Peptomyc announces the approval of its Phase 1b trial testing OMO-103 in combination with standard of care in PDAC patients

On July 27, 2023 Peptomyc S.L., a biotech company specialized in the development of protein therapeutics for cancer treatment, reported that it has received full study approval for its new Phase 1b clinical trial, which will evaluate the combination of the first-in-class MYC inhibitor, OMO103, together with the standard of care (SoC) regimen Gemcitabine and Nab-Paclitaxel in metastatic Pancreatic Ductal Adenocarcinoma (PDAC) patients in first line (Press release, Peptomyc, JUL 27, 2023, View Source [SID1234633483]). PDAC is the most common form of pancreatic cancer. Currently, it is the fourth highest cause of cancer mortality worldwide and its incidence is rising steeply. Current therapies offer limited chance of a lasting cure and the five-year survival rate is one of the lowest among the most commonly occurring cancers.

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MYC is an oncoprotein deregulated in most –if not all- types of cancers. It is activated in many PDAC cases, where it contributes to aggressiveness of the disease and resistance to treatments. The company recently completed a FIH Phase 1 study of OMO103 in all-comers solid tumor patients, demonstrating excellent safety and promising anti-tumor activity.

Manuela Niewel, MD, PhD, Chief Medical Officer of the company, leading Peptomyc’s clinical development, regulatory and medical affairs activities, says: "I am excited to be able to start our new Phase 1b study in PDAC patients. With OMO-103, we hope to address this devastating disease and one of the highest unmet medical needs in the oncology field".

This Phase 1b combination study will be conducted in four sites in Spain. The lead PI will be Dr. Teresa Macarulla at the Vall d’Hebron Institute of Oncology in Barcelona, and she will collaborate with Dr. Andres Muñoz at the Hospital Gregorio Marañon in Madrid, Dr. Mariona Calvo at the ICO-Hospitalet in Barcelona, and Dr. Roberto Pazo at the Hospital Miguel Servet in Zaragoza. The study is planned to start in Q3/2023.

Takeda Reports Strong First Quarter FY2023 Results, Driven by Growth & Launch Products

On July 27, 2023 Takeda (TOKYO:4502/NYSE:TAK) reported strong financial results for the first quarter of fiscal year 2023 (period ended June 30, 2023) (Press release, Takeda, JUL 27, 2023, View Source [SID1234633475]).

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Takeda chief financial officer, Costa Saroukos, commented:
"Our Growth & Launch Products continued to drive revenue growth in the first quarter of FY2023, contributing to a core operating profit margin of 30.8%. While we still anticipate headwinds affecting our business this fiscal year, largely due to generic competition, we remain confident of a return to growth in the near-term.

"Our growing product portfolio and innovative pipeline demonstrate the value of our strategic investments to strengthen our long-term competitiveness and enable our vision of discovering and delivering life-transforming treatments."

FINANCIAL HIGHLIGHTS

Results for FY2023 Q1 Ended June 30, 2023

(Billion yen,
except
percentages and
per share amounts)

REPORTED

CORE(c)

(Non-IFRS)(a)

FY2023 Q1

vs. PRIOR YEAR

(AER % change(d))

FY2023 Q1

vs. PRIOR YEAR

(AER % change(d))

vs. PRIOR YEAR

(CER % change(d))

Revenue

1,058.6

+8.9%

1,058.6

+8.9%

+3.7%

Operating Profit

168.6

+12.0%

326.3

+2.3%

-2.0%

Margin

15.9%

+0.4pp

30.8%

-2.0pp

Net Profit

89.4

-14.9%

233.4

+4.1%

+0.9%

EPS (yen)

58

-15.4%

150

+3.5%

+0.3%

Operating Cash Flow

92.4

+9.7%

Free Cash Flow

(Non-IFRS)(a)(b)

-207.5

N/A

(a) Further information regarding certain of Takeda’s Non-IFRS measures is posted on Takeda’s investor relations website at View Source

(b) We define Free Cash Flow as cash flows from operating activities, subtracting acquisition of property, plant and equipment ("PP&E"), intangible assets and investments as well as removing any other cash that is not available to Takeda’s immediate or general business use, and adding proceeds from sales of PP&E, as well as from sales of investments and businesses, net of cash and cash equivalents divested.

(c) Core results adjust our reported results calculated and presented pursuant to IFRS to exclude the effect of items unrelated to Takeda’s core operations, such as, to the extent applicable for each line item, non-recurring items, purchase accounting effects and transaction related costs, as well as amortization and impairment of intangible assets and other operating income and expenses.

(d) Actual Exchange Rate is presented in "AER" (which is presented in accordance with IFRS). Constant Exchange Rate (CER) change eliminates the effect of foreign exchange rates from year-over-year comparisons by translating Reported or Core results for the current period using corresponding exchange rates in the same period of the previous fiscal year.

FY2023 Outlook (unchanged from May 2023)

(Billion yen)

FY2023
FORECAST

FY2023
MANAGEMENT GUIDANCE
Core Change at CER
(Non-IFRS)

Revenue

3,840.0

Core Revenue

3,840.0

Low-single-digit % decline

Reported Operating Profit

349.0

Core Operating Profit

1,015.0

Low-10s % decline

Reported Net Profit

142.0

Reported EPS (Yen)

91

Core EPS (Yen)

434

Low-20s % decline

Free Cash Flow*

400.0 – 500.0

Annual Dividend per Share (Yen)

188

*Free Cash Flow guidance reflects expenditures related to the acquisition of TAK-279 from Nimbus (USD 1.0 billion) and in-licensing of fruquintinib from HUTCHMED (USD 400 million). In FY2022, Takeda paid USD 3.0 billion out of USD 4.0 billion upfront payment related to the acquisition of TAK-279. For the remaining USD 1.0 billion payment, Takeda paid USD 0.9 billion in April 2023, with USD 0.1 billion to be paid in August 2023.

Additional Information About Takeda’s Q1 Earnings Results
For more details on Takeda’s FY2023 Q1 results and other financial information, including key assumptions in FY2023 forecast and management guidance, please visit: View Source
For more information on Takeda’s commercial progress across the five key business areas and pipeline updates, please visit: View Source

Novocure Reports Second Quarter 2023 Financial Results

On July 27, 2023 Novocure (NASDAQ: NVCR) reported financial results for the quarter ended June 30, 2023 (Press release, NovoCure, JUL 27, 2023, View Source [SID1234633474]). Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields (TTFields).

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"With three more phase 3 trials set to read out by the end of 2024 and a new generation of trials slated to launch, our determination and commitment are strengthened by the prospect of potentially treating many more patients across a number of new indications in the coming years."

"The second quarter was a period of sound execution and expansion at Novocure," said Asaf Danziger, Novocure’s Chief Executive Officer. "Our restructured commercial organization has begun driving greater penetration in key markets, the launch of Optune in France has been a resounding success, and we are preparing to introduce our next generation arrays in more markets later this year. We believe there are many more patients who can benefit from TTFields therapy and we are determined to reach them."

"The positive results from the LUNAR trial in non-small cell lung cancer mark the beginning of the next chapter at Novocure, as we strive to extend survival for patients diagnosed with difficult-to-treat tumors," said William Doyle, Novocure’s Executive Chairman. "With three more phase 3 trials set to read out by the end of 2024 and a new generation of trials slated to launch, our determination and commitment are strengthened by the prospect of potentially treating many more patients across a number of new indications in the coming years."

Financial updates for the second quarter ended June 30, 2023:

Total net revenues for the quarter were $126.1 million, a decrease of 11% compared to the same period in 2022. The decrease resulted primarily from $13.4 million in reduced collections from previously denied or appealed claims in the U.S.
The United States, Germany and Japan contributed $87.0 million, $15.7 million and $7.9 million in quarterly net revenues, respectively, with our other active markets contributing $8.7 million.
Revenue in Greater China from Novocure’s partnership with Zai Lab totaled $6.8 million.
Gross margin for the quarter was 73%.
Research, development and clinical studies expenses for the quarter were $55.4 million, a decrease of 3% from the same period in 2022. This primarily reflects reduced costs associated with recently completed trials in the quarter. Total clinical trial expenses can fluctuate quarter-to-quarter dependent upon the amount of clinical research organization services delivered, clinical materials procured and number of trials actively underway. As our current phase 3 clinical trials near completion, we expect to backfill our clinical trial pipeline with new phase 2 and 3 trials.
Sales and marketing expenses for the quarter were $58.5 million, an increase of 31% compared to the same period in 2022. This increase reflects increased investments associated with geographic expansion and pre-launch activities intended to increase awareness of TTFields therapy in anticipation of future approvals in new indications.
General and administrative expenses for the quarter were $40.8 million, an increase of 29% compared to the same period in 2022. This reflects increased personnel and project costs to support larger patient populations, new geographic launches, supply chain expansion and information technology enhancements.
Net loss for the quarter was $57.4 million with loss per share of $0.54.
Adjusted EBITDA* for the quarter was $(27.2) million.
Cash, cash equivalents and short-term investments were $940.8 million as of June 30, 2023.
Operational updates for the second quarter ended June 30, 2023:

1,556 prescriptions were received in the quarter, an increase of 13% compared to the same period in 2022. Prescriptions from the United States, Germany and Japan contributed 981, 204 and 92 prescriptions, respectively, with our other active markets contributing 279 prescriptions.
As of June 30, 2023, there were 3,571 active patients on therapy. Active patients from the United States, Germany and Japan contributed 2,200, 499 and 352 active patients, respectively, with the remaining 520 active patients contributed by our other active markets.
Quarterly updates and achievements:

In June, we presented positive results from the phase 3 LUNAR trial evaluating the use of TTFields therapy together with standard therapies for the treatment of metastatic non-small cell lung cancer (NSCLC) following platinum-failure. The LUNAR trial met its primary endpoint with a statistically significant and clinically meaningful 3-month improvement in median overall survival (OS) with TTFields therapy added to standard therapies (HR=0.74, P=0.035). Patients randomized to receive TTFields therapy together with standard therapies demonstrated median OS of 13.2 months compared to 9.9 months in patients treated with standard therapies alone. Patients randomized to receive TTFields therapy and physician’s choice immune checkpoint inhibitor (ICI) demonstrated a median OS of 18.5 months, a profound extension compared to the median OS of 10.8 months demonstrated by patients that received ICI alone (HR=0.63; P=0.03). Patients randomized to receive TTFields therapy and docetaxel had a positive survival trend with a median OS of 11.1 months vs 8.7 months. TTFields therapy was well-tolerated with no added systemic toxicities and few grade 3 (no grade 4 or 5) device-related adverse events. These data are expected to serve as the basis for a PMA submission to the FDA in the second half of 2023.
In July, we announced that an independent data monitoring committee (DMC) reviewed the safety and efficacy data for all patients in the fully enrolled PANOVA-3 clinical trial. The interim analysis resulted in a DMC recommendation that the clinical trial proceed to final analysis. The PANOVA-3 study accrued 556 patients as of February 2023 and data will be reviewed in 2024, following an 18-month follow-up period.
In July, the U.S. Food and Drug Administration accepted the investigation device exemption for the LUNAR-2 clinical trial, a randomized, phase 3 study testing the safety and effectiveness of TTFields therapy concomitant with pembrolizumab and platinum-based chemotherapy in patients with metastatic NSCLC. The two primary endpoints of LUNAR-2 are overall survival and progression-free survival. LUNAR-2 is designed to accrue 734 patients with a 21-month follow-up following the enrollment of the last patient.
Anticipated clinical milestones:

Data from phase 3 INNOVATE-3 clinical trial in recurrent ovarian cancer (2H 2023)
Top-line data from phase 3 METIS clinical trial in brain metastases (Q1 2024)
Data from phase 3 PANOVA-3 clinical trial in locally advanced pancreatic cancer (2H 2024)
Conference call details

Novocure will host a conference call and webcast to discuss second quarter 2023 financial results at 8 a.m. EDT today, Thursday, July 27, 2023. To access the conference call by phone, use the following conference call registration link and dial-in details will be provided. To access the webcast, use the following webcast registration link.

The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

Synthekine Doses First Patient in Phase 1 Clinical Trial of Orthogonal IL-2 and CD19 CAR-T Combination Therapy, STK-009 + SYNCAR-001, for Treatment of CD19+ Hematologic Malignancies

On July 27, 2023 Synthekine Inc., a leader in engineered cytokine therapeutics, reported that the first patient has been dosed in a Phase 1 clinical trial of its orthogonal IL-2 and CD19 CAR-T combination therapy, STK-009 + SYNCAR-001, in adults with relapsed or refractory CD19+ hematologic malignancies (Press release, Synthekine, JUL 27, 2023, View Source [SID1234633473]). STK-009 + SYNCAR-001 is a two-component therapy consisting of SYNCAR-001, a CD19-targeting chimeric antigen receptor T cell (CAR-T) which expresses an engineered IL-2 receptor allowing it to selectively receive a signal from STK-009, an engineered pegylated IL-2 cytokine.

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"SYNCAR-001 + STK-009 is our second clinical stage program at Synthekine and represents another key milestone in our journey to develop novel cytokine therapeutics for patients with refractory cancers," said Debanjan Ray, chief executive officer of Synthekine. "Our orthogonal IL-2 system is a unique platform that breaks new ground in the field of cell therapy. Combining STK-009 with SYNCAR-001 has the potential to overcome key limitations associated with current CD19 CAR-T cell therapies, such as poor expansion and limited persistence following T cell transfer. These limitations have been shown to correlate with poor response and relapse, particularly in challenging hematological malignancies. We look forward to further evaluating STK-009’s ability to offer a safer approach with improved efficacy and durability."

Published results from preclinical studies show that treatment with STK-009 resulted in both systemic and intratumoral expansion and activation of SYNCAR-001 cells. As a result, even with substantially reduced cell doses, SYNCAR-001 together with STK-009 treatment was able to drive complete responses in large lymphoma tumors in preclinical models.

"While CAR-T cell therapies have had a major impact for patients with CD19+ hematologic malignancies, only a subset of these patients have durable benefit," said Naiyer Rizvi, M.D., chief medical officer of Synthekine. "We believe that controllable, selective and sustained cytokine support is central to the advancement of this class of treatment, and STK-009 + SYNCAR-001 has the potential to drive deeper and more durable responses while avoiding serious toxicities related to uncontrolled CAR-T cell expansion for patients in need of better treatment options."

The first-in-human Phase 1 clinical trial of SYNCAR-001 + STK-009 is an open-label, multi-center study enrolling patients with CD19+ hematologic malignancies. For additional information about the trial, please visit www.clinicaltrials.gov using the identifier NCT05665062.

Novel Chimeric Antigen Receptor Design Technology for CAR-T Cells Published and Licensed Exclusively to Cellinfinity Bio

On July 27, 2023 Cellinfinity Bio, a privately held biotechnology company developing first/best-in-class cell therapy products against solid tumors and other diseases, reported the publication of a novel CAR enhancement, the addition of CTLA-4 intracellular domain to improve anti-tumor CAR-T activity (Press release, Cellinfinity Bio, JUL 27, 2023, View Source [SID1234633472]). This technology is published in Nature Immunology on July 27. Trogocytosis is a process where cancer antigens are transferred onto immune cells, including T-cells; if the cancer antigen is a target of CAR-Ts, this results in CAR-Ts killing each other or fratricide. The addition of intracellular domain of CTLA-4 prevented trogocytosis and resulted in improved CAR-T survival, including maintenance of central memory T-cells and increased T-cell persistence – all leading to stronger anti-tumor efficacy. Cellinfinity Bio has an exclusive license of the published CTLA-4 tail technology from Yale University and Professor Sidi Chen’s lab, Founder of Cellinfinity Bio and Associate Professor of Genetics, Yale University.

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"Trogocytosis and fratricide substantially limit current CAR-T cell therapy response. We found a portion of CTLA-4 intracellular domain when fused onto CAR sequence optimizes CAR-T function and results in low trogocytosis, greater durability, and more potent in vitro and in vivo anti-tumor efficacy," said Sidi Chen. "Furthermore, the simple addition of CTLA-4 tail onto CARs has potential to improve CAR-Ts against diverse targets."

"Cellinfinity has identified several important gene enhancements and CAR modifications that improve anti-tumor activity of adoptive cell therapy," added Premal Patel, M.D., Ph.D., Chief Executive Officer, Cellinfinity Bio. "We intend to evaluate this CTLA-4 tail technology across diverse next generation of CAR-Ts targeting variety of cancers." Cellinfinity holds exclusive rights to the CTLA-4 tail technology, as well as exclusive license to other novel cell evolution technologies from the Chen lab at Yale University, that allow whole-genome gain-of-function and loss-of-function genetic screens.