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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Sorrento Therapeutics reports positive Parkinson’s disease study, shares rise

Sorrento Therapeutics announces top line results from Parkinson’s disease study


Sorrento Therapeutics announces top line results from Parkinson’s disease study, Stockwinners

Sorrento Therapeutics (SRNE) announced top line results in a discovery study aimed at exploring potential benefits of resiniferatoxin in controlling neuro-inflammatory processes associated with Parkinson’s disease in a rodent model.

Many clinical and pathological studies have shown that the disease extends beyond the substantia nigra and involves non-dopaminergic neurotransmitter systems that mediate both motor and non-motor symptoms.

Several therapeutic strategies have been proposed to treat such symptoms. However, despite the significant morbidity associated with these symptoms, research into and drug development for these problems remain scarce.

Transient receptor potential vanilloid 1, or TRPV1, is involved in pain perception and is highly expressed in sensory neurons. TRPV1 is also present in the brain where it may play a role in modulating neuronal function, controlling motor behavior and regulating neuroinflammation.

TRPV1 receptors are considered an important modulator of basal ganglia functions, and pharmacological changes to cells expressing those receptors might lead to protective therapies in Parkinson-induced motor symptoms.

Intrathecal administration of RTX at 7 days after AAV-A53T resulted in improved motor function analyzed by fine motor kinematic analysis when compared to control mice administered with vehicle for RTX.

No significant adverse effects after RTX infusion were observed. However, no significant effects in dopamine and its metabolites levels in striatum were observed.

The findings demonstrated activity of intrathecal administration of RTX at the early intervention paradigm to rescue motor deficits in AAV-A53T model.

The lack of effect on dopamine levels suggest that behavioral recovery is driven by mechanisms other than direct protection of nigrostriatal dopaminergic neurons, including modulation of neuroinflammation and/or analgesic effect of RTX after IT delivery.

A provisional patent has been filed for non-dopaminergic therapeutic approach with resiniferatoxin for symptomatic relief of motor symptoms associated with Parkinson’s disease.

The full results of the study will be released in publication in a scientific journal and presented at an upcoming neurosciences conference later this year.

Enabling preclinical work will continue with the goal of filing an IND by the end of the year. If results are confirmed, human clinical trials to start soon after. announces top line results from Parkinson’s disease study

Sorrento Therapeutics announced top line results in a discovery study aimed at exploring potential benefits of resiniferatoxin in controlling neuro-inflammatory processes associated with Parkinson’s disease in a rodent model.

Many clinical and pathological studies have shown that the disease extends beyond the substantia nigra and involves non-dopaminergic neurotransmitter systems that mediate both motor and non-motor symptoms.

Several therapeutic strategies have been proposed to treat such symptoms. However, despite the significant morbidity associated with these symptoms, research into and drug development for these problems remain scarce.

Transient receptor potential vanilloid 1, or TRPV1, is involved in pain perception and is highly expressed in sensory neurons.

TRPV1 is also present in the brain where it may play a role in modulating neuronal function, controlling motor behavior and regulating neuroinflammation. TRPV1 receptors are considered an important modulator of basal ganglia functions, and pharmacological changes to cells expressing those receptors might lead to protective therapies in Parkinson-induced motor symptoms.

Intrathecal administration of RTX at 7 days after AAV-A53T resulted in improved motor function analyzed by fine motor kinematic analysis when compared to control mice administered with vehicle for RTX.

No significant adverse effects after RTX infusion were observed. However, no significant effects in dopamine and its metabolites levels in striatum were observed.

The findings demonstrated activity of intrathecal administration of RTX at the early intervention paradigm to rescue motor deficits in AAV-A53T model.

The lack of effect on dopamine levels suggest that behavioral recovery is driven by mechanisms other than direct protection of nigrostriatal dopaminergic neurons, including modulation of neuroinflammation and/or analgesic effect of RTX after IT delivery.

A provisional patent has been filed for non-dopaminergic therapeutic approach with resiniferatoxin for symptomatic relief of motor symptoms associated with Parkinson’s disease. The full results of the study will be released in publication in a scientific journal and presented at an upcoming neurosciences conference later this year.

Enabling preclinical work will continue with the goal of filing an IND by the end of the year. If results are confirmed, human clinical trials to start soon after.

SRNE closed at $4.09.

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Zogenix tumbles on FDA action

Zogenix gets refusal to file letter from FDA on Fintepla NDA


Zogenix gets refusal to file letter from FDA on Fintepla NDA , Stockwinners

Zogenix (ZGNX) announced that it received a Refusal to File, RTF, letter from the U.S. Food and Drug Administration regarding its New Drug Application for FINTEPLA, for the treatment of seizures associated with Dravet syndrome.

Dravet syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in the first year of life with frequent and/or prolonged seizures. Previously known as Severe Myoclonic Epilepsy of Infancy (SMEI), it affects 1:15,700 individuals, 80% of whom have a mutation in their SCN1A gene.

Upon its preliminary review, the FDA determined that the NDA, submitted on February 5, 2019, was not sufficiently complete to permit a substantive review.

In the letter, the FDA cited two reasons for the RTF decision: first, certain non-clinical studies were not submitted to allow assessment of the chronic administration of fenfluramine; and, second, the application contained an incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA.

The FDA has not requested or recommended additional clinical efficacy or safety studies.

The company will seek immediate guidance, including a Type A meeting with the FDA, to clarify and respond to the issues identified in the RTF letter.

“We remain highly confident in FINTEPLA’s clinical profile demonstrated in the Phase 3 program in Dravet syndrome and are committed to advancing the product candidate as a potential new treatment option for this and other rare and often catastrophic epileptic encephalopathies,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix.

“We are fully committed to working with the FDA as quickly as possible to address the open issues and clarify the path to successfully re-filing our application.”

Zogenix’s Marketing Authorization Application, MAA, for FINTEPLA for the treatment of seizures associated with Dravet syndrome was previously accepted for review by the European Medicines Agency, EMA, and the Company anticipates an approvability decision could be reached by the EMA in the first quarter of 2020.

Piper Jaffray

Piper Jaffray analyst Danielle Brill lowered her price target on Zogenix to $68 after it received a refusal to file letter from FDA on its Fintepla new drug application.

The analyst notes that while the issues mentioned by the FDA sound like a simple fix, if the agency requires the company to conduct new toxicity studies, the re-submission could be delayed another 12-15 months.

In spite of the “discouraging news”, longer term, Brill still keeps her Overweight rating on Zogenix and believes that “the company should be able to leverage existing ICH data with fenfluramine” based on their reported prior FDA interactions, modeling a launch in Q2 of 2020.

ZGNX closed at $51.85, it last traded at $35.00.

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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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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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Ascendis Pharma reports positive data, shares jump

Phase 3 heiGHt trial met primary objective in children with pediatric growth hormone deficiency

Ascendis Pharma (ASND) announced positive top-line results from the phase 3 heiGHt Trial, a randomized, open-label, active-controlled trial that compared once-weekly TransCon Growth Hormone to a daily growth hormone in children with pediatric growth hormone deficiency.

Ascendis Pharma reports positive data, shares jump, Stockwinners

The trial met its primary objective, demonstrating that TransCon hGH was observed to be non-inferior and, additionally, superior to the daily hGH on the primary endpoint of annualized height velocity at 52 weeks.

In the primary analysis of the intent-to-treat population using ANCOVA, TransCon hGH demonstrated an AHV of 11.2 cm/year compared to 10.3 cm/year for the daily hGH.

The treatment difference was 0.86 cm/year with a 95% confidence interval of 0.22 to 1.50 cm/year.

The AHV for TransCon hGH was significantly greater than the daily hGH.

The AHV was greater for TransCon hGH than for the daily hGH at each visit, with the treatment difference reaching statistical significance from and including week 26 onward.

The incidence of poor responders was 4% and 11% in the TransCon hGH and daily hGH arms, respectively. All sensitivity analyses completed from the trial support the primary outcome, indicating the robustness of these results.

Results from the trial indicate that TransCon hGH was generally safe and well-tolerated, with adverse events consistent with the type and frequency observed with daily hGH therapy and comparable between arms of the trial.

ASND is up $44.64 to $113.95.

Cantor Fitzgerald analyst Alethia Young raised her price target for Ascendis Pharma to $185 from $100 as she was “positively surprised” that the TransCon hGH study for growth hormone achieved a superior result on annualized growth velocity of .86cm.

The analyst thinks that the expectation had been non-inferiority with a slightly favorable numerical trend. Young also thinks safety was clean as well, which has been a challenge with other long acting growth hormones.

She would expect shares to trade up 50%-100% based on her model, and sees continued upside from shares as additional programs, such as TransCon PTH, achieve proof of concept and more candidates from the proprietary TransCon platform enter the clinic. The analyst reiterates an Overweight rating on the shares.

Growth hormone (GH) has been available for management of the short stature associated with growth hormone deficiency (GHD) for more than 60 years; recombinant DNA-derived human growth hormone (rhGH) has been available since 1985. While availability is no longer a problem, there still remains a number of difficulties with the diagnosis of GHD, resulting mainly from the lack of appropriate tools to make (or exclude) the diagnosis reliably.

The incidence of short stature associated with GHD has been estimated to be about 1:4000 to 1:10000 . It is the primary indication for GH treatment in childhood, which presently requires daily subcutaneous injections for the patient and substantial cost for the healthcare system. Based on these premises, it is clear that an accurate diagnosis is essential

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Verrica Pharmaceuticals reports positive data on its molluscum contagiosum drug

Verrica Pharmaceuticals presents results from Phase 3 clinical trials of VP-102

Verrica Pharmaceuticals reports positive data on its molluscum contagiosum drug, Stockwinners

Verrica Pharmaceuticals (VRCA) presented data from the company’s pivotal Phase 3 CAMP-1 and CAMP-2 trials of lead product candidate, VP-102, at the American Academy of Dermatology annual meeting being held in Washington, DC from March 1-5.

Both trials of VP-102 in patients with molluscum contagiosum successfully met their primary endpoints.

In each trial, a clinically and statistically significant proportion of patients treated with VP-102 demonstrated complete clearance of all treatable molluscum lesions in 12 weeks.

On average, molluscum can take approximately 13 months to resolve without treatment, and in some cases can remain unresolved for several years.

The two randomized, double-blind, multicenter, placebo-controlled trials evaluated the efficacy of dermal application of VP-102 compared to placebo in subjects with molluscum.

In total, the trials enrolled 528 subjects two years of age and older with molluscum at 31 centers in the U.S. Subjects were treated once every 21 days with topical solution of 0.7% cantharidin for up to four applications.

Complete clearance of molluscum lesions was evaluated by assessment of the number of lesions at study visits over 12 weeks. Results from CAMP-1 and CAMP-2 showed 46% and 54% of subjects treated with VP-102, respectively, achieved complete clearance of all treatable molluscum lesions at the end of the trials versus 18% and 13% of subjects in the placebo groups.

By Day 84, VP-102 treated subjects had a 69% and 83% mean reduction in the number of molluscum lesions, a pre-specified endpoint, in CAMP-1 and CAMP-2 respectively, compared to a 20% increase and a 19% reduction for subjects on placebo. VP-102 was well-tolerated in both trials, with no serious adverse events reported in VP-102 treated subjects.

The most frequently reported adverse events were application site reactions that are well-known, reversible side effects related to the mechanism of action of cantharidin, a blistering agent, which is the active ingredient in VP-102.

There were no treatment-related serious adverse events reported in CAMP-1 or CAMP-2. Verrica previously announced topline results from both trials on January 3, 2019.

Based on the positive results, the company plans to submit a New Drug Application for VP-102 in the second half of 2019. If approved, VP-102 would be the first FDA-approved treatment for molluscum contagiosum.


Molluscum contagiosum is a relatively common viral infection of the skin, mainly in children, Stockwinners.com

VRCA closed at $12.11.

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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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Bristol-Myers comments on Celgene’s purchase

Bristol-Myers sees ‘meaningful financial benefits’ from Celgene transaction.

Bristol-Myers treatment for colorectal cancer approved, Stockwinners

Bristol Meyers Comments on Celgene purchase, Stockwinners

Bristol-Myers Squibb (BMY) said an updated its investor presentation about the Celgene (CELG) transaction.

The company said, “The Celgene transaction is the natural next step in Bristol-Myers Squibb’s proven strategy that has consistently delivered results for over a decade.

Through a disciplined approach to driving innovation, focusing on high-value opportunities and sourcing innovation externally to complement its internal portfolio and pipeline, Bristol-Myers Squibb has generated consistently strong growth and increased its dividend for 10 consecutive years.

The combination with Celgene will create a leading biopharma with increased scale, while maintaining the same agility and a focus on delivering for patients in core disease areas of high-unmet medical need.

The pipeline of the combined company provides significant near-, medium- and long-term opportunities for value creation. Bristol-Myers Squibb is acquiring Celgene’s robust and complementary pipeline at an attractive price.

In addition to six expected near-term product launches representing more than $15B in revenue potential, the combination will greatly increase Bristol-Myers Squibb’s Phase I and II assets, which will provide the next set of registrational opportunities in core therapeutic areas.

With an expanded set of scientific platforms and research capabilities, Bristol-Myers Squibb will be well positioned to discover and develop highly innovative medicines and accelerate these new options to patients through one of the highest-performing commercial organizations in the industry.

Bristol-Myers Squibb is well positioned for 2025 and beyond with continued leadership across Oncology and a diversified portfolio of assets.

The combined company will have a broad, balanced and earlier life-cycle marketed portfolio with a significantly higher number of opportunities across multiple diseases to drive the growth of Bristol-Myers Squibb in the second half of the decade. These opportunities will support financial strength for continued investment and innovation.

The Celgene transaction is expected to generate meaningful financial benefits for all stockholders.

With more than $45B of expected free cash flow generation over the first three full years post-closing, the combination will enable rapid debt reduction to de-lever the balance sheet and strengthen Bristol-Myers Squibb’s credit profile.

Bristol-Myers Squibb expects to realize run-rate cost synergies of approximately $2.5B by 2022 from the combination, and the combined company is expected to grow revenue and EPS every year through 2025.”

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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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