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XLRN On Watch, BPMC Makes An Imprint, Osteoporosis Drug Evenity Faces FDA Panel

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Today's Daily Dose brings you news about miRagen's phase I trial results of Cobomarsen in mycosis fungoides patients; MEDALIST trial results; Apellis Pharma's paroxysmal nocturnal hemoglobinuria study results; Blueprint Medicines' EXPLORER trial results, and Celyad's progress in phase I trial of non-gene edited allogeneic CAR-T therapy.

Read on...

Amgen (AMGN) and UCB's Biologics License Application for EVENITY for the treatment of osteoporosis in postmenopausal women at high risk for fracture is slated to be reviewed by an FDA panel on January 16, 2019.

EVENITY was turned down by the FDA last July, with the companies being asked to integrate the efficacy and safety data from ARCH study into the Biologics License Application.

The ARCH study is a phase III trial, which compared Evenity to Merck & Co's Fosamax. The trial met both primary and secondary endpoints. However, the patient incidence of positively adjudicated cardiovascular serious adverse events at 12 months was 2.5 percent in the EVENITY group compared to 1.9 percent in the Fosamax group.

The companies resubmitted the EVENITY BLA in July of this year.

AMGN closed Friday's trading at $208.25, up 2.91%.

Apellis Pharmaceuticals Inc. (APLS) has reported encouraging interim data from its phase Ib study of APL-2 in treatment-naïve patients with paroxysmal nocturnal hemoglobinuria.

The ongoing phase Ib study, dubbed PADDOCK, is evaluating 270mg subcutaneous APL-2 administered daily. The interim data involves data from 19 patients at baseline, 15 patients on therapy at day 85 and 10 patients at day 169.

In the PADDOCK study, treatment with APL-2 in Soliris-naïve patients with paroxysmal nocturnal hemoglobinuria resulted in broad control of hemolysis and normalization of mean hemoglobin to 12.2 g/dL by day 85, an increase of 4.2 g/dL from baseline. Soliris is indicted for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.

In the study, previously transfusion-dependent patients did not require any transfusions during maintenance treatment with APL-2. Rapid and durable normalization of Lactate Dehydrogenase, Reticulocyte Count and Total Bilirubin was also achieved.

APLS closed Friday's trading at $15.34, up 1.39%.

Blueprint Medicines Corp. (BPMC) says that new data from its ongoing phase I study of Avapritinib in patients with systemic mastocytosis shows a significant decrease in symptom burden in patients.

Systemic mastocytosis results from the abnormal proliferation and survival of mast cells, which mediate allergic responses.

In the ongoing study, dubbed EXPLORER, the updated results showed an overall response rate (ORR) of 83 percent. Twenty-four percent of patients had a complete response with a full or partial recovery of peripheral blood counts (CR/CRh). Responses deepened over time, with a median time to initial response of two months and a median time to CR/CRh of nine months. The median duration of response (DoR) was not yet reached, and the 12-month DoR rate was 76 percent, added the Company.

At baseline, 22 systemic mastocytosis patients received steroids for mastocytosis symptoms. As of the data cutoff date, 18 patients (80 percent) decreased their steroid dose, including nine patients (41 percent) who were able to entirely discontinue their steroids, noted the Company.

Avapritinib is also under a phase II study in patients with advanced systemic mastocytosis, dubbed PATHFINDER.

Blueprint Medicines expects to initiate a phase II trial of Avapritinib in patients with indolent and smoldering systemic mastocytosis, by the end of 2018.

BPMC closed Friday's trading at $57.37, up 1.15%.

Celgene Corporation (CELG) and Acceleron Pharma Inc.'s (XLRN) phase III trial of Luspatercept in patients with Myelodysplastic Syndromes, dubbed MEDALIST, has demonstrated the potential clinical benefit of the compound in achieving red blood cell transfusion independence.

The MEDALIST trial evaluated the efficacy and safety of Luspatercept to treat patients with ring sideroblast (RS+) myelodysplastic syndromes (MDS)-associated anemia who require red blood cell transfusions and who had failed, were intolerant to, or ineligible for erythropoietin therapy.

The study met the primary endpoint of red blood cell transfusion independence (RBC-TI) for 8 or more weeks during the first 24 weeks of the study. Treatment with Luspatercept resulted in a statistically significantly greater proportion of patients achieving RBC-TI more than or equal to 8 weeks compared to placebo.

The study also found in secondary endpoints that treatment with Luspatercept resulted in a statistically significant higher percentage of patients achieving RBC-TI of 12 or more weeks in the first 24 or 48 weeks of the study, as well as hematologic improvement-erythroid (HI-E) of 8 or more weeks, according to the Company.

In the study, treatment-emergent adverse events (TEAEs) of Grade 3 or 4 were reported in 42.5% of patients receiving Luspatercept and 44.7% of patients receiving placebo. Five patients receiving Luspatercept (3.3%) and four patients receiving placebo (5.3%) experienced one or more treatment-emergent adverse events that resulted in death.

The companies are planning to file for regulatory approval of Luspatercept in the United States and Europe in the first half of 2019.

XLRN closed Friday's trading at $52.93, down 0.06%.

Celyad (CYAD) has dosed the first patient in its phase I trial of non-gene edited allogeneic CAR-T therapy, CYAD-101, administered concurrently with FOLFOX chemotherapy in the treatment of patients with unresectable metastatic colorectal cancer.

In the trial, dubbed alloSHRINK, patients will receive six cycles of FOLFOX chemotherapy every two weeks and three administrations of CYAD-101 every two weeks 48 hours after the initiation of chemotherapy cycles one, two and three. The three dose levels to be evaluated are 100 million, 300 million and 1 billion cells per injection, respectively.

The Company expects topline data from alloSHRINK trial in second half of 2019.

CYAD closed Friday's trading at $27.35, down 2.32%.

Coherus BioSciences Inc. (CHRS) has executed a 340B prime vendor program contract agreement with Apexus LLC., of which UDENYCA is a part.

The 340B Program enables hospitals, community health centers, clinics and other safety net providers to purchase outpatient pharmaceuticals at discounted pricing, thereby expanding access to care to low-income and vulnerable segments of the population. (Source: Apexus).

Coherus' UDENYCA is a biosimilar of Amgen's (AMGN) blockbuster Neulasta for treating side effects from chemotherapy.

In addition to entering into a UDENYCA contract agreement with Apexus, Coherus BioSciences has received Q-Code medical billing status for UDENYCA from the Centers for Medicare and Medicaid Services (CMS), which will be effective January 1, 2019. The Q-Code enables broad and expansive access to patients and providers.

CHRS closed Friday's trading at $11.06, up 2.69%.

miRagen Therapeutics Inc. (MGEN) says that its phase I clinical trial of Cobomarsen in mycosis fungoides patients continue to show that the compound is safe and well-tolerated, has relevant biological and clinical activity in patients and has the potential to impact the quality of their life.

Mycosis fungoides, or MF, is the most common form of cutaneous T-cell lymphoma.

According to the trial data, 92% of the mycosis fungoides subjects in the systemic administration cohorts had improvement in tumor burden as assessed by modified Severity Weighted Assessment Tool (mSWAT) score independent of concomitant therapies, and 52% of patients receiving more than six doses achieved a partial response (at least a 50% reduction) in mSWAT score; with 69% of these patients who achieved a partial response maintaining the response for at least four consecutive months.

Cobomarsen has been found to be safe and well-tolerated in the study.

Commenting on the study findings, William Marshall, President and CEO of miRagen Therapeutics, said, "The data from the Phase 1 clinical trial drove our decision to advance the investigation of cobomarsen in the Phase 2 SOLAR clinical trial for patients with MF. Based on our discussions with the U.S. Food and Drug Administration, we believe the results generated in the SOLAR trial could allow us to seek accelerated approval for cobomarsen in the United States."

MGEN closed Friday's trading at $3.48, down 1.14%.

Also of note are the trial results reported by SYRS,IMDZ,KURA, BLCM and CBIO.

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