Miragen Therapeutics shares soar on leukemia data

Miragen Therapeutics announces data from Phase 1 trial of cobomarsen

Miragen Therapeutics (MGEN) announced new efficacy and safety data from the ATLL arm of its ongoing Phase 1 clinical trial of cobomarsen, which will be presented at the 12th Annual T-Cell Lymphoma Forum in La Jolla, CA, which is taking place from January 30th to February 1st.

Miragen shares soar on its test data, Stockwinners

Of note are the data from the subtype of aggressive ATLL patients (Adult T-Cell Leukemia Lymphoma (ATLL)) who at study entry had persistent residual disease after chemotherapy or other therapy.

In the trial, six patients of this subtype had an MST of 26 months with three patients still on treatment at the time of data analysis.

Adult T-Cell Leukemia Lymphoma does not have a cure, Stockwinners

The Company was most encouraged, however, by the observed biomarker activity showing that disease stabilization is marked by a decrease in biomarkers of tumor cells activation and proliferation, providing evidence of the biological mechanism effect of cobomarsen on disease stabilization.

The Company is evaluating cobomarsen in an ongoing Phase 1 basket trial of cancers where the disease process appears to be correlated with an increase in miR-155 levels, including ATLL, diffuse large B-cell lymphoma, and chronic lymphocytic leukemia.

Today’s announcement includes results for the first 15 patients with aggressive subtypes of ATLL who were treated with three doses of cobomarsen by intravenous infusion, including 600, 900 and 1200 mg doses.

The preliminary results from this first-in-human Phase 1 clinical trial of the miR-155 inhibitor, cobomarsen, in patients with ATLL was observed to improve disease stabilization and reduce biomarker expression associated with ATLL cellular proliferation and activation in patients with persistent residual disease after chemotherapy and other therapies.

Of the 15 ATLL patients treated with cobomarsen, nine were actively relapsing at the time of screening, and six had residual nodal or circulating leukemic disease after chemotherapy or other systemic therapies.

For these six patients, the duration of cobomarsen treatment ranged from 4.5 to 23.7 months, with three patients still on study as of October 17, 2019.

MST of these patients was 26 months, compared with the 7.4 months MST from a retrospective external historical cohort based on a literature search of peer-reviewed papers. The Company compiled a historical external control which includes a series of studies with ATLL patients treated with standard of care over the past 10 years.

These studies included more than 6,000 ATLL patients. The Company calculated an MST from diagnosis of 7.4 months for patients with the aggressive ATLL subtypes, regardless of the type of therapy or number of therapies administered.

Five of these six patients treated with cobomarsen were alive as of the October 17, 2019 data analysis. In the trial, disease stabilization was marked by an observed decrease in Ki-67, a biomarker of cell proliferation, as well as other biomarkers of cell activation on circulating tumor cells, providing evidence of the biological mechanism effect of cobomarsen on disease stabilization.

For the clinical trial, the Company established a retrospective external historical cohort based on a literature search of peer-reviewed papers which reported MST from diagnosis and PFS on large cohorts of ATLL patients over the last decade.

A total of 16 papers describing unique cohorts of patients from Japan, the United States, South America and Europe were included in the MST retrospective analysis, of which 12 included data regarding MST for combined aggressive subtypes and eight reported MST for both acute and lymphomatous sub-types separately but not for the combined aggressive subtypes.

The number of patients included in each publication varied from 54 to 1,792, for a total of 6,440 patients.

Chronic administration of cobomarsen has been generally safe and well tolerated with a favorable safety profile over one year of dosing on a weekly basis.

There were 196 total reported adverse events as of October 17, 2019, with only 22% of the total AEs considered possibly related to study drug, and 86% of the total AEs considered mild or moderate. 14% of the total AEs were Grade 3 or 4 and most resolved within 11 days.

With no drug related deaths and only two serious adverse events occurring in the same patient and deemed possibly related to the study drug, the observed safety profile of cobomarsen in ATLL through October 17, 2019 appears to be benign and well tolerated with chronic dosing.

MGEN closed at $1.69, last traded at $2.80.

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The Medicines Co. sold for $9.7B

Novartis to pay $85 per share in cash for each MDCO share

The Medicines Company (MDCO) announced that it has entered into definitive agreement in which Novartis (NVS) will acquire the company for $85 per share in an all-cash transaction, implying a fully diluted equity value of $9.7B.

Novartis to pay $85 per share in cash for each MDCO share, Stockwinners

The stock closed Friday down $1.21 to $68.55. See our previous blog post.

The purchase price represents a premium of approximately 45% to The Medicines Company’s closing share price of $58.65 on November 18, the last trading day prior to news reports of a potential transaction between The Medicines Company and Novartis.

Novartis receives positive CHMP opinion for Kymriah, Stockwinners
Novartis to pay $85 per share in cash for each MDCO share, Stockwinners , Stockwinners

The transaction was unanimously approved by the boards of both companies. Under the terms of the merger agreement, Novartis will commence a tender offer to purchase all outstanding shares of The Medicines Company for $85 per share in cash.

Following the completion of the tender offer, a wholly owned subsidiary of Novartis will merge with The Medicines Company and shares of The Medicines Company that have not been tendered and purchased in the tender offer will be converted into the right to receive the same price per share.

Completion of the transaction is expected in Q1 of 2020, pending the successful completion of the tender offer and other customary closing conditions.

Until that time, The Medicines Company will continue to operate as a separate and independent company. The company expects to file regulatory submissions for inclisiran in the U.S. in Q4 of 2019 and in Europe in the first quarter of 2020.

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Bio-Path says prexigebersen plus LDAC shows anti-leukemic activity

Prexigebersen plus LDAC shows early anti-leukemic activity

Prexigebersen plus LDAC shows early anti-leukemic activity. Stockwinners
Prexigebersen plus LDAC shows early anti-leukemic activity.

Bio-Path Holdings (BPTH) announces that interim data from its Phase 2 study of prexigebersen in combination with low-dose cytarabine for the treatment of acute myeloid leukemia has demonstrated that the combination therapy continues to be well-tolerated and has shown early anti-leukemic activity in nearly 50% of evaluable AML patients including four patients with complete remission and four with stable disease to date in this study.

The open-label Phase 2 study is evaluating the efficacy and safety of prexigebersen in conjunction with LDAC, a therapeutic regimen well established in treatment of AML patients who cannot or elect not to be treated with more intensive chemotherapy.

The primary objective of the study is to determine whether the combination of prexigebersen and LDAC provides greater efficacy than what would be expected with LDAC alone in this de novo patient population.

The study had a pre-determined decision point at 19 evaluable patients in which the study would be terminated if less than 5 patients responded and the study would be expanded to 54 patients if five or more patients responded.

The interim analysis was performed on 17 evaluable patients instead of 19, since criteria to move to the next steps in the study had been met.

Of the 17 evaluable patients, there were four patients who achieved complete responses, one patient who achieved a morphologic leukemia free state, two patients who had significantly reduced bone marrow blasts and four patients with stable disease.

In total, 47% of the evaluable patients showed some form of response, including stable disease, to the combination treatment.

The average age of patients in the study was 73.5 years old.

Based on the recommendations of the principal investigators of the study, the company is amending the protocol to change the dosing schedule to that used in the Phase 1b study in relapsed and refractory AML patients in which a higher dose of prexigerbesen was administered prior to LDAC treatment starting at day 10 versus LDAC treatment starting on day four as was the case in the BP1001-201 study to date.

In addition, the investigators endorse the inclusion of a decitabine cohort based on relatively new and positive data with this compound.

BPTH closed at $1.77, it last traded at $2.96


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T-Cell Stocks in Focus after FDA Approves First Gene Therapy

FDA gives Novartis first gene therapy in U.S. with Kymriah approval

nvs

The FDA said it issued a “historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.”

The FDA approved #Kymriah for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL. ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill #leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

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The agency added, “Treatment with Kymriah has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events.”

The FDA granted approval of Kymriah to Novartis (NVS).

WHAT TO NOTE

On Monday, Gilead Sciences (GILD) and Kite Pharma (KITE) announced that the companies have entered into a definitive agreement pursuant to which Gilead will acquire Kite for $180.00 per share in cash.

Kite Pharma’s lead product candidate is KTE-C19, a chimeric antigen receptor (CAR)-based therapy that is in Phase 2 clinical trials for patients with relapsed or refractory aggressive diffuse large B cell lymphoma, primary mediastinal B cell lymphoma, and transformed follicular lymphoma. This is similar to what Novartis received approval for but for adults.

[youtube https://www.youtube.com/watch?v=qOusvjjc_Q0?rel=0&controls=0&w=560&h=315]

Other stocks in this space include Juno Therapeutics (JUNO), Novartis, and Gilead (GILD) since it now owns Kite Pharma (KITE).

NOVARTIS   STATEMENT

Kymriah will be manufactured for each individual patient using their own cells at the Novartis Morris Plains, New Jersey facility.

Novartis also announced what it calls “a novel collaboration” with the United States Centers for Medicare and Medicaid Services “focused on improving efficiencies in current regulatory requirements in order to deliver value-based care and ensure access for this specific patient population.”

This approach is intended to include indication-based pricing for medicines and supports payments for a medicine, such as Kymriah for its initial indication, based on the clinical outcomes achieved, which would eliminate inefficiencies from the healthcare system. Other value-based approaches related to future indications for Kymriah and CAR-T cell therapies are under discussion. Furthermore, Novartis is collaborating with CMS to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month.

Future potential indications would be reviewed for the most relevant outcomes-based approach.

STICKER  SHOCK

Bloomberg reports that Novartis sets price of Kymriah at $475,000 per treatment.


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TG Therapeutics’ Leukemia Drug Proves Effective

Adding TG-1101 to ibrutinib increased the number of patients with bone marrow confirmed CR’s, MRD negativity

The combination was well tolerated; the addition of TG-1101 did not appear to alter the safety profile of ibrutinib monotherapy

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American Society of Clinical Oncology’s #ASCO Annual Meeting is taking place June 2 to June 6 in Chicago. Several pharmaceutical firms and biotechnology companies are presenting their new drugs and/or present progress on the status of their drugs under development. TG Therapeutics is one of those companies.

#TG Therapeutics $TGTX announced positive results from its Phase 3 $GENUINE trial of #TG-1101 plus ibrutinib in patients with previously treated high risk Chronic #Lymphocytic #Leukemia.
The trial was powered to show a statistically significant improvement in ORR of 30%, with a minimal absolute detectable difference between the two arms of approximately 20%. The trial met its primary endpoint, demonstrating a statistically significant improvement in Overall Response Rate, as assessed by blinded independent central radiology and hematology review by iwCLL criteria, compared to ibrutinib alone in both the Intent to Treat population and Treated population.
The combination was well tolerated and, apart from infusion related reactions, the addition of TG-1101 did not appear to alter the safety profile of ibrutinib monotherapy.
#Neutropenia, occurring in 9% of patients, was the most commonly reported Grade 3/4 Adverse Event in the combination arm, followed by infusion related reactions and #anemia, each reported in 5% of patients.
Neutropenia or #neutropaenia, is an abnormally low concentration of neutrophils (a type of white blood cell) in the blood. Neutrophils make up the majority of circulating white blood cells and serve as the primary defense against infections by destroying bacteria, bacterial fragments and immunoglobulin-bound viruses in the blood.  Patients with neutropenia are more susceptible to bacterial infections and, without prompt medical attention, the condition may become life-threatening.
Notably, the majority of infusion related reactions were Grade 1 or 2 in severity, with only 5% Grade 3/4 IRR observed. Median follow-up for this study was approximately 11.4 months.
In addition to the improvements in ORR, CR and MRD-negativity, a trend in improvement of Progression Free Survival was observed in the combination arm of TG-1101 plus ibrutinib as compared to ibrutinib alone.
“In addition to increasing the overall number of patients that responded to treatment with ibrutinib, adding TG-1101 to ibrutinib increased the number of patients with bone marrow confirmed CR’s, MRD negativity in peripheral blood, deepened nodal responses, and resulted in fewer patients progressing on therapy. Collectively, we see the consistent pattern of enhanced benefit as providing a compelling case for combining TG-1101 with ibrutinib in these hard to treat patients with high-risk CLL. We look forward to sharing these data with the FDA later this year to discuss filing for accelerated approval,” the company noted.

Other stocks to watch in the group: $INCY, $TSRO, $AZN, $AMGN

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The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.