BeiGene announces acceptance of supplemental NDA in China for REVLIMID

BeiGene announces acceptance of supplemental NDA in China for REVLIMID for the treatment of lymphoma

BeiGene (BGNE) announced that the China National Medical Products Administration has accepted a supplemental new drug application for #REVLIMID, in combination with rituximab, for the treatment of patients with relapsed or refractory indolent lymphoma.

BeiGene shares have been active lately, Stockwinners

REVLIMID was first approved in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone, in adult patients who have received at least one prior therapy, and the label for the combination was expanded in 2018 to include adult patients with newly-diagnosed multiple myeloma who are not eligible for transplant.

REVLIMID used for the treatment of multiple myeloma, Stockwinners

It is currently marketed in China by BeiGene under an exclusive license from Celgene Logistics Sarl, a Bristol-Myers Squibb (BMY) company.

The sNDA is supported by a clinical, non-clinical, and chemistry, manufacturing and control data package, including the results from the pivotal Phase 3 AUGMENT study sponsored and conducted by Bristol-Myers Squibb.

Bristol-Myers treatment for colorectal cancer approved, Stockwinners
Bristol-Myers purchased Celgene and REVLIMID awhile back, Stockwinners

AUGMENT is a randomized, double-blind, multicenter trial in which a total of 358 patients with relapsed or refractory follicular or marginal zone lymphoma were randomized 1:1 to receive REVLIMID and rituximab or rituximab and placebo.

With a median follow-up of 28.3 months, R2 demonstrated clinically meaningful and statistically significant improvement in progression-free survival, evaluated by an independent review committee, relative to the control arm with a 54% reduction in the risk of progression or death.

The median PFS was 39.4 months for the R2 arm and 14.1 months for the control arm with an improvement by more than 2 years. Overall response rate, a secondary endpoint, was 78% in the R2 arm vs. 53% in the control arm, as assessed by the IRC.

Duration of response was significantly improved for R2 vs. control with median DoR of 37 vs. 22 months, respectively.

Bristol Meyers Comments on Celgene purchase, Stockwinners
Bristol Meyers Comments on Celgene purchase, Stockwinners

The most frequent adverse event in the R2 arm was neutropenia, vs. 22% in the control arm.

Additional commonly observed AEs in more than 20% of patients included diarrhea, constipation, cough, and fatigue. Adverse events that were reported at a higher rate in the R2 arm were neutropenia, constipation, leukopenia, anemia, thrombocytopenia and tumor flare.

BMY closed at $63.51. BGNE closed at $173.14.

See our other blogs about BeiGene.

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MorphoSys higher on lymphoma data

MorphoSys says tafasitamab B-MIND study successfully passed futility analysis

MorphoSys (MOR) announced that the ongoing tafasitamab phase 3 B-MIND study has successfully passed the pre-planned, event-driven interim analysis for futility.

MorphoSys higher on lymphoma data, Stockwinners

An independent data monitoring committee reviewed the data and recommended to increase the number of patients from currently 330 to 450.

B-MIND compares the efficacy of the CD19 antibody tafasitamab plus bendamustine with rituximab plus bendamustine in patients with relapsed or refractory diffuse large B cell lymphoma, or r/r DLBCL.

Tafasitamab (MOR208) is a humanized monoclonal antibody , Stockwinners

“Within the interim analysis for futility, data were assessed by the IDMC for the probability of a positive study at primary completion.

The IDMC assessed efficacy data in both the overall patient population as well as in the biomarker-positive subpopulation. The biomarker, described as patients with a low natural killer cell count at baseline, was implemented as a co-primary endpoint in an amendment of B-MIND in the first quarter 2019.

The recommendation to enroll more patients aims to increase statistical power of the study in the biomarker-described patient subpopulation as well as the overall patient population. Data of the analysis were not shared with MorphoSys.

As a continuation of the B-MIND study protocol, enrollment will proceed according to the original inclusion and exclusion criteria to allow for ongoing comparison of the efficacy in the overall and biomarker positive patient population.

Top line results are expected to be available in Q1 2022,” the company stated.

Piper Jaffray Comments

Piper Jaffray analyst Danielle Brill said news that MOR208 successfully passed the B-MIND trial futility analysis is “a positive clearing event for investors” that de-risks the stock, even though enrollment will be expanded from 300 to 450 patients and it is unclear how strong the efficacy signal was in the overall population.

She continues to expect FDA and EMA approval for MOR208 based on L-MIND data by mid-2020 and keeps an Overweight rating on MorphoSys shares.

MOR +$1.98 to $28.09.

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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.

[youtube https://www.youtube.com/watch?v=7nCvItKbEns?rel=0&controls=0&w=560&h=315]

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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Trump plans executive order on drug prices

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The Trump administration is preparing an executive order regarding U.S. drug costs, which may express support for value-based agreements via which drug companies and health insurers make arrangements to pay for products depending on how well they work, Bloomberg reported yesterday afternoon, citing people familiar with the matter.

A first executive order on drug prices may come out soon, followed by a second, more extensive one later, sources told the news service.

Top health and budget officials in the administration will meet today to discuss the issue, according to the people, who asked not to be identified because the session is private. Trump sought recommendations from the nation’s health agencies on reducing medication costs, Health and Human Services Secretary Tom Price told senators last week.

Unlike other advanced economies, the U.S. doesn’t directly regulate medicine prices. The pricing system is opaque, with list prices set by drugmakers and rebates negotiated in private with intermediaries like PBMs.

The industry wants the government to modify the law so that companies can reach more value-based payment agreements, whereby reimbursements are based on a drug’s results. Swiss pharma giant Novartis AG has such a “pay-for-performance” plan in place for heart failure treatment #Entresto, in which insurers pay more if the drug keeps patients out of the hospital and lowers associated costs.

Another idea discussed by the industry would allow insurers to pay by increments for very expensive drugs that essentially cure diseases.

The president has threatened on several occasions to force drugmakers to bid for government business as a way to reduce prices. He’s also talked about letting consumers import drugs from other countries with lower prices. Neither of those policies, which would likely require a change in law to be implemented in a meaningful way, are in drafts of the orders, according to one person familiar with the effort.

Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol-Myers (BMY), Eli Lilly (LLY), GlaxoSmithKline (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY).

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Takeda and Seattle Genetics Publish Positive Results on T-cell lymphoma

ADCETRIS is an antibody-drug conjugate directed to CD30 which is expressed on CTCL lesions in approximately 50 percent of patients with the disease

Based on the study results, Takeda plans to begin to submit data from the ALCANZA trial to regulatory agencies in its territories in 2017.

https://stockwinners.com/blog

#Takeda (TKPYY) and #SeattleGenetics (SGEN) announced that data from the randomized Phase 3 ALCANZA clinical trial evaluating ADCETRIS in patients with cutaneous T-cell #lymphoma were published in the journal #Lancet.

Lymphoma is the most common blood cancer. The two main forms of lymphoma are Hodgkin lymphoma and non-Hodgkin lymphoma (NHL). Lymphoma occurs when cells of the immune system called lymphocytes, a type of white blood cell, grow and multiply uncontrollably. Cancerous lymphocytes can travel to many parts of the body, including the lymph nodes, spleen, bone marrow, blood, or other organs, and form a mass called a tumor. The body has two main types of lymphocytes that can develop into lymphomas: B-lymphocytes (B-cells) and T-lymphocytes (T-cells).

Data were previously presented in an oral session at the 58th American Society of Hematology annual meeting in December 2016.

#ADCETRIS is an antibody-drug conjugate directed to CD30 which is expressed on CTCL lesions in approximately 50 percent of patients with the disease.

ADCETRIS is currently not approved for the treatment of CTCL.

ALCANZA is a randomized, open-label Phase 3 study designed to evaluate single-agent ADCETRIS versus a control arm of investigator’s choice of the standard of care therapies methotrexate or bexarotene, in patients with CD30-positive CTCL.

The manuscript highlights data from the trial which achieved its primary endpoint with the ADCETRIS treatment arm demonstrating a highly statistically significant improvement in the rate of objective response lasting at least four months (ORR4) versus the control arm as assessed by an independent review facility.

#ORR4, as assessed by Global Response Score, was 56.3 percent in the ADCETRIS arm compared to 12.5 percent in the control arm.

Key secondary endpoints specified in the protocol, including complete response rate, progression-free survival and reduction in the burden of symptoms during treatment (Skindex-29), were all highly statistically significant in favor of the ADCETRIS arm.

The safety profile associated with ADCETRIS from the ALCANZA trial was generally consistent with the existing prescribing information.

The most common adverse events of any grade include: peripheral neuropathy, nausea, diarrhea, fatigue, vomiting, alopecia, pruritis, pyrexia, decreased appetite and hypertriglyceridemia.

Based on the study results, Takeda plans to begin to submit data from the ALCANZA trial to regulatory agencies in its territories in 2017. The U.S. FDA granted Breakthrough Therapy Designation to ADCETRIS for the treatment of the most common subtypes of CTCL, mycosis fungoides and primary cutaneous anaplastic large cell lymphoma .

Seattle Genetics plans to submit these data as part of a supplemental Biologics License Application to the FDA in mid-2017. The ALCANZA trial received a Special Protocol Assessment agreement from the FDA and scientific advice from the European Medicines Agency.

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