Achaogen Announces Review of Strategic Alternatives and Corporate Restructuring

Achaogen, Inc. (NASDAQ: AKAO), a biopharmaceutical company discovering, developing and commercializing innovative antibacterial agents to address multi-drug resistant (MDR) gram-negative infections, today announced that it has begun a review of strategic alternatives to maximize shareholder value, including but not limited to the potential sale or merger of the Company or its assets. In connection with this review, the Company has engaged Evercore as an independent financial advisor.

The Company also announced a restructuring of its organization to preserve cash resources which is expected to reduce operating expenses by approximately 35-40 percent, excluding one-time charges. The restructuring is expected to be largely completed before the end of 2018.  The restructuring is designed to focus the company’s cash resources on the continued successful launch of ZEMDRI and advancing C-Scape.

“We are committed to evaluating strategic alternatives that enhance value for shareholders while maintaining our focus on the successful launch of ZEMDRI and the development of C-Scape as well as aggressively managing our operating expenses,” said Blake Wise, CEO of Achaogen. “We look forward to providing an update on ZEMDRI commercialization and launch at our upcoming third quarter earnings call.”

There can be no assurance that the strategic review will result in any transaction or other outcome. The Company does not currently intend to publicly discuss or disclose further developments of the strategic review unless and until its Board of Directors has approved a transaction or otherwise determined that further disclosure is appropriate.

Conference Call

The Company will host a conference call and webcast to review Third Quarter 2018 financial results and details of the corporate restructuring on November 8, 2018 at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time. To participate by telephone, please dial 866-952-8559 (domestic) or 785-424-1745 (international); conference ID: ACHAOGEN. A live and archived audio webcast can be accessed through the Investor Relations section of the Company’s website at www.achaogen.com. The archived audio webcast will remain available on the Company’s website for 30 days following the conference call.

https://www.marketscreener.com/ACHAOGEN-INC-15991095/news/Achaogen-Announces-Review-of-Strategic-Alternatives-and-Corporate-Restructuring-27548464/

Stemline to Present on cancer med at ASH meet

Stemline Therapeutics, Inc. (Nasdaq: STML), a biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, announced today that ELZONRIS (tagraxofusp; SL-401), a novel targeted therapeutic directed to CD123, will be featured in four presentations, including an oral presentation, at the 2018 American Society of Hematology (ASH) Annual Meeting and Exposition, to be held December 1-4, 2018 in San Diego, CA.

Additionally, the Company is hosting an investor/analyst event on December 3, 2018 and plans to provide updates on the progress of its pre-commercial activities, disease awareness campaign, and market expansion efforts.

Ivan Bergstein, M.D., CEO of Stemline Therapeutics, commented, ‘We are honored that ASH has selected the BPDCN pivotal results for oral presentation. This selection underscores the heightening awareness of the disease – BPDCN, the target – CD123, and the clinical impact of the drug candidate – ELZONRIS. In addition, our regulatory team continues to work diligently in an effort to make ELZONRIS available to patients as quickly as possible, and our commercial team continues to execute on our broad-based pre-launch initiatives. This includes the build-out of our sales, marketing and reimbursement teams as well as continuing to advance our disease awareness campaign. In parallel, we are excited to present updated clinical data from our ongoing clinical trials in patients with chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF). Based on these data, we are enthusiastic about our plans to implement pivotal trials, or cohorts, in these devastating malignancies.’

https://www.marketscreener.com/STEMLINE-THERAPEUTICS-INC-11168022/news/Stemline-Therapeutics-Announces-Four-ELZONRIS-Presentations-Including-an-Oral-Presentation-at-Up-27548934/

Sumitomo Dainippon Pharma to present on Anti-Cancer Agent at ASH 2018

Sumitomo Dainippon Pharma Co., Ltd. (Head Office: Osaka, Japan; Representative Director, President and CEO: Hiroshi Nomura) announced today that the clinical data for an investigational inhibitor of cyclin-dependent kinase 9 (CDK9), alvocidib (generic name), will be presented in an oral session at the American Society of Hematology(ASH) Annual Meeting and Exposition in San Diego from December 1 to 4, 2018.

For your information, the clinical data to be presented at the oral session are the stage-1 results of a phase 2 study, Zella 201, for alvocidib in patients with relapsed or refractory MCL-1- dependent Acute Myeloid Leukemia (NCT02520011).

The abstract is now available on the official website of ASH. (https://ash.confex.com/ash/2018/webprogram/start.html)

https://www.marketscreener.com/SUMITOMO-DAINIPPON-PHARMA-6492512/news/Sumitomo-Dainippon-Pharma-Sumitomo-Dainippon-Pharma-announces-the-Clinical-Data-of-Investigational-27548945/

Clearside Hit as Phase III Xipere Failure Forces the Company to Discontinue Study

Shares of Alpharetta, Georgia-based Clearside Biomedical, Inc. are down more than 50 percent in premarket trading after the company announced its late-stage eye disease treatment added to Regeneron’s Eylea failed to distinguish itself against the established drug in the treatment of retinal vein occlusion.

Shares fell from Friday’s close of $5.56 to $2.65 after the company made the announcement. Clearside was investigating its treatment Xipere (formerly known as suprachoroidal CLS-TA) used in conjunction with Eylea, an anti-VEGF agent, against Eylea alone, in the treatment of retinal vein occlusion. In its announcement, Clearside said the primary endpoint of the trial was the improvement in best corrected visual acuity from baseline of at least 15 letters on the Early Treatment Diabetic Retinopathy Study between the two treatment arms. The company said that the Phase III SAPPHIRE trial showed that approximately 50 percent of patients in both arms showed at least a 15 letter improvement in vision. The company said there was no additional benefit for patients receiving Xipere alongside intravitreal Eylea.

With that data in hand, Clearside Chief Executive Officer Daniel White said the company plans to discontinue clinical development of the combination therapy for retinal vein occlusion. Not only will the SAPPHIRE trial be discontinued, but White said the companion Phase III clinical trial known as TOPAZ would also be shut down. The TOPAZ trial aimed to demonstrate the combination of Xipere and Eylea was a superior treatment to Eylea alone in patients with macular edema secondary to retinal vein occlusion.

“We believe the opportunity in our primary indication, uveitis, remains very attractive. Awareness and acceptance of the strong clinical profile of Xipere as a potential monotherapy in treating uveitic macular edema is growing, and we remain on track to submit our NDA for this indication before the end of this year,” White added in a statement.

Xipere is a proprietary suspension of the corticosteroid triamcinolone acetonide formulated for administration to the back of the eye via the suprachoroidal space. The approach is designed to enable rapid dispersion of medicine to the back of the eye so that adequate medicine reaches and stays at the site of disease and has potential to act longer, according to Clearside data. Xipere is being studied for unmet or underserved sight-threatening eye diseases that manifest in the retina and the choroid.

In the company’s Phase III treatment for uveitic MD, at week 24, 40.9 percent of patients experienced resolution in the Xipere arm, compared to 0 percent of patients in the control arm who underwent a sham procedure, the company said last week at the American Academy of Ophthalmology meeting. Additionally, in the Xipere arm, 68 percent of patients with any baseline level of vitreous haze, 72 percent of patients with anterior chamber cell inflammation and 74 percent of patients with anterior chamber flare  had their inflammation resolve, compared to 23 percent, 17 percent and 20 percent, respectively, of patients in the control arm, the company said.

https://www.biospace.com/article/clearside-biomedical-shares-plunge-as-phase-iii-xipere-failure-forces-the-company-to-discontinue-study/

Takeda Licenses Oncology Target from Crescendo Biologics

Acting on an option out of 2016 collaboration deal, Japan’s Takeda Pharmaceutical licensed Humabodies directed to one of its oncology targets from Cambridge, UK’s Crescendo Biologics.

Crescendo developed multi-functional Humabody therapeutics based on a transgenic mouse platform that generates 100 percent human VH domain building blocks (Humabody VH). These molecules can be configured to engage therapeutic targets, which results in larger therapeutic windows compared to typical IgG approaches.

Humabodies are small and can quickly penetrate and accumulate in tissues and tumors while quickly clearing from tumors and tissues. This minimizes systemic toxicity.

In 2016, the two companies formed a collaboration and license agreement. In it, Takeda has the right to develop and commercialize Humabody-based therapeutics that came out of the collaboration. Crescendo is eligible for clinical development, regulatory and sales-based milestones up to $754 million in addition to royalties on Humabody-based product sales by Takeda.

“The team at Crescendo has made great progress on our Humabody programs, working closely with the Takeda team,” stated Peter Pack, chief executive officer of Crescendo “To date, we have met all the technical milestones on time or earlier than planned, which is proof of our excellent collaboration. We are delighted that the option to license has been taken by Takeda ahead of schedule and look forward to further future successes.”

The decision to exercise the option comes earlier than expected.

Crescendo’s lead compound is CB307, a novel bispecific PSMA-targeted T-cell enhancer for the selective activation of tumor-specific T-cells exclusively within the tumor microenvironment. This avoids systemic toxicity. The company states that, “This highly modular format can be reconfigured to create a pipeline of multiple therapeutic candidates each treating a different cancer indication, by targeting any of a range of alternative tumor-specific markers.”

In mid-August, Crescendo announced it had hit another technical milestone in the Takeda collaboration. Although the company did not disclose the amount of the milestone payments, it did indicate it had delivered another diverse panel of functional Humabody leads against Takeda’s second selected target. A previous announce about an equivalent milestone was announced in April 2018.

At the time, Pack stated, “Our highly productive relationship draws together Takeda’s deep oncology experience with Crescendo’s expertise in developing optimally configured Humabodies. Together we are fast progressing towards our goal of developing next generation, highly modular and multi-functional biologics against cancer. This is another important step forward for Crescendo and further validates the power of our innovative technology.”

The companies did not specify the targets or the specific Humabody. Crescendo lists three compounds on its website that are available for partnering or out-licensing. They are all in oncology. CB201 is a differentiated biparatropic PD-1 antagonist that has shown enhanced in vivo efficacy over commercial IgG-based PD-1 antagonists. CB213 is a novel bispecific PD-1 X LAG-3 antagonist that delivers potent simultaneous dual checkpoint blockade in patients that don’t respond to PD-1 blockade alone. And CB108, a half-life extended biparatropic PSMA-specific Humabody Drug Conjugate (HDC) that has shown superior tumor targeting, enhanced internalization and highly efficient accumulation in target-positive tumors.

https://www.biospace.com/article/takeda-licenses-oncology-target-from-crescendo-biologics/

Novartis Plans for 60 Regulatory Filings Over the Next Three Years

Days after Novartis axed one-fifth of its research programs, the Swiss pharma giant said it intends to seek regulatory approval for 60 new treatments between 2019 and 2021.

Novartis made the announcement today during a research and development update in London. The company said the conference provides investors with a deeper insight into its pipeline and the potential therapeutics that could be on the market over the next few years. In its announcement this morning, Novartis said the company has 26 potential blockbusters in confirmatory development and 13 projects in clinical development across Cell, Gene & Radioligand therapies.

With a bold prediction of a potential 26 blockbuster drugs, Novartis’ secondary multiple sclerosis treatment Mayzent is expected to lead the way. Mayzent (siponimod) is currently under review by the U.S. Food and Drug Administration (FDA), as well as the European Medicines Agency (EMA). In April, Novartis released additional data from its Phase III EXPAND trial that showed siponimod-dosed patients gained a significant benefit in cognitive processing speed. That data came about one month after Novartis published data that showed multiple sclerosis drug siponimod generated significant improvements in patients, including a 21 percent decrease in the risk of disease progression. If approved, Novartis said it anticipates the launch of Mayzent in the first quarter of 2019. Mayzent would bolster its MS pipeline that is expected to see some losses due to generic competition for its blockbuster drug Gilenya.

In addition to Mayzent, Novartis pointed to several other drugs that have the potential to be blockbusters. Novartis’ ofatumumab is a next-generation B-cell depletor with a potentially favorable safety profile from faster b-cell repletion and preserved immunity, and with a convenient monthly sub-cutaneous dosing. It is also being studied as a potential therapy for multiple sclerosis. If approved, it could be seen as a rival for Roche’s Ocrevus. Ofatumumab has already been approved as a treatment for chronic lymphocytic leukemia under the brand name Arzerra.

Novartis also highlighted a few other drugs that have the potential to be blockbusters, including moderate-to-severe asthma treatment fevipiprant; nAMD treatment brolucizumab, which Novartis said has the potential to reduce treatment burden by drying the retina better with fewer injections; and in sickle cell disease, the therapeutic crizanlizumab has shown itself to be effective in clinical trials and the company anticipates regulatory filing in 2019.

During the Monday showcase, Novartis pointed to the potential launch of its therapy for spinal muscular atrophy type I, AVXS101. The company said it was on track for a launch in the first half of 2019 and clinical development is ongoing for all other SMA subtypes. Novartis gained AVXS101 through its $8.7 billion acquisition of AveXis earlier this year. Novartis said it anticipates regulatory approvals for AAVXS101 early next year in the United States, Europe and Japan.

Novartis also pointed to some other highlights in its Radioligand therapy. Last year, Novartis acquired Advanced Accelerator Applications for $3.9 billion and successfully launched Lutathera in neuroendocrine tumors (NET) and has projects in development for indications beyond NET. In October Novartis announced the acquisition of Endocyte for $2.1 billion to expand its radiopharmaceuticals business.

Novartis also announced that it is pursuing new indications for some of its established brand medications such as Cosentyx, Entresto and Gilenya. The company said Cosentyx is expected to be Novartis’ largest drug next year, with robust growth in all three approved indications.

https://www.biospace.com/article/novartis-plans-for-60-regulatory-filings-over-the-next-three-years/

FDA Chief Outlines Reasons Why Powerful Opioid Pain Med Was Approved

Two days after the U.S. Food and Drug Administration approved a powerful new sublingual opioid medication, FDA Commissioner Scott Gottlieb used his Twitter account to defend the approval amid concerns that the medication is susceptible to abuse.

Developed by AcelRx Pharmaceuticals, Dsuvia was approved Friday for the management of acute pain in adults that is severe enough to require an analgesic in certified medically supervised healthcare settings, such as hospitals, surgical centers and emergency departments. Dsuvia, which is reportedly 1,000 times more powerful than morphine, is in tablet form and is meant to be used in a medically supervised setting, however, there is significant concern that the drug could be abused by addicts, particularly in the light of the opioid epidemic sweeping the United States.

On Sunday, Gottlieb took to Twitter, as well as posting a blog on the FDA’s website, to outline why the drug was approved in a time when the country is reeling from an opioid crisis that takes the lives of 116 Americans daily, according to the U.S. Department of Health and Human Services.

Despite the abundance of opioid medications on the market, Gottlieb said the FDA should consider the approval of new opioid pain medications that can help fill targeted medical needs. In the case of Dsuvia, Gottlieb said the drug has some unique aspects that fill a void in the market. He said the sublingual form of the drug makes it easy for some patients to take, particularly those who had difficulty swallowing a pill or receiving intravenous treatment. Specifically, he pointed to the potential use of Dsuvia on the battlefield. Gottlieb said the U.S. Department of Defense worked closely with AcelRx to develop the medication with that goal in mind.

“This opioid formulation, along with Dsuvia’s unique delivery device, was a priority medical product for the Pentagon because it fills a specific and important, but limited, unmet medical need in treating our nation’s soldiers on the battlefield. The involvement and needs of the DoD in treating soldiers on the battlefield were discussed by the advisory committee,” Gottlieb said.

While the potency of Dsuvia raises concerns over its potential for abuse, Gottlieb said the FDA set certain guidance on the administration of the drug. Dsuvia is not for home use and should not be used for more than 72 hours. Additionally, Gottlieb said it will only be administered by a certified healthcare provider using a single-dose applicator.

“These are important steps to help prevent abuse, misuse and potential diversion. The drug is also reserved for use in patients for whom alternative pain treatment options have not been tolerated or provided adequate relief; or are not expected to do so,” Gottlieb added.

Friday’s approval of Dsuvia comes one year after the FDA rejected approval of the drug. In October 2017, the FDA issued a Complete Response Letter to AcelRx calling for additional safety data. The regulatory agency wanted additional safety data for the maximum dose of the drug from at least 50 patients who participated in late-stage clinical trials. AcelRx filed its NDA based off two open-label Phase III studies that demonstrated a clinically significant drop in pain levels for patients.

AcelRx Chief Executive Officer Vince Angotti said the features of Dsuvia and the treatment settings are an important step in the management of acute pain. Angotti said the company is committed to the “safe and effective administration” of the newly-approved Dsuvia through the company’s FDA-approved Risk Evaluation and Mitigation Strategies program.

Pamela Palmer, co-founder and chief medical officer of AcelRx, said the approval of Dsuvia is the “culmination of nearly 15 years of research to improve the standard of care for managing acute pain in medically supervised settings.”

AcelRx plans to launch Dsuvia in the first quarter of 2019. Shares of AcelRx jumped more than 15 percent in Friday trading to close at $4.80 per share. The stock is up slightly in pre-market trading.

https://www.biospace.com/article/fda-commissioner-gottlieb-outlines-reasons-why-a-powerful-opioid-pain-treatment-was-approved/