs for three targets, including an upfront payment to Sutro of $90 million, which was received in July 2022, and $422.5 million in potential milestones per product candidate. Sutro will also receive financial support for its research efforts and has an option to co-develop and co-commercialize product candidates in the U.S.
A $10 million milestone payment from Merck was triggered in July 2022 upon the first patient dosed in a Phase 1 study of MK-1484, a selective IL-2 agonist, under the existing cytokine derivative collaboration.
Sutro is manufacturing initial drug supply for the clinical development of Merck’s MK-1484, currently in a Phase 1 study; clinical trial materials for Bristol Myers Squibb’s (BMS) CC-99712, a BCMA-targeting ADC for treatment of multiple myeloma, in Phase 1 studies; and clinical trial materials for M1231, a MUC1-EGFR-targeting bispecific ADC, for Merck KGaA, Darmstadt, Germany, known as EMD Serono in the U.S. and Canada (EMD Serono), in Phase 1 studies.
Sutro supplies cell-free extract to Vaxcyte for the manufacture of clinical trial materials for VAX-24, which is designed to prevent invasive pneumococcal disease. Vaxcyte announced in July 2022 that it had completed enrollment of the Phase 1/2 clinical proof-of-concept study of VAX-24. Sutro is eligible to receive four percent (4%) royalties on worldwide net sales of VAX-24 and any licensed vaccine candidates.
BioNova announced in July 2022 that it had submitted its IND for BN301 (STRO-001) to the National Medical Products Administration for the treatment of hematologic malignancies. Sutro is providing clinical drug supply to BioNova for clinical studies in Greater China.
Sutro is currently supporting Tasly for initiation of clinical development activities and IND filing in Greater China for STRO-002 and will provide initial clinical drug supply.
Second Quarter 2022 Financial Highlights

Cash, Cash Equivalents and Marketable Securities
As of June 30, 2022, Sutro had cash, cash equivalents and marketable securities of $191.6 million, as compared to $192.1 million as of March 31, 2022, which, together with the $90 million upfront payment received from Astellas in July 2022, provides a projected cash runway into the first half of 2024, based on current business plans and assumptions. The above balances do not include the value associated with Sutro’s holdings of Vaxcyte common stock.

Unrealized Loss from Decrease in Value of Vaxcyte Common Stock
As of June 30, 2022, Sutro held approximately 1.6 million shares of Vaxcyte common stock, with a fair value of $34.0 million. The non-operating, unrealized loss of $3.7 million in the second quarter of 2022 was due to the decrease since March 31, 2022 in the estimated fair value of Sutro’s holdings of Vaxcyte common stock. Vaxcyte common stock held by Sutro will be remeasured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any non-operating, unrealized gains and losses recorded in Sutro’s statements of operations.

Revenue
Revenue was $28.1 million for the quarter ended June 30, 2022, as compared to $28.0 million for the same period in 2021, related principally to recognition of the upfront payment from Tasly in the second quarter of 2022 and the Merck, BMS, and EMD Serono collaborations in both years. Future collaboration revenue from Astellas, Tasly, Merck, BMS, and EMD Serono, and from any additional collaboration partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other collaboration agreement payments.

Operating Expenses
Total operating expenses for the quarter ended June 30, 2022 were $47.5 million, as compared to $37.9 million for the same period in 2021. The second quarter of 2022 includes non-cash expenses for stock-based compensation of $6.7 million and depreciation and amortization of $1.4 million, as compared to $5.9 million and $1.1 million, respectively, in the comparable 2021 period. Total operating expenses for the quarter ended June 30, 2022 were comprised of research and development expenses of $32.3 million and general and administrative expenses of $15.1 million, which are expected to increase in 2022 as Sutro’s internal product candidates advance in clinical development and additional general and administrative expenses are incurred as a public company.

On August 8, 2022 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 and provided business highlights for the second quarter and first six months of 2022 (Press release, Prothena, AUG 8, 2022, View Source [SID1234617847]).

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"We are closing the first half of 2022 from a position of strength, with multiple clinical programs advancing and a solid cash position supporting a robust set of milestones anticipated in 2022 and beyond. We are grateful for the opportunity to quickly progress the development of PRX012, a next-generation anti-Aβ antibody, under FDA’s Fast Track designation, further reinforcing our commitment to Alzheimer’s disease patients and their families," said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena. "We believe our anticipated progress in the second half of the year will continue to validate our biology-directed R&D strategy, with antibodies that have been engineered to target pathogenic proteins in a manner we believe will be most impactful for the treatment of disease. We expect to achieve multiple milestones, including topline Phase 1 data for PRX005, the initiation of a Phase 1 multiple ascending dose study of PRX012 and the continued advancement of the Phase 2 study of PRX004 by Novo Nordisk. Additionally, we remain focused on our confirmatory Phase 3 AFFIRM-AL study of birtamimab, the first potential therapy to show survival benefit in Mayo Stage IV patients with AL amyloidosis."

Second Quarter and Recent Business Highlights and Upcoming Milestones

Neurodegenerative Diseases Portfolio

Alzheimer’s Disease (AD)

PRX012, a potential best-in-class, next-generation treatment for AD, is an investigational monoclonal antibody targeting a key epitope at the N-terminus of amyloid beta (Aβ) with high binding potency supporting subcutaneous administration

In April, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for PRX012 for the treatment of AD
Phase 1 multiple ascending dose (MAD) study initiation expected by year-end 2022
Topline data from Phase 1 study expected in 2023
PRX005, a potential best-in-class treatment for AD, is an investigational antibody 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 the global neuroscience research and development collaboration with Bristol Myers Squibb

Topline data from Phase 1 study expected in 2022
PRX123, a potential first-in-class dual Aβ/tau vaccine treatment and prevention therapy for AD, is a dual-target vaccine targeting key epitopes within the Aβ and tau proteins to promote amyloid clearance and blockade of pathogenic tau

IND filing expected in 2023
Parkinson’s Disease (PD)

Prasinezumab, a potential first-in-class treatment for PD, is a humanized monoclonal antibody designed to target key epitopes within the C-terminus of alpha-synuclein and is the focus of the worldwide collaboration with Roche

Phase 2b PADOVA study results expected in 2024
Rare Peripheral Amyloid Diseases Portfolio

AL Amyloidosis

Birtamimab, a potential best-in-class amyloid depleter treatment for AL amyloidosis, is an investigational humanized monoclonal antibody designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid that causes organ dysfunction and failure

Abstract accepted for poster presentation on Monday September 5, 2022 at the XVIII International Symposium on Amyloidosis (ISA) titled: Birtamimab in Patients with Mayo Stage IV AL Amyloidosis: Rationale for Confirmatory AFFIRM-AL Phase 3 Study (Poster P040)
Confirmatory Phase 3 AFFIRM-AL study results expected in 2024
ATTR Amyloidosis

NNC6019 (previously PRX004), a potential first-in-class treatment for ATTR amyloidosis, is a humanized monoclonal antibody designed to deplete the pathogenic, non-native forms of the transthyretin (TTR) protein, that is being developed by Novo Nordisk for the treatment of ATTR cardiomyopathy

Phase 2 trial of NNC6019 in patients with ATTR cardiomyopathy is being conducted by Novo Nordisk (NCT05442047)
Second Quarter and First Six Months of 2022 Financial Results

For the second quarter and first six months of 2022, Prothena reported a net loss of $41.2 million and $77.5 million, as compared to a net income of $27.6 million and a net loss of $9.1 million for the second quarter and first six months of 2021. Net loss per share for the second quarter of 2022 and first six months of 2022 was $0.88, and $1.66, respectively, as compared to net income per share on a diluted basis of $0.58 and net loss per share of $0.21 for the second quarter and first six months of 2021, respectively.

Prothena reported total revenue of $1.3 million and $2.5 million for the second quarter and first six months of 2022, respectively, primarily from collaboration revenue from BMS. As compared to total revenue of $60.1 million and $60.2 million for the second quarter and first six months of 2021, from collaboration and license revenue from Roche.

Research and development (R&D) expenses totaled $31.6 million and $58.8 million for the second quarter and first six months of 2022, respectively, as compared to $21.1 million and $42.2 million for the second quarter and first six months of 2021, respectively. The increase in R&D expense for the second quarter and first six months of 2022 compared to the same periods in the prior year was primarily due to higher personnel related expenses, higher clinical trial expenses primarily related to the PRX012 and birtamimab programs, higher manufacturing costs primarily related to the birtamimab, PRX019 and PRX123 programs, and higher other R&D expense; offset in part by lower collaboration expenses related to the prasinezumab program with Roche as a result of the cost share opt-out exercised in May 2021 and lower manufacturing expenses related to the NNC6019/PRX004 and PRX005 programs. R&D expenses included non-cash share-based compensation expense of $3.8 million and $7.1 million for the second quarter and first six months of 2022, respectively, as compared to $2.2 million and $4.2 million for the second quarter and first six months of 2021, respectively.

General and administrative (G&A) expenses totaled $13.0 million and $24.8 million for the second quarter and first six months of 2022, respectively, as compared to $11.0 million and $22.2 million for the second quarter and first six months of 2021, respectively. The increase in G&A expenses for the second quarter and first six months of 2022 compared to the same periods in the prior year was primarily related to higher personnel related expenses and higher consulting expenses; offset in part by lower legal expenses. G&A expenses included non-cash share-based compensation expense of $4.5 million and $8.8 million for the second quarter and first six months of 2022, respectively, as compared to $3.3 million and $7.5 million for the second quarter and first six months of 2021, respectively.

Total non-cash share-based compensation expense was $8.3 million and $15.9 million for the second quarter and first six months of 2022, respectively, as compared to $5.5 million and $11.7 million for the second quarter and first six months of 2021, respectively.

As of June 30, 2022, Prothena had $510.1 million in cash, cash equivalents and restricted cash, and no debt.

As of July 29, 2022, Prothena had approximately 46.9 million ordinary shares outstanding.

2022 Financial Guidance

The Company continues to expect the full year 2022 net cash used in operating and investing activities to be $120 to $132 million, which includes an expected $40 million clinical milestone payment from Novo Nordisk and expects to end the year with approximately $454 million in cash, cash equivalents and restricted cash (midpoint). The estimated full year 2022 net cash used in operating and investing activities is primarily driven by an estimated net loss of $154 to $170 million, which includes an estimated $32 million of non-cash share-based compensation expense.