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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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.

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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.

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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.

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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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Shares of Aravive soar on its ovarian cancer drug

Aravive reports data from ongoing Phase 1b portion of AVB-500 trial

Aravive (ARAV) announced new data from the ongoing Phase 1b portion of the Phase 1b/2 clinical trial of AVB-500 in platinum-resistant recurrent ovarian cancer patients.

Ovarian cancer data sends shares sharply higher, Stockwinners

The data from the first 31 patients treated at the 10 mg/kg dose are maturing and affirm earlier findings on the relationship between AVB-500 levels and anti-tumor response.

In this data analysis, high serum drug levels of AVB-500 were strongly predictive of anti-tumor activity with statistically significant correlation to progression-free survival.

PFS is the primary endpoint for platinum-resistant ovarian cancer clinical trials.

At the 10 mg/kg dose, patients that met or exceeded the minimal efficacious concentration of AVB-500 demonstrated a greater than four-fold increase in median PFS over those with low exposure and approximately two-fold improvement in overall response rate, including one complete response.

How AVB-500 works, Stockwinners

Patients who achieved sufficient AVB-500 exposure also showed improvements in duration of response and clinical benefit rate, with reduced chance of progressing by 3.2-fold.

The open-label, Phase 1b portion of the Phase 1b/2 clinical trial of AVB-500 enrolled patients with platinum-resistant recurrent ovarian cancer in two cohorts, one investigating a combination of AVB-500 with pegylated liposomal doxorubicin and the other a combination with paclitaxel.

All patients were treated with 10mg/kg AVB-500 every other week.

The company previously reported drug exposure-response relationship among the initial patients receiving 10 mg/kg.

The study identified a minimal efficacious concentration that is consistent with at least 95% target engagement based on independent pharmacokinetic modeling.

At the 10 mg/kg dose, 17 of 31 patients in the study achieved the minimal efficacious concentration after the first dose of AVB-500. The baseline characteristics, demographics and safety parameters were comparable between patients who achieved the minimal efficacious concentration and those who fell below that threshold.

The analysis shows that the clinical benefit at this dose level in the study can be primarily attributed to AVB-500 exposure.

Other notable findings: The patient with CR that is pending confirmation achieved the MEC of AVB-500 and was on paclitaxel. She had a baseline serum GAS6 level typical of the platinum-resistant ovarian cancer population and two-fold higher than that observed in healthy volunteers. She also exhibited poor prognostic factors, including two prior lines of therapy and platinum-free interval of less than three months.

Among the patients who responded to AVB-500, four of seven remain responders and continue on study, and two patients remain on AVB-500 as a single agent.

All four patients achieved the minimal efficacious AVB-500 concentration. Among the 13 patients whose best response was SD, four achieved the MEC of AVB-500 and remain on study. One SD patient, whose trough level was below the minimal efficacious concentration, did not progress but withdrew consent.

Because the study is still ongoing and includes only patients above the MEC, duration of response and PFS may continue to evolve.

These data confirm the company’s strategy to investigate higher doses in the current Phase 1b study, to determine if a greater proportion of patients can exceed the MEC.

According to our modeling, a dose of 20 mg/kg should allow greater than 90% of the patients to achieve the MEC.

It is anticipated 6 to 12 patients will be treated with 15mg/kg and an additional 12 patients will be treated with 20mg/kg.

An independent safety monitoring group will review data from the 15mg/kg group prior to escalation to the 20 mg/kg dose.

AVB-500 continues to be well-tolerated and there have been no serious and unexpected adverse reactions or dose-limiting toxicities to date.

ARAV closed at $6.51, last traded at $17.20.

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BeiGene drug wins FDA approval for rare form of lymphoma

The FDA granted accelerated approval to the capsules for treatment of adult patients with mantle cell lymphoma, who have received at least one prior therapy.

The U.S. Food and Drug Administration approved BeiGene Ltd’s lymphoma drug, accepting the Chinese drugmaker’s strategy of largely using data from trials held outside the United States to file for approval.

FDA granted accelerated approval to the capsules for treatment of adult patients with mantle cell lymphoma
FDA granted approval to the capsules for treatment of aduls with mantle cell lymphoma, Stockwinners

The company tested the treatment, Brukinsa, in 118 patients with mantle cell lymphoma enrolled in two studies. About three-quarters were Asian, 21% Caucasian, and between 10% to 15% were from the United States, BeiGene said.

The FDA granted accelerated approval to the capsules for treatment of adult patients with mantle cell lymphoma, who have received at least one prior therapy.

Mantle cell lymphoma is a rare, aggressive form of non-Hodgkin lymphoma, a blood cancer that most often affects men aged over 60. The company estimates between 3,000 and 4,000 new patients were diagnosed in the United States in 2015.

Last month, BeiGene (BGNE) and Amgen (AMGN) announced a global strategic oncology collaboration for the commercialization and development in China of Amgen’s XGEVA, KYPROLIS, and BLINCYTO, and the joint global development of 20 oncology assets in Amgen’s pipeline, with BeiGene responsible for development and commercialization in China.

In connection with the collaboration, Amgen said it will purchase a 20.5% stake in BeiGene for approximately $2.7B in cash at $174.85 per American Depositary Share, or ADS.

BGNE closed at $196.40.

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Watch shares of Mirati Therapeutics

Mirati presents data from sitravatinib in combination with nivolumab trials

Mirati Therapeutics (MRTX) announced the presentation of initial data from its ongoing Phase 2 clinical trial of sitravatinib in combination with nivolumab in metastatic urothelial cancer patients with documented progression on a platinum-chemotherapy and checkpoint inhibitor.

Mirati is in focus on cancer data, Stockwinners

The data were presented in an oral presentation at the Society of Immunotherapy of Cancer 34th Annual Meeting.

Preliminary results from the ongoing Phase 1 study of neoadjuvant sitravatinib combined with nivolumab in patients with resectable squamous cell carcinoma of the oral cavity, SNOW trial, were also presented in a poster session.

The preliminary data suggest that the combination of neoadjuvant sitravatinib and nivolumab is safe and active in patients with squamous cell carcinoma of the oral cavity who are candidates for resection.

Chart shows Sitravatinib in action, Stockwinners

Tumor reduction was observed in all eight patients who were eligible for evaluation, including one complete pathological response.

All patients received postoperative radiation therapy, and none required postoperative chemotherapy.

With a median follow-up of 31.4 weeks, all patients are alive with no disease recurrence to date.

In most patients, treatment with sitravatinib led to a decrease in myeloid-derived suppressor cells and a shift towards M1-type macrophages in the tumor microenvironment, supporting previous preclinical findings.

“Sitravatinib is a spectrum-selective kinase inhibitor that potently inhibits receptor tyrosine kinases, including TAM family receptors that has the potential to increase responsiveness in patients whose tumors are resistant to checkpoint inhibitors. The initial efficacy data from the Phase 2 clinical trial presented today in patients with checkpoint refractory mUC is promising and extends the clinical benefit data beyond what has already been demonstrated by sitravatinib combined with nivolumab in checkpoint refractory non-small cell lung cancer,” said Charles Baum, M.D., CEO of Mirati.

Sitravatinib offers hope for cancer patients, Stockwinners

“In addition, we are evaluating sitravatinib in patients who have progressed on checkpoint therapy, including those with NSCLC and renal cell cancer, and we continue to expand development efforts of sitravatinib through our collaboration with BeiGene in multiple indications including NSCLC, renal cell cancer, hepatocellular cancer, ovarian cancer, and gastric cancer.”

MRTX closed at $104.78.

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Nektar Therapeutics is in focus

Nektar presents new clinical, preclinical data from immuno-oncology pipeline

Nektar Therapeutics (NKTR) announced the presentation of five clinical and preclinical data abstracts focused on its immuno-oncology portfolio at the 2019 Society for Immunotherapy of Cancer Annual Meeting.

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Nektar Therapeutics is in focus, Stockwinners

New clinical results from the PIVOT-02 Phase 1/2 study were shared in an oral presentation titled, “Clinical activity of BEMPEG plus NIVO in previously untreated patients with metastatic melanoma: updated results from the Phase 1/2 PIVOT-02 Study” during the Combination Phase 1-2 Clinical Trials Session on Saturday, November 9th.

Additional preclinical data presented at the annual meeting highlighted NKTR-255, an IL-15 agonist discovered by Nektar.

The presentations demonstrated that NKTR-255 enhanced activity of antibody-dependent cellular cytotoxicity against tumor cells in vitro, and that it also enhanced in vivo efficacy of ADCC-inducing antibodies in models of human solid tumors.

NKTR-255 is designed to engage the IL-15 pathway to stimulate and expand natural killer cells and promote the survival and expansion of memory CD8+ T cells without inducing suppressive regulatory T cells.

Nektar product pipeline, Stockwinners

NKTR-255 is currently being evaluated in a Phase 1/2 clinical trial in patients with either relapsed or refractory Non-Hodgkin’s lymphoma or multiple myeloma.

“The data presented at this year’s SITC meeting continue to showcase the potential of our I-O portfolio, most notably our key IL-2 pathway program, bempeg, and our new IL-15 pathway program, NKTR-255,” said Jonathan Zalevsky, Ph.D., Chief Research & Development Officer at Nektar.

“The 18-month follow-up data presentation for the Stage IV melanoma patients in our PIVOT-02 study reinforces the promise of BEMPEG and NIVO to work synergistically to achieve a deepening of response over time, while maintaining a favorable safety and tolerability profile. We’re pleased that at this 18 month timepoint, 85% of patients who achieved responses have ongoing responses and median PFS has not yet been reached.”

Separately, Nektar Therapeutics (NKTR) announced updated results from the first-in-human Phase 1a study of NKTR-358, a novel T regulatory cell stimulator in development for the treatment of autoimmune and other chronic inflammatory conditions.

Nektar reports new data, Stockwinners

The data, which were presented at the 2019 Annual Meeting of the American College of Rheumatology in Atlanta, show that treatment with NKTR-358 led to a marked and selective dose-dependent expansion in the numbers and proliferative capacity of FoxP3+CD25bright Treg cells, and a measurable activation of Treg cells.

These data are a continuation of initial results reported at 2019 Annual European Congress of Rheumatology in June 2019.

NKTR-358 is designed to treat autoimmune and inflammatory conditions by correcting the immune system imbalance that results from reduced numbers and impaired function of immune-regulating Treg cells.

NKTR-358 works by targeting the interleukin-2 receptor complex to stimulate the proliferation and activation of Treg cells.

NKTR-358 was discovered by Nektar and is being co-developed and commercialized in partnership with Eli Lilly (LLY).

Eli Lilly announces Alimta label expanded by FDA, Stockwinners
Eli Lilly and Nektar report new data, Stockwinners

“We’re pleased to report that final results from our first-in-human Phase 1a study continue to support the positive safety and tolerability profile of NKTR-358, while reinforcing its selective and measurable impact on the numbers, expansion and activation of regulatory T cells or Tregs,” said Brian Kotzin, M.D., senior VP, Clinical Development and NKTR-358 Program Lead at Nektar.

“Autoimmune and inflammatory diseases are marked by an imbalance in the body’s self-tolerance and self-regulatory immune pathways, and the ability of NKTR-358 to expand functional Tregs could help restore normal balance.

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Aveo Pharma. shares tumble following FDA meeting

Aveo Pharmaceuticals provides update after FDA meeting discussing TIVO-3 results

Aveo Pharmaceuticals (AVEO) provided a regulatory update following a meeting with the FDA to discuss results from the August overall survival, or OS, analysis of the TIVO-3 trial and the company’s proposal to proceed with a new drug application, or NDA, for tivozanib. TIVO-3 is the company’s Phase 3 randomized, controlled, multi-center, open-label study to compare tivozanib, the company’s vascular endothelial growth factor receptor tyrosine kinase inhibitor, or VEGFR-TKI, to sorafenib in 350 subjects with highly refractory metastatic renal cell carcinoma, or RCC.

AVEO shares tumble following FDA meeting, Stockwinners

The TIVO-3 trial was designed to address the FDA’s concerns regarding the OS trend in the TIVO-1 trial. In the TIVO-1 trial, the company’s initial RCC pivotal trial, the FDA found that the inconsistent progression free survival, or PFS, and OS results and imbalance in post study treatments made the trial results uninterpretable and inconclusive when making a risk-benefit assessment necessary for drug approval.

The company previously announced that the TIVO-3 trial met its primary endpoint of demonstrating a significant improvement in PFS. The study also demonstrated a significant improvement in the secondary endpoint of overall response rate.

The August analysis of the secondary endpoint of OS was the second prespecified interim OS analysis of the TIVO-3 trial, and showed an updated OS hazard ratio, or HR, of 0.99 at two years from the last patient enrolled in the study.

In the FDA’s preliminary feedback, based on its assessment of the totality of evidence presented to date, the FDA recommended that the company not submit an NDA at this time.

The FDA stated that it remained concerned about the results of TIVO-3 in the context of the overall development of tivozanib. The FDA noted that the company’s current interim OS results do not abrogate the FDA’s concerns over detriment and that those results may worsen with final analysis at 263 events and that the median OS for tivozanib is worse than that of sorafenib.

In view of the changing first-line treatment landscape as well as the FDA’s continued concerns, the company informed the FDA that it intends to narrow its proposed indication to relapsed/refractory RCC.

At the meeting, the FDA acknowledged AVEO’s responses and reiterated its concerns about the survival information and the totality of data.

The FDA noted that the choice to submit the data is the company’s, and that a discussion with the Oncologic Drug Advisory Committee will likely be required.

The FDA said that if AVEO wishes to proceed with a revised OS analysis in June 2020, AVEO should submit an updated statistical analysis plan, or SAP, with a planned OS update based on the projected number of events at that time.

AVEO intends to submit to the FDA an update to the SAP for the final OS analysis consistent with these discussions, followed by an NDA submission in Q1 of 2020.

AVEO expects to report the final OS analysis in June 2020 based on a May 1, 2020 cutoff, at which point the company estimates that the study will have reached approximately 263 OS events, as discussed with the FDA.

The FDA and the company agreed that if the final analysis yields an OS HR above 1.00, the company will withdraw its NDA application.

AVEO shares are down 35% to $0.59.

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BeiGene shares soar on Amgen stake

Amgen to buy 20.5% stake in BeiGene for $2.7B at $174.85 per ADS

BeiGene (BGNE) and Amgen (AMGN) announced a global strategic oncology collaboration for the commercialization and development in China of Amgen’s XGEVA, KYPROLIS, and BLINCYTO, and the joint global development of 20 oncology assets in Amgen’s pipeline, with BeiGene responsible for development and commercialization in China.

Amgen takes 20% stake in BeiGene, Stockwinners

In connection with the collaboration, Amgen will purchase a 20.5% stake in BeiGene for approximately $2.7B in cash at $174.85 per American Depositary Share, or ADS.

Amgen will receive one seat on BeiGene’s Board of Directors.

Under the agreement, BeiGene will commercialize XGEVA, KYPROLIS and BLINCYTO in China for five or seven years, during which time the parties will equally share profits and losses.

Amgen enters Chinese market by taking stake in BeiGene, Stockwinners

Following the commercialization period, BeiGene will have the right to retain one product and will be entitled to receive royalties on sales in China for an additional five years on the products not retained; and XGEVA was approved in China in 2019 for patients with giant cell tumor of the bone and is in development for prevention of skeletal-related events in cancer patients with bone metastases.

Blincyto is indicated for acute lymphoblastic leukemia, Stockwinners

KYPROLIS is in late-stage development in China for patients with multiple myeloma, and BLINCYTO is in late-stage development in China as a treatment for adult patients with relapsed or refractory acute lymphoblastic leukemia.

Kyprolis is indicated for multiple myeloma , Stockwinners

BeiGene has agreed to jointly develop 20 Amgen oncology pipeline assets globally, which include targeted small-molecule agents such as AMG 510, a first-in-class investigational KRAS G12C inhibitor, as well as BiTE antibodies, for solid and hematologic malignancies; Amgen and BeiGene will co-fund global development costs, with BeiGene contributing up to $1.25B worth of development services and cash over the term of the collaboration.

BeiGene is entitled to receive royalties from global sales of each product outside of China, with the exception of AMG 510; For each pipeline asset that is approved in China, BeiGene will receive commercial rights for seven years from approval, during which time the parties will share equally in profits and losses.

BeiGene is also entitled to receive royalties from sales in China for five years after the seven-year commercial term; and BeiGene will also have the right to retain approximately one of every three approved pipeline assets, up to a total of six, other than AMG 510, for commercialization in China, during which time the parties will share in profits and losses.

The transactions have been approved by the boards of directors of both companies and are expected to close in the first quarter of 2020, subject to approval by a majority vote of BeiGene’s shareholders pursuant to the listing rules of the Hong Kong Stock Exchange, the expiration or termination of applicable waiting periods under applicable antitrust laws, and satisfaction of other customary closing conditions.

BeiGene has already received commitments from shareholders holding approximately 40% of its outstanding shares to vote in favor of the transactions.

BGNE closed at $138.34, last traded at $174.58.

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Assembly Biosciences reports chronic hepatitis data, shares jump

Assembly Biosciences announces presentation of ABI-H0731, ABI-H2158 data

Assembly Biosciences (ASMB) announced that data on its lead HBV core inhibitor candidates, ABI-H0731 and ABI-H2158 for the treatment of chronic HBV will be featured in a late-breaking poster session during the American Association for the Study of Liver Diseases Annual Meeting.

Shares jump on hepatitis data, Stockwinners

A hepatitis B infection can result in either an acute infection or a chronic infection. When a person is first infected with the hepatitis B virus, it is called an “acute infection” (or a new infection). Most healthy adults that are infected do not have any symptoms and are able to get rid of the virus without any problems. Some adults are unable to get rid of the virus after six months and they are diagnosed as having a “chronic infection.”

Title: Continued Therapy with ABI-H0731+Nrtl Results in Sequential Reduction/Loss of HBV DNA, HBV RNA, HBeAg, HBcrAg and HBsAg in HBeAg-Positive Patients.

Abstract Summary: Final results from Phase 2a are reported for HBeAg+ patients with chronic HBV infection treated with 731+Nrtl for 24 weeks.

In Study 202, greater mean log10 declines in HBV DNA and RNA were achieved with 731+Nrtl versus entecavir alone.

In Study 201, the proportion of patients on 731+Nrtl versus Nrtl alone achieving DNA target not detected was 69% vs 0%, and the proportion of patients achieving RNA less than35 U/mL whose RNA was greater than or equal to35 U/mL at baseline was 52% vs 0% respectively.

In Study 211, there are 64 HBeAg+ patients currently on extended treatment beyond 24 weeks. Among the 27 HBeAg+ patients receiving 731+Nrtl in Study 201, 41% have now achieved DNA TND along with RNA less than35 U/mL and HBeAg less than1 IU/mL.

At their last time point, Study 202 patients now in Study 211 have demonstrated mean DNA and RNA declines of 6.1 and 3.0 logs, respectively, with observed mean log changes of greater than or equal to 0.6 for HBeAg, greater than 0.8 log for HBcrAg and greater than or equal to 0.4 log for HBsAg.

731 continues to exhibit a favorable safety and tolerability profile in patients treated for up to 1 year, with only mild/moderate adverse events and lab abnormalities, and only a single discontinuation due to a Grade 1 rash.

The combination of 731+NrtI results in faster and deeper declines in HBV DNA and RNA than NrtI alone, as well as subsequent declines in the surrogate markers of cccDNA predictive of cccDNA pool depletion, and HBsAg.

A Visual Guide to Hepatitis, Stockwinners

The emergent data supports the continued development of 731.

Abstract data are as of the time of submission; the poster is expected to include updated safety and efficacy results. Title: The Second-Generation Hepatitis B Virus Core Inhibitor ABI-H2158 is Associated with Potent Antiviral Activity in a 14-Day Monotherapy Study in HBeAg-positive Patients with Chronic Hepatitis B.

Abstract Summary: The Phase 1b study is enrolling sequential cohorts of 9 patients and each cohort will be randomized to receive 2158 or placebo QD for 14 days in a blinded manner.

Dosing in the 1st cohort has been completed. In patients receiving 2158, mean declines from Baseline to Day 15 in HBV DNA and RNA levels were 2.3 log10 and 2.1 log10 IU/mL respectively.

No serious AEs, dose limiting toxicities or premature discontinuations were reported.

Three patients reported a total of 5 mild, drug-related AEs that recovered without intervention; dizziness, fatigue, rash, headache and upper abdominal pain.

Treatment emergent laboratory abnormalities were infrequent, mild and transient, with no ALT elevations Grade greater than or equal to 1 in severity.

Day 14 plasma 2158 Cmax and AUC0-24hr were 3,390 ng/mL and 46,100 hr*ng/mL, respectively.

Results from the initial 100 mg low dose of ABI-H2158 cohort demonstrated potent antiviral activity, a favourable safety profile when administered for 14 days, and support once daily dosing in CHB patients.

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Celgene sells its Otezla to Amgen for $13.4 billion

Amgen to acquire Otezla from Celgene for $13.4B in cash

Amgen (AMGN) announced that it has entered into an agreement with Celgene (CELG) in connection with its previously announced merger with Bristol-Myers (BMY) to acquire worldwide rights to Otezla, “the only oral, non-biologic treatment for psoriasis and psoriatic arthritis,” and certain related assets and liabilities, for $13.4B in cash, or approximately $11.2B, net of the present value of $2.2B in anticipated future cash tax benefits.

Bristol Meyers Comments on Celgene purchase, Stockwinners
Celgene sells Otezla to pave the way for its merger with Bristol-Meyers, Stockwinners

Otezla (apremilast) is a prescription medicine approved for the treatment of patients with moderate to severe plaque psoriasis for whom phototherapy or systemic therapy is appropriate. Otezla is a prescription medicine approved for the treatment of adult patients with active psoriatic arthritis. Otezla is a prescription medicine approved for the treatment of adult patients with oral ulcers associated with Behçet’s Disease.

Amgen goes shopping, Stockwinners

Amgen believes that the acquisition of Otezla offers many benefits including: A strong strategic fit with Amgen’s long-standing expertise in psoriasis and inflammation; A differentiated, oral therapy complementary to Amgen’s existing inflammation franchise of innovative biologics and biosimilar products; At least low double-digit Otezla sales growth, on average, over the next five years; Acceleration of Amgen’s near- and long-term revenue growth; Immediate non-GAAP EPS accretion; Intellectual Property exclusivity through at least 2028 in the U.S.; Worldwide rights which fit well with Amgen’s international presence and global expansion objectives; Support of increased R&D investment in 2020 to advance Amgen’s innovative pipeline of first-in-class molecules; No interruption in deployment of Amgen’s capital allocation priorities. Sales of Otezla in 2018 were $1.6B driven by strong volume growth.

Bristol-Myers treatment for colorectal cancer approved, Stockwinners
Bristol-Myers is in the process of buying Celgene, Stockwinners

The closing of the acquisition is contingent on Bristol-Myers entering into a consent decree with the Federal Trade Commission in connection with the pending Celgene merger, the closing of the pending merger with Celgene and the satisfaction of other customary closing conditions.

The transaction is expected to close by the end of 2019.

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Pancreatic cancer data sends shares of Tyme Technologies higher

Tyme Technologies presents updated data from TYME-88-Panc Phase II study

Tyme Techs. shares jump of data, Stockwinners

Tyme Technologies (TYME) announced that its multicenter open-label Phase II TYME-88-Panc study evaluating SM-88 as an oral monotherapy in patients with advanced pancreatic cancer continues to demonstrate encouraging results and a well-tolerated safety profile.

The data from the TYME-88-Panc study were presented at the European Society of Medical Oncology 21st World Congress on Gastrointestinal Cancer.

The Annual meeting is held in Barcelona this year, Stockwinners

Updated results from the ongoing multicenter open-label Phase II TYME-88-Panc study involved 49 heavily pretreated patients with radiographically progressive metastatic pancreatic cancer who had significant disease related morbidity before receiving TYME’s investigational agent SM-88.

More than 80% of patients had received at least two prior lines of therapy. Of the 49 patients, 38 patients were evaluable for efficacy, as defined in the protocol.

Pancreatic cancer, Stockwinners

TYME-88-Panc is a two-part study in which Part 1 was intended to determine optimal dosing and assess if early clinical benefit supported further development of SM-88 in pancreatic cancer.

This study is being performed under a TYME IND with input from the FDA prior to study initiation. In this study, based on information available as of April 25, 2019, the median overall survival of evaluable patients was 6.4 months.

Certain efficacy indicators correlate A RECIST clinical benefit rate of stable disease or better was achieved by 44% of patients with available imaging. Notably, patients achieving stable disease or better demonstrated a statistically significant improvement in survival with a 92% reduction in risk of death.

The CBR was durable with majority of these patients remaining in stable disease or better at more than 7 months after receiving treatment with SM-88.

The measurement of CTCs is emerging as an important prognostic indicator in patients with pancreatic cancer. This is now the second TYME study in cancer patients showing that SM-88 reduces CTCs.

In a previous study of patients with prostate cancer, SM-88 treatment was also associated with a reduction in CTC count. In the TYME-88-Panc study, a median reduction of 63% in CTC burden was observed in evaluable patients. Importantly, patients with available results reaching an 80% reduction or greater in CTCs demonstrated a 60% decrease in risk of death.

In addition to these findings from the TYME-88-Panc study, data were also presented on subgroup analyses. TYME identified several screening criteria that were associated with rapidly declining prognostic factors defined as greater than 2 lines of prior therapy; age greater than 75 years old; albumin less than 3.5 g/dl. Patients with no indicators of poor prognosis had a better trend in survival.

TYME identified key sub-groups of patients who performed better. Patients with 1 or 2 prior lines of therapy had a better trend in survival. Female patients had a statistically significant trend toward better survival. These encouraging findings warrant further clinical evaluation of these subgroups. As of April 25, 2019, the study reported that SM-88 was well tolerated with only 4.0% of patients who experienced serious adverse events deemed at least possibly related to SM-88. One patient with reported SAEs continued on treatment.

The TYME-88-Panc research results are from an investigational study. SM-88 is not approved for the treatment of patients with any disease condition.

TYME is up 30 cents to $1.52.

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Karyopharm Therapeutics shares jump on FDA decision

Karyopharm announces FDA approval of XPOVIO-dexamethasone combination for multiple myeloma

Karyopharm Therapeutics shares soar on FDA approval, Stockwinners

Karyopharm Therapeutics Inc. (KPTI) announced that the U.S. Food and Drug Administration has approved oral XPOVIO, a nuclear export inhibitor, in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.

This indication is approved under accelerated approval based on response rate.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

XPOVIO to become commercially available in the U.S. on or before July 10, 2019, Stockwinners

The ongoing, randomized Phase 3 BOSTON study evaluating selinexor in combination with Velcade and low-dose dexamethasone will serve as the confirmatory trial.

The FDA’s Accelerated Approval Program was developed to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need.

Karyopharm expects XPOVIO to become commercially available in the U.S. on or before July 10, 2019.

A Marketing Authorization Application for selinexor is also currently under review by the European Medicines Agency.

The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Xpovio.

Women who are pregnant or breastfeeding should not take Xpovio because it may cause harm to a developing fetus or newborn baby. Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

The FDA granted this application Fast Track designation. Xpovio also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Executive Changes

In a regulatory filing, Karyopharm disclosed that on July 2, Karyopharm Therapeutics promoted Perry Monaco to Senior Vice President, Sales responsible for the company’s sales function under the direction of Michael Kauffman, Chief Executive Officer of the company.

On July 2, following the buildout of the company’s commercial organization and development of the company’s product launch strategy, Anand Varadan resigned as Executive Vice President, Chief Commercial Officer of the company, effective July 5.

KPTI last traded at $8.15.

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