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LPCN Awaits FDA Word, NKTR On Track, All's Well With Five Prime's FPT155

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Today's Daily Dose brings you news about Alnylam's new positive efficacy results from its ongoing phase II open-label extension (OLE) study of Lumasiran for the treatment of primary hyperoxaluria type; Five Prime's initial safety data from its phase I clinical trial of FPT155 in patients with advanced solid tumors; Lipocine's much-awaited catalyst; Mirati's initial efficacy data from a phase II trial of Sitravatinib in metastatic urothelial cancer patients and Nektar's 18-month follow-up data from its PIVOT-02 study.

Read on…

Alnylam Pharmaceuticals Inc. (ALNY) announced new positive efficacy results from its ongoing phase II open-label extension (OLE) study of Lumasiran for the treatment of primary hyperoxaluria type 1.

Primary hyperoxaluria type 1 (PH1) is a rare disorder that mainly affects the kidneys. It results from the buildup of a substance called oxalate, which normally is filtered through the kidneys and excreted in the urine (Source: NIH).

The open-label extension phase II study of Lumasiran demonstrated a 76 percent mean maximal reduction in urinary oxalate excretion, with all patients achieving a urinary oxalate level at or below 1.5 times the upper limit of normal, and 68 percent of patients achieving a urinary oxalate level within the normal range (less than or equal to 0.46 mmol/24hr/1.73m2).

The Company has also initiated a new global phase III study of Lumasiran for the Treatment of advanced Primary Hyperoxaluria Type 1, dubbed ILLUMINATE-C. The primary study endpoint is set as the percent reduction in plasma oxalate from baseline to six months.

Alnylam expects to report initial results from the ILLUMINATE-C trial in late 2020.

ALNY closed Friday's trading at $93.03, up 1.63%.

Five Prime Therapeutics Inc. (FPRX) has said that initial safety data from its phase I clinical trial of FPT155 in patients with advanced solid tumors suggest that it may not cause the same safety issues seen with a prior molecule targeting CD28.

FPT155 is a novel therapeutic CD80-Fc fusion protein that directly engages CD28 to enhance its co-stimulatory T-cell activation activity without inducing super agonism.

In 2006, in a phase I trial of TGN1412, a compound that targets CD28, 6 healthy volunteers had experienced serious toxicity including multi-organ failure. The trial later came to be referred to as the "elephant man drug trial" because of the disastrous side effects like severe swelling of the head, the trial participants had suffered.

Five Prime noted that FPT155 was well tolerated at all dose levels, with no dose-limiting toxicities and no grade 4 or higher adverse events. Dose escalation with FPT155 is ongoing with the study currently enrolling patients at the 70 mg dose level.

FPRX closed Friday's trading at $3.89, unchanged from the previous day's close.

Lipocine Inc.'s (LPCN) testosterone replacement therapy TLANDO is still awaiting word from the FDA regarding approval. The regulatory agency's decision on TLANDO was on November 9, 2019.

TLANDO, formerly known as LPCN 1021, is a twice-a-day oral testosterone product candidate that is designed to help restore normal testosterone levels in hypogonadal men.

The FDA had turned down TLANDO in June 2016, citing deficiencies related to the dosing algorithm for the proposed label, and in May 2018, saying that the product could not be approved in its current form.

It remains to be seen if the third time will prove lucky for TLANDO.

LPCN closed Friday's trading at $2.73, down 2.50%.

Mirati Therapeutics Inc.'s (MRTX) initial efficacy data from a phase II trial of Sitravatinib in combination with OPDIVO in metastatic urothelial cancer patients with documented progression on a platinum-chemotherapy and checkpoint inhibitor, presented on Saturday, has shown that the compound has the potential to increase responsiveness in patients whose tumors are resistant to checkpoint inhibitors.

According to the Company, out of the 22 metastatic urothelial cancer patients who were evaluable for response with at least one radiographic scan, 1 patient achieved a confirmed Complete Response and 6 archived Partial Response. Twenty-one of the twenty-two evaluable patients achieved a Complete Response, Partial Response or stable disease.

The preliminary results from an ongoing phase I study of neoadjuvant Sitravatinib combined with OPDIVO in patients with resectable squamous cell carcinoma of the oral cavity, dubbed SNOW, showed that there was tumor reduction in all eight patients who were eligible for evaluation, including one complete pathological response. With a median follow-up of 31.4 weeks, all patients are alive with no disease recurrence to date, noted the Company.

MRTX closed Friday's trading at $104.78, up 6.92%.

Nektar Therapeutics (NKTR), on Saturday, presented 18-month follow-up data from a phase I/II study of Bempegaldesleukin in combination with Opdivo in previously untreated metastatic melanoma patients, dubbed PIVOT-02.

At a median time of follow-up of 18.6 months for 38 efficacy-evaluable patients, the confirmed objective response rate (ORR) was 53%, with 34% of patients achieving confirmed complete responses. Forty-two percent of patients achieved a maximum reduction of 100% in target lesions and DCR, also known as disease control rate (CR+ Partial Response + Stable Disease), was 74%.

The median percent reduction of target lesions from baseline was 61.5%. At a median time of follow-up of 18.6 months, the median duration of response has not been reached, with 85% of patients with responses having ongoing responses, noted the Company.

A phase III trial evaluating Bempegaldesleukin in combination with Opdivo versus Opdivo in first-line advanced melanoma patients is currently recruiting patients.

NKTR closed Friday's trading at $20.04, up 7.17%.

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