On August 13, 2024, Inovio Pharmaceuticals, Inc. (the "Company") entered into an Equity Distribution Agreement (the "Sales Agreement") with Oppenheimer & Co. Inc., as sales agent (the "Sales Agent") under which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $0.001 per share (the "Common Stock"), through the Sales Agent (Filing, 8-K, Inovio, AUG 13, 2024, View Source [SID1234645810]).

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Pursuant to the Sales Agreement, sales of the Common Stock, if any, will be made under the Company’s previously filed and effective Registration Statement on Form S-3 (File No. 333-275445). The Company will file a prospectus supplement for the offer and sale of its Common Stock pursuant to the Sales Agreement having an aggregate offering price of up to $60,000,000.

Subject to the terms and conditions of the Sales Agreement, the Sales Agent may sell the Common Stock by any method permitted by law deemed to be an "at the market offering" as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended. The Sales Agent will use commercially reasonable efforts to sell the Common Stock from time to time, based upon instructions from the Company, including any price, time or size limits or other customary parameters or conditions the Company may impose. The Company will pay the Sales Agent a commission of up to three percent (3.0%) of the gross sales proceeds of any Common Stock sold through the Sales Agent under the Sales Agreement, and the Company has provided the Sales Agent with certain indemnification rights. The Company also will reimburse the Sales Agent for certain specified expenses in connection with entering into the Sales Agreement. The Company has no obligation to sell any of the Common Stock under the Sales Agreement and may at any time and from time to time suspend the offering of the Common Stock under the Sales Agreement.

The foregoing description of the Sales Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sales Agreement, a copy of which is filed as Exhibit 1.1 to this Current Report on Form 8-K.

The legal opinion of Cooley LLP relating to the validity of the Common Stock being offered pursuant to the Sales Agreement is filed as Exhibit 5.1 to this Current Report on Form 8-K.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.

On August 13, 2024 AstraZeneca reported that Imfinzi (durvalumab) and Lynparza (olaparib) have been approved in the European Union (EU) as treatment for certain patients with primary advanced or recurrent endometrial cancer (Press release, AstraZeneca, AUG 13, 2024, View Source [SID1234645826]). Imfinzi plus chemotherapy as 1st-line treatment followed by Lynparza and Imfinzi has been approved for patients with mismatch repair proficient (pMMR) disease. Imfinzi plus chemotherapy followed by Imfinzi alone has been approved for patients with mismatch repair deficient (dMMR) disease.

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The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on a prespecified exploratory subgroup analysis by mismatch repair (MMR) status from the DUO-E Phase III trial, which was published in the Journal of Clinical Oncology.

In the trial, the Lynparza and Imfinzi regimen reduced the risk of disease progression or death for patients with pMMR disease by 43% (median 15.0 months versus 9.7 months, hazard ratio [HR] 0.57; 95% confidence interval [CI] 0.44-0.73) versus the control arm.1 The Imfinzi regimen reduced the risk of disease progression or death among patients with dMMR disease by 58% (median not reached versus 7.0 months, HR 0.42; 95% CI 0.22-0.80) versus the control arm.1

Els Van Nieuwenhuysen, Gynaecological Oncologist at the UZ Leuven, Belgium and DUO-E trial investigator, said: "This approval is welcome news for patients with advanced or recurrent endometrial cancer in Europe, especially those with mismatch repair proficient disease who have limited options. The olaparib and durvalumab as well as the durvalumab regimens now have the potential to improve outcomes for all patients in this setting in Europe, regardless of mismatch repair status."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: "This approval of Imfinzi and Lynparza regimens marks the first-ever approval for a combination of an immunotherapy and PARP inhibitor in endometrial cancer and a major step forward for patients. In Europe, endometrial cancer is the fourth most common cancer in women, and until now, the 70 to 80 per cent of patients who have mismatch repair proficient disease have had few available treatment options."

The safety profiles of both regimens were generally manageable, well-tolerated and broadly consistent with the known profiles of the individual agents.1,2,3

Regulatory submissions for Imfinzi and Lynparza are currently under review in Japan and several other countries based on the DUO-E trial. Imfinzi plus chemotherapy was recently approved for dMMR patients with primary advanced or recurrent endometrial cancer in the US.

Notes

Endometrial cancer
Endometrial cancer is a highly heterogeneous disease that originates in the tissue lining of the uterus and is most common in women who have already been through menopause, with the average age at diagnosis being over 60 years old.4-7

The majority of patients with endometrial cancer are diagnosed at an early stage of disease, where the cancer is confined to the uterus.8 They are typically treated with surgery and/or radiation, and the five-year survival rate is high (approximately 80-90%).9 Patients with advanced disease (Stage III-IV) usually have a much poorer prognosis, with the five-year survival rate falling to less than 20%.10 Immunotherapy combined with chemotherapy is emerging as a new standard of care for advanced endometrial cancer, particularly for patients with dMMR disease, who make up approximately 20-30% of all patients.11-14 There is a significant need for new treatment options, especially for the 70-80% of patients with pMMR disease.15,16

In Europe, nearly 125,000 women were diagnosed with endometrial cancer in 2022.17,18

DUO-E
The DUO-E trial (GOG 3041/ENGOT-EN10) is a three-arm, randomised, double-blind, placebo-controlled, multicentre Phase III trial of 1st-line Imfinzi (durvalumab) plus platinum-based chemotherapy (carboplatin and paclitaxel) followed by either Imfinzi monotherapy or Imfinzi plus Lynparza (olaparib) as maintenance therapy versus platinum-based chemotherapy alone as a treatment for patients with newly diagnosed advanced or recurrent endometrial cancer.

The DUO-E trial randomised 699 patients with newly diagnosed advanced or recurrent epithelial endometrial carcinoma to receive either Imfinzi (1120mg) or placebo, given every three weeks in addition to standard-of-care platinum-based chemotherapy. After 4-6 cycles of chemotherapy, patients (whose disease had not progressed) then received either Imfinzi (1500mg) or placebo every four weeks as maintenance, plus 300mg Lynparza (300mg BID [2x150mg tablets, twice a day]) or placebo until disease progression.

The dual primary endpoint was progression-free survival (PFS) of each treatment arm versus standard-of-care chemotherapy alone, and both arms demonstrated a statistically significant and clinically meaningful improvement in PFS compared to standard of care in patients with newly diagnosed advanced or recurrent endometrial cancer.1 Key secondary endpoints included overall survival (OS), safety and tolerability. The trial continues to assess OS for both arms in the overall trial population. Mismatch repair (MMR) status, recurrence status and geographic location were stratification factors. The trial was sponsored independently by AstraZeneca and conducted in 253 study locations across 22 countries including the US, Europe, South America and Asia.

For more information about the trial, please visit ClinicalTrials.gov.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is the only approved immunotherapy and the global standard of care in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy. Imfinzi is also approved for the treatment of extensive-stage SCLC and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC.

Imfinzi also demonstrated statistically significant and clinically meaningful event-free survival results in patients with resectable early-stage NSCLC based on the AEGEAN Phase III trial. Imfinzi in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery is approved for patients in Switzerland and the UK based on this trial.

In limited-stage SCLC, Imfinzi demonstrated statistically significant and clinically meaningful improvements in the dual primary endpoints of OS and PFS compared to placebo in patients who had not progressed following standard-of-care concurrent chemoradiotherapy in the ADRIATIC Phase III trial.

In addition to its indications in lung cancers, Imfinzi is approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU and in combination with chemotherapy (carboplatin and paclitaxel) followed by Imfinzi monotherapy in primary advanced or recurrent endometrial cancer that is mismatch repair deficient in the US.

Since the first approval in May 2017, more than 220,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, breast cancer, bladder cancer, several gastrointestinal and gynaecologic cancers and other solid tumours.

Lynparza
Lynparza is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination-related (HRR) genes, such as those with mutations in BRCA1 and/or BRCA2, or those where deficiency is induced by other agents (such as new hormonal agents [NHAs]).

Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza may also help enhance immunogenicity and increase the impact of anti-tumour immune responses.

Lynparza is currently approved in a number of countries across multiple tumour types, including maintenance treatment of platinum-sensitive relapsed ovarian cancer and as both monotherapy and in combination with bevacizumab for the 1st-line maintenance treatment of BRCA-mutated (BRCAm) and homologous recombination repair deficient (HRD)-positive advanced ovarian cancer, respectively; for germline BRCA mutation (gBRCAm), HER2-negative metastatic breast cancer (in the EU and Japan, this includes locally advanced breast cancer); for gBRCAm, HER2-negative high-risk early breast cancer (in Japan, this includes all BRCAm HER2-negative high-risk early breast cancer); for gBRCAm metastatic pancreatic cancer; in combination with abiraterone for the treatment of metastatic castration-resistant prostate cancer (mCRPC) when chemotherapy is not clinically indicated (EU only) and for BRCAm mCRPC (US and Japan); and as monotherapy for HRR gene-mutated mCRPC in patients who have progressed on prior NHA treatment (BRCAm only in the EU and Japan). In China, Lynparza is approved for the treatment of BRCA-mutated mCRPC as well as 1st-line maintenance treatment with bevacizumab for HRD-positive advanced ovarian cancer.

Lynparza is being jointly developed and commercialised by AstraZeneca and MSD, both as a monotherapy and in combination with other potential medicines. Independently, the companies are developing and will commercialise Lynparza in combination with their respective PD-L1 and PD-1 medicines, Imfinzi (durvalumab) and Keytruda (pembrolizumab). Lynparza has been used to treat approximately 140,000 patients worldwide. Lynparza has a broad clinical trial development programme, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

On August 13, 2024 Instil Bio, Inc. ("Instil") (Nasdaq: TIL), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel therapies, reported its second quarter 2024 financial results and provided a corporate update (Press release, Instil Bio, AUG 13, 2024, View Source [SID1234645811]).

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"We have expanded our pipeline with a pair of clinical-stage, potentially best-in-class therapeutics by in-licensing SYN-2510 and SYN-27M," said Bronson Crouch, CEO of Instil. "By executing a 15-year lease of our Tarzana cell therapy manufacturing facility, we have strengthened our financial foundation to support Instil’s near-term clinical development of these assets."

Recent Highlights:

•In-licensed SYN-2510 and SYN-27M: In August 2024, SynBioTx, Inc., a wholly owned subsidiary of Instil, entered into a license and collaboration agreement with ImmuneOnco (HKEX:1541) for the exclusive global development and commercialization rights outside of Greater China of SYN-2510, a potentially best-in-class PD-L1xVEGF bispecific antibody, and SYN-27M, a next-generation ADCC-enhanced CTLA-4 antibody. SYN-2510 and SYN-27M have completed Phase 1a dose escalation studies in multiple solid tumor types in China, and ImmuneOnco is continuing patient enrollment in both programs to support dose optimization and dose expansion.

•Executed lease of our cell therapy manufacturing facility to AstraZeneca Pharmaceuticals LP: In July 2024, Instil reported the execution of a lease of its U.S. cell therapy manufacturing facility to AstraZeneca Pharmaceuticals LP. Under the terms of the agreement, initial base rent is greater than $7.5 million annually, and escalates at 3% per annum, with the tenant also required to pay certain operating expenses and tax expenses, subject to certain rent abatement in the first year of the 15-year lease term.

•Exploring further opportunities to in-license or acquire novel therapeutic candidates: Instil continues to explore further opportunities to in-license or otherwise acquire novel therapeutic candidates with first-in-class or best-in-class potential.
•Cash runway expected beyond 2026.

Second Quarter 2024 Financial and Operating Results:

As of June 30, 2024, Instil had cash, cash equivalents, marketable securities and long-term investments of $152.6 million, which consisted of $6.8 million in cash and cash equivalents, $141.8 million in marketable securities, and $4.0 million in long-term investments, compared to $175.0 million in cash, cash equivalents, marketable securities and long-term investments as of December 31, 2023, consisting of $9.2 million in cash and cash equivalents, $1.5 million in restricted cash, $141.2 million in marketable securities, and $23.2 million in long-term investments. Instil expects that its cash, cash equivalents, marketable securities and long-term investments as of June 30, 2024 will enable it to fund its operating plan beyond 2026.

Research and development expenses were $2.9 million and $10.2 million for the three and six months ended June 30, 2024, respectively, compared to $8.5 million and $29.1 million for the three and six months ended June 30, 2023, respectively.

General and administrative expenses were $10.7 million and $23.1 million for the three and six months ended June 30, 2024, respectively, compared to $11.5 million and $24.7 million for the three and six months ended June 30, 2023, respectively.

Restructuring and impairment charges were $0.5 million and $4.8 million for the three and six months ended June 30, 2024, respectively, compared to $1.0 million and $25.6 million for three and six months ended June 30, 2023, respectively.

Net loss per share, basic and diluted were $2.29 and $6.03 for the three and six months ended June 30, 2024, respectively, compared to $2.87 and $11.64 for the three and six months ended June 30, 2023, respectively. Non-GAAP net loss per share, basic and diluted, were $1.57 and $3.95 for the three and six months ended June 30, 2024, respectively, compared to $2.03 and $6.33 for the three and six months ended June 30, 2023, respectively.

On August 13, 2024 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 second quarter of 2024, its recent business highlights, and a preview of select anticipated milestones (Press release, Sutro Biopharma, AUG 13, 2024, View Source [SID1234645827]).

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"We continue to make meaningful progress with the development of luvelta across multiple indications, including enrollment of a patient expansion cohort in combination with bevacizumab, nearing initiation of our second registration-enabling trial, REFRaME-P1, for pediatric patients with a rare form of acute myeloid leukemia (AML) and approaching site activation of a Phase 2 trial in non-small cell lung cancer (NSCLC)," said Bill Newell, Sutro’s Chief Executive Officer. "We plan to share supplemental data from our Phase 1b trial of luvelta in combination with bevacizumab at the ESMO (Free ESMO Whitepaper) meeting in September."

Mr. Newell added, "We are off to strong start in our new partnership with Ipsen for STRO-003 and continue to advance our preclinical pipeline of next-generation ADCs, including our tissue-factor targeting exatecan ADC, STRO-004. In parallel, we are exploring new partnership opportunities to maximize the potential of our platform and pipeline, led by our new Chief Business Development Officer Barbara Leyman. Additionally, we are delighted to welcome Sukhi Jagpal to our Board, as he brings a wealth of invaluable financial and strategic expertise."

Recent Business Highlights and Select Anticipated Milestones

Luveltamab Tazevibulin (luvelta), FRα-Targeting ADC Franchise:

Sutro will present updated data from the Phase 1b study of luvelta in combination with bevacizumab for patients with ovarian cancer in a poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024 to be held September 13-17 in Barcelona, Spain.
Title: Luveltamab tazevibulin, an antifolate receptor alpha (FRα) antibody-drug conjugate (ADC), in combination with bevacizumab (bev) in patients with recurrent high-grade epithelial ovarian cancer (EOC): STRO-002-GM2 phase 1 study
Date: Saturday, September 14, 2024
Part 2 (randomized portion) of the Phase 3 trial, REFRαME-O1, for treatment of platinum-resistant ovarian cancer (PROC), is underway.
REFRαME-P1, a registration-enabling trial for pediatric patients with CBFA2T3::GLIS2 (CBF/GLIS; RAM phenotype) AML, is expected to be initiated in the second half of 2024.
A Phase 2 expansion study in combination with bevacizumab is ongoing, with data expected in the first half of 2025.
A Phase 2 trial for the treatment of NSCLC is expected to initiate in the second half of 2024, with initial data expected in the first half of 2025.
Additional Pipeline Development and Collaboration Updates:

In April 2024, Sutro announced a global licensing agreement for STRO-003, a ROR1-targeting ADC, with Ipsen.
Sutro plans to submit an IND for STRO-004 in 2025.
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 $970 million in payments through June 30, 2024, including equity investments.
Corporate Updates:

In August, Sutro strengthened its Board of Directors with the appointment of Sukhi Jagpal, MBA, CPA, CBV. Mr. Jagpal brings 20 years of experience in the life sciences industry, with expertise in financial management, communication, and organizational effectiveness, including financial analysis, mergers and acquisitions, and cost optimization.
In July, Sutro appointed Barbara Leyman, Ph.D., as Chief Business Development Officer, with a focus on building value and executing the Company’s business development strategy, in addition to serving on Sutro’s senior management team.
Upcoming Events: Sutro will participate in two upcoming investor conferences. Webcasts of the presentations will be accessible through the News & Events page of the Investor Relations section of the Company’s website at www.sutrobio.com. Archived replays will be available for at least 30 days after the events.

Wedbush PacGrow Healthcare Conference in New York, August 13-14, 2024
Wells Fargo Healthcare Conference in Boston, September 4-6, 2024
Second Quarter 2024 Financial Highlights

Cash, Cash Equivalents and Marketable Securities and Vaxcyte Common Stock
As of June 30, 2024, Sutro had $426.0 million, composed of cash, cash equivalents and marketable securities of $375.6 million and approximately 0.7 million shares of Vaxcyte common stock with a fair value of $50.4 million.

Unrealized Gain from Increase in Value of Vaxcyte Common Stock
The non-operating, unrealized gain of $4.8 million for the quarter ended June 30, 2024 was due to the increase since March 31, 2024 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 $25.7 million for the quarter ended June 30, 2024, as compared to $10.4 million for the same period in 2023, with the 2024 amount related principally to the Astellas collaboration and the Vaxcyte agreement. Future collaboration and license revenue under existing agreements, and from any additional collaboration and license partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other agreement payments.

Operating Expenses
Total operating expenses for the quarter ended June 30, 2024 were $74.4 million, as compared to $56.6 million for the same period in 2023. The 2024 quarter includes non-cash expenses for stock-based compensation of $6.2 million and depreciation and amortization of $1.8 million, as compared to $6.7 million and $1.7 million, respectively, in the comparable 2023 period. Total operating expenses for the quarter ended June 30, 2024 were comprised of research and development expenses of $62.0 million and general and administrative expenses of $12.4 million.

On August 13, 2024 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases and cancer, reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Kezar Life Sciences, AUG 13, 2024, View Source [SID1234645812]).

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"We are thrilled to announce completion of enrollment to our PORTOLA trial and look forward to sharing topline results earlier than expected in the first half of 2025. This important milestone brings us one step closer to delivering zetomipzomib as a new treatment option for patients suffering from autoimmune hepatitis, a disease of significant unmet medical need," said Chris Kirk, PhD, Kezar’s Co-founder and Chief Executive Officer. "In addition, we are continuing to see strong enrollment activity in our global PALIZADE trial and look to continue this momentum by focusing our clinical resources on zetomipzomib development programs going forward. I want to thank our team for their hard work and commitment across all of our clinical trials and share our gratitude to the patients, their families and the study investigators, for their participation in these studies."

Zetomipzomib: Selective Immunoproteasome Inhibitor

PALIZADE – Phase 2b clinical trial of zetomipzomib in patients with active lupus nephritis (LN) (ClinicalTrials.gov: NCT05781750)

•
PALIZADE is a global, placebo-controlled, randomized, double-blind Phase 2b clinical trial evaluating the efficacy and safety of two dose-levels of zetomipzomib in patients with active LN. Target enrollment will be 279 patients, randomly assigned (1:1:1) to receive 30 mg of zetomipzomib, 60 mg of zetomipzomib or placebo subcutaneously once weekly for 52 weeks, in addition to standard background therapy. Background therapy can, but will not be mandated to, include standard induction therapy. Over the initial 16 weeks, there will be a mandatory corticosteroid taper to 5 mg per day or less. End-of-treatment assessments will occur at Week 53. The primary efficacy endpoint is the proportion of patients who achieve a complete renal response (CRR) at Week 37, including a urine protein-to-creatine ratio (UPCR) of 0.5 or less without receiving rescue or prohibited medications.
•
Our partner, Everest Medicines, announced that the first patient in China was dosed with zetomipzomib as part of our global PALIZADE trial. Kezar entered into a collaboration and license agreement with Everest Medicines in September 2023 to develop and commercialize zetomipzomib in Greater China, South Korea and Southeast Asia.

•
Kezar expects to report topline data from PALIZADE in mid-2026.

PORTOLA – Phase 2a clinical trial of zetomipzomib in patients with autoimmune hepatitis (AIH) (ClinicalTrials.gov: NCT05569759)

•
PORTOLA is a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of zetomipzomib in patients with AIH that are insufficiently responding to standard of care or have relapsed. The study has completed enrollment of 24 patients, randomized (2:1) to receive 60 mg of zetomipzomib or placebo in addition to background therapy for 24 weeks, with a protocol-suggested steroid taper. The primary efficacy endpoint will measure the proportion of patients who achieve a complete biochemical response by Week 24 measured as normalization of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and Immunoglobulin G (IgG) values (if elevated at baseline), with steroid dose levels not higher than baseline.
•
Kezar expects to report topline data from PORTOLA in the first half of 2025.

KZR-261: Broad-Spectrum Sec61 Translocon Inhibitor

KZR-261-101– Phase 1 clinical trial of KZR-261 in patients with locally advanced or metastatic solid malignancies (ClinicalTrials.gov: NCT05047536)

•
The Phase 1 clinical trial of KZR-261 is being conducted in two parts: dose escalation and dose expansion in tumor-specific solid tumors. The study is designed to evaluate safety and tolerability, pharmacokinetics and pharmacodynamics, identify a recommended Phase 2 dose and explore the preliminary anti-tumor activity of KZR-261 in patients with locally advanced or metastatic disease.
•
Enrollment has been stopped in the KZR-261 Phase 1 study, and clinical resources are being reallocated toward development of zetomipzomib in AIH and LN. Patients already enrolled in the study will continue to have access to KZR-261.
•
A total of 61 patients enrolled across the dose-escalation and dose expansion portions of the study, which included seven patients enrolled in the melanoma cohort of the dose-expansion portion at a dose level of 60 mg/m2.
•
No objective responses have been observed to date in the study. Five patients (two with melanoma) within the dose escalation portion of the study experienced stable disease for four months or longer, of which two patients (melanoma; head and neck) experienced stable disease for twelve months or longer.
•
KZR-261 has demonstrated consistent pharmacokinetics across all dose levels to date, and evidence of dose-dependent inhibition of Sec61 has been observed in patient blood samples.
•
Kezar plans to report full data at a medical conference following completion of the study.

Financial Results

•
Cash, cash equivalents and marketable securities totaled $164.2 million as of June 30, 2024, compared to $201.4 million as of December 31, 2023. The decrease was primarily attributable to cash used in operations to advance clinical-stage programs.

•
Research and development (R&D) expenses for the second quarter of 2024 decreased by $4.7 million to $16.3 million, compared to $21.0 million in the second quarter of 2023. This decrease was primarily due to the Company’s strategic restructuring in October 2023 to prioritize its clinical-stage programs, reducing personnel-related costs and spending in its early-stage research activities. The decrease was partially offset by the increased clinical trial costs related to the PALIZADE and PORTOLA trials.
•
General and administrative (G&A) expenses for the second quarter of 2024 decreased by $0.2 million to $5.6 million compared to $5.8 million in the second quarter of 2023. The decrease was primarily due to a decrease in legal and professional service expenses.
•
Restructuring and impairment charges for the second quarter of 2024 were $1.5 million. The charges were primarily related to an impairment loss of the right-of-use asset related to vacated space in the company’s leased office facilities.
•
Net loss for the second quarter of 2024 was $21.5 million, or $0.30 per basic and diluted common share, compared to a net loss of $24.3 million, or $0.34 per basic and diluted common share, for the second quarter of 2023.
•
Total shares of common stock outstandingwere 72.9 million shares as of June 30, 2024. Additionally, there were options to purchase 15.3 million shares of common stock at a weighted-average exercise price of $2.18 per share and 0.2 million restricted stock units outstanding as of June 30, 2024.