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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Zosano reports positive migraine data, Shares jump

Zosano says Qtrypta long-term study shows ‘well-tolerated safety profile’

Zosano says Qtrypta long-term study shows 'well-tolerated safety profile', See Stockwinners for more

Zosano says Qtrypta long-term study shows ‘well-tolerated safety profile’ , Stockwinners

Zosano Pharma (ZSAN) announced earlier today the completion of the second and final goal of the long-term safety study for Qtrypta, in which patients treated migraine attacks over a one year period.

“The long-term data generated in this trial reinforced the well-tolerated safety profile and strong efficacy results previously reported in the six-month dosing portion of this safety study and in the randomized Phase 2/3 ZOTRIP pivotal study,” the company said in a statement.

Throughout the clinical program, over 5,800 migraine attacks have been treated with Qtrypta to date, it added.

The Qtrypta long-term safety trial is an open-label study evaluating the safety of the 3.8 mg dose of intracutaneous zolmitriptan in adults with migraine who have historically experienced at least two migraine attacks per month.

The study evaluated over 150 adults with migraine disease for six months, and more than 50 patients for a year at 31 sites in the U.S.

Of more than 5,800 migraines treated, investigators reported 832 adverse events, of which 298 were reported as application site reactions and 161 were reported as triptan related adverse events.

Observational efficacy parameters continued to demonstrate a rate of pain freedom at two hours following patch application of approximately 44% and most bothersome symptom freedom of approximately 68%, while pain relief at two hours was reported at 81% of migraine attacks treated, said Zosano.

The company expects to file an New Drug Application for Qtrypta in the fourth quarter of 2019.

ZSAN is up 95% to $4.41.

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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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Clovis Oncology is in focus

Clovis receives Breakthrough Therapy Designation for Rubraca

CLVS to submit NDA to FDA
Clovis is in focus

 

Clovis Oncology (CLVS) announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation for Rubraca as a monotherapy treatment of adult patients with BRCA1/2-mutated mCRPC who have received at least one prior androgen receptor-directed therapy and taxane-based chemotherapy.

Breakthrough Therapy designation is granted by the FDA to investigational agents intended to treat a serious or life-threatening disease or condition and whose preliminary clinical evidence may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy.

The FDA previously granted Breakthrough Therapy designation to Rubraca for the monotherapy treatment of certain advanced ovarian cancer patients and then in December 2016 approved Rubraca for the treatment of certain adult patients with deleterious BRCA mutation associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies.

The FDA subsequently approved Rubraca in a second indication, the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy, in April 2018.

This most recent Breakthrough Therapy designation was granted to Rubraca based on initial efficacy and safety results from TRITON2, the Phase 2 study of Rubraca in men with advanced prostate cancer with BRCA 1/2 mutations and deleterious mutations of other homologous recombination repair genes, in the metastatic castration-resistant setting.

JP Morgan Comments

JPMorgan analyst Cory Kasimov is encouraged by Clovis Oncology’s announcement this morning that rucaparib received breakthrough designation from the FDA for the third line treatment of patients with BRCA mutated metastatic castrate-resistant prostate cancer on the basis of data from the Triton-2 study.

The analyst says the news further increases his confidence in the potential for rucaparib to produce response rates that are meaningfully better than currently available options.

His model implies $9 per share for prostate, assuming a 55% probability of success and $450M in peak unadjusted sales. #Kasimov keeps an Overweight rating on Clovis.

CLVS closed at $29.14, it last traded at $31.50.


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Amarin sharply higher on data

Amarin soars after REDUCE-IT study meets primary endpoint

Amarin sharply higher on data, Stockwinners
Amarin sharply higher on data, Stockwinners

Amarin (AMRN) announced topline results from the Vascepa cardiovascular outcomes trial, REDUCE-IT, a global study of 8,179 statin-treated adults with elevated CV risk.

REDUCE-IT met its primary endpoint demonstrating an approximately 25% relative risk reduction, to a high degree of statistical significance, in major adverse CV events in the intent-to-treat patient population with use of Vascepa 4 grams/day as compared to placebo, Amarin said in a statement.

Patients enrolled in REDUCE-IT had LDL-C between 41-100 mg/dL controlled by statin therapy and various cardiovascular risk factors including persistent elevated triglycerides between 150-499 mg/dL and either established cardiovascular disease or diabetes mellitus and at least one other CV risk factor. Key topline results include approximately 25% relative risk reduction, demonstrated to a high degree of statistical significance, in the primary endpoint composite of the first occurrence of MACE, including cardiovascular death, nonfatal myocardial infarction, nonfatal stroke, coronary revascularization, or unstable angina requiring hospitalization.

This result was supported by robust demonstrations of efficacy across multiple secondary endpoints, the company said.

It added that Vascepa was well tolerated with a safety profile consistent with clinical experience associated with omega-3 fatty acids and current FDA-approved labeling.

The proportions of patients experiencing adverse events and serious adverse events in REDUCE-IT were similar between the active and placebo treatment groups.

Median follow-up time in REDUCE-IT was 4.9 years. Amarin said it is “eager to share REDUCE-IT data in greater detail with both the medical community and regulatory authorities.”

REDUCE-IT results have been accepted for presentation at the 2018 Scientific Sessions of the American Heart Association on November 10, 2018 in Chicago, Illinois.

“We are delighted with these topline study results,” said John Thero, president and CEO of Amarin.

“Given Vascepa is affordably priced, orally administered and has a favorable safety profile, REDUCE-IT results could lead to a new paradigm in treatment to further reduce the significant cardiovascular risk that remains in millions of patients with LDL-C controlled by statin therapy, as studied in REDUCE-IT.”

It notes, “As previously described, given the successful topline results of REDUCE-IT, Amarin is in the process of increasing the number of company sales representatives promoting Vascepa to over 400 people in the United States.”

Shares of Amarin (AMRN) closed at $2.99, it last traded at $12.30.


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Today’s stock disaster

GTx announces placebo-controlled ASTRID trial did not meet primary endpoint

GTx Phase 2 trial of enobosarm meets primary endpoint. Stockwinners.com
Today’s stock disaster

GTx (GTXI) announced that the ASTRID Trial, a Phase 2 double-blind, placebo-controlled clinical trial of orally-administered enobosarm in post-menopausal women with stress urinary incontinence, did not achieve statistical significance on the primary endpoint of the proportion of patients with a greater than 50% reduction in incontinence episodes per day compared to placebo.

The percentage of patients with a greater than 50% reduction after 12 weeks of enobosarm treatment was 58.9% for 3mg, 57.7% for 1mg and 52.7% for placebo.

Enobosarm was generally safe and well tolerated.

Reported adverse events were minimal and similar across all treatment groups.

“We are very disappointed that the ASTRID Trial did not achieve its primary endpoint,” said Robert Wills, executive chairman of GTx.

“We plan to conduct a full review of all the data. We want to thank the patients, physicians, study coordinators and the entire GTx team for their support of this novel study. We have an ongoing preclinical program assessing the potential of SARDs, our novel selective androgen receptor degrader technology, to treat castration-resistant prostate cancer. We are currently on target to have development candidates by year end, which we potentially plan to take into IND-enabling studies.”

GTXI closed at $23.29, it last traded at $2.00.


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Incyte reports positive data

Incyte announces Phase 2b trial of ruxolitinib cream met primary endpoint

Incyte says REACH1 trial met primary endpoint, Stockwinners
Incyte reports positive data, Stockwinners

Incyte Corporation (INCY) announced positive results from its randomized, dose-ranging, vehicle- and active-controlled Phase 2b study evaluating ruxolitinib cream in patients with atopic dermatitis who are candidates for topical therapy.

The study, part of the True-AD clinical trial program, met its primary endpoint, demonstrating that ruxolitinib cream 1.5% administered twice daily significantly improved Eczema Area and Severity Index scores – a measurement of the extent and severity of AD – from baseline versus vehicle control at Week 4.

Additionally, treatment with ruxolitinib cream 1.5% BID resulted in a rapid and sustained reduction in itch versus vehicle, a key secondary endpoint.

These results were shared in an oral presentation today at the 27th European Academy of Dermatology and Venerology Congress in Paris, France.

Key study results included: Significantly improved EASI score in the ruxolitinib cream 1.5% BID arm versus vehicle at Week 4, the primary endpoint, and improvement in EASI score versus the active control, triamcinolone 0.1% cream, at Week 4, a secondary endpoint.

Significantly improved EASI scores in the ruxolitinib cream 1.5% BID arm versus vehicle at Weeks 2 and 8. Significantly greater changes in EASI score in the once daily ruxolitinib cream 1.5% and 0.5% arms versus vehicle at Week 4.

Significantly more Investigator’s Global Assessment responders – a measure of disease severity – in the ruxolitinib cream 1.5% BID arm versus vehicle at Week 4, and greater IGA response rates across other ruxolitinib arms versus vehicle.

Rapid and sustained reductions in itch numerical rating scale score observed as early as within two days from the initiation of therapy, and a more pronounced reduction in itch with ruxolitinib cream 1.5% BID and QD than with triamcinolone cream 0.1% BID.

Ruxolitinib cream was well-tolerated at all dosage strengths and was not associated with clinically-significant application site reactions.

All treatment-related adverse events were Grade 1 or Grade 2 in severity. Ruxolitinib cream is the first JAK1/JAK2 inhibitor to exhibit positive results as a topical monotherapy in the AD patient population.

Over-activity of the JAK signaling pathway has been shown to drive inflammation involved in the pathogenesis of AD.

These data support the planned initiation of a global, pivotal Phase 3 program, for which preparations are already underway.

INCY closed at $66.51.


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K2M Group sold for $1.4B

Stryker to acquire K2M Group for $27.50 per share

K2M Group sold for $1.4B , Stockwinners
K2M Group sold for $1.4B , Stockwinners

Stryker (SYK) announced a definitive merger agreement to acquire all of the issued and outstanding shares of common stock of K2M Group (KTWO) for $27.50 per share, or a total equity value of approximately $1.4B.

The combined business will have a competitive portfolio across Stryker’s Spine product categories and leverage a more powerful commercial engine.

With the addition of K2M’s proven product portfolio, consistent track record of execution and robust pipeline, Stryker Spine’s business will be well-positioned to sustain innovation and provide its customers and employees with proven products.

Upon closing of the transaction, it is expected that Eric Major will serve as President of Stryker’s Spine division and lead the combined business in its continued growth and innovation.

Bradley Paddock, the current President of Stryker’s Spine division, will assist with transitioning his responsibilities to Major while also supporting the integration efforts.

The acquisition of K2M is expected to close late in the fourth quarter of this year and is expected to have an immaterial dilutive impact to Stryker’s net EPS and adjusted net EPS in 2018.

There is no change to Stryker’s previously announced expected adjusted net EPS for the full year, which is a range of $7.22-$7.27.

For 2019, and beyond, Stryker reaffirms its previously stated long-term financial goals for sales, operating margins and adjusted net EPS.


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Spark Therapeutics tumbles on hemophilia study

Spark says Phase 1/2 data for SPK-8011 shows a 97% reduction in ABR

Spark tumbles on hemophilia study , Stockwinners
Spark tumbles on hemophilia study , Stockwinners

 

Shares of Spark Therapeutics (ONCE) are sinking after the company announced preliminary Phase 1/2 data for its investigational gene therapy candidate SPK-8011 for hemophilia A. A dose response as demonstrated by FVIII expression ranged from 16% to 49%, with a mean of 30% post 12 weeks in five of the participants in the 2×1012 vg/kg cohort, Spark announced in its Q2 earnings release.

As of the July 13, 2018, data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 vector genomes /kg body weight, three at a dose of 1×1012 vg/kg and seven at a dose of 2×1012 vg/kg.

Across all participants, at all three doses, beginning four weeks after vector infusion, there has been a 97-percent reduction in annualized bleeding rate and a 97-percent reduction in annualized infusion rate.

The first two trial participants, who have been followed for greater than one year, have shown stable FVIII activity levels since reaching plateau for up to 66 weeks, with follow up ongoing.

Additionally, there is evidence of a dose-dependent increase in mean FVIII activity levels across the three dose cohorts.

Five of the participants in the 2×1012 vg/kg cohort have FVIII activity levels between 16 and 49 percent, with follow-up ranging from 12 to 30 weeks.

The mean FVIII activity for these five participants is 30 percent, based on average FVIII levels post-12 weeks after vector infusion. These five participants have reduced their overall ABR by 100 percent and reduced their overall AIR by 100 percent.

The other two participants in the 2×1012 vg/kg cohort had an immune response that caused their FVIII levels to decline to less than 5 percent. Clinically, both participants have moved from prophylactic to on-demand treatment and have seen meaningful reductions in their bleeding and infusion rates.

One of these participants did not rapidly respond to oral steroids and he elected to be admitted to the hospital to receive two intravenous methylprednisolone infusions rather than have the infusions on an outpatient basis.

The event was subsequently resolved. The admission to hospital for these infusions met the criteria for a serious adverse event.

Of note, across the study, seven of the 12 participants received a tapering course of oral steroids in response to an alanine aminotransferase elevation above patient baseline, declining FVIII levels and/or positive IFN-g enzyme-linked immunospots.

For these seven participants, steroids led to normalization of ALT and ELISPOTs.

For all but the two above mentioned 2×1012 vg/kg cohort participants, oral steroids led to stabilization of target FVIII levels. Based on the totality of the results to date, Spark Therapeutics intends to initiate a Phase 3 run-in study in the fourth quarter of 2018. Following completion of the run-in study, Phase 3 participants are expected to receive 2×1012 vg/kg of SPK-8011.

Additional details on the Phase 3 trial design will be determined following continued discussions with FDA and EMA, which are expected in the fourth quarter.

Finally, the company has successfully scaled-up its mammalian-based manufacturing process in suspension to a capacity level of 200 liters and amended its agreement with Brammer Bio to secure a dedicated manufacturing suite, both of which will enable Spark Therapeutics to meet supply needs for Phase 3 clinical development as well as expected commercial requirements.

ONCE closed at $77.61, it last traded at $56.00.


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ResTORbio higher on data

ResTORbio announces ‘positive’ topline results in Phase 2b trial of RTB101

ResTORbio higher on data, Stockwinners
ResTORbio higher on data, Stockwinners

ResTORbio (TORC) announced positive topline results from its dose-ranging Phase 2b clinical trial that enrolled 652 elderly patients at increased risk of morbidity and mortality associated with respiratory tract infections.

In this trial, RTB101, an oral, selective, and potent inhibitor of target of rapamycin complex 1, demonstrated a statistically significant and clinically meaningful reduction in the percentage of patients with one or more laboratory-confirmed RTIs during the 16-week treatment period compared to placebo, the primary endpoint of the study, with the 10 mg once daily dose.

Greater TORC1 inhibition with RTB101 10 mg in combination with everolimus 0.1 mg did not meet the primary endpoint, suggesting that that less TORC1 inhibition with RTB101 10 mg once daily may have greater benefit in high-risk elderly patients.

The Phase 2b trial was a two-part, randomized, double-blind, placebo-controlled clinical trial conducted during the winter cold and flu season in the southern hemisphere and northern hemisphere.

Patients enrolled were those at increased risk of morbidity and mortality from RTIs including patients who were: 85 years of age or older, or 65 years of age or older with asthma, type 2 diabetes mellitus, chronic obstructive pulmonary disease, or current smokers.

The doses investigated in Part 1 were RTB101 5 mg and RTB101 10 mg once daily. The doses investigated in Part 2 were RTB101 10 mg once daily, RTB101 10 mg twice daily and RTB101 10 mg in combination with everolimus 0.1 mg once daily.

The following was observed in an analysis of the primary endpoint: A 30.6% decrease relative to placebo in the percentage of all patients treated with RTB101 10 mg once daily who developed one or more laboratory-confirmed RTs. A 20.6% decrease relative to placebo in the percentage of all patients treated with RTB101 5 mg once daily who developed one or more laboratory-confirmed RTIs.

No decrease relative to placebo in the percentage of patients treated with either RTB101 10 mg twice daily or the combination of RTB101 10 mg + everolimus 0.1 mg once daily who developed one or more laboratory-confirmed RTIs, suggesting that less TORC1 inhibition with RTB101 10 mg once daily may have greater benefit in high-risk elderly patients.

To better understand the activity observed in the RTB101 10 mg once daily cohort, a pre-specified analysis of each patient subgroup enrolled in the study was conducted.

The following decreases in the percentage of patients with laboratory-confirmed RTIs were observed in the RTB101 10 mg once daily cohort as compared to the placebo cohort: A 68.4% decrease in all asthma patients.

A 66.7% decrease in all patients 85 years of age and older. A 26.9% decrease in all T2DM patients.

No decrease was observed in either COPD patients or current smokers; a 42.0% decrease in all patients was observed when excluding patients with COPD and a 43.9% decrease in all patients was observed when excluding current smokers.

All doses were observed to be well-tolerated. Data from the RTB101 10 mg once daily cohort are as follows: Adverse events were balanced between the RTB101 10 mg once daily and placebo treatment groups. 4.5% of subjects in the RTB101 10 mg once daily cohort and 7.2% of subjects in the placebo cohort had a serious adverse event, none of which were considered related to study drug. 4.5% of subjects in the RTB101 10 mg once daily cohort and 6.1% of subjects in the placebo cohort discontinued study drug due to an AE.

All AEs were mild or moderate except for 11 severe AEs in the RTB101 10 mg once daily cohort and 22 severe AEs in the placebo cohort.

TORC closed at $9.01, it last traded at $24.49.


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Zogenix sharply higher on data

Zogenix says primary endpoint achieved in second Phase 3 clinical trial of ZX008

Zogenix sharply higher on data, Stockwinners
Zogenix sharply higher on data, Stockwinners

Zogenix (ZGNX) reported positive top-line results from its second confirmatory Phase 3 study for its investigational drug, ZX008, for the treatment of children and young adults 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 one out of 15,700 individuals, 80% of whom have a mutation in their SCN1A gene.

The study results, which are consistent with those reported in Study 1, Zogenix’s first pivotal Phase 3 study, successfully met the primary endpoint and all key secondary endpoints, demonstrating that ZX008, at a dose of 0.5 mg/kg/day, is superior to placebo when added to a stiripentol regimen. Key Findings: Patients taking ZX008 achieved a 54.7% greater reduction in mean monthly convulsive seizures compared to placebo.

The median reduction in monthly convulsive seizure frequency was 62.7% in the ZX008 group compared to 1.2% in placebo patients. ZX008 also demonstrated statistically significant improvement versus placebo in both key secondary measures, including patients with clinically meaningful reductions in seizure frequency and longest seizure-free interval.

ZX008 was generally well-tolerated in this study with the adverse events consistent with those observed in Study 1 and the known safety profile of fenfluramine.

No patient exhibited cardiac valvulopathy or pulmonary hypertension at any time in the study.

Secondary endpoints assessed ZX008 compared to placebo in terms of the proportions of patients who achieved greater than or equal to 50% reductions and greater than or equal to 75% reductions in monthly convulsive seizures, as well as the median of the longest convulsive seizure-free interval.

ZX008 was generally well-tolerated in this study, with the adverse events consistent with those observed in Study 1 and the known safety profile of fenfluramine.

The incidence of treatment emergent adverse events was similar in both the treatment and placebo groups, with 97.7% of patients receiving ZX008 experiencing at least one treatment emergent adverse event compared to 95.5% of patients in the placebo group.

The most common adverse events in the ZX008 group were decreased appetite, diarrhea, pyrexia, fatigue, and nasopharyngitis.

The incidence of serious adverse events was similar in both the treatment and placebo groups, with 14% of patients in the ZX008 group experiencing at least one treatment emergent serious adverse event compared to 15.9% of patients in the placebo group. T

wo patients in the ZX008 group had an adverse event leading to study discontinuation compared to one in the placebo group.

ZGNX closed at $46.30, it last traded at $55.30.


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Bristol-Myers treatment for colorectal cancer approved

Bristol-Myers’ Opdivo approved for MSI-H/dMMR metastatic colorectal cancer

Bristol-Myers treatment for colorectal cancer approved, Stockwinners
Bristol-Myers treatment for colorectal cancer approved, Stockwinners

Bristol-Myers (BMY) announced Opdivo – nivolumab – 3 mg/kg plus low-dose Yervoy – ipilimumab – 1 mg/kg injections for intravenous use received approval from the FDA for the treatment of adult and pediatric patients 12 years and older with microsatellite instability high or mismatch repair deficient metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan.

Approval for this indication has been granted under accelerated approval.

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

The application was granted Priority Review and Breakthrough Therapy Designation by the FDA.

Among the 82 patients who received prior treatment with a fluoropyrimidine, oxaliplatin and irinotecan, 46% responded to treatment with Opdivo + Yervoy.

The percentage of these patients with a complete response was 3.7%, and the percentage of patients with a partial response was 43%.

Among these 38 responders, the median DOR was not reached; 89% of those patients had responses of six months or longer, and 21% had responses of 12 months or longer.

This trial is ongoing. Among all enrolled patients, 49% responded to treatment with Opdivo + Yervoy; 4.2% experienced a complete response, while 45% experienced a partial response.

Opdivo was discontinued in 13% of patients and delayed in 45% of patients due to an adverse reaction.

Serious adverse reactions occurred in 47% of patients.

The Opdivo + Yervoy combination is also approved in two other tumor types, advanced renal cell carcinoma and unresectable or metastatic melanoma.

Continued approval for these accelerated approval indications may be contingent upon verification and description of clinical benefit in the confirmatory trials.

BMY closed at $56.18.


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Spero Therapeutics higher on data

Spero says clinical data demonstrates SPR994 to be well-tolerated 

Spero Therapeutics higher on data, Stockwinners
Spero Therapeutics higher on data, Stockwinners

Spero Therapeutics (SPRO) reported positive results from an interim analysis of its ongoing single ascending dose and multiple ascending dose Phase 1 clinical trial of SPR994, its investigational oral carbapenem product candidate designed for the treatment of Gram-negative infections, in healthy volunteers.

The data demonstrate that SPR994 has a favorable safety, pharmacokinetic and pharmacodynamic profile that supports the advancement of SPR994 to a pivotal Phase 3 clinical trial at a 300 mg dose administered three times per day.

The Phase 1 clinical trial of SPR994 is assessing the safety, tolerability and pharmacokinetics of orally administered SPR994.

Interim Phase 1 results from the SAD portion of the trial demonstrate SPR994 to be well tolerated at doses ranging from 100 mg to 900 mg daily, with linear human pharmacokinetics over the dose range tested.

The data also show that the mean free drug plasma concentrations of tebipenem, the active metabolite of SPR994, remained above the MIC90 for the relevant bacterial pathogens for greater than 50% of an 8-hour dosing interval, consistent with the targeted therapeutic range observed in Spero’s hollow fiber experiments.

The pharmacokinetic profile of SPR994 observed to date in both the SAD and MAD portions of the trial provides evidence supporting SPR994’s administration without regard to meals.

Urine concentration data support the use of SPR994 to treat cUTI, with peak urine concentrations 100-fold higher than maximum concentrations in plasma.

No serious adverse events have been reported in the Phase 1 clinical trial to date.

Oral administration of SPR994 has been well tolerated at all doses tested and results are consistent with available clinical and post-marketing data for Orapenem and other approved IV carbapenem antibiotics.

Orapenem is currently approved in Japan for the treatment of pediatric infections and has the same orally bioavailable active ingredient as SPR994.

To date, the Phase 1 clinical trial has enrolled 115 healthy adult volunteers into 14 SAD cohorts and 1 MAD cohort. The SAD portion of the Phase 1 clinical trial is evaluating formulations of SPR994 at single doses ranging from 100 mg to 900 mg daily.

The MAD portion of the trial is designed to assess the safety, tolerability and pharmacokinetics of SPR994 administered orally to healthy volunteers for 14 days.

The initial MAD cohort received 300 mg of SPR994 administered three times per day and the MAD portion of the trial will continue to dose escalate to determine the maximum tolerated dose.

SPRO closed at $16.89, it last traded at $18.21.


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