On November 29, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported that the independent Data Safety Monitoring Board (DSMB) of the Phase 3 ASPIRE clinical trial for patients with untreated metastatic pancreatic ductal adenocarcinoma has completed its pre-specified review of safety data for treated patients which included 214 patients in the trial (Press release, Panbela Therapeutics, NOV 29, 2023, View Source [SID1234638044]). The DSMB has recommended that the study continue without modification. The ASPIRE Trial is a global randomized, doubleblind placebo-controlled clinical trial to evaluate ivospemin in combination with gemcitabine and nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma. "We are pleased that no safety concerns were identified and the DSMB’s recommendation is to proceed without modification to the ASPIRE Trial. With a focus on enrollment and completing site initiations, we are looking forward to the interim analysis in mid-2024." said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela.

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About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention, ovarian cancer and diabetes. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101 Ivospemin

Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. For more information, please visit View Source

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increasing polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X Eflornithine

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigator-initiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.

On November 29, 2023 Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, reported that the US Patent and Trademark Office (USPTO) has issued patent US11/730,732 for KIZFIZO, the first pediatric and ready-to-use drinkable formulation of temozolomide (Press release, ORPHELIA Pharma, NOV 29, 2023, https://www.orphelia-pharma.eu/news/orphelia-pharma-secures-us-patent-covering-kizfizo-first-drinkable-formulation-of-temozolomide/?utm_source=rss&utm_medium=rss&utm_campaign=orphelia-pharma-secures-us-patent-covering-kizfizo-first-drinkable-formulation-of-temozolomide [SID1234638043]).

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KIZFIZO (temozolomide oral suspension, 40 mg/ml), known as Ped-TMZ or KIMOZO during its clinical development and ongoing early access programs, is specifically designed for use in the treatment of children with relapsed or refractory high-risk neuroblastoma, oncology indications with a very poor prognosis. This taste-masked oral suspension was developed for children: it allows a precise dose to be administered orally or via a nasogastric tube in a small volume. Orphelia Pharma has been developing KIZFIZO in collaboration with Gustave Roussy, the leading European cancer center, for the last six years.

"Following the issuance of the European patent in 2021, this grant of the US patent once again highlights the innovation in the formulation of KIZFIZO, which lies in the discovery of an essential excipient improving the stability and the rheological properties of the suspension," said Jeremy Bastid, chief development officer at Orphelia Pharma. "This US patent complements the European one already issued and the Orphan Drug Designation (ODD) obtained in the US and Europe for temozolomide in the treatment of neuroblastoma. Therefore, we benefit from a double exclusivity protection for KIZFIZO in these territories."

"There is a major hospital need for an oral formulation of temozolomide, but attempts to generate such liquid formulations have been unsatisfactory, with low concentrations leading to large administration volumes, short shelf-lives and erratic recrystallization," said Maxime Annereau, pharmacist at Gustave Roussy and co-inventor of the patent. "Our work, carried out jointly with the Orphelia Pharma team, led to the identification of a formulation with a high concentration of temozolomide while controlling its polymorphic transition process."

Jeremy Bastid added: "Temozolomide is an essential component in the treatment armamentarium of refractory or relapsed high-risk neuroblastoma, a disease that affects very young children. KIZFIZO addresses a major unmet medical need. We will now focus our efforts on making KIZFIZO, which is already accessible under the ongoing early access program, available quickly to all patients; we have recently submitted an application for marketing authorization in Europe."

The granted US (US11/730,732) and European (EP3613436) patents ‘oral suspension of temozolomide’ are co-owned by Orphelia Pharma and Gustave Roussy. Based on the filing date of the priority application, the patent protection is expected to last until 2038. This protection complements the ODDs already obtained in these territories.

About KIZFIZO 40 mg/ml
KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.
In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.
KIZFIZO has received an ODD from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.

On November 29, 2023 Ferring Pharmaceuticals reported the presentation of new 36-month follow-up data from the Phase 3 study at the 24th Annual Meeting of the Society of Urologic Oncology (SUO) demonstrating a sustained durability of response with ADSTILADRIN (nadofaragene firadenovec-vncg) in adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1) (Press release, Ferring, NOV 29, 2023, View Source [SID1234638042]). ADSTILADRIN is the first and only intravesical gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population.

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"These new data continue to build on the body of evidence supporting the efficacy and safety of ADSTILADRIN, adding to the data on its sustained durable response following treatment," said Elizabeth Garner, M.D., M.P.H., Chief Scientific Officer, Ferring Pharmaceuticals, U.S. "Patients with BCG-unresponsive NMIBC are at high risk for recurrence and disease progression, which can often lead to bladder removal surgery, or cystectomy. ADSTILADRIN is an innovative therapy that we believe will transform the current standard of care for these patients, providing an effective alternative therapy."

The new interim data are from a 36-month follow-up analysis from the Phase 3 study, which is ongoing with a planned five-year treatment and monitoring phase. From the outset, patients with NMIBC CIS±Ta/T1 received ADSTILADRIN 75 mL intravesical instillation (3 x 1011 vp) once every three months for up to 12 months (four doses) or until unacceptable toxicity or recurrent high-grade (HG) NMIBC. Patients without evidence of HG recurrence were allowed to continue ADSTILADRIN treatment every three months as part of an ongoing follow-up analysis. Among patients who continued treatment after the 12-month Phase 3 period, 25.5% (14/55 patients) remained HG recurrence-free at 36 months following the start of treatment. The Kaplan-Meier (KM)-estimated probability of duration of complete response (CR) for at least 12, 24 and 36 months was 46.5%, 36.6% and 34.2%, respectively.1

In the overall CIS±Ta/T1 cohort of participants (N=103), the KM-estimated median (95% confidence interval [CI]) duration of HG recurrence-free survival was six months (3.4, 8.3), with a 30.1% (21.55%, 39.2%) probability of (95% CI) of HG recurrence-free survival for at least 12 months. At 36 months, the KM-estimated cystectomy-free survival (95% CI) was 53.8% (43.3%, 63.1%), and the three-year overall survival was 90.4% (82.3%, 94.9%). Two patients (1.9%) discontinued ADSTILADRIN treatment due to adverse events, while four (3.9%) experienced progression to muscle-invasive disease.1

"The management of patients with continued high-grade NMIBC after treatment with BCG remains challenging," said Stephen A. Boorjian M.D., who is the Carl Rosen Professor and David and Anne Luther Chair of the Department of Urology at Mayo Clinic, and was the lead investigator on the ADSTILADRIN clinical trial presented at the conference. "These new ADSTILADRIN data emphasize the importance of obtaining long-term follow up from novel therapies to establish treatment expectations."

Abstract and Presentation Time:

Efficacy of Intravesical Nadofaragene Firadenovec for Patients with BCG-Unresponsive Carcinoma in Situ of the Bladder: 36-Month Follow-Up From a Phase 3 Trial

Poster #164, Thursday, Nov. 30 from 4:15 – 5:15 p.m. EST

About ADSTILADRIN
ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high quantities of interferon alfa-2b protein, a naturally-occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).2

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.3 Bladder cancer is the sixth most common cancer in the U.S., and it is estimated that there were approximately 81,180 new cases of bladder cancer in the U.S. in 2022,4 75% of which present as NMIBC.5 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard of care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.3 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).6

INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

On November 29, 2023 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported that members of the management team will participate in a virtual fireside chat at the JMP Securities Hematology and Oncology Summit on Wednesday, December 6, 2023 at 1:30 PM ET (Press release, Deciphera Pharmaceuticals, NOV 29, 2023, View Source [SID1234638041]).

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A live webcast of the fireside chat will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On November 29, 2023 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported two poster presentations at the upcoming 2023 San Antonio Breast Cancer Symposium (SABCS), taking place December 5-9, 2023 at the Henry B. González Convention Center in San Antonio, Texas (Press release, Cogent Biosciences, NOV 29, 2023, View Source [SID1234638040]).

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Poster Details
The posters will be available on the ‘Posters and Publications’ page of Cogent’s website when they are presented.

Presentation ID: PO3-26-02
Title: Identification of a novel, brain penetrant, EGFR sparing, ErbB2 inhibitor with activity against oncogenic ErbB2 mutations
Session: Poster Session 3
Date: Thursday, December 7, 2023
Time: 12:00 PM – 2:00 PM CT (1:00 PM – 3:00 PM ET)

Presentation ID: PO3-26-01
Title: Preclinical in vitro and in vivo characterization of a novel, wild-type-sparing, PI3Kα H1047R mutant-selective inhibitor
Session: Poster Session 3
Date: Thursday, December 7, 2023
Time: 12:00 PM – 2:00 PM CT (1:00 PM – 3:00 PM ET)