Watch PTC Therapeutics into FDA meeting

Watch PTC Therapeutics ahead of FDA panel meeting

Updated with FDA staff comments on September 26, 2017

Watch PTCT ahead of FDA Meeting. See Stockwinners.com Market Radar for details

The U.S. Food and Drug Administration has scheduled an advisory panel meeting for next week to review PTC Therapeutics’ (PTCT) ataluren for the treatment of Duchenne muscular dystrophy.

Commenting ahead of the meeting, Citi analyst Joel Beatty said that U.S. Congressman Collin Peterson is expected to voice his support for the drug.

The analyst argued that PTC Therapeutics could be worth about $59 per share if ataluren, which is marketed with the trade name #Translarna, is approved in the U.S. and about $8 per share if it is not.

TRANSLARNA

PTC Therapeutics’ lead product candidate ataluren is a novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation.

Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation, or nmDMD.

The European Medicines Agency has designated ataluren as an orphan medicinal product and the FDA has granted orphan drug designation to ataluren for the treatment of nmDMD.

Back in June, PTC Therapeutics announced that the FDA had notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28 to review the new drug application, or NDA, for ataluren.

The company’s NDA submission was granted standard review by the FDA on March 6, with the FDA setting a Prescription Drug User Fee, or PDUFA, goal date of October 24 for completion of its review of the ataluren NDA.

POLITICAL SUPPORT

Earlier this month, Citi analyst Joel Beatty said he had found comments online from U.S. Congressman Collin Peterson that voice support for approval of PTC Therapeutics’ DMD treatment.

The document appeared to have been posted in response to the FDA’s request for comments regarding its advisory panel meeting scheduled for September 28, and is written in the form of a speech to be given at the meeting, Beatty told investors in a research note.

The analyst confirmed with the Congressman’s office that a legislative assistant plans to read the letter on behalf of Representative Peterson at the advisory meeting.

The comments mentioned a constituent with DMD who will be in the audience and for whom “access to ataluren has allowed him to live and thrive well into his 20s with no side effects,” Beatty cited the statement as saying.

The analyst believes political support could play a role in the FDA’s review of ataluren.

Back in June, Beatty had told investors that he was increasing his probability of approval to 25% from 20% following the advisory meeting announcement, but said he still viewed U.S. approval at less than 50/50.

The analyst also pointed out that he believes PTC Therapeutics is worth about $59 per share if Translarna is approved in the U.S. and about $8 per share if Translarna does not receive a U.S. approval.

JP  MORGAN

JPMorgan analyst Anupam #Rama says he remains “somewhat cautious” on shares of PTC Therapeutics (PTCT) with the FDA’s Peripheral and Central Nervous Systems Drugs Advisory Committee convening on September 28 to review the company’s Translarna for the treatment of non-sense mutation Duchenne muscular dystrophy.

Translarna has an FDA action date of October 24. The analyst cites the drug’s “mixed” Phase 2 and Phase 3 data sets and prior refuse-to-file letter for his “somewhat cautious” stance.

For Sarepta Therapeutics (SRPT), Rama sees a net positive read-through from a favorable panel, on the potential for an accelerated path forward for golodirsen in Exon 53 DMD.

Further, a negative panel is likely to be viewed as Translarna-specific, playing out as a net neutral for Sarepta, the analyst tells investors in a research note. He does not see “major fundamental downside risk” for Sarepta shares going into PTC’s panel. Rama keeps a Neutral rating on PTC

FDA STAFF COMMENTS

In the briefing documents, FDA staff wrote: “The Office of Clinical Pharmacology has reviewed the information contained in NDA 200896. The OCP review team does not consider the exposure-response analyses and in vitro data as supportive evidence of effectiveness. See Link for the complete comments and response!

PRICE ACTION

In Monday’s trading, shares of PTC Therapeutics are up 11 cents to $18.64.

Since June 6, the day PTC reported the FDA’s tentative scheduling of the advisory committee meeting, the stock is up 34%.


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NewLink Genetics Higher on Collaboration with AstraZeneca

NewLink Genetics enters clinical collaboration with AstraZeneca

NewLink Genetics enters clinical collaboration with AstraZeneca. See Stockwinners.com Market Radar

NewLink Genetics (NLNK) announced that it has entered into a clinical collaboration agreement with AstraZeneca (AZN) to evaluate the combination of indoximod, NewLink Genetics’ small molecule IDO pathway inhibitor, and durvalumab, AstraZeneca’s anti-PD-L1 monoclonal antibody, along with standard of care chemotherapy for patients with metastatic pancreatic cancer.

The primary objective for this randomized placebo-controlled, Phase 2 study is to evaluate the efficacy and safety of the immuno-oncology-based combination compared to gemcitabine/ABRAXANE alone.

Patients will also be enrolled into a smaller cohort evaluating the combination of #durvalumab with gemcitabine/ABRAXANE.

The Phase 2 trial will be funded equally by both companies, with NewLink Genetics serving as the study sponsor. NewLink Genetics’ share of the aggregate expense of the trial is not expected to have a material effect on its financial position.

On September 7th, NLNK shares jumped 45% after firm reported data from its ongoing phase 2 trial of IDO-inhibitor indoximod in advanced melanoma. NewLink Genetics thinks that combining indoximod with PD-1 drugs can deliver even better outcomes for patients because IDO-inhibitors prevent cancer cells from hijacking proteins that suppress immune system responses, and PD-1 drugs improve the ability of T-cells to spot and destroy cancer cells.

PRICE  ACTION

NLNK has a 52-weeks trading range of $5.90 – $25.17. Shares last traded at $11.55 in pre-market trading, up 55 cents on the news.


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Alnylam Jumps Following Results

Sanofi and Alnylam report topline results of patisiran in ATTR amyloidosis

ALNY-LOGO

Sanofi’s (SNY) specialty care global business unit, Sanofi Genzyme, and Alnylam (ALNY) announced that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint and all secondary endpoints.

The primary endpoint for the study was the change from baseline in the modified neuropathy impairment score at 18 months.

The key secondary endpoint was improvement in quality of life.

In healthy people, normal, so-called “wild-type” TTR functions as a transporter of thyroid hormone and vitamin A (retinol) within the bloodstream. People with mutations in the TTR gene produce abnormal, amyloidogenic, “variant” TTR throughout their lives.

The APOLLO trial enrolled 225 hATTR amyloidosis patients with polyneuropathy.

The overall safety profile of patisiran was “encouraging”.

Based on these positive results, Alnylam expects to file its first New Drug Application in late 2017 and first Marketing Authorisation Application shortly thereafter.

Sanofi Genzyme is currently preparing for regulatory filings for patisiran in Japan, Brazil and other countries, to begin in 1H18. Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.


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AVEO Oncology is in focus

AVEO Oncology, EUSA Pharma announce TiNivo combination study opt-in

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AVEO Oncology (AVEO) and EUSA Pharma announced that EUSA Pharma, under its multi-territory licensing agreement with AVEO for FOTIVDA, has opted into the Phase 1/2 TiNivo study.

Under terms of the agreement, EUSA may utilize data from the study for regulatory or commercial purposes in exchange for a research and development funding payment totaling $2M.

EUSA’s decision follows approval in August of tivozanib by the European Commission for the treatment of adult patients with advanced renal cell carcinoma in the European Union plus Norway and Iceland.

The TiNivo trial is a Phase 1/2 trial of tivozanib in combination with Bristol-Myers Squibb’s (BMY) OPDIVO, an immune checkpoint, or PD-1, inhibitor, for the treatment of RCC. The TiNivo trial is being led by the Institut Gustave Roussy in Paris under the direction of Bernard Escudier, MD, Chairman of the Genitourinary Oncology Committee.

In June, AVEO announced the advancement of the trial into the Phase 2 expansion portion following successful completion of the Phase 1 dose escalation portion. The combination was well tolerated to the full dose and schedule of single agent tivozanib, with no dose limiting toxicities.

The expansion portion of the trial is expected to enroll an additional 20 subjects. Phase 1 results from the ongoing study have been submitted for presentation at a scientific meeting taking place in the fourth quarter. Under the terms of their December 2015 agreement, EUSA Pharma has agreed to pay AVEO up to $388M in future milestone payments and research and development funding, assuming successful achievement of specified development, regulatory and commercialization objectives.

In addition, a tiered royalty will be due to AVEO ranging from a low double-digit up to mid-twenty percent on net sales of tivozanib in the agreement’s territories. With European approval, AVEO will be eligible for up to $12M in milestones from EUSA based on reimbursement and regulatory approvals.

In the territories licensed to EUSA, 30% of milestone and royalty payments received by AVEO, excluding research and development payments such as the one announced today, are due to Kyowa Hakko Kirin as a sublicensing fee. In the territories retained by AVEO, the royalty obligation to KHK ranges from the low- to mid-teens on net sales.


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FDA Approves Innoviva’s drug for COPD

Innoviva, GlaxoSmithKline confirm FDA approval of Trelegy Ellipta

GlaxoSmithKline (GSK) and Innoviva (INVA) announced that the U.S. FDA has approved once-daily, single inhaler triple therapy fluticasone furoate/umeclidinium/vilanterol, under the brand name Trelegy Ellipta, for the long-term, once-daily, maintenance treatment of patients with chronic obstructive pulmonary disease, including chronic bronchitis and/or emphysema, who are on a fixed-dose combination of fluticasone furoate and vilanterol for airflow obstruction and reducing exacerbations in whom additional treatment of airflow obstruction is desired or patients who are on umeclidinium and a fixed-dose combination of fluticasone furoate and vilanterol.

COPD is a disease that affects the lungs, causing reduced airflow, which makes it hard to breathe. It is also progressive, which means it worsens over time. COPD can include emphysema, chronic bronchitis, or both. Roughly 15 million adults in the U.S. have been diagnosed with COPD, while millions more who have it may not even know it.

Trelegy Ellipta is not indicated for relief of acute bronchospasm or the treatment of asthma.

Following this approval by the FDA, Trelegy Ellipta will be available in the US shortly.

Regulatory applications have been submitted and are undergoing assessment in a number of other countries, including the European Union, Australia and Canada.

INVA closed at $13.81. The stock has a 52-week trading range of $8.67 – $14.20. GSK closed at $40.05.


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Dimension Therapeutics sold for $138 million

Ultragenyx to acquire Dimension Therapeutics for $5.50 per share in cash

Ultragenyx to acquire Dimension Therapeutics for $5.50 per share in cash. See Stockwinners.com for details

Ultragenyx Pharmaceutical (RARE) announced that it has made a proposal to acquire all of the outstanding shares of common stock of Dimension Therapeutics (DMTX) for $5.50 per share, or approximately $138 million, in cash at close to be effectuated via a tender offer.

The Ultragenyx offer represents a premium of over 358% to Dimension’s unaffected share price as of August 24, 2017 and premiums of 24% and 48% over the implied value of the all-stock consideration to be received by Dimension stockholders pursuant to the announced acquisition of Dimension by REGENXBIO (RGNX), based on REGENXBIO’s last closing price and trailing 20-trading day volume-weighted average price as of September 15, 2017, respectively.

As such, the proposal would provide Dimension stockholders with an immediate and certain return on their investment in Dimension and constitutes a superior alternative to the REGENXBIO transaction.

The proposal has been approved by the Board of Directors of Ultragenyx. Ultragenyx would fund the transaction from cash resources on its balance sheet and anticipates that customary closing conditions to the transaction could be satisfied so that the tender offer could complete as soon as 25 business days after merger agreement signing.


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Teva sells its women’s health portfolio assets for $1.38B

Teva announces sale of global women’s health portfolio assets for $1.38B

Teva rallies after finding experienced CEO. See Stockwinners.com for details

Teva Pharmaceutical (TEVA) announced it has entered into two agreements to sell the remaining assets of its specialty global women’s health business for $1.38B.

Proceeds from these sales, combined with proceeds from the recently announced sale of PARAGARD total $2.48B and will be used by Teva to progress repayment of term loan debt.

Teva has entered into a definitive agreement under which CVC Capital Partners Fund VI will acquire a portfolio of products within its global women’s health business across contraception, fertility, menopause and osteoporosis for $703M in cash.

The portfolio of products, which is marketed and sold outside of the U.S., includes Ovaleap, Zoely, Seasonique, Colpotrophine, Actonel and additional products.

Teva has also entered into a definitive agreement under which Foundation Consumer Healthcare will acquire Plan B One-Step and Teva’s value brands of emergency contraception, Take Action, Aftera, and Next Choice One Dose for $675M in cash.

Completion of the transactions is subject to customary conditions, including antitrust clearance in the U.S. and EU respectively, together with employee consultations.

The transactions are expected to close before the end of 2017. Until the transactions are completed, Teva will continue to market the products in the normal course, providing full support to manage the business and to meet the needs of customers and patients.- Teva Pharmaceutical announced it has entered into two agreements to sell the remaining assets of its specialty global women’s health business for $1.38B.

Proceeds from these sales, combined with proceeds from the recently announced sale of PARAGARD total $2.48B and will be used by Teva to progress repayment of term loan debt.

Teva has entered into a definitive agreement under which CVC Capital Partners Fund VI will acquire a portfolio of products within its global women’s health business across contraception, fertility, menopause and osteoporosis for $703M in cash.

The portfolio of products, which is marketed and sold outside of the U.S., includes Ovaleap, Zoely, Seasonique, Colpotrophine, Actonel and additional products. Teva has also entered into a definitive agreement under which Foundation Consumer Healthcare will acquire Plan B One-Step and Teva’s value brands of emergency contraception, Take Action, Aftera, and Next Choice One Dose for $675M in cash.

Completion of the transactions is subject to customary conditions, including antitrust clearance in the U.S. and EU respectively, together with employee consultations.

The transactions are expected to close before the end of 2017. Until the transactions are completed, Teva will continue to market the products in the normal course, providing full support to manage the business and to meet the needs of customers and patients.


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Halozyme is on the move!

Halozyme to receive $150M from Bristol-Myers for Enhanze technology partnership

halozyme is on the move. See Stockwinners.com for details

Bristol-Myers (BMY) and Halozyme (HALO) announced a global collaboration and license agreement to develop subcutaneously administered Bristol-Myers Squibb immuno-oncology medicines using Halozyme’s Enhanze drug-delivery technology.

The Halozyme Enhanze technology is based on a proprietary recombinant human hyaluronidase enzyme to aid in the dispersion and absorption of other injected therapeutic drugs.

Halozyme will receive an initial $105M for access to the Enhanze technology. Bristol-Myers Squibb has an option to select additional targets within five years from the effective date. The collaboration may extend to a maximum of 11 targets.

Halozyme has the potential to earn milestone payments of up to $160M for each of the nominated collaboration targets and additional milestone payments for combination products.

In addition, Bristol-Myers Squibb will pay Halozyme royalties on sales of products using the Enhanze technology developed under the collaboration. For Bristol-Myers, the transaction is expected to be dilutive to non-GAAP EPS in 2017 and 2018 by approximately 1c, and by approximately 5c in 2019.

ROCHE  DEAL

Separately, Halozyme (HALO) licensed its Enhanze drug-delivery technology to Roche (RHHBY) for exclusive development of an undisclosed therapeutic target.

Halozyme will receive an initial $30M with the potential to earn additional payments of up to $160M subject to achievement of specified development, regulatory and sales-based milestones.

Halozyme will also receive tiered, mid-single digit royalties on sales of commercialized products. The Halozyme/Roche relationship dates back to the original global collaboration and licensing agreement for the ENHANZE technology signed in 2006.

RAISED   GUIDANCE

Halozyme raised its FY17 revenue view to $245M-$260M from $115M-$130M vs FY17 consensus $131.9M. Reflecting the portion of upfront payments from the new agreements expected to be recorded as revenue in 2017.

PRICE  ACTION

HALO closed at $13.18. It has a 52-weeks trading range of $8.18 – $15.20. Shares last traded at $14.80 in pre-market trading.


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Sage Therapeutics Trial Fails, Shares Tumble

Sage Therapeutics announces results from Sage-547 trial did not meet endpoint

Sage Therapeutics announces results from Sage-547. See Stockwinners.com for details

Sage Therapeutics (SAGE) reported top-line results from its Phase 3 STATUS Trial of brexanolone, or SAGE-547, in the treatment super-refractory status epilepticus, or SRSE.

The trial was testing Sage’s drug, brexanolone, plus standard of care in patients with super-refractory status epilepticus (SRSE) whose seizures persisted despite earlier treatments, versus a placebo plus standard of care.

The study did not meet the primary endpoint, comparing success in weaning of third-line agents and resolution of potentially life-threatening status epilepticus with brexanolone vs. placebo when added to standard-of-care.

The company is also testing the same drug in post-partum depression, with results of a two late-stage trials in such patients expected before the end of the year. But Tuesday’s failure is raising some doubts about whether that trial will succeed.

There are no treatments for SRSE currently approved by the FDA.

PRICE ACTION:

SAGE closed at $88.52. It last traded at $66.09.


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Short Squeeze in Teva following CEO Announcement

Teva rallies after finding experienced CEO

Teva rallies after finding experienced CEO. See Stockwinners.com for details

Shares of Teva Pharmaceutical (TEVA) surged in morning trading after naming a permanent chief executive officer, ending a months long search for a new chief. The new CEO brings 30 years of global pharmaceutical and healthcare experience.

CEO APPOINTMENT

Teva this morning said that it has named named Kare Schultz to be its new president and CEO. Schultz will relocate to Israel and will be based out of the company’s headquarters in Petach Tikva, the company stated.

He will succeed Yitzhak Peterburg, who has been serving as interim CEO since Erez Vigodman stepped down in February.

Schultz joins Teva from H. Lundbeck (HLUYY), where he has served as president and CEO since 2015. Prior to joining Lundbeck, Schultz worked for nearly three decades at Novo Nordisk (NVO), where he once served as the company’s chief operations officer.

Sol Barer, Teva’s chairman, told the Wall Street Journal that the company searched for months for a CEO to “make sure we got the right person,” adding that “this is a critical time in Teva’s history.”

WHAT’S NOTABLE

In July, rumors had swirled that Teva would name AstraZeneca (AZN) CEO Pascal Soriot as its new CEO.

According to a Calcalist report at the time, Soriot met with Teva’s chairman and search committee and “expressed his agreement” to serve as the next CEO. Soriot later told Bloomberg that he plans on staying at AstraZeneca “for the foreseeable future.”

In addition to Vigodman’s departure in February, Teva CFO Eyal Desheh left the company at the end of June, being replaced on an interim basis by Michael McClellan.

In August, shares of Teva dropped after the company cut its full year outlook and its dividend, citing the impact of increased price erosion in its U.S. Generics business.

DEBT BURDEN

Teva, which is looking to pay down more than $5B of debt this year, is looking for “a series of partners,” a spokeswoman confirmed to Reuters last month, adding that its intent is “not to fund the whole pipeline, just some projects in it. A small part of it.

” The drugmaker is also pursuing the sale of certain non-core assets to extract synergies related to the Actavis Generics transaction, Peterburg has said.

Looking ahead, new CEO Schultz is likely to face pressure from investors to split the company into two businesses.

NEW CEO A “POSITIVE CATALYST”

This morning, BTIG analyst Timothy Chiang upgraded Teva to Buy from Neutral, calling the CEO appointment a “positive catalyst that should help create a floor for the shares and provide a boost to near-term investor sentiment.” #Chiang is upbeat on Teva’s decision to hire “an experienced pharmaceutical executive” as its new chief and is bullish on fremanezumab, Teva’s migraine treatment.

Raymond James analyst Elliot Wilbur said Teva strength today likely reflects the company landing a name brand CEO in Kare Schultz. The analyst said Schultz’s arrival likely marks a short-term bottom in shares, but does not change the need for bold strategic action and heavy investment spend in order to resurrect top line growth in the face of generic headwinds. #Wilbur said financial flexibility remains limited and much remains to be seen in terms of how the company will be reshaped, and doesn’t see favorable risk/reward scenarios even at current depressed levels. Wilbur rates Teva a Market Perform.

SHORT RATIO

The short ratio, short interest ratio (SIR) or float short for a public company is the ratio of tradable shares being shorted to shares in the market, or the float. It is an indirect metric of investor sentiment. When short interest is high, above 40%, it implies company investors hope shares will decline in value.

According to the latest data, 41.9 million shares of TEVA have been shorted as of August 15th. The stock has a 26 million daily average trading volume of 26 million,giving it a short ratio of 1.62.

A short squeeze is a rapid increase in the price of a stock that occurs when there is a lack of supply and an excess of demand for the stock. Short squeezes result when short sellers cover their positions on a stock, resulting in buying volume that drives the stock price up.

PRICE ACTION

Teva is up nearly 16% in Monday’s trading to $17.89. Year-to-date, however, shares are down about 52%.


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Integra LifeSciences Sold for $47M

Natus Medical to acquire Integra LifeSciences for $47M in cash 

Natus Medical to acquire Integra LifeSciences for $47M in cash. See Stockwinners.com for details

Natus Medical (BABY) announced that it has entered into a definitive agreement with Integra LifeSciences (IART) under which Natus will acquire certain neurosurgery business assets from Integra LifeSciences in an all cash transaction for $47.5M.

With current annual revenue of approximately $50M, the acquisition marks Natus’ entry into the $2B global neurosurgery market.

The divestiture by Integra is contingent on the consummation of Integra’s proposed acquisition of Codman Neurosurgery.

As part of the transaction, Natus will acquire the global Camino ICP monitoring product line, including its San Diego manufacturing facility, from Integra.

The sale also includes the U.S. rights relating to Integra’s fixed pressure shunts, as well as U.S. rights to Codman’s DURAFORM dural graft implant, standard EVD catheters and CSF collection systems.

Integra is divesting these assets in connection with the review by the Federal Trade Commission of Integra’s proposed acquisition of Johnson & Johnson’s Codman Neurosurgery assets. Both the divestiture and the pending acquisition of Codman Neurosurgery remain subject to final regulatory approvals and satisfaction of other customary closing conditions.

Both transactions are expected to close in October 2017 after securing regulatory clearance. Natus will use cash on hand and available from its credit facility to fund the acquisition.


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Intra-Cellular Higher on Schizophrenia Data

Intra-Cellular reports positive data for lumateperone in schizophrenia

Intra-Cellular to move forward with lumateperone long-term safety study. See Stockwinners.com Market Radar for details

Intra-Cellular (ITCI) announced “positive” topline data from the first part of an open-label safety switching study in which 302 patients with stable symptoms of schizophrenia were switched from standard-of-care antipsychotic medications to lumateperone with no dose titration of #lumateperone required for a 6-week treatment duration, then switched back to standard-of-care.

In this study, lumateperone was generally well tolerated with a favorable safety profile.

Statistically significant improvements from standard-of-care baseline were observed in body weight, cardiometabolic and endocrine parameters in patients with stable symptoms of schizophrenia when switched to lumateperone and worsened again when switched back to standard-of-care medication.

Additionally, treatment with lumateperone was not associated with the motor or cardiovascular disturbances often associated with other antipsychotic medications. Statistically significant improvement from baseline was observed in the Positive and Negative Syndrome Scale mean total score.

Greater improvements were observed in subgroups of patients with elevated symptomatology such as those with comorbid symptoms of depression and those with prominent negative symptoms.

Related Blogs

You may recall last month ITCI reported positive data and that pushed shares higher. Today’s report should help push shares higher.


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Alnylam Tumbles after Patient Dies

Alnylam suspends dosing in all ongoing fitusiran studies after patient death

ALNY-LOGO

Alnylam Pharmaceuticals (ALNY) announced today an update on the company’s fitusiran and givosiran investigational RNAi therapeutic programs.

With fitusiran, an RNAi therapeutic in development for the treatment of hemophilia A and B with or without inhibitors, Alnylam is reporting a fatal thrombotic event in a patient with hemophilia A without inhibitors in the Phase 2 open-label extension study of fitusiran.

As a result, the company has suspended dosing in all ongoing #fitusiran studies pending further review of the safety event and development of a risk mitigation strategy. “Based on overall consideration of fitusiran’s benefit-risk profile, Alnylam is guiding that it aims to resume dosing as soon as possible upon agreement with global regulatory authorities and with appropriate protocol amendments in place for enhanced patient safety monitoring.”

With givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyrias, Alnylam said it has reached alignment with the FDA on a Phase 3 study design which includes an interim analysis based on reduction of a urinary biomarker, aminolevulinic acid, as a surrogate endpoint reasonably likely to predict clinical benefit.

Based on the new givosiran Phase 3 design, the company is now guiding that pending FDA review of the program at the time of interim analysis and assuming positive results, it expects to submit a new drug application at or around year-end 2018.

Based on today’s update, Alnylam will postpone its fitusiran RNAi Roundtable webinar previously scheduled for September 12 until a later date.

“We are deeply saddened to learn of this patient’s death, and we extend our sympathies to his family,” said Akshay Vaishnaw, M.D., Ph.D, Executive Vice President of R&D at Alnylam.

“We believe that fitusiran holds great promise as a potential treatment option for patients with hemophilia, and we remain fully committed to its ongoing development.

Following further investigation of this safety finding, implementation of a risk mitigation strategy, and alignment with global regulatory authorities, we expect to resume fitusiran dosing in our clinical studies as soon as possible, potentially as early as late 2017, with a goal of advancing this innovative investigational medicine to hemophilia patients in need.”

PRICE  ACTION

ALNY has a 52-weeks trading range of $31.38 – $89.45. It closed at $86.02, last traded at $77.00.


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Sarepta Therapeutics Reports Positive DMD Data

Sarepta announces ‘positive’ results in 4053-101 DMD study

 Sarepta announces positive DMD results. See Stockwinners.com for details.

Sarepta Therapeutics (SRPT) announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53.

Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.

The study comprised two parts.

In Part 1, 12 patients were randomized to receive a dose titration of golodirsen or placebo. At the end of Part 1, all 12 patients continued on golodirsen and an additional 13 patients started golodirsen.

In Part 2, all 25 patients were treated for an additional 48 weeks at the time of muscle biopsy.

The analysis included biopsies of the bicep muscle at baseline and on-treatment at the Part 2 Week 48 time point. All 25 participants displayed an increase in skipping exon 53 over baseline levels, representing a 100 percent response rate as measured by RT-PCR and demonstrating proof of mechanism.

Mean dystrophin protein increased to 1.019 percent of normal compared to a mean baseline of 0.095 percent of normal as measured by Western blot, the primary biological endpoint in the study, representing a 10.7 fold increase from baseline.

The study also showed a statistically significant increase in dystrophin immunofluorescence as measured by immunohistochemistry, the secondary biological endpoint in the study, confirming sarcolemma-associated protein expression and distribution.

Francesco Muntoni, principal investigator for this study and Pediatric Neurologist, Great Ormond Street Hospital for Children NHS Foundation Trust and the UCL Great Ormond Street Institute of Child Health, said,

“All treated boys showed the anticipated exon skipping after treatment and this resulted in a mean increase of dystrophin protein, as measured by Western blot, from 0.095 percent at baseline to 1.019 percent of normal after at least one-year of treatment with golodirsen. These data were also supported by the highly statistically significant increase of dystrophin expression at the sarcolemma, as measured by recently developed validated methodology. This is now the second exon-skipping agent to have shown a statistically significant increase in dystrophin production, validating the exon-skipping approach to treating DMD boys with amenable mutations.”

PRICE ACTION

SRPT closed at $41.08. It last traded at $46.50 in pre-market trading.


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Insmed Could be Sold

Insmed seen as potential target after inhaled antibiotic study succeeds

Insmed seen as potential target after inhaled antibiotic study succeeds. See Stockwinners.com Market Radar for details

Insmed (INSM) has announced results from a phase 3 clinical trial, saying its inhaled antibiotic successfully treated patients with a rare lung disease caused by a bacterial infection.

Following the news, Citi analyst Liav #Abraham argued that the positive headline data could increase the likelihood of Insmed being a viable takeover target.

STUDY RESULTS

This morning, Insmed announced top-line data from its Phase 3 CONVERT study, saying it met its primary endpoint of culture conversion by month 6 with statistical and clinical significance.

The study demonstrated that the addition of ALIS to guideline-based therapy eliminated evidence of nontuberculous mycobacterial, or NTM, lung disease caused by MAC in sputum by month 6 in 29% of patients, compared to 9% of patients on GBT alone.

Insmed plans to pursue accelerated approval of ALIS under subpart H based on the data from the CONVERT study, which will be reviewed by the Division of Anti-Infective Products, the company noted.

TAKEOUT TARGET

In a research note this morning, Citi’s Abraham told investors that the positive headline data for Insmed’s ALIS for the treatment of NTM lung disease bode well for the potential approval of the therapy.

The analyst highlighted the clinical meaningfulness of the data, with a 20% difference in culture conversion reported between the active and control arms of the trial.

Additionally, Abraham pointed out that the debate on the stock is likely to shift to the commercial outlook for ALIS, with pricing also being key to the uptake of the drug. The positive headline data “could well increase the likelihood of the company as a viable takeout target,” she argued. Abraham reiterated a Neutral rating on the shares.

ACCELERATED APPROVAL

Also commenting on the announcement, Leerink analyst Joseph Schwartz told investors in a research note of his own that after achieving statistical significance in the primary endpoint for ALIS, Insmed’s management intends on pursuing accelerated approval, in line with the expectations laid out during the company’s recent analyst day.

The higher reported adverse events in the ALIS cohort were expected, and while the secondary endpoint of six-minute walk test did not achieve statistical significance overall, patients who culture converted showed a statistical significant improvement, which was a pre-specified analysis, #Schwartz contended.

The analyst reiterated an Outperform rating on the shares.

Meanwhile, his peer at #Evercore ISI increased his price target for Insmed to $40 from $28 following the clinical data. Analyst Josh #Schimmer noted, however, that the commercial outlook is still not well defined and will likely evolve over time as more is learned about the CONVERT study results, the companion 312 study and the company’s efforts to expand labelled use. Schimmer reiterated an Outperform rating on the shares.

PRICE ACTION

In Tuesday’s trading, shares of Insmed (INSM) are up $14 to $26.


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This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.