Watch shares of iCAD

iCAD announces first metastatic brain tumor treated with Xoft Axxent eBx System

iCAD (ICAD) announced the first metastatic brain tumor was treated in the U.S. with intraoperative radiation therapy, or IORT, using the Xoft Axxent Electronic Brachytherapy, or eBx, System.

This procedure is the start of a clinical trial on IORT for patients with large brain metastases treated with neurological resection with the Xoft System.

iCAD reports positive brain tumor data, Stockwinners

The Xoft System is also currently being studied for the treatment of other types of brain tumors in institutions worldwide, including the European Medical Center.

Positive preliminary clinical data on Xoft IORT for the treatment of recurrent glioblastoma, or GBM, was presented at the European Association of Neurosurgical Societies, or EANS.

The Xoft System is made by iCAD, Stockwinners

In a matched pair study, 30 patients were treated for recurrent GBM.

The IORT group was treated with a single fraction of radiation immediately following surgical resection, without chemotherapy or temozolomide following surgery.

The comparison group was treated with routine postoperative adjuvant chemotherapy +/- concomitant or sequential EBRT. Median overall survival, or OS, in group A was 24 months; OS for group B was 21 months.

As of September 2019, nine patients were still alive from group A, whereas none of the patients in group B survived.

A retrospective analysis published in World Neurosurgery examined the repeat resection and the various methods of IORT for the treatment of malignant brain gliomas, including high-energy linear accelerators and modern, integrated brachytherapy solutions using solid and balloon applicators.

iCAD, Inc. provides image analysis, workflow solutions, and radiation therapy for the early identification and treatment of cancer in the United States and internationally. It operates through two segments, Cancer Detection and Cancer Therapy. The company provides electronic brachytherapy (eBX) products, including Axxent eBx systems for the treatment of early stage breast cancer, endometrial cancer, cervical cancer, and skin cancer, as well as for treating other cancers or conditions where radiation therapy is indicated comprising intraoperative radiation therapy.

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Argenx issues positive guidance, shares rise

Argenx says beginning 2020 in an ‘exciting position’

In a regulatory filing, Argenx (ARGX) provided strategic outlook for 2020 outlining key priorities for its broad pipeline and path towards achieving its ‘argenx 2021’ integrated commercial vision.

Argenx issues positive guidance, Stockwinners

“We begin 2020 in an exciting position, having met all our objectives for our clinical programs.

This includes the completion of enrollment of our Phase 3 ADAPT trial of efgartigimod in gMG, the launch of key efgartigimod clinical trials in ITP and CIDP, and the initiation of cusatuzumab clinical trials in two AML settings with Janssen.

In addition, we’re announcing today positive proof-of-concept data for efgartigimod in PV, our third ‘beachhead’ indication, further demonstrating our initial development strategy of targeting pathogenic autoantibodies and creating commercial opportunities in several therapeutic areas.

Looking forward to the remainder of 2020, we plan up to five registrational efgartigimod trials and further expansion of the cusatuzumab global development plan with Janssen,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

“Most importantly, we are continuing to execute on the ‘argenx 2021’ vision to become a global, integrated immunology company with our first launch of efgartigimod in gMG expected in 2021.

At the core of this growth strategy is a commitment to expanding our early-stage pipeline with immunology breakthroughs and advancing our late-stage candidates while extending our reach to bring first-in-class medicines to patients,” continued Van Hauwermeiren.

As part of its 2021 vision, Argenx highlights: leadership in FcRn and its therapeutics immunology potential; launch of MyRealWorld MG; and a “strong” financial foundation. In addition, Argenx reported “positive” proof-of-concept data in PV, the third beachhead indication as part of the broad efgartigimod development strategy.

Argenx has a close relationship with Janssen, Stockwinners

Within its neuromuscular franchise, Argenx is evaluating efgartigimod in gMG and efgartigmod in CIDP; within its hematology/oncology franchise, it is evaluating efgartigimdo in ITP and cusatuzumab in collaboration with Janssen (JNJ).

ARGX shares are up 5.2% to $165.00 in Thursday’s trading.

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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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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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Audentes Therapeutics sold for $3 billion

Astellas to acquire Audentes for $60 per share in cash

Astellas Pharma (ALPMY) and Audentes Therapeutics (BOLD) announced that they have entered into a definitive agreement for Astellas to acquire Audentes at a price of $60.00 per share in cash, representing a total equity value of approximately $3B.

Under the agreement, which has been unanimously approved by the boards of directors of both Astellas and Audentes, Astellas will acquire Audentes through Asilomar Acquisition Corp., a wholly-owned subsidiary of Astellas US Holding, Inc.

Gene Therapy pays off nicely for Audentes Therapeutics, Stockwinners

Asilomar will commence a tender offer for all outstanding shares of common stock of Audentes, for a price of $60.00 per share in cash.

Promptly upon successful completion of the Tender Offer, Asilomar will be merged into Audentes, and any remaining shares of common stock of Audentes will be canceled and converted into the right to receive the same $60.00 per share price.

Astellas pays $3 billion for Audentes Therapeutics, Stockwinners

The board of directors of Audentes has resolved to recommend that Audentes stockholders tender their shares to Astellas. Consummation of the transaction is subject to customary closing conditions, including US antitrust clearance and the tender of a majority of Audentes’ outstanding shares of common stock.

The offer price represents a premium of 110% to Audentes’ closing share price of $28.61 on December 2, 2019.

The all-cash transaction is valued at approximately $3B including the purchase of all common shares, options, restricted stock units and other securities.

The Tender Offer period is expected to commence in the next few weeks and to expire 20 business days after its commencement, unless otherwise extended.

If the Tender Offer conditions are not satisfied, Astellas may be required to extend the Tender Offer under certain circumstances. Astellas is still reviewing the impact of a consummation of the transaction on its financial results for the fiscal year ending March 31, 2020.

Audentes Therapeutics, Inc. focuses on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects.

The company is developing AT132, which is in Phase I/II clinical studies for the treatment of X-linked myotubular myopathy (XLMTM); AT342 that is in Phase I/II clinical studies to treat crigler-najjar syndrome; AT845, which is in preclinical studies for the treatment of pompe disease; and AT307 to treat CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. 

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ChemoCentryx shares soar on data

ChemoCentryx, VFMCRP say pivotal phase-III ADVOCATE trial met primary endpoints

Vifor Fresenius Medical Care Renal Pharma, or VFMCRP, and ChemoCentryx (CCXI) announced positive topline data from the pivotal phase-III ADVOCATE trial of avacopan, an orally administered selective complement 5a receptor inhibitor, for the treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis).

ChemoCentryx shares soar on its ANCA drug, Stockwinners

ANCA vasculitis is an autoimmune disease affecting small blood vessels in the body. It is caused by autoantibodies called ANCAs, or Anti-Neutrophilic Cytoplasmic Autoantibodies.

This global study, in which a total of 331 patients with ANCA vasculitis were enrolled, met both of its primary endpoints, disease remission at 26 weeks and sustained remission at 52 weeks, as assessed by the Birmingham Vasculitis Activity Score, or BVAS.

Remission was defined as a BVAS score of zero and being off glucocorticoid treatment for ANCA vasculitis for at least the preceding four weeks.

BVAS remission at week 26 was achieved in 72.3% of the avacopan treated subjects vs. 70.1% of subjects in the glucocorticoid standard of care control group.

Sustained remission at 52 weeks was observed in 65.7% of the avacopan treated patients vs. 54.9% in the glucocorticoid standard of care control group, achieving both non-inferiority and superiority to glucocorticoid standard of care.

Importantly, subjects who received avacopan experienced additional benefits compared to those in the glucocorticoid standard of care control group.

These benefits, assessed as pre-specified secondary endpoints, include: Significant reduction in glucocorticoid-related toxicity; Significant improvement in kidney function in patients with renal disease at baseline; Improvements in health-related quality of life metrics.

The topline safety results revealed an acceptable safety profile in this serious and life threatening disease with fewer subjects having serious adverse events in the avacopan group than in the glucocorticoid standard of care control group.

A full analysis of the data is underway and expanded results are expected to be announced in the coming weeks. “These results exceed our expectations,” said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx.

“Today we mark the dawn of a new and historic period in the lives of ANCA vasculitis patients. This day we have for the first time demonstrated that a highly targeted therapy aimed at the very center of the ANCA disease process is superior to the traditional approach of broad immune suppression therapy; a therapy which the present findings may make obsolete. Until now ANCA vasculitis patients have had to endure regimens that contain chronic high doses of steroids and all their noxious effects, but with today’s data it is clear that the time of making patients sick with steroid therapy in an attempt to make their acute vasculitis better may at last be over. Working with our partner VFMCRP, we plan to make regulatory submissions for full marketing approval to both the European Medicines Agency and the FDA in 2020.”

CCXI is up $26.68 to $34.65

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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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CRISPR Therapeutics sharply higher on gene editing data

Crispr, Vertex announce interim data from first two patients treated with CTX001

CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Incorporated (VRTX) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials.

Gene Editing shows promises in treating patients, Stockwinners

One patient with transfusion-dependent beta thalassemia received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up.

Stockwinners offers stocks to buy, stocks to watch, upgrades, downgrades, earnings, Stocks to Avoid
Vertex and CRISPR report positive data, Stockwinners

One patient with severe sickle cell disease received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up.

These studies are ongoing and patients will be followed for approximately two years following infusion.

Several additional patients have been enrolled and have had drug product manufactured across the two studies.

The patient with TDT has the beta0/IVS-I-110 genotype and required 16.5 transfusions per year before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion.

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Two serious adverse events occurred, neither of which the principal investigator considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved.

At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells.

The patient with SCD experienced seven vaso-occlusive crises per year before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion.

Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain, all of which resolved.

At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells.

William Blair

The firm upgrades Crispr Therapeutics to Outperform after ‘impressive’ data .

William Blair analyst Raju Prasad upgraded Crispr Therapeutics to Outperform from Market Perform after the company presented initial results from the ongoing Phase I/II trials of CTX001 for the treatment of beta thalassemia and sickle cell disease.

The analyst says today’s data cut was “impressive” as it exceeds the 30% fetal hemoglobin threshold that he viewed as critical.

To date, Crispr has shown initial proof-of-concept in beta thalassemia and sickle cell that exceeded expectations and de-risks its wholly owned immuno-oncology platform, Prasad tells investors in a research note. Further, given the “optionality” of the CRISPR-Cas9 platform and potential cost-effectiveness when compared with lentiviral-based therapies, Crispr could be a potential takeout candidate, adds the analyst.

Stockwinners

Goldman Sachs

Goldman Sachs analyst Salveen Richter raised his 12-month price target for Crispr Therapeutics (CRSP) to $75 from $52 while keeping a Neutral rating on the shares.

The first clinical results from the ongoing CTX001 trial impress, Richter tells investors in a research note. The analyst, who cautions the data represents small patient numbers, is “highly encouraged” with the profile of CTX001 and sees today’s positive data as initial validation of Crispr’s ex-vivo gene editing platform.

Roth Capital

Roth Capital analyst Tony Butler raised his price target for Crispr Therapeutics to $100 from $65 and maintained a Buy rating after the company and partner Vertex Pharmaceuticals (VRTX) announced the first CTX001 early clinical data in two patients.

In a research note to investors, Butler says that though only two patients have been treated with CTX001, these data provide a hint that CRISPR-Cas9 could be curative for hemoglobinopathies, adding that gene-editing is “clearly not a fiction.”

The results utilizing gene-editing with CRISPR-Cas9 to create allogenic CAR-T cells against various cancers may also increase its probability of success based on successful editing in the CLIMB trials, he said.

Cantor Fitzgerald

Cantor Fitzgerald analyst Alethia Young raised her price target for Vertex Pharmaceuticals (VRTX) to $229 from $217 after the company and partner Crispr Therapeutics (CRSP) announced the first CTX001 early clinical data in two patients.

The analyst finds the data “highly encouraging” and thinks this could be an “exciting pipeline program of focus for Vertex by this time next year.” She increased her probability of success for CTX001 to 20% from 10% and reiterates an Overweight rating on shares of Vertex.

Piper Jaffray

Piper Jaffray analyst Edward Tenthoff raised his price target for Crispr Therapeutics (CRSP) to $107 from $100 after the company and partner Vertex Pharmaceuticals (VRTX) reported first-ever CTX001 data on one beta thalassemia and one sickle cell disease patient. Tenthoff is “impressed by these early results showing potential curative effect and look for more patients and longer follow-up next year.” He reiterates an Overweight rating on Crispr Therapeutics.

CRSP is up $10 to $68.55. VRTX is up $5.07 to $215.07

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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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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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Epizyme jumps on $100M investment

Royalty Pharma to purchase future royalties on tazemetostat from Eisai

Royalty Pharma announced that it has agreed to pay $330M to purchase Eisai Co.’s (ESALY) royalties on future worldwide sales of tazemetostat, Epizyme’s (EPZM) lead investigational agent, outside of Japan, and made an equity investment in Epizyme of $100M, with options to invest up to an additional $100M in Epizyme common stock.

In addition, investment funds managed by Pharmakon Advisors agreed to provide $70M in senior-secured loans with the possibility to fund up to $370M over time.

Tazemetostat is a first-in-class, oral EZH2 inhibitor in clinical development for certain oncology indications, including epithelioid sarcoma and follicular lymphoma.

Epizyme receives a $100M investment, Stockwinners

Under a collaboration agreement between Epizyme and Eisai, Epizyme is responsible for the development and worldwide commercialization of tazemetostat (outside of Japan) and Eisai is responsible for the development and commercialization of tazemetostat in Japan.

As part of the agreement, Epizyme owes milestones and royalties on sales of tazemetostat outside of Japan to Eisai, and Eisai owes royalties on sales of tazemetostat in Japan to Epizyme.

Under the terms of its agreement with Eisai, Royalty Pharma has acquired Eisai’s future worldwide royalties on net sales by Epizyme of tazemetostat outside of Japan, for an upfront payment of $110M plus up to an additional $220M for the remainder of the royalty upon FDA approval of tazemetostat for certain indications.

Under the terms of its agreement with Epizyme, Royalty Pharma will make an upfront payment of $100M for shares of Epizyme common stock based on a price of $15 per share.

Royalty Pharma makes $100M investment in Epizyme, Stockwinners

Epizyme has an 18-month option to sell an additional $50M of its common stock to Royalty Pharma at then prevailing prices, not to exceed $20 per share, and Royalty Pharma has a three-year option to purchase an additional 2.5M shares of Epizyme common stock at $20 per share.

In addition, Royalty Pharma will make additional payments to Epizyme if annual net sales of tazemetostat outside of Japan exceed certain thresholds, and Epizyme has assigned to Royalty Pharma the future royalty streams on tazemetostat sales in Japan previously owed to Epizyme by Eisai.

Epizyme has also agreed to appoint a representative from Royalty Pharma to its board of directors. Under the terms of the loan agreement with Epizyme, investment funds managed by Pharmakon Advisors will fund $25M at closing and up to an additional $45M in two tranches.

In addition, the loan agreement contemplates the potential for an additional $300M, subject to mutual agreement of the parties. The loans will have a coupon of LIBOR + 7.75% and a 5-year maturity.

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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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