FDA rejects Intercept’s drug application, shares plunge

Intercept receives FDA CRL for obeticholic acid recommending additional data

Intercept Pharmaceuticals (ICPT) announced that the U.S. Food and Drug Administration has issued a Complete Response Letter regarding the New Drug Application for obeticholic acid for the treatment of fibrosis due to nonalcoholic steatohepatitis.

stockwinners.com ICPT
Shares plunge following FDA’s comments

The CRL indicated that, based on the data the FDA has reviewed to date, the Agency has determined that the predicted benefit of OCA based on a surrogate histopathologic endpoint remains uncertain and does not sufficiently outweigh the potential risks to support accelerated approval for the treatment of patients with liver fibrosis due to NASH.

NASH is the severe form of Non-Alcoholic Fatty Liver Disease (NAFLD). It can lead to liver cancer, cirrhosis, and liver transplants.

The FDA recommends that Intercept submit additional post-interim analysis efficacy and safety data from the ongoing REGENERATE study in support of potential accelerated approval and that the long-term outcomes phase of the study should continue.

Intercept had previously disclosed that, based on the FDA’s decision to postpone a tentatively scheduled advisory committee meeting, it was expected that the Agency’s review of its NDA would extend beyond the PDUFA goal date and that the FDA would move forward with rescheduling the Adcom.

The NDA submission for OCA is the first for NASH and was based on data from 35 clinical trials and more than 1,700 NASH patients treated with the drug.

OCA is the only investigational NASH drug with Breakthrough Therapy designation and has uniquely demonstrated reproducible ability to reverse or otherwise stabilize liver fibrosis in patients with advanced fibrosis due to NASH.

According to the FDA draft guidance for NASH fibrosis, of the histologic features of NASH, fibrosis is considered the strongest predictor of adverse clinical outcomes, including liver-related death.

Estimated number of people suffering from fatty liver, Stockwinners

There is currently no approved therapy for this devastating disease, which has become a leading cause of liver failure and resulting poor clinical outcomes.

A number of other companies working on NASH treatments are also moving lower, with CymaBay (CBAY) down about 3% and NGM Biopharmaceuticals (NGM) fractionally lower.

Other companies exploring NASH treatments include Madrigal Pharmaceuticals (MDGL), Novo Nordisk (NVO), Genfit (GNFT), Eli Lilly (LLY), and Alnylam Pharmaceuticals (ALNY).

ICPT closed at $77.49, last traded at $48.00, down 38%.

STOCKWINNERS

To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This 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.

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.

STOCKWINNERS

To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This 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.

Epizyme’s Tazemetostat receives FDA’s okay

Epizyme says FDA advisory committee votes unanimously in favor of tazemetostat

Epizyme (EPZM) announced that the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration voted 11 – 0 in favor of the benefit-risk profile of tazemetostat as a treatment for patients with metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery.

Epizyme shares halted ahead of FDA meeting, Stockwinners

“Today’s ODAC outcome is a significant step toward addressing the critical needs of ES patients.

This is a remarkable achievement marking the culmination of years of hard work by the entire Epizyme team. If approved, we will have the opportunity to change how patients with this devastating cancer are treated.

Our commercial-readiness is complete, and we look forward to finalizing our dialog with the FDA,” said Robert Bazemore, president and CEO of Epizyme.

Epizyme’s New Drug Application, or NDA, for tazemetostat is, an oral, first-in-class EZH2 inhibitor, for the treatment of patients with metastatic or locally advanced epithelioid sarcoma (ES) who are not eligible for curative surgery.

Epithelioid Sarcoma is a rare form of cancer, Stockwinners

The advisory committee vote will be non-binding, but FDA takes its recommendations into consideration when reviewing related applications for marketing approval.

ES is a rare and aggressive soft tissue sarcoma characterized by a loss of the INI1 protein. Patients are most commonly diagnosed as young adults, between 20 and 40 years of age, typically with no patients living past five years from diagnosis. ES becomes more aggressive after recurrence or once it has metastasized, with a typical survival of less than one year for patients with metastatic disease.

Epizyme, Inc. discovers, develops, and commercializes novel epigenetic medicines for patients with cancer and other diseases primarily in the United States.

EPZM halted at $18.19.

STOCKWINNERS

To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This 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.

Synthorx sold for $2.5 billion

Sanofi to acquire Synthorx for $68 per share in cash

Sanofi (SNY) and Synthorx (THOR) entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Synthorx for $68 per share in cash, which represents an aggregate equity value of approximately $2.5B, on a fully diluted basis.

Synthorx sold for $2.5 billion, Stockwinners

The transaction was unanimously approved by both the Sanofi and Synthorx boards.

Under the terms of the merger agreement, Sanofi will commence a cash tender offer to acquire all of the outstanding shares of Synthorx common stock for $68 per share in cash.

The $68 per share acquisition price represents a 172% premium to Synthorx’s closing price on December 6.

Following the successful completion of the tender offer, a wholly owned subsidiary of Sanofi will merge with Synthorx and the outstanding Synthorx shares not tendered in the tender offer will be converted into the right to receive the same $68 per share in cash paid in the tender offer.

The tender offer is expected to commence in December.

Sanofi plans to finance the transaction with cash on hand. Subject to the satisfaction or waiver of customary closing conditions, Sanofi expects to complete the acquisition in the first quarter of 2020.

“Synthorx’s Expanded Genetic Alphabet platform is expected to be a source for developing a differentiated therapeutic pipeline. Alone and in combination with other existing Sanofi platforms, including the Nanobody technology, it will enable the company to develop a wide range of novel biologics, including drug conjugates, protein fusions, and multi-specific biologics, with applications beyond oncology and extending to other therapeutic areas… The addition of THOR-707 and Synthorx’s other earlier-stage cytokine programs to Sanofi’s pipeline will enhance Sanofi’s position in oncology, and in immuno-oncology. We expect IL-2 to become a foundation of future IO-IO combinations as well as offering multiple combination opportunities with Sanofi’s clinical and pre-clinical oncology assets, including with PD-1, CD-38, and molecules that modulate effector T-cells and natural killer cells.”

THOR closed at $25.04, it last traded at $67.43. SNY closed at $46.03.

STOCKWINNERS

To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This 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.

ArQule sold for $2.7 billion

Merck to acquire ArQule for $20 per share

Merck (MRK) and ArQule (ARQL) announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire ArQule for $20 per share in cash for an approximate total equity value of $2.7B.

Merck buys ArQule for $2.7B, Stockwinners

ArQule’s lead investigational candidate, ARQ 531, is a novel, oral Bruton’s tyrosine kinase, or BTK, inhibitor currently in a Phase 2 dose expansion study for the treatment of B-cell malignancies.

BTK inhibition has been shown to prevent B-cell receptor signaling that is critical for the survival and proliferation of leukemic cells in many B-cell malignancies.

Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners
Merck buys ArQule, Stockwinners

ARQ 531 is a selective, reversible inhibitor that blocks both wild-type BTK and the C481S mutant form of the enzyme that is commonly associated with resistance to other BTK inhibitors.

In early clinical trials, ARQ 531 demonstrated a manageable safety profile and early signs of anti-tumor activity for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia, or CLL, and Richter’s Transformation.

ArQule lead investigative drug is ARQ531

Final data from the Phase 1 study of ARQ 531 will be presented on December 9 at the 61st American Society of Hematology, or ASH.

Under the terms of the acquisition agreement announced, Merck, through a subsidiary, will initiate a tender offer to acquire all outstanding shares of ArQule.

The closing of the tender offer will be subject to certain conditions, including the tender of shares representing at least a majority of the total number of ArQule’s outstanding shares, the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions.

Upon the successful completion of the tender offer, Merck’s acquisition subsidiary will be merged into ArQule, and any remaining shares of common stock of ArQule will be canceled and converted into the right to receive the same $20 per share price payable in the tender offer.

The transaction is expected to close early in Q1 of 2020.

ARQL closed at $9.66, last traded at $19.90.

STOCKWINNERS

To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This 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.

Translate »