AbbVie’s antidepressant achieves positive results

AbbVie’s cariprazine met primary endpoint in Phase 3 study

AbbVie (ABBV) announced top-line results from two Phase 3 clinical trials, Study 3111-301-001 and Study 3111-302-001, evaluating the efficacy and safety of cariprazine as an adjunctive treatment for patients with major depressive disorder.

Cariprazine, sold under the brand names Vraylar in the United States and Reagila in the European Union, is an atypical antipsychotic which is used in the treatment of schizophrenia, bipolar mania, and bipolar depression.

Drug pipeline looks very promising for Abbvie

In Study 3111-301-001, cariprazine showed a statistically significant change from baseline to week six in the Montgomery-Asberg Depression Rating Scale total score compared with placebo.

The Montgomery–Asberg Depression Rating Scale (MADRS) is a ten-item diagnostic questionnaire which psychiatrists use to measure the severity of depressive episodes in patients with mood disorders. 

Patients treated with cariprazine at 1.5 mg/day achieved improved MADRS total score at week six compared to placebo.

Patients treated with cariprazine at 3.0 mg/day demonstrated improvement in MADRS total score at week six over placebo but did not meet statistical significance.

In Study 3111-302-001, cariprazine demonstrated numerical improvement in depressive symptoms from baseline to week six in MADRS total score compared with placebo but did not meet its primary endpoint for either the 1.5 mg/day or 3.0 mg/day dose.

In a previously published Phase 2/3 registration-enabling study, RGH-MD-75, patients treated with cariprazine flexible doses of 2.0-4.5 mg/day in addition to ongoing antidepressant therapy met the primary endpoint and achieved improved MADRS total scores at week eight compared to placebo.

Based on the positive results of studies 3111-301-001 and RGH-MD-75, and the totality of data reported, AbbVie intends to submit a supplemental New Drug Application with the U.S. FDA for the expanded use of cariprazine for the adjunctive treatment of MDD.

Separately, AbbVie reported Q3 Global Humira sales of $5.425B up 5.6% on reported basis.

In Q3, Global net revenues from the immunology portfolio were $6.674 billion, an increase of 15.3 percent on a reported basis, or 14.9 percent on an operational basis.

Global Humira net revenues of $5.425 billion increased 5.6 percent on a reported basis, or 5.2 percent on an operational basis.

U.S. Humira net revenues were $4.613 billion, an increase of 10.1 percent.

Internationally, Humira net revenues were $812 million, a decrease of 14.6 percent on a reported basis, or 16.7 percent on an operational basis, due to biosimilar competition.

Treatment for Psoriasis

Global Skyrizi net revenues were $796 million. Global Rinvoq net revenues were $453 million.

Treatment for moderate to severe rheumatoid arthritis

ABBV is up $5 to $114.70.

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FDA Approves Biogen’s Alzheimer’s Drug

FDA approves Biogen Alzheimer’s drug, says benefits outweigh risks

The FDA approved Biogen’s (BIIB) Aduhelm to treat patients with Alzheimer’s disease.

“This approval is significant in many ways. Aduhelm is the first novel therapy approved for Alzheimer’s disease since 2003.

Perhaps more significantly, Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain.

The clinical trials for Aduhelm were the first to show that a reduction in these plaques – a hallmark finding in the brain of patients with Alzheimer’s – is expected to lead to a reduction in the clinical decline of this devastating form of dementia,” the FDA said in a statement.

Eli Lilly announces Alimta label expanded by FDA, Stockwinners
Eli Lilly is a partner with Biogen

It added, “We ultimately decided to use the Accelerated Approval pathway – a pathway intended to provide earlier access to potentially valuable therapies for patients with serious diseases where there is an unmet need, and where there is an expectation of clinical benefit despite some residual uncertainty regarding that benefit.

Brain of an Alzheimer patient

In determining that the application met the requirements for Accelerated Approval, the Agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy.”

The FDA said in its approval statement: “Additionally, FDA is requiring Biogen to conduct a post-approval clinical trial to verify the drug’s clinical benefit. If the drug does not work as intended, we can take steps to remove it from the market. But hopefully, we will see further evidence of benefit in the clinical trial and as greater numbers of people receive Aduhelm. As an agency, we will also continue to work to foster drug development for this catastrophic disease.”

STIFEL

Stifel analyst Paul Matteis reiterates his Buy rating on Biogen shares following the FDA granting accelerated approval of aducanumab, now to be called “Aduhelm,” for the treatment of Alzheimer’s disease.

Approval based on amyloid plaque as a “surrogate” is “definitely unexpected” and appears to be a way for FDA to work around the contentious advisory committee meeting, argues Matteis, who adds that the approval “is a big win.” How investors will risk-adjust revenues that are modeled after completion of a Phase 4 trial and how insurers will treat access for a drug approved based on a biomarker are “highly interesting” questions that will now “be debated at a materially higher stock valuation,” added Matteis. Biogen shares remain halted for trading at midday following news of the FDA approval.

JEFFRIES

Jefferies analyst Andrew Tsai said news of Biogen (BIIB) being granted FDA approval for aducanumab is likely to spark investor enthusiasm across all Alzheimer’s names and he believes the longer-term setup for Athira Pharma (ATHA) looks more attractive now. Given what he views as “the FDA tailwind,” he would buy on strength as he believes the FDA’s aducanumab decision “clearly has a positive readthrough” to Athira, whose Phase I data suggests ATHA-1017 could produce “a profound cognitive benefit” in Phase 2/3 studies expected to read out in 2022, Tsai tells investors.

In that context, he thinks a 25%-50% short-term move for Athira shares “seems reasonable” relative to the company’s current market cap.

Shares of Biogen (BIIB) remain halted while Eli Lilly (LLY), who has an Alzheimer’s disease drug in its pipeline, is up 4% to $210.78 following the news.

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FDA Approves J&J’s one shot Covid-19 Vaccine

Johnson & Johnson Covid vaccine granted emergency approval from FDA 

The Food and Drug Administration issued an emergency use authorization for the third vaccine for the prevention of coronavirus disease. The FDA has determined that the Janssen COVID-19 Vaccine has met the statutory criteria for issuance of an EUA. The totality of the available data “provides clear evidence that the Janssen COVID-19 Vaccine may be effective in preventing COVID-19,” the agency said in a statement.

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The Janssen COVID-19 Vaccine is manufactured using a specific type of virus called adenovirus type 26. The vaccine uses Ad26 to deliver a piece of the DNA, or genetic material, that is used to make the distinctive “spike” protein of the SARS-CoV-2 virus, the FDA said. While adenoviruses are a group of viruses that are relatively common, Ad26, which can cause cold symptoms and pink eye, has been modified for the vaccine so that it cannot replicate in the human body to cause illness, it added. After a person receives this vaccine, the body can temporarily make the spike protein, which does not cause disease, but triggers the immune system to learn to react defensively, producing an immune response against SARS-CoV-2.

The EUA allows Johnson & Johnson’s (JNJ) Janssen COVID-19 vaccine to be distributed in the U.S for use in individuals 18 years of age and older.

Meanwhile, Johnson & Johnson also announced that the U.S. Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices has recommended its single-shot COVID-19 vaccine.

The ACIP recommendation will be forwarded to the Director of the CDC and the U.S. Department of Health and Human Services for review and adoption.

Johnson & Johnson has begun shipping its COVID-19 vaccine and expects to deliver enough single-shot vaccines by the end of March to enable the full vaccination of more than 20M people in the U.S.

The company plans to deliver 100M single-shot vaccines to the U.S. during the first half of 2021. The U.S. government will manage allocation and distribution of the vaccine in the U.S.

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Big Rock Partners buys NeuroRx

NeuroRx to trade on Nasdaq following Big Rock Partners Acquisition merger

Big Rock Partners Acquisition (BRPA) announced that it has entered into an agreement and plan of merger with NeuroRx, a clinical stage, small molecule pharmaceutical company.

Big Rock shares jump on purchase of NeuroRx

NeuroRx develops novel therapeutics for the treatment of COVID-19 and Bipolar Depression.

Under the terms of the transaction, Big Rock and NeuroRx will merge and the company is expected to continue to trade on the Nasdaq Stock Market under the symbol (NRXP).

The transaction is expected to occur in the first or second quarter of 2021.

As a public Nasdaq-listed company, NeuroRx expects to have increased access to capital to continue development of its drug pipeline targeting Central Nervous System/Psychiatry and Respiratory Disease.

NeuroRx is a clinical stage, small molecule pharmaceutical company which develops novel therapeutics for the treatment of central nervous system disorders and life-threatening pulmonary disease.

NeuroRx’s two main drugs are Zyesami which is an application for COVID-related respiratory failure and NRX-101, which focuses on suicidal bipolar depression and PTSD.

Zyesami is a synthetic human vasoactive intestinal peptide, or VIP, a 28 amino-acid natural peptide with 50 years of research. NRX-101 is a fixed-dose combination of D-cycloserine and lurasidone that has advanced to phase 3 with FDA Breakthrough Therapy Designation, a Special Protocol Agreement, Biomarker Letter of Support, and Fast Track Designation.

NeuroRx’s management team is comprised of industry veterans, led by founder, Chairman & CEO Jonathan C. Javitt, MD, MPH, Robert Besthof, MIM (Chief Commercial Officer), William Fricker, MBA, CPA (Chief Financial Officer) and Alessandra Daigneault, JD (Corporate Secretary), who are expected to continue to run the combined company, post-transaction.

All officers and members of the board of Big Rock will resign in connection with the closing of the transactions.

The board of the combined company will initially consist of seven members, including Prof. Jonathan Javitt.

Under the terms of the transaction, Big Rock will issue to NeuroRx’s current equity holders an aggregate of 50M shares of Big Rock common stock for their interests in NeuroRx, representing $500M of equity consideration, assuming a value of $10.00 per common share.

Subject to certain conditions, an aggregate of 25M additional shares of Big Rock common stock will be issued to NeuroRx pre-merger equity holders if, prior to December 31, 2022, RLF-100 receives emergency use authorization by the FDA and the FDA accepts the company’s filing of its application to approve RLF-100.

In addition, subject to certain conditions, a $100M cash earnout may be payable to NeuroRx pre-merger equity holders if, prior to December 31, 2022, either FDA approval of the company’s COVID-19 Drug is obtained and the company’s COVID-19 Drug is listed in the FDA’s or FDA approval of the company’s Antidepressant Drug Regimen is obtained and the company’s Antidepressant Drug Regimen is listed in the FDA’s “Orange Book”.

The boards of both NeuroRx and Big Rock have unanimously approved the proposed transaction.

Completion of the transaction is subject to approval by stockholders of NeuroRx and Big Rock and other customary closing conditions.

BRPA is up 30% to $15.50.

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Sage Therapeutics shares jump on it depression drug

Sage Therapeutics, Biogen announce collaboration on SAGE-217, SAGE-324

Biogen (BIIB) and Sage Therapeutics (SAGE) announced that they have executed a global collaboration and license agreement to jointly develop and commercialize zuranolone for major depressive disorder, postpartum depression and other psychiatric disorders and SAGE-324 for essential tremor and other neurological disorders.

Sage receives a large investment from Biogen

Zuranolone, a potential first-in-class, two-week, once-daily oral therapy in development for the treatment of MDD and PPD, is currently in Phase 3 development as part of the LANDSCAPE and NEST clinical programs.

Zuranolone has breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for MDD and, if successfully developed and approved, has the potential to be a novel treatment paradigm in depression.

Biogen gambles on Sage’s depression drug

The vision for zuranolone in MDD and PPD is based on its potential, being evaluated in the LANDSCAPE and NEST development programs, to work rapidly and to continue providing sustained benefit beyond the period of dosing.

Together, these two features, if supported by positive clinical efficacy and safety data, could provide an alternative option to how depression is treated today based on a target profile of an “as-needed” short course of treatment for a depressive episode, with rapid and sustained efficacy and favorable tolerability.

The development of an “as-needed” treatment for depression may help ease the difficulties associated with chronic use of antidepressants and may enhance quality of life and patient adherence.

To date, two positive pivotal studies have been completed with zuranolone 30 mg, one in MDD (MDD-201) and one in PPD.

Additionally, while the Phase 3 MOUNTAIN Study of zuranolone in MDD did not meet its primary endpoint, the encouraging data from the recently announced MOUNTAIN six-month follow-up period and the topline interim SHORELINE Study analysis, suggest the potential for zuranolone, if successfully developed and approved, to be uniquely positioned as a disruptive, distinct and novel treatment approach for patients.

Biogen and Sage believe that zuranolone is clinically active in MDD based on the data compiled to date and look forward to planned data readouts in 2021.

Sage is pursuing three development pathways for zuranolone in the U.S.: PPD; acute rapid response therapy in MDD when co-initiated with new standard antidepressant therapy; and “as-needed,” or episodic, treatment of MDD.

As a result, Sage is advancing four additional pivotal studies evaluating a 50 mg dose of zuranolone: a Phase 3 study in PPD, a Phase 3 study of use as an acute RRT in patients with MDD when co-initiated with new standard antidepressant therapy , a Phase 3 study in the acute treatment of MDD and an open label Phase 3 study evaluating the long-term safety, tolerability and efficacy of “as-needed” repeat treatment. Data from these studies are expected in 2021.

Upon closing of the transaction, Biogen and Sage will collaborate to further define the development and commercialization strategy for zuranolone.

Beyond PPD and MDD, zuranolone may also have potential in other psychiatric disorders including bipolar disorder and generalized anxiety disorder. SAGE-324 is a next-generation positive allosteric modulator of GABAA receptors in Phase 2 development for essential tremor with potential in other neurological conditions such as epilepsy and PD.

Essential tremor is one of the most common movement disorders estimated to affect over six million patients in the U.S., and current standard of care may be inadequate for many.

Following encouraging results from a Phase 1 open-label study in essential tremor, Sage advanced SAGE-324 to the Phase 2a KINETIC Study, which Sage is currently conducting.

The KINETIC Study is a 28-day placebo-controlled study in patients with essential tremor expected to read out in 2021.

Upon closing of the transaction, Biogen and Sage will collaborate to further define the development and commercialization strategy for SAGE-324 in essential tremor and, as appropriate, for potential expansion into other neurological disorders.

The strategic collaboration is global in scope and under the terms of the agreement, Sage will receive $1.525 billion in cash to be comprised of an upfront payment of $875 million and a $650 million equity investment in Sage from the purchase of approximately 6.2 million newly issued shares of Sage common stock at a price of $104.14 per share, representing a premium of 40 percent over the 30-day volume-weighted average share price of $74.39 per share as of November 25, 2020.

Should the zuranolone and SAGE-324 programs achieve certain development and commercial milestones, Sage will be eligible to receive up to approximately $1.6 billion in potential milestone payments. Biogen and Sage will share responsibility and costs for development as well as profits and losses for commercialization in the U.S.. Outside the U.S., Biogen will be responsible for development and commercialization, excluding Japan, Taiwan and South Korea with respect to zuranolone, and will pay Sage tiered royalties in the high teens to low twenties. Closing of the transaction is contingent on completion of review under antitrust laws, including the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in the U.S., and other customary closing conditions. The transaction is expected to close by the end of January 2021.

BIIB last traded at $241.75. SAGE last traded at $84.00.

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Watch Jazz Pharmaceuticals!

Jazz Pharmaceuticals receives EU Marketing Authorization for Sunosi

Jazz Pharmaceuticals (JAZZ) announced that the European Commission approved Sunosi to improve wakefulness and reduce excessive daytime sleepiness in adults with narcolepsy or obstructive sleep apnea whose EDS has not been satisfactorily treated by primary OSA therapy, such as continuous positive airway pressure.

Jazz should be in play, Stockwinners

Sunosi is the first dual-acting dopamine and norepinephrine reuptake inhibitor approved to treat EDS in adults living with narcolepsy or OSA and the only licensed therapy in the European Union for the treatment of EDS in adults living with OSA.

Once-daily Sunosi is approved with doses of 75 mg and 150 mg for people with narcolepsy and doses of 37.5 mg, 75 mg and 150 mg for patients with OSA.

Sunosi is approved in Europe, Stockwinners

At Week 12 of the Phase 3 clinical trial, 150 mg of solriamfetol for narcolepsy patients and both 75 mg and 150 mg doses for OSA patients demonstrated improvements in wakefulness compared to placebo as assessed via the maintenance of wakefulness test from approximately one hour post-dose through approximately nine hours post-dose.

The European Commission approval extends to all European Union Member States, as well as Iceland, Norway and Liechtenstein.

The Marketing Authorization Application for Sunosi is based on data from four randomized placebo-controlled studies included in the Treatment of Obstructive sleep apnea and Narcolepsy Excessive Sleepiness clinical trial program.

Data from the studies in the TONES program demonstrated the superiority of solriamfetol relative to placebo.

The Marketing Authorisation Application (MAA) for Sunosi is based on data from four randomised placebo-controlled studies included in the Treatment of Obstructive sleep apnea and Narcolepsy Excessive Sleepiness (TONES) clinical trial program. Data from the studies in the TONES program demonstrated the superiority of solriamfetol relative to placebo.

JAZZ closed at $151.05.

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Epizyme’s Tazemetostat receives FDA’s okay

Epizyme says FDA advisory committee votes unanimously in favor of tazemetostat

Epizyme (EPZM) announced that the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration voted 11 – 0 in favor of the benefit-risk profile of tazemetostat as a treatment for patients with metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery.

Epizyme shares halted ahead of FDA meeting, Stockwinners

“Today’s ODAC outcome is a significant step toward addressing the critical needs of ES patients.

This is a remarkable achievement marking the culmination of years of hard work by the entire Epizyme team. If approved, we will have the opportunity to change how patients with this devastating cancer are treated.

Our commercial-readiness is complete, and we look forward to finalizing our dialog with the FDA,” said Robert Bazemore, president and CEO of Epizyme.

Epizyme’s New Drug Application, or NDA, for tazemetostat is, an oral, first-in-class EZH2 inhibitor, for the treatment of patients with metastatic or locally advanced epithelioid sarcoma (ES) who are not eligible for curative surgery.

Epithelioid Sarcoma is a rare form of cancer, Stockwinners

The advisory committee vote will be non-binding, but FDA takes its recommendations into consideration when reviewing related applications for marketing approval.

ES is a rare and aggressive soft tissue sarcoma characterized by a loss of the INI1 protein. Patients are most commonly diagnosed as young adults, between 20 and 40 years of age, typically with no patients living past five years from diagnosis. ES becomes more aggressive after recurrence or once it has metastasized, with a typical survival of less than one year for patients with metastatic disease.

Epizyme, Inc. discovers, develops, and commercializes novel epigenetic medicines for patients with cancer and other diseases primarily in the United States.

EPZM halted at $18.19.

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Axsome shares soar on its depression drug

Axsome Therapeutics: AXS-05 achieves primary endpoint in Phase 3 trial, Shares jump 77%

Axsome Therapeutics (AXSM) announced that AXS-05, a novel, oral, investigational NMDA receptor antagonist with multimodal activity, met the primary endpoint and rapidly and significantly improved symptoms of depression in the GEMINI Phase 3 trial in major depressive disorder.

Axsome Therapeutics shares soar on its Gemini depression drug study, Stockwinners

The GEMINI study was a randomized, double-blind, placebo-controlled, multi-center, U.S. trial, in which 327 adult patients with confirmed moderate to severe MDD were randomized to treatment with either AXS-05 or placebo once daily for the first 3 days and twice daily thereafter for a total of 6 weeks.

AXS-05 met the primary endpoint by demonstrating a highly statistically significant reduction in the Montgomery-Asberg Depression Rating Scale total score compared to placebo at Week 6, with mean reductions from baseline of 16.6 points for AXS-05 and 11.9 points for placebo.

AXS-05 rapidly and durably improved depressive symptoms as compared to placebo with statistical significance on the MADRS total score demonstrated at Week 1, the earliest time point assessed, and at all time points thereafter.

Rates of remission from depression were statistically significantly greater for AXS-05 compared to placebo at Week 2 and at every time point thereafter, being achieved by 39.5% of AXS-05 patients compared to 17.3% of placebo patients at Week 6.

AXS-05 demonstrated rapid onset of action with statistically significant improvement as compared to placebo on numerous endpoints at Week 1, or only 4 days after the start of twice daily dosing.

Statistically significant improvements at Week 1 were observed for MADRS total score; Patient Global Impression-Improvement; Clinical Global Impression-Severity; Clinical Global Impression-Improvement; Quick Inventory of Depressive Symptomatology-Self-Rated; Quality of Life Enjoyment and Satisfaction Questionnaire-Short Form; and other endpoints.

On all secondary endpoints including the following, AXS-05 demonstrated statistically significant improvement at Week 6 compared to placebo, reflecting increasing treatment effects over time: clinical response on the MADRS total score; PGI-I; CGI-S; CGI-I; QIDS-SR-16; Sheehan Disability Scale; and Q-LES-Q-SF.

AXSM is up 77% to $82.66.

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Synthorx sold for $2.5 billion

Sanofi to acquire Synthorx for $68 per share in cash

Sanofi (SNY) and Synthorx (THOR) entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Synthorx for $68 per share in cash, which represents an aggregate equity value of approximately $2.5B, on a fully diluted basis.

Synthorx sold for $2.5 billion, Stockwinners

The transaction was unanimously approved by both the Sanofi and Synthorx boards.

Under the terms of the merger agreement, Sanofi will commence a cash tender offer to acquire all of the outstanding shares of Synthorx common stock for $68 per share in cash.

The $68 per share acquisition price represents a 172% premium to Synthorx’s closing price on December 6.

Following the successful completion of the tender offer, a wholly owned subsidiary of Sanofi will merge with Synthorx and the outstanding Synthorx shares not tendered in the tender offer will be converted into the right to receive the same $68 per share in cash paid in the tender offer.

The tender offer is expected to commence in December.

Sanofi plans to finance the transaction with cash on hand. Subject to the satisfaction or waiver of customary closing conditions, Sanofi expects to complete the acquisition in the first quarter of 2020.

“Synthorx’s Expanded Genetic Alphabet platform is expected to be a source for developing a differentiated therapeutic pipeline. Alone and in combination with other existing Sanofi platforms, including the Nanobody technology, it will enable the company to develop a wide range of novel biologics, including drug conjugates, protein fusions, and multi-specific biologics, with applications beyond oncology and extending to other therapeutic areas… The addition of THOR-707 and Synthorx’s other earlier-stage cytokine programs to Sanofi’s pipeline will enhance Sanofi’s position in oncology, and in immuno-oncology. We expect IL-2 to become a foundation of future IO-IO combinations as well as offering multiple combination opportunities with Sanofi’s clinical and pre-clinical oncology assets, including with PD-1, CD-38, and molecules that modulate effector T-cells and natural killer cells.”

THOR closed at $25.04, it last traded at $67.43. SNY closed at $46.03.

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Epizyme jumps on $100M investment

Royalty Pharma to purchase future royalties on tazemetostat from Eisai

Royalty Pharma announced that it has agreed to pay $330M to purchase Eisai Co.’s (ESALY) royalties on future worldwide sales of tazemetostat, Epizyme’s (EPZM) lead investigational agent, outside of Japan, and made an equity investment in Epizyme of $100M, with options to invest up to an additional $100M in Epizyme common stock.

In addition, investment funds managed by Pharmakon Advisors agreed to provide $70M in senior-secured loans with the possibility to fund up to $370M over time.

Tazemetostat is a first-in-class, oral EZH2 inhibitor in clinical development for certain oncology indications, including epithelioid sarcoma and follicular lymphoma.

Epizyme receives a $100M investment, Stockwinners

Under a collaboration agreement between Epizyme and Eisai, Epizyme is responsible for the development and worldwide commercialization of tazemetostat (outside of Japan) and Eisai is responsible for the development and commercialization of tazemetostat in Japan.

As part of the agreement, Epizyme owes milestones and royalties on sales of tazemetostat outside of Japan to Eisai, and Eisai owes royalties on sales of tazemetostat in Japan to Epizyme.

Under the terms of its agreement with Eisai, Royalty Pharma has acquired Eisai’s future worldwide royalties on net sales by Epizyme of tazemetostat outside of Japan, for an upfront payment of $110M plus up to an additional $220M for the remainder of the royalty upon FDA approval of tazemetostat for certain indications.

Under the terms of its agreement with Epizyme, Royalty Pharma will make an upfront payment of $100M for shares of Epizyme common stock based on a price of $15 per share.

Royalty Pharma makes $100M investment in Epizyme, Stockwinners

Epizyme has an 18-month option to sell an additional $50M of its common stock to Royalty Pharma at then prevailing prices, not to exceed $20 per share, and Royalty Pharma has a three-year option to purchase an additional 2.5M shares of Epizyme common stock at $20 per share.

In addition, Royalty Pharma will make additional payments to Epizyme if annual net sales of tazemetostat outside of Japan exceed certain thresholds, and Epizyme has assigned to Royalty Pharma the future royalty streams on tazemetostat sales in Japan previously owed to Epizyme by Eisai.

Epizyme has also agreed to appoint a representative from Royalty Pharma to its board of directors. Under the terms of the loan agreement with Epizyme, investment funds managed by Pharmakon Advisors will fund $25M at closing and up to an additional $45M in two tranches.

In addition, the loan agreement contemplates the potential for an additional $300M, subject to mutual agreement of the parties. The loans will have a coupon of LIBOR + 7.75% and a 5-year maturity.

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Amag Pharma announces publication of PROLONG trial in journal

AMAG Pharmaceuticals (AMAG) announced that the results of the PROLONG trial were published online in the American Journal of Perinatology.

Amag Pharma shares rise ahead of FDA meeting, Stockwinners

AMAG Pharmaceuticals, Inc. (AMAG) announced that the results of PROLONG (Progestin’s Role in Optimizing Neonatal Gestation) were published online in the American Journal of Perinatology. PROLONG was a randomized, double-blind, placebo-controlled clinical trial evaluating 17-OHPC (17-α-hydroxyprogesterone caproate or Makena®) in patients with a history of a prior spontaneous singleton preterm delivery. The PROLONG trial was conducted as part of an approval commitment under the Food & Drug Administration’s (FDA) “Subpart H” accelerated approval process. 

As previously announced, the PROLONG trial did not meet the two pre-specified co-primary endpoints of a reduction of preterm birth and a reduction in the neonatal morbidity and mortality index, in contrast to the original Meis trial results published in the New England Journal of Medicine.

PROLONG reaffirmed that the 17-OHPC safety profile is comparable to placebo, the company said.

Amag Pharma (AMAG) shares rise ahead of FDA meeting, Stockwinners

Makena is a progestin indicated to reduce the risk of preterm birth in women pregnant with a single baby who have a history of singleton spontaneous preterm birth.

Makena was approved by the FDA in February 2011 and was granted orphan drug exclusivity through February 3, 2018. In February of 2018, AMAG introduced the prefilled Makena auto-injector containing a short, thin, non-visible needle for subcutaneous use, offering patients and providers a new administration option.

The FDA will hold a meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee on October 29 to “better understand and interpret the PROLONG trial data,” AMAG added.

Analysts’ Comments

Piper Jaffray analyst Christoper Raymond said the briefing documents posted this morning ahead of Tuesday’s advisory committee meeting to discuss Makena’s failed confirmatory PROLONG trial “leave the door open” for the continued marketing of the drug.

Draft questions propose pursuing withdrawal of Makena’s approval, leaving Makena on the market but requiring a new confirmatory trial, or leaving it on the market without a new confirmatory trial and he thinks it appears that the FDA “is leaning toward the latter two options.”

However, he also thinks that the detailed PROLONG data that have now been provided for the first time “make a strong argument for the drug’s withdrawal,” Raymond tells investors.

The analyst sees the full PROLONG data having “some chilling effect” on overall use whether the FDA pulls the drug or not and he keeps a Neutral rating on Amag Pharmaceuticals shares.

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Karyopharm Therapeutics shares jump on FDA decision

Karyopharm announces FDA approval of XPOVIO-dexamethasone combination for multiple myeloma

Karyopharm Therapeutics shares soar on FDA approval, Stockwinners

Karyopharm Therapeutics Inc. (KPTI) announced that the U.S. Food and Drug Administration has approved oral XPOVIO, a nuclear export inhibitor, in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody.

This indication is approved under accelerated approval based on response rate.

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

XPOVIO to become commercially available in the U.S. on or before July 10, 2019, Stockwinners

The ongoing, randomized Phase 3 BOSTON study evaluating selinexor in combination with Velcade and low-dose dexamethasone will serve as the confirmatory trial.

The FDA’s Accelerated Approval Program was developed to allow for expedited approval of drugs that treat serious conditions and that fill an unmet medical need.

Karyopharm expects XPOVIO to become commercially available in the U.S. on or before July 10, 2019.

A Marketing Authorization Application for selinexor is also currently under review by the European Medicines Agency.

The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Xpovio.

Women who are pregnant or breastfeeding should not take Xpovio because it may cause harm to a developing fetus or newborn baby. Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

The FDA granted this application Fast Track designation. Xpovio also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

Executive Changes

In a regulatory filing, Karyopharm disclosed that on July 2, Karyopharm Therapeutics promoted Perry Monaco to Senior Vice President, Sales responsible for the company’s sales function under the direction of Michael Kauffman, Chief Executive Officer of the company.

On July 2, following the buildout of the company’s commercial organization and development of the company’s product launch strategy, Anand Varadan resigned as Executive Vice President, Chief Commercial Officer of the company, effective July 5.

KPTI last traded at $8.15.

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Array BioPharma sold for $11.4 billion

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

Array BioPharma sold for $11.4 billion, Stockwinners

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

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

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

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

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

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

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

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

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

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

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

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

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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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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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