On August 14, 2024 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported second quarter and first half 2024 financial results and business updates (Press release, Precigen, AUG 14, 2024, View Source [SID1234645926]).

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"We are all in on PRGN-2012 given the immense unmet need for RRP patients and our groundbreaking pivotal data supporting the potential of what we hope to be the first-ever FDA approved therapy to treat RRP," said Helen Sabzevari, PhD, President and CEO of Precigen. "By strategically focusing our portfolio, streamlining resources and recent public offering, we have optimized the company to rapidly prepare for submission of a rolling biologics license application under an accelerated approval pathway. We are excited to have initiated enrollment in the confirmatory clinical trial and will continue to accelerate our commercial readiness campaign for a potential launch in 2025 under the leadership of our newly hired Chief Commercial Officer. Additionally, we plan to maximize portfolio value by focusing on strategic partnerships to further advance our highly promising UltraCAR-T programs."

"Our recent reprioritization and public offering is expected to fund our operations into early 2025 allowing us to focus on advancement of PRGN-2012 while continuing to explore potential non-dilutive financing opportunities for future liquidity," said Harry Thomasian Jr., CFO of Precigen.

Key Program Highlights

· PRGN-2012 AdenoVerse Gene Therapy in RRP: PRGN-2012 is an investigational off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). PRGN-2012 received Breakthrough Therapy Designation from the US Food and Drug Administration (FDA). PRGN-2012 also received Orphan Drug Designation from the FDA and Orphan Drug Designation from the European Commission.

o Results from the pivotal clinical study of PRGN-2012 for the treatment of RRP were presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in a late-breaking oral presentation titled, "PRGN-2012, a novel gorilla adenovirus-based immunotherapy, provides the first treatment that leads to complete and durable responses in recurrent respiratory papillomatosis patients."

§ Pivotal study met primary safety and efficacy endpoints.

§ 51% (18 out of 35) of patients achieved Complete Response, requiring no surgeries after treatment with PRGN-2012; Complete Responses have been durable beyond 12 months with median duration of follow up of 20 months as of the May 20, 2024 data cutoff.

§ 86% of patients (30 out of 35) had a decrease in surgical interventions in the year after PRGN-2012 treatment compared to the year prior to treatment; RRP surgeries reduced from a median of 4 (range: 3-10) pre-treatment to 0 (range: 0-7) post-treatment.

§ PRGN-2012 was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.

§ PRGN-2012 treatment induced HPV 6/11-specific T cell responses in RRP patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with non-responders.

§ PRGN-2012 significantly (p < 0.0001) improved Derkay and quality of life scores in complete responders.

o A rolling Biologics License Application (BLA) submission under an accelerated approval pathway is anticipated in the second half of 2024.

o The Company has initiated enrollment in the confirmatory clinical trial, in accordance with the guidance from the FDA, prior to submission of the BLA.

o The Company and the Recurrent Respiratory Papillomatosis Foundation held the inaugural RRP Awareness Day on June 11, 2024. The multi-stakeholder event raised awareness by bringing together individuals living with RRP, caregivers, clinicians, and government officials.

· Strategic Prioritization: In August 2024, the Company announced a strategic prioritization of its clinical portfolio and associated streamlining of resources, including a reduction of over 20% of its workforce, to focus on potential commercialization of PRGN-2012.

o PRGN-2009 AdenoVerse Gene Therapy Clinical Trials

§ The Company plans to continue PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer.

§ PRGN-2009 cervical cancer clinical trial enrollment at non-NCI clinical sites will be paused.

o UltraCAR-T Clinical Programs

§ The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the FDA, and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps.

§ The Company will pause the PRGN-3005 and PRGN-3007 clinical trials.

§ The Company will minimize UltraCAR-T spend and focus on strategic partnerships to further advance UltraCAR-T programs.

o Preclinical Programs

§ The Company will pause all preclinical programs.

o ActoBio

§ The Company has initiated shutdown of its Belgium-based ActoBio subsidiary operations, including planned elimination of all ActoBio personnel.

§ In conjunction with this shutdown, ActoBio’s portfolio of intellectual property will be made available for prospective transactions.

Financial Highlights

· Strategic prioritization resulted in non-cash impairment charges of $32.9 million, net of tax, in the second quarter and severance charges of $3.0 million, of which $2.1 million was recorded in the second quarter and $0.9 million is expected to be recorded in the third quarter.

· The Company closed a public offering of its common stock in August 2024, resulting in net proceeds of approximately $31.4 million.

Second Quarter 2024 Financial Results Compared to Prior Year Period

Research and development expenses increased $3.8 million, or 32%, compared to the three months ended June 30, 2023. Salaries, benefits, and other personnel costs increased $2.1 million primarily due to severance charges related to the shutdown of the Company’s ActoBio subsidiary. Additionally, fees paid to contract research organizations related to the start of the PRGN-2012 confirmatory clinical trial and close out of the PRGN-2012 pivotal clinical trial activities and professional fees incurred related to our manufacturing facility readiness for anticipated BLA submission increased compared to the same period in 2023.

SG&A expenses increased by $1.0 million, or 11%, compared to the three months ended June 30, 2023. This increase was primarily driven by severance costs incurred related to the suspension of ActoBio operations of $0.4 million, increased costs associated with PRGN-2012 commercial readiness as well as increased professional fees incurred related to general corporate matters compared to the same period in 2023.

In conjunction with the suspension of ActoBio’s operations, the Company recorded $34.5 million of impairment charges related to goodwill and other noncurrent assets in the second quarter of 2024, as well as a related tax benefit of $1.7 million.

Total revenues decreased $1.1 million, or 59%, compared to the three months ended June 30, 2023. This decrease was related to reductions in product and service revenues at Exemplar.

Net loss was $58.8 million, or $(0.23) per basic and diluted share, compared to net loss of $20.3 million, or $(0.08) per basic and diluted share, in period ended June 30, 2023.

First Half 2024 Financial Results Compared to Prior Year Period

Research and development expenses increased $5.9 million, or 25%, compared to the six months ended June 30, 2023. Salaries, benefits, and other personnel costs increased by $3.3 million primarily due to $2.1 million of severance charges related to the shutdown of the Company’s ActoBio subsidiary and an increase in the hiring of employees related to the advancement of PRGN-2012 in 2023 at Precigen. Additionally, fees paid to contract research organizations related to the start of the PRGN-2012 confirmatory clinical trial and close out of the PRGN-2012 pivotal clinical trial activities and professional fees incurred related to our manufacturing facility readiness for anticipated BLA submission increased compared to the prior year period. These increases were offset by lower costs incurred at contract research organizations for other programs compared to the same period in 2023.

SG&A expenses decreased by $0.5 million, or 2%, compared to the six months ended June 30, 2023. This decrease was primarily due to lower stock compensation and insurance expenses in 2024 compared to the same period in 2023. These decreases were offset by severance costs incurred in the second quarter of 2024 related to the suspension of ActoBio’s operations, and increased costs related to PRGN-2012 commercial readiness compared to the same period in 2023.

In conjunction with the suspension of ActoBio’s operations, the Company recorded $34.5 million of impairment charges related to goodwill and other noncurrent assets in the second quarter of 2024, as well as a related tax benefit of $1.7 million.

Total revenues decreased $1.9 million, or 51%, compared to the six months ended June 30, 2023. This decrease was related to reductions in product and service revenues at Exemplar.

Net loss was $82.5 million, or $(0.33) per basic and diluted share, compared to net loss of $43.1 million, or $(0.18) per basic and diluted share, in period ended June 30, 2023.

On August 14, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported the ESMO (Free ESMO Whitepaper) Congress decided to move C4T’s previously accepted preliminary monotherapy Phase 1 abstract for CFT1946, a novel BiDAC degrader in mutant BRAF V600 solid tumors, to an oral presentation (Press release, C4 Therapeutics, AUG 14, 2024, View Source [SID1234645892]). This oral presentation session at the ESMO (Free ESMO Whitepaper) Congress 2024 is scheduled for Friday, September 13, 2024, at 4:00 pm to 5:30 pm CEST. Additionally, C4T announced it will host an investor webcast on Friday, September 13, 2024.

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Updated Details for ESMO (Free ESMO Whitepaper) Congress 2024 Presentation
Title: Preliminary Results from a Phase 1 Study of CFT1946, a Novel BiDAC Degrader in Mutant BRAF V600 Solid Tumors
Presentation Date and Time: Friday, September 13, 2024, 4:00 – 5:30 pm CEST
Final Publication Number: 612O
Presenter: Maria Vieito, M.D., Msc (Barcelona, Spain)

C4T Webcast for Analysts and Investors
C4T will host an investor webcast on Friday, September 13, 2024 to discuss the CFT1946 monotherapy data from ongoing CFT1946 Phase 1 trial in BRAF V600 solid tumors. The time of the webcast and access information will be shared closer to the webcast event.

On August 14, 2024 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that allowance for the Company’s "proenzyme composition" patent was received from the Canadian Intellectual Property Office (CIPO) (Press release, Propanc, AUG 14, 2024, View Source [SID1234645927]). The patent broadly captures both high dose and high ratio claims for future clinical doses of the company’s lead asset, PRP. This is the second Canadian patent either allowed or granted in this important North American jurisdiction. Currently, the Company’s intellectual property portfolio consists of 93 patents filed in major jurisdictions relating to the use of PRP against solid tumors.

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The proenzymes composition patent is an important part of the IP portfolio covering possible future clinical dosage ranges for PRP, as the Company advances to a Phase 1, First-In-Human (FIH) study in advanced cancer patients suffering from solid tumors. The patent has been granted in major jurisdictions such as Europe, Japan and South East Asia, and is currently under examination in the United States. PRP is targeting the global metastatic cancer treatment market, projected to be worth US$111.2 Billion by 2027, according to current analysis by Emergen Research.

"We continue to grow our intellectual property portfolio in key global jurisdictions," said Mr. James Nathanielsz, Propanc’s Chief Executive Officer. "Our lead asset, PRP, is a novel method to prevent and treat metastatic cancer from solid tumors, but without the severe, or even serious side effects normally associated with standard therapies. The proenzymes composition patent will cover future PRP clinical doses as a welcome addition to the treatment process, such as when resistant tumors could be pretreated by PRP, as a chemo-sensitizing agent. This practical application of our patent portfolio provides a strong indication of our commercial embodiment for future licensing partners, which we believe can potentially revolutionize the way we treat metastatic cancer from solid tumors. Today, metastatic cancer remains the main cause of patient death for sufferers. We look forward to updating our shareholders as we progress."

About PRP:

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas, administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include pancreatic, ovarian, kidney, breast, brain, prostate, colorectal, lung, liver, uterine, and skin cancers. Orphan Drug Designation status of PRP has been granted from the US Food and Drug Administration (FDA) for treatment of pancreatic cancer.

To view the Company’s "Mechanism of Action" video on the Company’s lead asset, PRP, please click on the following link: View Source

On August 14, 2024 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported financial results for the second quarter ended June 30, 2024 and other recent business developments (Press release, Celcuity, AUG 14, 2024, View Source [SID1234645893]).

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"We made significant strides advancing the clinical development of gedatolisib this quarter. Overall enrollment in VIKTORIA-1 remains robust and on-track relative to our previous projections," said Brian Sullivan, CEO and co-founder of Celcuity. "We also initiated efforts to launch VIKTORIA-2, a Phase 3 study to evaluate gedatolisib as a first-line treatment option for patients with HR+, HER2- advanced breast cancer."

"In our VIKTORIA-1 study, while overall enrollment is on track, the proportion of patients who have PIK3CA wild-type tumors, versus those with PIK3CA mutations, has recently shifted lower than our original estimates. As a result, we expect to reach the enrollment target for the PIK3CA wild-type cohort in the fourth quarter, rather than the third quarter, as we originally forecasted. In light of this, we expect topline data for the PIK3CA WT cohort to shift to sometime between late Q4 2024 and Q1 2025."

Second Quarter 2024 Business Highlights and Other Recent Developments

● The VIKTORIA-1 Phase 3 trial expects to provide topline data for the PIK3CA wild-type cohort in late Q4 2024 or Q1 2025 and for the PIK3CA mutant cohort in the first half of 2025.

○ VIKTORIA-1 is evaluating gedatolisib in combination with fulvestrant with and without palbociclib in adults with HR+, HER2- advanced breast cancer who have received prior treatment with a CDK4/6 inhibitor.
○ Enrollment of the PIK3CA wild-type cohort is more than 80% complete and expected to reach the enrollment target during Q4 2024. The PIK3CA wild-type cohort represents approximately 60% of the total patients enrolled to date in VIKTORIA-1.

● In May, the Company announced its plan to initiate VIKTORIA-2, a Phase 3 study to evaluate the efficacy and safety of gedatolisib in combination with fulvestrant plus a CDK4/6 inhibitor, either ribociclib or palbociclib, in comparison to fulvestrant plus a CDK4/6 inhibitor as a first-line treatment for patients with HR+/HER2- advanced breast cancer.

○ A safety run-in study to evaluate the safety of gedatolisib combined with ribociclib and fulvestrant will precede initiation of the Phase 3 portion of the study.
○ The Phase 3 portion of the study is expected to enroll approximately 638 patients at up to 200 sites across North America, Europe, Latin America, and Asia.
○ First patient enrollment is expected in the second quarter of 2025.

● During the quarter, the Company secured a combined total of $129 million in gross proceeds from equity and debt financings, which extended the cash runway for current clinical development program activities through 2026.

● The Phase 1b/2 trial, evaluating gedatolisib in combination with darolutamide for the treatment of patients with metastatic castration resistant prostate cancer (mCRPC), remains on track to report preliminary data in the first half of 2025.

○ Enrollment is ongoing and the trial is expected to enroll up to 54 patients with mCRPC whose disease progressed after treatment with an androgen receptor signaling inhibitor.

● Three manuscripts reporting clinical and nonclinical results for gedatolisib were published recently.

○ In April, The Lancet Oncology published results from the dose expansion groups of its Phase 1b study evaluating gedatolisib in combination with palbociclib and endocrine therapy in HR+/HER2- advanced breast cancer. The published manuscript is available online and on the publications section of Celcuity’s website.
○ In June, npj Breast Cancer published results of nonclinical studies showing gedatolisib’s superior potency and efficacy versus single-node PI3K/AKT/mTOR inhibitors in breast cancer models. The article is available online and on the publications section of Celcuity’s website.
○ In August, Molecular Oncology published results of nonclinical studies in prostate cancer models showing gedatolisib’s superior potency and efficacy versus single-node PI3K/AKT/mTOR inhibitors. The article is available online and will soon be available on the publications section of Celcuity’s website.

Second Quarter 2024 Financial Results

Unless otherwise stated, all comparisons are for the second quarter ended June 30, 2024, compared to the second quarter ended June 30, 2023.

Total operating expenses were $24.3 million for the second quarter of 2024, compared to $15.1 million for the second quarter of 2023.

Research and development (R&D) expenses were $22.5 million for the second quarter of 2024, compared to $13.8 million for the prior-year period. Of the approximately $8.7 million increase in R&D expenses, $6.6 million primarily related to activities supporting the VIKTORIA-1 Phase 3 trial and the initiation of the CELC-G-201 Phase 1b/2 clinical trial, and $2.1 million was related to increased employee and consulting expenses.

General and administrative (G&A) expenses were $1.8 million for the second quarter of 2024, compared to $1.3 million for the prior-year period. Employee and consulting related expenses accounted for $0.3 million of the increase. Professional fees and other administrative expenses accounted for the remaining increase of approximately $0.2 million.

Net loss for the second quarter of 2024 was $23.7 million, or $0.62 loss per share, compared to a net loss of $14.6 million, or $0.66 loss per share, for the second quarter of 2023. Non-GAAP adjusted net loss for the second quarter of 2024 was $22.2 million, or $0.58 loss per share, compared to non-GAAP adjusted net loss of $11.1 million, or $0.51 loss per share, for the second quarter of 2023. Non-GAAP adjusted net loss excludes stock-based compensation expense, non-cash interest expense, and non-cash interest income. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the second quarter of 2024 was $18.1 million, compared to $9.7 million for the second quarter of 2023.

At June 30, 2024, Celcuity reported cash, cash equivalents and short-term investments of $283.1 million.

Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the second quarter 2024 financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-800-717-1738 or 1-646-307-1865. A live webcast presentation can also be accessed using this weblink: View Source;tp_key=c55c86e8c3. A replay of the webcast will be available on the Celcuity website following the live event.

On August 14, 2024 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical stage company focused on developing next generation therapeutics to target difficult-to-treat cancers, reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, Pyxis Oncology, AUG 14, 2024, View Source [SID1234645928]). The Company ended the second quarter of 2024 with $157.2 million in cash, cash equivalents, restricted cash and short-term investments, which is expected to provide cash runway into the second half of 2026 and enable the Company to fund the next phase of PYX-201 clinical development, which the Company plans to announce in the fall of 2024.

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"I’m thrilled with our team’s continued operational and clinical execution prowess that keeps us on track to deliver preliminary data from our ongoing Phase 1 trial of PYX-201, a first-in-concept tumor stroma targeting antibody-drug conjugate (ADC) against the stromal Extradomain-B Fibronectin (EDB+FN) target, this fall," said Lara S. Sullivan, M.D., President and Chief Executive Officer of Pyxis Oncology.

Dr. Sullivan added, "To date, we have dosed 72 subjects in the PYX-201 dose escalation study with a continued focus on head and neck squamous cell carcinoma (HNSCC), non-small cell lung cancer (NSCLC), ovarian cancer, soft tissue sarcoma, and pancreatic ductal adenocarcinoma cancer (PDAC) based on an assessment of factors including immunohistochemistry target expression, stromal volume, unmet medical need, and clinical investigator judgment. PYX-201 safety data observed to date continues to support go-forward monotherapy and potential combination clinical development strategies, both of which we believe could have the potential to provide additional treatment options to patients with difficult-to treat-cancers."

Recent Clinical Program Updates

PYX-201

PYX-201, an ADC that uniquely targets EDB+FN within the tumor stroma, is the Company’s lead clinical program being evaluated in an ongoing Phase 1 trial in multiple types of solid tumors.

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To date, 72 subjects have been dosed with PYX-201 in this Phase 1 trial. Dose escalation and safety monitoring remain ongoing for the trial.

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The Company expects to announce preliminary data from the Phase 1 trial of PYX-201, including efficacy, safety, pharmacokinetics (PK), and provide an update on future development plans in the fall of 2024.

PYX-106

PYX-106, a fully human Siglec-15-targeting antibody designed to block suppression of T-cell proliferation and function, is being evaluated in ongoing Phase 1 clinical studies in multiple types of solid tumors.

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Dose escalation of PYX-106 and safety monitoring is ongoing with 33 subjects dosed to date in the Phase 1 trial.

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The Company expects to report preliminary data from the Phase 1 trial of PYX-106, including PK/pharmacodynamic results, by year-end 2024.

Second Quarter 2024 Financial Results

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As of June 30, 2024, Pyxis Oncology had cash and cash equivalents, including restricted cash and short-term investments of $157.2 million. The Company believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its operations into the second half of 2026, including the Company’s current projections for PYX-201’s next phase of clinical development.

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Research and development expenses were $13.9 million for the quarter ended June 30, 2024, compared to $11.4 million for the quarter ended June 30, 2023. The period-over-period increase was primarily due to increased clinical trial-related expenses, including manufacturing of drug product and drug substance for our ongoing Phase 1 clinical trials of PYX-201 and PYX-106.

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General and administrative expenses were $6.1 million for the quarter ended June 30, 2024, compared to $6.7 million for the quarter ended June 30, 2023. The period-over-period decline was primarily due to lower professional and consultant fees.

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Net loss was $17.3 million, or ($0.29) per common share, for the quarter ended June 30, 2024, compared to $15.9 million, or ($0.41) per common share, for the quarter ended June 30, 2023. Net losses for the quarters ended June 30, 2024 and 2023 included $2.9 million and $3.7 million, respectively, related to non-cash stock-based compensation expense.

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As of August 14, 2024, the outstanding number of shares of common stock of Pyxis Oncology was 58,942,243.