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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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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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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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.

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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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