AstraZeneca and Merck report prostate cancer data

AstraZeneca, Merck presents results from Phase 3 PROfound trial of LYNPARZA

AstraZeneca (AZN) and Merck (MRK) presented detailed results from the Phase 3 PROfound trial in 387 men with metastatic castration-resistant prostate cancer who have a mutation in their homologous recombination repair genes and whose disease had progressed on prior treatment with new hormonal agent treatments e.g. abiraterone or enzalutamide.

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AstraZeneca and Merck report prostate cancer, Stockwinners

The trial was designed to analyze men with mCRPC harboring HRR-mutated genes in two cohorts: the primary endpoint was in those with mutations in BRCA1/2 or ATM genes and then, if LYNPARZA showed clinical benefit, a formal analysis was performed of the overall trial population of men with HRRm genes.

Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners
AstraZeneca and Merck report prostate cancer , Stockwinners

Results showed a statistically-significant and clinically-meaningful improvement with LYNPARZA in the primary endpoint of radiographic progression-free survival in BRCA1/2 or ATM-mutated tumors reducing the risk of disease progression or death by a median of 7.4 months versus 3.6 months for those receiving abiraterone or enzalutamide.

LYNPARZA reduced the risk of disease progression or death by 66% for these men. The trial also met the key secondary endpoint of rPFS in the overall HRRm population, where LYNPARZA reduced the risk of disease progression or death by 51% and improved rPFS to a median of 5.8 months vs. 3.5 months for those receiving abiraterone or enzalutamide.

In the key secondary endpoint of time to pain progression, median TTPP was not reached with LYNPARZA and was 9.92 months with abiraterone and enzalutamide in patients with BRCA1/2 or ATM mutations.

Results also showed a trend at this interim analysis time point for improvement in overall survival, another key secondary endpoint. LYNPARZA extended OS to a median of 18.5 months versus 15.1 months for abiraterone or enzalutamide in men with BRCA1/2 or ATM-mutated tumors, of which 81% started on abiraterone or enzalutamide and, following confirmed disease progression, then switched to LYNPARZA.

At this interim analysis, the OS endpoint did not meet statistical significance. In an exploratory analysis, a similar trend in OS was observed at this interim analysis in the HRRm population with a median of 17.5 months for men treated with LYNPARZA vs. 14.3 months for those receiving abiraterone or enzalutamide.

The trial showed a confirmed overall response rate a key secondary endpoint of 33.3% for LYNPARZA vs. 2.3% for abiraterone or enzalutamide in patients with BRCA1/2 or ATM mutations.

In an exploratory analysis of patients in the overall HRRm population, confirmed ORR was 21.7 % for LYNPARZA vs. 4.5% for patients receiving abiraterone or enzalutamide. The safety and tolerability profile of LYNPARZA in the PROfound trial was in line with that observed in prior clinical trials.

The most common adverse events greater than or equal to20% for LYNPARZA compared to abiraterone or enzalutamide were anemia, nausea, fatigue and asthenia, decreased appetite, and diarrhea. Grade 3 or above AEs were anemia, fatigue and asthenia, vomiting, dyspnea, urinary tract infection, nausea, decreased appetite, diarrhea, and back pain. AEs led to discontinuation of treatment in 16% of patients on LYNPARZA vs. 9% on abiraterone and enzalutamide.

AstraZeneca and Merck are also exploring additional trials in prostate cancer, including the ongoing Phase 3 PROpel trial, evaluating LYNPARZA as a first-line therapy in mCRPC for patients with or without HRR mutations, in combination with abiraterone acetate.

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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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Array BioPharma sold for $11.4 billion

Pfizer to acquire Array BioPharma for $48.00 per share in cash, or $11.4B

Array BioPharma sold for $11.4 billion, Stockwinners

Pfizer (PFE) and Array BioPharma (ARRY) announced that they have entered into a definitive merger agreement under which Pfizer will acquire Array, a commercial stage biopharmaceutical company focused on the discovery, development and commercialization of targeted small molecule medicines to treat cancer and other diseases of high unmet need.

Array BioPharma Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule drugs to treat patients with cancer and other diseases. It provides BRAFTOVITM (encorafenib) capsules in combination with MEKTOVI (binimetinib) tablets for the treatment of patients with unresectable or metastatic melanoma with a BRAF mutation. The company’s lead clinical programs include encorafenib and binimetinib that are investigated in approximately 30 clinical trials for various solid tumor indications, including a Phase III trial in BRAF-mutant colorectal cancer. Its product pipeline also includes ipatasertib, an AKT inhibitor that is in Phase III trial to treat prostate or breast cancers; selumetinib, a MEK inhibitor for cancer; larotrectinib, a PanTrk inhibitor that is in a Phase II/registration clinical trial for cancer; tucatinib, a HER2 inhibitor for breast cancer, which is in Phase II/registration trial; and ARRY-797 that is in Phase III clinical trial for Lamin A/C-related dilated cardiomyopathy. 

Pfizer has agreed to acquire Array for $48 per share in cash, for a total enterprise value of approximately $11.4B.

The Boards of Directors of both companies have approved the merger. Upon the close of the transaction,

Array’s employees will join Pfizer and continue to be located in Cambridge, Massachusetts and Morrisville, North Carolina, as well as Boulder, Colorado, which becomes part of Pfizer’s Oncology Research & Development network in addition to La Jolla, California and Pearl River, New York.

Pfizer expects to finance the majority of the transaction with debt and the balance with existing cash.

The transaction is expected to be dilutive to Pfizer’s Adjusted Diluted EPS by 4c-5c in 2019, 4c-5c in 2020, neutral in 2021, and accretive beginning in 2022, with additional accretion and growth anticipated thereafter.

Pfizer will provide any appropriate updates to its current 2019 guidance in conjunction with its third quarter 2019 earnings release.

Under the terms of the merger agreement, a subsidiary of Pfizer will commence a cash tender offer to purchase all outstanding shares of Array common stock for $48 per share in cash for a total enterprise value of approximately $11.4B.

The closing of the tender offer is subject to customary closing conditions, including regulatory approvals and the tender of a majority of the outstanding shares of Array common stock.

The merger agreement contemplates that Pfizer will acquire any shares of Array that are not tendered into the offer through a second-step merger, which will be completed promptly following the closing of the tender offer.

Pfizer expects to complete the acquisition in the second half of 2019.

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Bempegaldesleukin data should send Nektar shares higher

Nektar presents biomarker, clinical data from PIVOT-02 Phase 2 study

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Nektar presents biomarker, clinical data from PIVOT-02 Phase 2 study, Stockwinners

Nektar Therapeutics (NKTR) announced that biomarker and clinical data from PIVOT-02 was presented at the 2019 American Society of Clinical Oncology Meeting in Chicago, Illinois.

#Bempegaldesleukin is an investigational, CD122-preferential IL-2 pathway agonist designed to provide sustained signaling through the IL-2 beta-gamma receptor.

Bempegaldesleukin is under investigation in clinical trial NCT03729245 (A Study of NKTR-214 in Combination With Nivolumab Compared With the Investigator’s Choice of a Tyrosine Kinase Inhibitor (TKI) Therapy (Either Sunitinib or Cabozantinib Monotherapy) for Advanced Metastatic Renal Cell Carcinoma (RCC)).

PIVOT-02 is an ongoing Phase 2 study evaluating bempeg in combination with nivolumab in solid tumors.

Exploratory biomarker analyses of PIVOT-02 baseline tumor biopsies identified immune signatures that potentially enrich for response in patients with 1L metastatic melanoma and not 1L metastatic urothelial carcinoma.

Notable response rates were seen in both 1L metastatic melanoma and 1L metastatic urothelial cancer patients, regardless of PD-L1 status or unfavorable tumor microenvironments.

At a median time of follow-up of 12.7 months, confirmed objective response rate was 53% in efficacy-evaluable patients, with 34% of patients achieving confirmed complete responses. 42% of patients achieved a maximum reduction of 100% in target lesions. DCR, also known as disease control rate was 74%.

Median time to response was 2 months. Median duration of response was not reached. At the 12.7 month median follow-up, data were too immature to calculate median progression-free survival. 80% of patients with responses have ongoing responses. Amongst the 35 patients with known pre-treatment PD-L1 status, ORR in PD-L1 negative patients was 6/14 and in PD-L1 positive patients was 13/21.

One of three patients with unknown PD-L1 baseline status experienced a CR.

A total of 6/41 of patients experienced a Grade 3 or higher TRAE with 4/41 patients discontinuing treatment due to a TRAE. A total of 41 patients have been treated at the RP2D with 3 patients discontinuing prior to 1st scan due to an unrelated treatment-emergent adverse event and patient decision.

A Phase 3 trial evaluating bempeg in combination with nivolumab versus nivolumab in first-line advanced melanoma patients is currently recruiting patients.

A Phase 2 pivotal trial evaluating bempeg in combination with nivolumab in first-line metastatic urothelial cancer is currently recruiting patients.

 Piper Jaffray

Piper Jaffray analyst Tyler Van Buren reiterates an Overweight rating and $100 price target on Nektar, and believes shares should be up significantly on Monday following updated data from the bempegaldesleukin + nivo PIVOT-02 melanoma cohort.

The increase in complete response rate from 24% to 34% at just beyond 12 months is more than the analyst and investors were anticipating and “the waterfall plot is like nothing [he has] ever seen in solid tumors.”

Ultimately, Van Buren believes the high quality of responses and the ability to maintain patients on therapy is contributing to robust durability, which increases his confidence in the ultimate outcome of the Phase III trial which will have a final mPFS evaluation around Q3 of 2020.

NKTR closed at $31.32.

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Incyte to present at ASCO meeting in Chicago

Incyte announces abstracts featuring genomic profiling data for ASCO

Incyte Corp. (INCY) announces that multiple abstracts highlighting data from its oncology portfolio will be presented at the upcoming 2019 American Society of Clinical Oncology Annual Meeting, to be held from May 31-June 4, in Chicago, Illinois; and the 24th Congress of the European Hematology Association, to be held June 13-16, in Amsterdam, the Netherlands.

Abstracts accepted for presentation at ASCO feature genomic profiling data from Incyte’s ongoing Phase 2 FIGHT-202 trial evaluating its selective fibroblast growth factor receptor inhibitor, pemigatinib, in patients with cholangiocarcinoma, as well as efficacy and safety data from the Novartis-sponsored GEOMETRY mono-1 trial of capmatinib, the investigational selective MET inhibitor licensed to Novartis (NVS) by Incyte.

Additionally, data to be presented at EHA will showcase the continued study of Incyte’s JAK1/JAK2 inhibitor, #ruxolitinib, in myeloproliferative neoplasms.

“Our presence at ASCO and EHA illustrates Incyte’s ongoing commitment to discovering and developing therapeutic options that address significant unmet medical needs for patients,” said Steven Stein, M.D., Chief Medical Officer, Incyte.

ASCO’s annual meeting is May 31-June 4 in Chicago, Stockwinners

“We are pleased to highlight new data on two investigational medicines – pemigatinib and capmatinib – that were discovered by Incyte scientists and for which we anticipate applications for initial U.S. regulatory approvals later this year, as well as data that furthers our understanding of the treatment of MPNs.”

Incyte Corporation focuses on the discovery, development, and commercialization of various therapeutics in the United States. The company offers JAKAFI, a drug for the treatment of myelofibrosis and polycythemia vera cancers; and Iclusig, a kinase inhibitor to treat chronic myeloid leukemia and philadelphia-chromosome positive acute lymphoblastic leukemia.

Its clinical stage products include ruxolitinib, a drug in Phase III clinical trial for steroid-refractory acute graft-versus-host-diseases (GVHD); and Phase II trial for the treatment of essential thrombocythemia and refractory myelofibrosis.

In addition, the company engages in the development of itacitinib, which is in Phase III clinical trial to treat naïve acute and chronic GVHD, as well as Phase I/II clinical trial in combination with osimertinib for non-small cell lung cancer (NSCLC); and pemigatinib that is in Phase II clinical trial for treating bladder cancer, cholangiocarcinoma, and 8p11 myeloproliferative syndrome, as well as a pivotal program for solid tumors with driver activations of FGF/FGFR.

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Mustang Bio soars on its gene therapy data

NEJM reports ‘medical breakthrough’ in Mustang Bio cell and gene therapy

Mustang Bio (MBIO) announced that the New England Journal of Medicine has published data from St. Jude Children’s Research Hospital, the nation’s “leading hospital” for understanding, treating and curing childhood cancer and other life-threatening diseases.

Mustang Bio soars on its gene therapy data, Stockwinners

The data comes from a Phase 1/2 clinical trial of a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID, also referred to as SCID-X1 and commonly known as “bubble boy disease.”

Under a licensing agreement with St. Jude, Mustang will develop the lentiviral gene therapy for commercial use as MB-107.

The multi-center Phase 1/2 clinical trial is evaluating the safety and efficacy of a lentiviral vector to transfer a normal copy of the IL2RG gene to bone marrow stem cells in newly diagnosed infants under the age of two with XSCID, preceded by low exposure-targeted busulfan conditioning.

A total of 10 infants have received the therapy to date in this clinical trial. Among the data highlights, bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.

In seven of the eight cases, normalization of naive T-cell and natural killer cell numbers occurred within three to four months after treatment, accompanied by vector marking in T, B, NK and myeloid cells and marrow progenitors.

All patients cleared previous infections and are growing normally. Seven of the eight infants treated have developed normal IgM levels to date.

Most patients were discharged from the hospital within one month.

Data Highlights:

  • Bone marrow harvest, busulfan conditioning and cell infusion were well tolerated.
  • In seven of the eight cases, normalization of CD3+, CD4+ and CD4+ naïve T-cell and natural killer (“NK”) cell numbers occurred within three to four months after treatment, accompanied by vector marking in T, B, NK and myeloid cells and marrow progenitors.
    • The eighth infant had insufficient T cells initially, but normalization of T cells occurred following an unconditioned boost of gene-corrected cells, and the patient is progressing favorably.
  • All patients cleared previous infections and are growing normally.
  • Seven of the eight infants treated have developed normal IgM levels to date.
    • Four of these seven infants have discontinued monthly infusions of intravenous immunoglobulin (IVIG) therapy to date.
    • Three of those four infants that discontinued monthly IVIG infusions have responded to vaccines to date.

MBIO closed at $2.66, it last traded at $8.91.

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Bio-Path Holdings presents positive pancreatic cancer data

Bio-Path Holdings presents BP1003 data at AACR

Bio-Path Holdings presents positive pancreatic cancer data, Stockwinners

Bio-Path Holdings (BPTH) announced that data from pre-clinical studies supporting the potential of BP1003, a liposome-incorporated STAT3 oligodeoxynucleotide inhibitor, for the treatment of pancreatic cancer, non-small cell lung cancer, or NSCLC, and acute myelogenous leukemia, or AML, were presented in a poster at the American Association for Cancer Research, or AACR.

The poster highlights four antisense oligo sequences directed against STAT3 mRNA identified by Bio-Path and manufactured using DNAbilize antisense RNAi nanoparticle technology.

Cell viability tests and western blots were conducted to determine the inhibitory effects of liposome-incorporated STAT3 antisense oligo on NSCLC and AML cells.

An ex vivo live tissue sensitivity assay was performed with a panel of 20 pancreatic ductal adenocarcinoma patient-derived xenografts to study the overall activity of BP1003 alone, and in combination with gemcitabine.

Using previously defined criteria, tissue slice viability inhibition greater than 30% and with a less than 0.05 value was considered to be a response.

For validation of ex vivo results, tumor bearing mice were administered BP1003 and gemcitabine twice a week for 28 days. Tumor volumes were monitored for up to 49 days.

The most potent liposome-incorporated STAT3 antisense sequence in decreasing NSCLC cell viability was selected as the drug candidate BP1003.

Further validation in AML cells demonstrated that BP1003 inhibited cell viability and STAT3 protein expression. In the ex vivo LTSA assay, BP1003 at a dose of 10 microM significantly inhibited the tissue slice viability in 9 out of 18 PDAC PDXs by more than 30%.

The combination of BP1003 and gemcitabine further enhanced ex vivo efficacy of BP1003 in a subset of PDXs.

In the in vivo study, a combination of BP1003 and gemcitabine caused tumor regression during the 28-day drug treatment period.

This anti-cancer activity was maintained for another 21 days, even when drug treatment had ceased.

Preclinical pancreatic cancer models demonstrated that BP1003 successfully penetrated the stroma into pancreatic tumors.

Finally, the results in pancreatic cancer showed that BP1003 inhibited tumor slice viability in nine of 18.

BPTH closed at $19.68, it last traded at $20.61.

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Advaxis jumps on its prostate cancer drug data

Advaxis says ADXS-PSA in combination with KEYTRUDA prolonged survival in mCRPC

Advaxis jumps on its prostate cancer drug data, Stockwinners

Advaxis (ADXS) announced updated data from the Phase 1/2 KEYNOTE-046 study in metastatic, castration-resistant prostate cancer.

Castrate-resistant prostate cancer refers to prostate cancer that keeps growing even when the amount of testosterone in the body is reduced to very low levels. Many early-stage prostate cancers need normal levels of testosterone to grow, but castrate-resistant prostate cancers do not. 

Androgens are required for normal growth and function of the prostate. Androgens are also necessary for prostate cancers to grow. Androgens promote the growth of both normal and cancerous prostate cells by binding to and activating the androgen receptor, a protein that is expressed in prostate cells Once activated, the androgen receptor stimulates the expression of specific genes that cause prostate cells to grow .

Early in their development, prostate cancers need relatively high levels of androgens to grow. Such prostate cancers are called castration sensitive, androgen dependent, or androgen sensitive because treatments that decrease androgen levels or block androgen activity can inhibit their growth.

This trial is being conducted in conjunction with Merck (MRK) and is evaluating ADXS-PSA, one of Advaxis’ Listeria monocytogenes-based immunotherapies, alone and in combination with KEYTRUDA, Merck’s anti-PD-1 therapy.

Findings will be highlighted in a poster discussion entitled “Effects of ADXS-PSA with or without Pembrolizumab on Survival and Antigen Spreading in Metastatic, Castration-Resistant Prostate Cancer Patients” at the American Association for Cancer Research Annual Meeting underway in Atlanta.

KEYNOTE-046 is an open-label, multicenter, dose-determining safety and tolerability Phase 1/2 trial of 50 heavily pretreated patients conducted in two parts, with a Phase 2 expansion cohort. The objective of the study is to evaluate ADXS-PSA alone and in combination with KEYTRUDA for primary endpoints that include safety, tolerability and dosing.

Secondary endpoints include anti-tumor activity and progression-free survival, and exploratory endpoints include associations between biomarkers of immunologic response with clinical outcomes.

Key findings from the combination arm of KEYNOTE-046 include the following: The majority of treatment-related adverse events consisted of transient and reversible Grade 1-2 chills/rigors, fever, hypotension, nausea and fatigue.

The combination of ADXS-PSA and pembrolizumab has been well-tolerated, to date, with no additive toxicity observed. Median overall survival was 21.1 months at data cutoff in this dataset of 37 patients.

Correlative immune analyses showed T-cell responses against PSA in 75% of subjects and antigen spreading in 85% of subjects. Broader immune stimulation, including B-cell activation, was observed in the combination arm than in the ADXS-PSA monotherapy arm.

ADXS closed at $5.91, it last traded at $8.15. MRK closed at $83.17.

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Dare Bioscience posts positive data, shares jump

Dare Bioscience announces publication of clinical findings for DARE-VVA1

Dare Bioscience publishes positive data on tamoxifen, Stockwinners

Dare Bioscience (DARE) announced the publication of clinical findings for vaginally-administered tamoxifen in Clinical and Experimental Obstetrics and Gynecology, an international journal for publication of research focused on the development of new therapeutic interventions for obstetrics and gynecology.

Dare’s product candidate, DARE-VVA1, incorporates tamoxifen in a proprietary formulation designed for vaginal delivery.

Dare holds the exclusive worldwide rights to patents issued in the U.S. and Japan covering the use and delivery of DARE-VVA1 for vulvar and vaginal atrophy, or VVA, and a U.S. patent covering composition, use and delivery of DARE-VVA1 for VVA.

The publication reported that a self-administered vaginal suppository containing tamoxifen, dosed daily for one week followed by twice weekly for three months, administered to four healthy postmenopausal women with VVA showed significant improvements in reducing vaginal pH and vaginal dryness without significant systemic absorption of tamoxifen.

This exploratory study demonstrated that tamoxifen was effective when delivered intravaginally for three months in postmenopausal women suffering with VVA.

The median vaginal pH at the time of enrollment was 7.1. At the end of month 3, the median vaginal pH was 5.0. The median paired difference between baseline and month 3 was -2.0, with a range of -2.5 to -1.5.

The self-assessment of vaginal dryness improved between baseline and month 3. Vaginal dryness was rated using a visual analogue scale, or VAS, that ranged from 0 to 10.


Tamoxifen commonly is used as a breast cancer treatment

At baseline, the median vaginal dryness rating was 8.0, with a range of 7.5 to 9.0. At the end of month 3, the median vaginal dryness rating was 3.0, with a range of 2.0 to 3.0. The median change between baseline and month 3 was -5.5.

In addition, systemic exposure was at least an order of magnitude lower following vaginal administration compared with oral tamoxifen. After eight weeks of study treatment, median plasma concentration of tamoxifen was 5.8 ng/ml, with a range of 1.0 to 10.0 ng/ml.

In comparison, after three months of oral administration of 20-mg tamoxifen once daily, the average steady state plasma concentration of tamoxifen is 122 ng/ml, with a range of 71 to 183 ng/ml. VVA is an inflammation of the vaginal epithelium due to the reduction in levels of circulating estrogen. Historically, estrogen-based therapies delivered through creams, rings and tablet supplements have been prescribed for the treatment of VVA symptoms.

However, estrogen-based products can be worrisome for women undergoing treatment for hormone-receptor positive breast cancer and are often contraindicated in such breast cancer patients and in patients with a genetic predisposition or history of familial disease, because of the concern that estrogen use will promote recurrence of disease.

Many breast cancer survivors undergo menopausal symptoms as a direct consequence of cancer treatment.

Breast cancer patients treated with aromatase inhibitors refer to VVA as one of the most unpleasant side effects of treatment. Tamoxifen has been a commonly used treatment for breast cancer and is systemically metabolized to active metabolite 4-hydroxy-N-desmethyl-tamoxifen, otherwise known as endoxifen. In breast tissue, tamoxifen acts as an estrogen antagonist.

In other tissue, including vaginal tissue, tamoxifen has been reported to exert an estrogen-like response on vaginal cytology by a mechanism yet to be understood and not expected based upon its an anti-estrogen activity.

This exploratory study demonstrated that vaginal administration of tamoxifen for three months in postmenopausal women with VVA is a possible new, non-estrogen-based treatment approach.

Dare is currently conducting activities in preparation for future clinical work with DARE-VVA1, its proprietary vaginal formulation of tamoxifen.

If successful, DARE-VVA1 could be the first and only vaginally administered tamoxifen product approved by the FDA for the treatment of VVA in hormone-receptor positive breast cancer patients.

DARE closed at $0.90, last traded at $2.70.

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Axovant higher on data

Axovant jumps following update on Tay-Sachs Disease gene therapy program

Axovant higher on gene data, Stockwinners

Shares of Axovant (AXGT) have surged higher today following the company’s report of three-month data from an investigator-initiated study administering investigational AXO-AAV-GM2 gene therapy in a patient with advanced infantile Tay-Sachs disease, a rare and fatal pediatric eurodegenerative genetic disorder.

Tay-Sachs disease (TSD) is a fatal genetic disorder, most commonly occurring in children, that results in progressive destruction of the nervous system. Tay-Sachs is caused by the absence of a vital enzyme called hexosaminidase-A (Hex-A). Without Hex-A, a fatty substance, or lipid, called GM2 ganglioside accumulates abnormally in cells, especially in the nerve cells of the brain. This ongoing accumulation causes progressive damage to the cells.

In children, the destructive process begins in the fetus early in pregnancy. However, a baby with Tay-Sachs disease appears normal until about six months of age when its development slows. By about two years of age, most children experience recurrent seizures and diminishing mental function. The infant gradually regresses, and is eventually unable to crawl, turn over, sit or reach out. Eventually, the child becomes blind, cognitively impaired, paralyzed and non-responsive. By the time a child with Tay-Sachs is three or four years old, the nervous system is so badly affected that death usually results by age five.

The study is evaluating a total dose of 1.0x 1014 vg of AXO-AAV-GM2 in a 30-month-old child with advanced infantile Tay-Sachs disease.

AXO-AAV-GM2 was administered into the cisterna magna and lumbar spinal canal only.

Due to the patient’s advanced disease, a co-delivered intrathalamic injection of AXO-AAV-GM2 was not administered.

Future patients in the program, who are expected to be treated earlier in their disease course, will receive AXO-AAV-GM2 co-delivered into the thalamus bilaterally as well as into the cisterna magna and spinal canal.

“AXO-AAV-GM2 was generally well-tolerated and no serious adverse events have been reported as of the 3-month visit. At 3 months, no clinically relevant laboratory abnormalities were observed following AXO-AAV-GM2 administration.

The patient’s clinical condition was stable from baseline to month 3 without clinical deterioration observed on neurological exam.

Furthermore, there was no significant deterioration in the condition from the pre-treatment magnetic resonance imaging of the brain at baseline to the post-treatment MRI at month 3,” the company announced in press release earlier this morning.

Additionally, Chardan has upgraded Axovant to Buy from Neutral following the data report.

In morning trading, Axovant shares are up 46c, or 31%, to $1.94.

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Spark Therapeutics sold for $4.8 billion

Roche acquires Spark Therapeutics for $114.50 per share, a 122% premium

Spark tumbles on hemophilia study , Stockwinners

Roche acquires Spark Therapeutics for $114.50 per share , Stockwinners

Spark Therapeutics (ONCE) announced that it has entered into a definitive merger agreement for Roche (RHHBY) to fully acquire Spark Therapeutics at a price of $114.50 per share in an all-cash transaction.

This corresponds to a total equity value of approximately $4.8B on a fully diluted basis, inclusive of approximately $500M of projected net cash expected at close.

The per share price represents a premium of 122% to Spark’s closing price on Feb. 22, 2019. The merger agreement has been unanimously approved by the boards of both Spark and Roche.

Under the terms of the merger agreement, Roche will commence a tender offer to acquire all outstanding shares of Spark’s common stock, and Spark will file a recommendation statement containing the unanimous recommendation of the Spark board that Spark shareholders tender their shares to Roche.

Spark Therapeutics will continue its operations in Philadelphia as an independent company within the Roche Group.

The closing of the transaction is expected to take place in Q2 of 2019.

Bernstein analyst Vincent #Chen notes that he has long seen Spark Therapeutics (ONCE) as a takeout candidate in gene therapy after the Wall Street Journal reported Roche (RHHBY) is near acquiring the company for close to $5B.

The analyst thinks the deal makes sense as adding hemophilia gene therapy to Hemlibra sets up Roche as a leader in next-gen non-factor hemophilia drugs, CNS gene therapy pipeline is a good fit for Roche’s growing neuroscience franchise, and as it serves as a cornerstone in establishing Roche as a gene therapy leader, to rival the likes of Novartis (NVS).


Credit Suisse analyst Martin Auster notes that the Wall Street Journal recently reported that Roche (RHHBY) is nearing a deal with Spark Therapeutics (ONCE) for $5B.

The analyst believes the transaction value implies the re-emergence of Spark’s hemophilia A gene therapy program with a competitive profile and substantial value assigned to additional pipeline programs, such as SPK-3006 for Pompe, and the technology platform.

Spark’s acquisition would further strengthen the company’s position in the hemophilia space, he contends. Further, Auster argues that the deal may increase M&A interest among other gene therapy names in his coverage universe, which include BioMarin (BMRN), Sarepta (SRPT), Ultragenyx (RARE), PTC (PTC) and Solid Biosciences (SLDB). He sees particularly strong read-through to Sarepta.


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Achieve Life Sciences higher on smoking data

Achieve Life Sciences announces final data from cytisinicline study

Achieve Life Sciences (ACHV) announced final data from their Phase I/II multi-dose, pharmacokinetic and pharmacodynamics, or PK/PD, clinical study of cytisinicline in smokers.

The study evaluated the repeat-dose PK and PD effects of 1.5 mg and 3 mg cytisinicline in 26 healthy volunteer smokers when administered over the standard 25-day course of treatment.

Smokers in the study had a mean age of 39 years, smoked on average 17.2 cigarettes a day, and were not required to quit smoking or have a predetermined quit date while on study.

All subjects had a significant and immediate reduction in cigarettes smoked within 2 days of initiating cytisinicline treatment.

By Day 26, subjects had an average 80% reduction in cigarettes smoked, 82% reduction in expired carbon monoxide, and 46% had stopped smoking.

The biochemically verified smoking cessation rates were 39% and 54% in the 1.5 mg and 3.0 mg cytisinicline treated groups, respectively.

The PK results indicated expected increases in plasma concentration between the standard 1.5 mg and higher 3.0 mg doses of cytisinicline with no evidence of drug accumulation.

Cytisinicline at either dose was well tolerated with only transient, mild-to-moderate headache as the most common adverse event, which was not treatment limiting. No adverse events were severe, serious, or led to withdrawal from the study.

Cindy Jacobs, CMO at Achieve commented, “Given the short 25-day treatment period, the abstinence rates observed are impressive, particularly since subjects were not required to commit to quitting and received minimal behavioral support during the study. These results continue to support our belief that cytisinicline could be a well-tolerated and effective potential treatment option for the millions of people who are battling nicotine addiction.”

ACHV closed at $1.81, it last traded at $2.88.

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Sarepta gene therapy showed functional improvements

Sarepta gene therapy showed functional improvements across all measures 

Sarepta drops after U.K. trial halt,
Sarepta gene therapy showed functional improvements

Sarepta Therapeutics (SRPT) announced  at the 23rd International Congress of the World Muscle Society in Mendoza, Argentina, Jerry Mendell, M.D., of Nationwide Children’s Hospital presented updated results from its gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy.

Dr. Mendell presented the following updated data on the four patients enrolled in the study: All patients showed robust expression of transduced micro-dystrophin, which is properly localized to the muscle sarcolemma, as measured by immunohistochemistry.

Mean gene expression for the study, as measured by percentage of micro-dystrophin positive fibers was 81.2% and the mean intensity of the fibers was 96.0% compared to normal control.

All post-treatment biopsies showed robust levels of micro-dystrophin as measured by Western blot, with a mean of 74.3% compared to normal utilizing Sarepta’s method, or 95.8% compared to normal pursuant to Nationwide Children’s quantification of Sarepta’s method that adjusts for fat and fibrotic tissue.

Gene expression for the fourth patient was robust, as follows: As measured by immunohistochemistry, micro-dystrophin positive fibers was 96.2% and the mean intensity of the fibers was 160.0% compared to normal control. As measured by Western blot, patient 4 showed robust levels of micro-dystrophin, with a mean of 182.7% compared to normal utilizing Sarepta’s method, or 222% compared to normal pursuant to Nationwide Children’s quantification of Sarepta’s method that adjusts for fat and fibrotic tissue.

In all patients, expression of micro-dystrophin was associated with significant expression and up-regulation of the dystrophin-associated protein complex, an additional indication of functionality of dystrophin.

All patients showed significant decreases of serum creatine kinase levels at last measure, with a mean reduction of CK of over 78% from baseline.

Doug Ingram, Sarepta’s president and CEO, said, “The encouraging results that we previously saw and reinforced in the fourth patient strengthen our resolve to rapidly move to a confirming trial and, assuming successful, to bring this therapy to the Duchenne community around the world with a sense of urgency.”

SRPT closed at $147.38; it last traded at $152.50.


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