Merek’s KEYTRUDA Improved Overall Survival in Patients with Non-small Cell Lung Cancer

Merck announced that the pivotal Phase 3 KEYNOTE-189 trial investigating KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in combination with pemetrexed (Alimta) and cisplatin or carboplatin, for the first-line treatment of patients with metastatic non-squamous non-small cell lung cancer (NSCLC), met its dual primary endpoints of overall survival (OS) and progression-free survival (PFS). Based on an interim analysis conducted by the independent Data Monitoring Committee, treatment with KEYTRUDA in combination with pemetrexed plus platinum chemotherapy resulted in significantly longer OS and PFS than pemetrexed plus platinum chemotherapy alone. The safety profile of KEYTRUDA in this combination was consistent with that previously observed.

Results from KEYNOTE-189 will be presented at an upcoming medical meeting and submitted to regulatory authorities.

“KEYNOTE-189 showed significant improvement in overall survival and progression-free survival for patients receiving KEYTRUDA in the first-line setting in combination with traditional chemotherapy, compared with those receiving chemotherapy alone,” said Dr. Roger M. Perlmutter, president, Merck Research Laboratories. “We are deeply grateful to the KEYNOTE-189 patients and investigators for their important contributions to this landmark study, and we look forward to presenting the data in the near future.”

KEYNOTE-189 is a randomized, double blind, placebo controlled, Phase 3 study (, NCT02578680) investigating KEYTRUDA (pembrolizumab) in combination with pemetrexed and cisplatin or carboplatin compared with pemetrexed and cisplatin or carboplatin alone in patients with advanced or metastatic nonsquamous non-small cell lung cancer, regardless of PD-L1 expression. Patients had no EGFR or ALK genomic tumor aberrations, and had not previously received systemic therapy for advanced disease. The KEYNOTE-189 study was done in collaboration with Eli Lilly and Company, the makers of pemetrexed. The dual primary endpoints are OS and PFS; secondary endpoints include overall response rate (ORR) and duration of response (DOR). The study enrolled 614 patients randomized 2:1 to receive either KEYTRUDA (200 mg fixed dose every three weeks) plus pemetrexed (500 mg/m) (with vitamin supplementation) plus cisplatin (75 mg/m2) or carboplatin AUC 5 on day 1 every 3 weeks (Q3W) for 4 cycles followed by KEYTRUDA 200 mg plus pemetrexed (500 mg/m) Q3W or KEYTRUDA placebo 200 mg plus pemetrexed (500 mg/m2) (with vitamin supplementation) plus cisplatin (75 mg/m2) or carboplatin AUC 5 on day 1 every 3 weeks (Q3W) for 4 cycles followed by KEYTRUDA placebo 200 mg plus pemetrexed (500 mg/m2) Q3W until disease progression, unacceptable toxicity, physician decision or consent withdrawal. Patients on the control arm who experienced disease progression, verified by central independent review, were permitted to undergo treatment assignment unblinding and crossover to receive open-label KEYTRUDA.

Gilead to buy Kite Pharma in $11.9 billion deal

Reuters — Gilead Sciences agreed to buy Kite Pharma in a $11.9 billion deal on Monday, as it looks to fuel growth with an emerging class of cancer immunotherapies that are expected to generate billions.

Gilead will pay $180 per share in an all-cash deal, representing a 29.4 percent premium over Kite’s Friday close. Kite’s shares were trading up at $178.15 before the bell.

Santa Monica, California-based Kite is developing a CAR-T, or chimeric antigen receptor T-cell therapy, which harnesses the body’s own immune cells to recognize and attack malignant cells.

Gilead’s growth has been fueled by its pricey but revolutionary hepatitis C drugs but with fewer eligible patients and rising competition, sales have begun to fall.

Second-quarter sales of its hepatitis C drugs — Sovaldi, Harvoni and Epclusa — totaled $2.9 billion, down from $4 billion a year earlier.

Wall Street and Gilead shareholders have long been expecting Gilead to use its cash pile for a big-ticket acquisition.

The deal for Kite, which has been approved by the boards of both companies, is expected to close in the fourth quarter.

Kite is one of the leading players in the emerging field of CAR-T, and is competing with rivals Novartis, Juno Therapeutics, and Bluebird bio in a race to get the first approved therapy.

If approved, these drugs are expected to cost up to $500,000 and generate billions of dollars. Success would also help advance a cancer-fighting technique that scientists have been trying to perfect for decades.

The U.S. Food and Drug Administration (FDA) is expected to decide by Nov. 29 whether to approve Kite’s CAR-T, axi-cel, for treatment of adults with advanced lymphoma.

Gilead has a market value of $96.36 billion, according to Thomson Reuters data. The company was once the world’s largest maker of HIV drugs, and in 2011 agreed to acquire hep C drug developer Pharmasset for $11 billion.

Last year, Gilead generated total sales of $30.39 billion, of which $14.8 billion came from hep C treatments.

Bank of America/Merrill Lynch and Lazard are acting as financial advisers to Gilead, while Centerview Partners is Kite’s exclusive financial adviser.

Skadden, Arps, Slate, Meagher & Flom is the legal counsel to Gilead and Sullivan & Cromwell and Cooley for Kite.

The Wall Street Journal reported first reported the deal.

Gilead’s shares were little changed in premarket trading, while Juno’s shares rose nearly 14 percent and Bluebird’s 2.6 percent.

Data Showing Anti-Addictive/Pain Relief Benefits of Cannabinoids to be Presented at Upcoming Society of Neuroscience Meeting

Data recently obtained from Nemus Bioscience‘s research and development partner, the University of Mississippi (UM) will show the superiority of the NEMUS proprietary analogue of CBD, NB2222, versus plant-derived CBD in ameliorating pain in a validated mouse (murine) model of chemotherapy-induced peripheral neuropathy using an opioid as an active comparator.  The data was accepted for a presentation at the 2017 Annual Meeting of the Society of Neuroscience to held in Washington, D.C. on November 11th-15th.

Additional data will also detail the anti-addictive potential of NB2222 in an animal model of opioid addiction. The in vivo research was led by Professor Kenneth Sufka of the University of Mississippi based on the molecular cannabinoid discovery work performed by the team at ElSohly Laboratories, Inc. (ELI) in collaboration with the University, spearheaded by Dr. Mahmoud ElSohly, who also holds a faculty appointment at the University.

“NEMUS has previously reported that our proprietary analogue of CBD, NB2222, has exhibited a competitive bioavailability profile when compared to plant-derived CBD, penetrating the blood-brain barrier to enter the central nervous system, as well as all major organs, especially the liver, in animal studies conducted to-date,” commented Brian Murphy, MD, MBA, the NEMUS CEO and Chief Medical Officer. “The data being presented by Dr. Sufka is a pivotal start to developing the analogue of CBD to potentially address medical indications in multiple organ systems, especially the eye, brain and diseases of the liver.”

Dr. Kenneth Sufka, professor of Psychology and Pharmacology and Research Professor with the National Center for Natural Products Research at UM stated, “We have been able to demonstrate that NB2222 was able to deliver analgesia comparable to morphine, which has been implicated in the current global opioid abuse epidemic. The research team then moved to the next step using a validated animal model of addiction to opioids to assess the anti-abuse potential of NB2222 as a therapeutic. We look forward to presenting the data this fall.”

“The discovery team is very pleased with the performance of this analogue of CBD in the studies that have been performed so far,” reported Dr. Mahmoud ElSohly, professor at the National Center for Natural Products Research at the University of Mississippi School of Pharmacy and co-inventor of the CBD analogue. “We believe that bio-engineered cannabinoids offer a unique opportunity to potentially enhance the efficacy and safety profile of this class of therapeutics. We look forward to working with NEMUS in advancing other cannabinoid-based therapies across a spectrum of diseases.”

Dr. Murphy noted, “It appears that NB2222 could have a number of uses beyond pain management and ocular conditions, and moving forward, Nemus will consider strategic collaborations for this CBD analogue.”

Forge Therapeutics Raises $15M Series A Financing to Develop First Novel Gram-Negative Antibiotic in Decades

Forge Therapeutics, Inc., a biotechnology company discovering first-in-class antibiotics using a breakthrough drug discovery platform, announced today the completion of a $15M Series A financing. The round is led by MagnaSci Ventures, with participation from Evotec AG, Alexandria Venture Investments, MP Healthcare Venture Management, Red Apple Group, and WS Investments. Forge has used its enabling technology to identify a novel LpxC inhibitor effective against multi-drug resistant bacteria ‘superbugs,’ and the funding will support the program into clinical studies.

“This financing is an important step forward to solving the ‘superbug’ epidemic, an urgent global health issue in desperate need of innovation. We’ve been impressed with the strength of the Forge team, their technologies and their commitment to innovating the antibiotic space,” said Brian T. Dorsey, Founding Partner at MagnaSci Ventures. “With our investment and resources, we look forward to working together on developing the first novel antibiotic against Gram-negative bacteria in decades.” In connection with the Series A financing, Mr. Dorsey will be joining Forge’s Board of Directors.

“We are pleased to have such quality investors join us in our pursuit to eradicate deadly ‘superbug’ infections with novel antibiotics stemming from our robust drug discovery engine,” said Zachary A. Zimmerman, Ph.D., CEO of Forge. “The proceeds from this financing, coupled with the non-dilutive monies received from government agencies CARB-X and NIH/NIAID, will advance our LpxC inhibitor into clinical studies.”

With its proprietary chemistry approach, Forge develops small molecule inhibitors targeting metalloenzymes.  Forge’s lead effort is focused on LpxC, a zinc metalloenzyme found only in Gram-negative bacteria and which is essential for bacteria to grow. Forge has discovered novel small molecule inhibitors of LpxC that are potent in vitro, efficacious in vivo, and effective against drug resistant Gram-negative bacteria ‘superbugs.’

Sangamo Therapeutics and Pfizer Announce Collaboration for Hemophilia A Gene Therapy

Sangamo Therapeutics, Inc. and Pfizer Inc. announced this week, an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter.

“Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A,” said Mikael Dolsten, MD, PhD, President of Worldwide Research and Development at Pfizer. “Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing. We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.”

“With a long-standing heritage in rare disease, including hemophilia, Pfizer is an ideal partner for our Hemophilia A program,” said Dr. Sandy Macrae, Sangamo’s Chief Executive Officer. “We believe Pfizer’s end-to-end gene therapy capabilities will enable comprehensive development and commercialization of SB-525, which could potentially benefit Hemophilia A patients around the world. This collaboration also marks an important milestone for Sangamo as we continue to make progress in the translation of our ground-breaking research into new genomic therapies to treat serious, genetically tractable diseases.”

Under the terms of the collaboration agreement, Sangamo will receive a $70 million upfront payment from Pfizer. Sangamo will be responsible for conducting the SB-525 Phase 1/2 clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization activities for SB-525 and additional products, if any. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional Hemophilia A gene therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations utilizing viral delivery vectors.

Gene therapy is a potentially transformational technology for patients, focused on highly specialized, one-time, treatments that address the root cause of diseases caused by genetic mutation. The technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient’s cells to compensate for a defective one. The genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector such as rAAV. There have been no gene therapy products approved in the U.S. to date.

Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A. SB-525 is comprised of a rAAV vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has cleared initiation of human clinical trials for SB-525, which also has been granted orphan drug designation. Sangamo is on track this quarter to start a Phase 1/2 clinical trial to evaluate safety and to measure blood levels of Factor VIII protein and other efficacy endpoints.


Licensing Agreement for the Development of Drug to Treat Rare Pediatric Disorder Announced

A new treatment in development looks to treat rare condition that robs approximately 20,000 US children per year of their ability to speak. Q BioMed Inc.  and ASDERA LLC announced a licensing agreement that provides Q Biomed with the worldwide exclusive rights to ASDERA’s ASD-002, which is being developed to treat a rare pediatric nonverbal disorder.

Among the more than 60,000 US children who develop autism spectrum disorders (ASD) every year, 20,000 become nonverbal or lose the ability to speak. The numbers are similar in Europe and this nonverbal group will have to rely on assisted living for the rest of their life.

Denis Corin, CEO of Q Biomed said, “Given the severity of this disorder, and the immense emotional toll on these children and their families, our goal is to move the product forward quickly by using all the regulatory tools available to us to expedite the advancement of this drug candidate.”

The cost for treatment and assisted living in the US alone can equal or exceed ten million dollars per patient over a lifetime. The estimated cost to the US healthcare system and lost productivity is estimated at 200 billion dollars each. Currently there is no treatment for this disorder.  EEG, behavioral, and  genetic testing can identify a very targeted population of children in their second year of life that we believe would respond to this treatment.

Research published in 2014 by Wittkowski et al in the Nature journal Translational Psychiatry and independently confirmed in 2015 by Gugliemi et al indicated that certain ion channels were not active enough in this targeted population. ASD-002 is designed to ameliorate this age-specific condition by activating these ion channels. If the second year of life treatment window is missed, many of these children may lose the ability to speak while others may never start to speak at all.

“We are very excited about the potential of ASD-002 and hopefully this will allow thousands of children each year to develop speech and live independent and productive lives,” added Corin. Under the terms of the agreement, Q Biomed receives global rights to develop and commercialize the drug in the rare pediatric disease market.

“ASDERA’s mission is to focus on developing targeted therapies for diseases where there is a high unmet need,” said Dr. Knut Wittkowski. “We are grateful to be working with Q Biomed, which immediately saw the potential of ASD-002 and are committed to ensuring that ASD-002 reaches its intended target patient population.”

Early physiological and behavioral signs of becoming nonverbal appear around nine months of age and include: excessive crying, abnormal eye tracking, and epileptiform EEG. These children may never speak or might begin speaking a few words and then “regress” and lose their ability to speak, while developing on the autism spectrum. Pathological symptoms manifest at the age of 24 months, at which age post-mortem scans  reveal patchy regions of disorganization in cortical brain regions.

Research Papers:

  • Wittkowski KM, Sonakya V, Bigio B, Tonn MK, Shic F, Ascano M, Nasca C, Gold-Von Simson G. A novel computational biostatistics approach implies impaired dephosphorylation of growth factor receptors as associated with severity of autism. Transl Psychiatry. 2014;4:e354. PMCID: 3905234. Available from:
  • Guglielmi L, Servettini I, Caramia M, Catacuzzeno L, Franciolini F, D’Adamo MC, Pessia M. Update on the implication of potassium channels in autism: K(+) channelautism spectrum disorder. Front Cell Neurosci. 2015;9:34. PMCID: 4345917. Available from:

ASDERA is a privately held biotech company, whose mission is to provide solutions for diseases with high unmet needs. The results leading to ASD-002 were derived using a proprietary discovery platform that uniquely analyzes genetic data to identify collections of functionally related genes and, thus, possible drugs to modulate these functions. This platform has also been successfully applied to analyze clinical trials to identify targeted patient populations more likely to respond to therapy.

Q BioMed Inc. is a biomedical acceleration and development company focused on licensing and acquiring biomedical assets across the healthcare spectrum. It is dedicated to providing these target assets the strategic resources, developmental support, and expansion capital needed to ensure they meet their potential, enabling them to provide products to patients in need‏.


Q BioMed Gears up for Production of Cancer Pain Palliation Drug with Commercial Launch Expected in Q2 2017

Q BioMed Inc, a NYC-based biomedical acceleration and development company has begun process validation for the manufacturing of a non-narcotic analgesic treatment for pain associated with metastatic bone cancer. The drug, a generic Strontium Chloride 89 injection, provides long-lasting relief for patients suffering from debilitating bone pain due to metastatic cancer, typically caused by advanced-stage breast, prostate or lung cancer. It has been proven to provide a long-term effect — resulting in cancer pain relief and enhanced quality of life.

There are approximately 350,000 cases of patients living with bone metastases in the U.S. alone. In addition, 380,000 new diagnoses of patients with breast, prostate and lung cancer occur every year and approximately 1 in 3 of those will develop bone metastases.

The delivery of an affordable, non-narcotic pain therapy is a much needed and underutilized option for this patient population and coincides well with the recently passed 21st Century Cures Act combating opiate overuse and abuse. The Act, provides $1 billion in funding over the next two years for opioid addiction prevention and treatment programs to develop, promote and use non-narcotic alternative therapies.

The commercialization of the drug allows Q BioMed to deliver an effective and much needed alternative to hundreds of thousands of suffering patients. The company said in a statement that they are very pleased to offer this U.S. Food and Drug Administration approved therapy to patients in the very near term and are currently negotiating with US-based contract manufacturing organization (CMO) agreements and expect to have those completed along with the commercial launch in Q2 2017.

The company recently closed on its 2nd tranche of the $4,000,000 funding announced on November 30th 2016. The company received $1,000,000 on the 2nd closing bringing the total received to date to $2.5MM and expect to receive the remaining $1.5MM upon effectiveness of their recently filed registration statement on Form S-1.

Q BioMed is focused on licensing and acquiring biomedical assets across the healthcare spectrum. The company provides these target assets the strategic resources, developmental support, and expansion capital they need to ensure they meet their developmental potential, enabling them to provide products to patients in need.

Athersys Gets Nod from PMDA in Japan to Start Trial for the Treatment of Ischemic Stroke with MultiStem®

Athersys, Inc. got a major nod from Japan’s Pharmaceutical and Medical Devices Agency (PMDA) review of the Clinical Trial Notification (CTN), allowing the commencement by HEALIOS K.K. (Healios) of a confirmatory clinical trial evaluating the safety and efficacy of administration of MultiStem®, Athersys’ novel cell therapy product, for the treatment of ischemic stroke in Japan (also designated by Healios as HLCM051 in Japan).

In accordance with the regulatory system in Japan, a CTN is equivalent to an Investigational New Drug application, or IND, under the regulatory system used in the United States.  This clinical trial to be conducted in Japan is part of a partnership and license agreement between Healios and Athersys, focused on the development and commercialization in Japan of novel cellular therapies, including MultiStem, for the treatment of ischemic stroke and potentially other indications.  The study design was accepted as proposed to PMDA in the CTN.

“This announcement demonstrates exciting and important progress, achieved within less than eight months of launching the collaboration, and reflects the tremendous effort on the part of the teams at both organizations that are working together to advance this program in a highly focused and efficient manner,” said Gil Van Bokkelen, Ph.D., Chairman and Chief Executive Officer of Athersys.  “The development approach being taken by Healios and Athersys in Japan is consistent with the approach we plan to implement in a separate international study. Both studies are designed to provide the confirmatory evidence that we believe will put us in a strong position to obtain approval in one of the greatest areas of unmet clinical need in medicine today.”

The planned study will be a randomized, double-blind, placebo-controlled clinical trial conducted at hospitals in Japan that have extensive experience at providing care for stroke victims.  Based on the experience from the B01-02 study, subjects enrolled in the trial will receive either a single dose of MultiStem or placebo, administered within 18–36 hours of the occurrence of the stroke, in addition to standard of care.  As previously disclosed, the study will evaluate patient recovery through approximately 90 days following initial treatment based on Excellent Outcome and other neurological, functional and clinical endpoints.  Additional patient follow up will occur through one year, and other design elements of the Japan trial are consistent with a planned international Phase 3 study, and what has been described previously at the Stroke 2016 conference held in Sapporo earlier this year.

The trial in Japan follows a Phase 2 study completed by Athersys, referred to as the B01-02 trial, which was conducted at 33 clinical sites in the United States and United Kingdom.  The study evaluated the safety and effectiveness of the intravenous administration of MultiStem cells within 24–48 hours after the occurrence of a moderate to severe stroke.  The evaluable patient population comprised 126 subjects who were treated with MultiStem therapy or placebo.

As disclosed previously, intravenous administration of MultiStem following an ischemic stroke was well tolerated, consistent with observations from other clinical trials evaluating the safety of MultiStem treatment.  Additionally, among all evaluable subjects in the B01-02 study, a greater percentage of subjects in the MultiStem group (15.4%) had an Excellent Outcome (mRS ≤1, NIHSS ≤1, and Barthel Index ≥95) at day 90 compared to the placebo group (6.6%) (p=0.10).  At one year, a greater percentage of subjects in the MultiStem group had an Excellent Outcome compared to the placebo group (23.1% vs. 8.2%, p=0.02). For the MultiStem subjects treated within 36 hours of the stroke (i.e. the target population in the planned Japan study), the difference in Excellent Outcome was even greater (e.g., at one year, 29.0% vs. 8.2%, p<0.01).  Likewise, other measures of functional recovery, including the mRS distribution or “shift” analysis, biomarker data and clinical outcomes, including shorter hospitalization, less time in the Intensive Care Unit, and lower rates of mortality and life threatening adverse events, suggest that MultiStem treated patients experienced an improved recovery from post-stroke neurological complications.

“We are excited about reaching this point and about the degree of enthusiasm expressed for the study at the clinical investigators meeting recently held in Tokyo.  With the CTN now in effect, the next steps in the process prior to the initiation of enrollment include obtaining Institutional Review Board approval and finalizing agreements with each of the participating clinical sites.  In the near term, Healios will be focusing on these activities, while Athersys provides support in other areas in preparation for the commencement of enrollment,” concluded Dr. Van Bokkelen.

Stroke represents an area where the clinical need is particularly significant, since it is a leading cause of death and serious disability worldwide, with a substantially impaired quality of life for many stroke victims. Currently, there are nearly 17 million people that suffer a stroke globally and more than two million stroke victims each year in the United States, Europe and Japan, combined. Ischemic strokes, which represent the most common form of stroke, are caused by a blockage of blood flow in the brain that cuts off the supply of oxygen and nutrients and can result in long-term or permanent disability due to neurological damage. Unfortunately, current therapeutic options for ischemic stroke victims are limited, since the only available therapies, administration of the clot dissolving agent tPA, or “thrombolytic,” or surgical intervention using mechanical reperfusion to remove the clot, must be conducted within several hours of the occurrence of the stroke. As a consequence of this limited time window, only a small percentage of stroke victims are treated with the currently available therapy—most simply receive supportive or “palliative” care. The long-term costs of stroke are substantial, with many patients requiring extended hospitalization, extended physical therapy or rehabilitation (for those patients that are capable of entering such programs), and many require long-term institutional or family care.

Q BioMed Closes on Licensing Agreement for FDA Approved Drug – vows no price hike

Q BioMed Inc., a New York-based biotechnology acceleration company recently acquired a FDA approved generic drug, Strontium Chloride (“SR89”). This licensed radiopharmaceutical agent is indicated for the treatment of pain associated with metastatic bone cancer. SR89 provides long lasting relief for patients suffering from bone pain due to metastatic cancer, typically caused by advanced-stage breast, prostate or lung cancer.

The drug is preferentially absorbed in bone metastases, it has been proven to provide a long-term effect resulting in non-narcotic cancer pain relief and enhanced quality of life.

The Company’s immediate efforts and resources will focus on the material procurement and manufacturing process as well as preparing the marketing plan and distribution strategy. The drug is expected to be revenue ready within a short time frame and Q BioMed’s aim is to generate sales within the first year.

According to the Company in a recent press release, “We will make every effort to make this drug as widely available as possible and ensure that the drug will be priced competitively at a cost to patients that is lower than what they are currently paying. In the current environment of skyrocketing drug and medical costs, we believe this is a welcome deviation from the recent headlines. Further, we believe there is an opportunity to invest additional resources into the program to grow the revenue potential significantly. We look forward to making additional details available as soon as practical.”

There are approximately 300,000 new cases of bone metastases in patients with breast and lung cancer per year in the U.S. alone. Approximately 80% of patients using SR89 have reported experiencing a substantial decrease in pain, an increase in physical activity and a reduction in the need for opiate analgesics, such as morphine.

Denis Corin, CEO of Q BioMed Inc. said, “We are very excited about the potential for this drug and look forward to bringing it to market as quickly as possible, delivering significant value to all stakeholders, including current and future patients. The revenue we hope to realize will be a significant milestone for us and substantially derisks our business. However, over and above the near term revenue, we are particularly enthusiastic about the opportunities for growth to increase the potential revenue exponentially. “

Novartis Dissolves Its Cell Therapy Unit; 120 Positions Eliminated

Novartis AG said it will fold its specialized cell and gene therapies unit into other parts of the company, leading to about 120 job cuts months before seeking approval for a new type of cancer treatment.

Cell and gene therapy development will no longer be housed in a separate division, the Basel, Switzerland-based company said Wednesday. The change won’t affect a plan to apply for U.S. approval early next year for a type of cell therapy, called a CAR-T, for children with an aggressive form of blood cancer, Novartis said. The therapy, known as CTL019, will be submitted for European approval later next year.

“Novartis is committed to the ongoing development of CAR-T therapies and remains well positioned to successfully launch CTL019,” the company wrote in a statement.

The Swiss drugmaker, Europe’s second-largest by sales, in May said it would create separate units for cancer treatments and for other medicines following the acquisition of oncology assets from GlaxoSmithKline Plc. While an isolated cell-therapy unit worked well under the previous structure, work on such medicines will be more efficient with the reorganization, Novartis said. Most of the affected jobs are in the U.S.

“Today’s news on Novartis is really a pattern seen with other bigger pharmaceuticals companies such as Pfizer.” stated Karine Kleinhaus, M.D., M.P.H., is Divisional Vice President, North America at Pluristem Therapeutics., a clinical-stage biotechnology company using placental cells

“Big pharma has found that focusing on licensing deals with smaller, focused biotechnology companies is the better route as these biotechs have focused their businesses, making them nimble and able to deal effectively with every aspect of the discovery and development of new therapies, including the complex manufacturing needed to produce highly sensitive biologics. With big pharma cutting down on R&D teams, smaller mid and late-stage firms like Pluristem, can step in with completed early R&D and commercial-grade manufacturing, as well as late-stage clinical trials that are under way,” add Dr. Kleinhaus.

Pfizer boosts cancer drug pipeline with $14 billion Medivation deal

BLOOMBERG — Pfizer Inc said on Monday it would buy U.S. cancer drug company Medivation Inc., in a deal valued at about $14 billion, adding blockbuster prostate cancer drug Xtandi to its portfolio.

Medivation shares were up 20 percent at $80.56 in premarket trade, just shy of the offer price of $81.50 per share in cash.

The offer is at a substantial premium to Sanofi SA’s (SASY.PA) initial offer to buy Medivation for $52.50 in April that pushed the San Francisco-based company to put itself up for sale.

The deal comes four months after Pfizer and Ireland-based Allergan Plc scrapped their $160 billion merger. Pfizer has since bought Anacor Pharmaceuticals Inc ANAC.O in a $5.2 billion deal to add an eczema gel to its portfolio.

The deals hints at a shift in Pfizer’s M&A strategy from lowering taxes – the rationale behind the failed Allergan deal – to strengthen its drugs portfolio ahead of a decision on selling or spinning off its generic drugs business by late 2016.

Pfizer, whose oncology offerings include breast cancer drug Ibrance and several other promising immuno-oncology products, will now get access to Xtandi as well as Talazoparib, another breast cancer treatment under development.

Xtandi, which generated U.S. net sales of $330.3 million in the second quarter, has posted double-digit growth, putting Medivation on track to achieve its target of more than 50 percent in revenue growth for the year.

Japanese drugmaker Astellas Pharma Inc (4503.T) owns the rights to sell Xtandi outside the United States.

“We believe the combination of Xtandi and talazoparib has the potential to be a uniquely value-added combination in the treatment of prostate cancer and should be closely watched,” Leerink analysts wrote in a client note.

Reuters had reported that Pfizer, Merck & Co Inc (MRK.N), Celgene Corp (CELG.O) and Gilead Sciences Inc (GILD.O) had submitted expressions of interest to buy Medivation.

“Given the already very high price being discussed, the difficult public relationship between Medivation and Sanofi … we see a higher bid as very unlikely, but not impossible,” RBC Capital Partners wrote in a client note ahead of the announcement.

Sanofi said while it recognized the potential strategic benefits of a combination with Medivation, it was a “disciplined acquirer and remained committed to acting in the best interests of Sanofi shareholders.”

Pfizer said it expects to complete the acquisition, which was approved by boards of both companies, in the third or the fourth quarter.

Pfizer’s financial advisers were Guggenheim Securities and Centerview Partners, with Ropes & Gray LLP providing legal counsel.

J.P. Morgan Securities and Evercore were Medivation’s financial advisers, while Cooley LLP and Wachtell, Lipton, Rosen & Katz served as its legal advisers.

Pfizer shares were marginally down at $34.80 in premarket.

Up to Friday’s close, Medivation shares had risen about 58 percent since Sanofi’s first offer.

(Reporting by Ankur Banerjee in Bengaluru; Editing by Don Sebastian and Anil D’Silva)

Bristol-Myers Squibb Drug Fails Lung-Cancer Study

A blockbuster cancer treatment failed in a key study as the drug’s maker, Bristol-Myers Squibb, attempts to extend its usage for lung cancer patients.

Shares of the New York company plunged 16 percent Friday, its biggest one-day drop in 14 years. Shares of rival Merck & Co., which makes a rival cancer drug, spiked 10 percent to reach an 18-year high.

Bristol’s drug, Opdivo, and Merck’s drug Keytruda are immunotherapies, which bolster the immune system so that patients can better fight cancer. Both drugs are already approved to treat melanoma and lung cancer, but only after chemotherapy.

In June, Merck reported positive results from a key study focusing on Keytruda as a lone treatment for lung cancer. The negative results from Bristol appear to put Merck in the lead for treating cancer patients without resorting to chemotherapy and its drastic side effects.

The latest late-stage study for Opdivo involved 541 patients who had received no prior treatment for lung cancer.

“We remain committed to improving patient outcomes through our comprehensive development program,” said Bristol-Myers CEO Dr. Giovanni Caforio.

-Associated Press

Pfizer acquires Bamboo Therapeutics to beef up gene therapy arsenal

(Reuters) – Pfizer Inc said it had acquired privately held gene therapy developer Bamboo Therapeutics Inc in a deal worth up to $645 million to boost its presence in the treatment of rare diseases.

Research into gene therapy, which aims to insert corrective genes into malfunctioning cells, goes back a quarter of a century but the field has experienced multiple setbacks and been plagued by safety concerns.

However, the discovery of better ways to carry replacement genes into cells is building optimism.

The U.S. Food and Drug Administration has yet to approve any gene therapies but Europe has approved two – a treatment from GlaxoSmithKline for a rare immune disorder in babies and one from uniQure NV for a serious blood condition.

Genetic material can be delivered to the cells by a variety of means, most frequently using a viral vector.

Bamboo was formed in 2014 to advance the work of Dr. Richard Jude Samulski, who is considered a pioneer in the field after he became the first to realize the potential of using adeno-associated virus’s (AAV) as a vehicle to replace a defective gene with a healthy gene.

Through the acquisition, Pfizer will gain access to Bamboo’s experimental gene therapies for rare diseases such as Duchenne Muscular Dystrophy (DMD), giant axonal neuropathy (GAN), Friedreich ataxia (FA) and Canavan disease.

Focused on neurological and neuromuscular diseases, Bamboo’s drugs are still in the preclinical or early stages of development. Pfizer is paying the Chapel Hill, North Carolina-based company $150 million upfront, and Bamboo stands to make $495 million in milestone payments.

Pfizer has been investing in gene therapies – touted as a one-time cure for intractable and expensive-to-treat diseases – in recent years.

In 2014, the drugmaker entered into a collaboration with Philadelphia-based Spark Therapeutics Inc to develop SPK-9001, a gene therapy for hemophilia B.

Earlier this year, Pfizer signed a collaboration and license agreement with Emeryville, California-based 4D Molecular Therapeutics to develop targeted vectors for cardiac disease.

Other big drugmakers have made similar investments. Bristol-Myers Squibb Co has a tie-up with uniQure to develop gene therapies for heart diseases, while Celgene Corp has teamed up with bluebird bio Inc for cancer.


Cancer Trial for Leukemia Halted after Death of Two Patients

The U.S. Food and Drug Administration placed a hold on a Juno Therapeutics clinical trial of a treatment for a form of leukemia following the death of two trial patients last week.

The Company said in a press announcement that both deaths occurred last week after the patients, who had relapsed or refractory B cell acute lymphoblastic leukemia, took the drug fludarabine before receiving the chimeric antigen receptor (CAR) T cells that Juno had taken from their bodies and re-engineered to better attack cancer cells. Another patient had died earlier in the trial, but “confounding factors” spurred Juno to continue with the study at that time

Juno announced that it has received notice from the U.S. Food and Drug Administration (FDA) that a clinical hold has been placed on the Phase II clinical trial of JCAR015 in adult patients with relapsed or refractory B cell acute lymphoblastic leukemia (r/r ALL), known as the “ROCKET” trial.

Juno has proposed to the FDA to continue the ROCKET trial using JCAR015 with cyclophosphamide pre-conditioning alone. In response, the FDA has requested that Juno submit, as a Complete Response to the Clinical Hold several items including a revised patient informed consent form, a revised investigator brochure, a revised trial protocol, and a copy of the presentation made to the agency yesterday. Juno stated they will submit the requested information to the FDA this week.

Juno’s trials and plans for its other CD19-directed CAR-T cell product candidates, including JCAR017, are not affected.

Pfizer to Acquire Anacor Citing Strong fit with Pfizer’s Inflammation and Immunology portfolio

Pfizer Inc. and Anacor Pharmaceuticals, Inc. today announced that they have entered into a definitive merger agreement under which Pfizer will acquire Anacor for $99.25 per Anacor share, in cash, for a total transaction value, net of cash, of approximately $5.2 billion, which assumes the conversion of Anacor’s outstanding convertible notes. The Boards of Directors of both companies have unanimously approved the transaction. Anacor’s flagship asset, crisaborole, a differentiated non-steroidal topical PDE4 inhibitor with anti-inflammatory properties, is currently under review by the U.S. FDA for the treatment of mild-to-moderate atopic dermatitis, commonly referred to as eczema.

“We believe the acquisition of Anacor represents an attractive opportunity to address a significant unmet medical need for a large patient population with mild-to-moderate atopic dermatitis, which currently has few safe topical treatments available”

“We believe the acquisition of Anacor represents an attractive opportunity to address a significant unmet medical need for a large patient population with mild-to-moderate atopic dermatitis, which currently has few safe topical treatments available,” said Albert Bourla, Group President of Pfizer’s Global Innovative Pharma and Global Vaccines, Oncology and Consumer Healthcare Businesses. “Crisaborole is a differentiated asset with compelling clinical data that, if approved, has the potential to be an important first-line treatment option for these patients and the physicians who treat them.”

“Anacor will be a strong fit with Pfizer’s innovative business, further supporting our strategic focus on Inflammation and Immunology, and is expected to enhance near-term revenue growth for the innovative business. Our dedicated Inflammation and Immunology group has strong existing in-market franchises with Enbrel and Xeljanz, as well as a robust mid-stage pipeline, and this acquisition has the potential to add a near-term U.S. product launch. We believe we are well positioned to maximize crisaborole’s commercial potential through our strong relationships with pediatricians and primary care physicians,” continued Bourla.

In both of its Phase 3 pivotal studies, crisaborole achieved statistically significant results on all primary and secondary endpoints and in March 2016, the FDA accepted for review Anacor’s New Drug Application seeking approval of crisaborole for the potential treatment of mild-to-moderate atopic dermatitis in children and adults. The Prescription Drug User Fee Act (PDUFA) goal date for the completion of the FDA’s review is January 7, 2017. If approved, Pfizer believes peak year sales for crisaborole have the potential to reach or exceed $2.0 billion.

“Today marks the beginning of an exciting new chapter for Anacor, which we believe will deliver significant value to our shareholders,” said Paul L. Berns, Anacor’s Chairman and Chief Executive Officer. “We have a deep respect for Pfizer, and it is clear that they share our commitment to addressing the significant unmet medical needs in inflammatory disease. We are proud of the innovative company that our team has built and are confident that Pfizer will help accelerate Anacor’s important mission given the strength of its global platform and resources.”

Atopic dermatitis is a common, relapsing, chronic, inflammatory skin disorder, with patients displaying a chronic rash characterized by inflammation and itching, often occurring in folds of the skin with symptoms lasting up to 14 days or more. Approximately 18 to 25 million people in the United States suffer from this condition, including between 8 and 18% of infants and children. Atopic dermatitis has been considerably underdiagnosed due to the lack of approved effective systemic agents, and limitations of current topical agents. There have been no new molecular entities for atopic dermatitis in the last 15 years.

Anacor also holds the rights to Kerydin, a topical treatment for onychomycosis (toenail fungus) that is distributed and commercialized by Sandoz Inc. in the U.S.

Pfizer anticipates financing the transaction through existing cash. Pfizer does not expect the transaction to impact its current 2016 financial guidance. Pfizer expects the transaction to be slightly dilutive to Adjusted Diluted Earnings Per Share (EPS)(1) in 2017 with accretion to Adjusted Diluted EPS(1) beginning in 2018 and increasing thereafter.

Under the terms of the merger agreement, a subsidiary of Pfizer will commence a cash tender offer to purchase all of the outstanding shares of Anacor common stock for $99.25 per share in cash. The closing of the tender offer is subject to customary closing conditions, including U.S. antitrust clearance and the tender of a majority of the outstanding shares of Anacor common stock. The merger agreement contemplates that Pfizer will acquire any shares of Anacor 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 third-quarter 2016.

Pharma News: Pfizer approaches Medivation about potential takeover

Reuters – Pfizer Inc has approached U.S. cancer drug maker Medivation Inc to express interest in an acquisition, raising the possibility of a bid rivaling a $9.3 billion offer by Sanofi SA, people familiar with the matter said on Tuesday.

Pfizer’s approach comes less than a week after Sanofi went public with its $52.50 per share cash offer, complaining that Medivation refused to engage. Medivation subsequently rejected the offer as too low. Its shares closed on Tuesday at $57.52.

Medivation has not yet decided whether it should engage with Pfizer in negotiations and is in discussions with its financial and legal advisers, the people said. There is no certainty that Pfizer will press ahead with a bid, they added.

Sanofi currently has no plans to raise its offer and is waiting for Medivation to launch an auction to sell itself before it makes any new bid, some of the people said.

The sources asked not to be identified because the matter is not public. Medivation, Sanofi and Pfizer declined to comment.

Based in San Francisco, Medivation is best known for its oncology drug Xtandi, which treats prostate cancer.

For Pfizer, a deal with Medivation would mark another attempt at building scale in patented drugs after it scrapped its $160 billion acquisition of Dublin-based Allergan Plc (AGN.N) last month.

The breakdown came days after the U.S. Treasury issued new rules that weighed on Pfizer’s ability to slash its tax bill by using the deal to redomicile in Ireland.

Earlier on Tuesday, Pfizer Chief Executive Ian Read said in an interview with Reuters that he would consider another merger of any size, as long as the deal makes sense. He did not comment on Medivation.

Sanofi is vying for Medivation in an attempt to expand in the lucrative oncology sector, as it struggles to compensate for declining revenues from a key diabetes drug that recently lost patent protection.

Sanofi’s unsolicited approach for Medivation has echoes of its bid for rare disease drug maker Genzyme in 2011. It took Sanofi nine months to overcome Genzyme’s resistance. It also offered Genzyme shareholders so-called contingent value rights, which offered them additional payments if the acquired company was able to achieve certain performance milestones.

Using contingent value rights in the case of Medivation may be more challenging for Sanofi, given its lackluster track record in cancer drugs. However, Sanofi has no plans to use contingent value rights in any new offer, according to the sources.

Reuters  – Reporting by Lauren Hirsch and Carl O’Donnell in New York; Additional reporting by Ben Hirschler in London and Greg Roumeliotis in New York; Editing by Dan Grebler


Q&A with Denis Corin, Q Bio Med Inc – Getting Past the “Pharma Bro” – Smaller Biotech Continue to Strive for Well-Being of Patients

There has been a vast amount of mixed feelings surrounding the biotech/drug company development of drugs and the final price consumers/patients are paying. Especially when one biotech CEO, Martin Shkreli (aka ‘Pharma Bro”) decided to massively increase of a very cheap drug vital to patients with HIV from $13.50 to $750. The actions of one CEO has cast an unjust dark shadow on peers in the industry.

According to Denis Corin, CEO of Q BioMed Inc, a biomedical acceleration and development company, Shkreli’s actions drowned the positive work of hundreds of thousands good corporate citizens in the industry – all judgment cast by one greedy arrogant pharma exec.

“The reason innovators, scientists, well-meaning drug company executives and entrepreneurs, do what we do, has to be rooted in the well- being of the patients we ultimately want to treat. We want to make their lives better. Period. Whether its adding a few healthy years with less pain, or perhaps it’s a life changing cure that will add months or years of comfortable life for patients and their families,” believes Corin.

Life-saving drugs are expensive to develop and for every one that makes it through an overly regulated FDA, over 500 fail. The money has to come from somewhere. Corin believes that there is a balance and what we have seen in the headlines this past while or so is way off the mark and can attest it’s not the way the biotech sector thinks or behaves.

Corin’s own company, Q BioMed Inc, has been working hard to find innovative products and drugs that can accelerate through the development process with the best human capital and least money possible. He recently spoke with The Bio Connection:

Q: What impact did Martin Shkreli (aka “Pharma Bro”) have to the industry?
Corin: I am not sure what the lasting impact is. For the moment its put a spotlight on the cost of drugs and the profit margins associated with those drugs. I think the increased focus on these issues and the overall cost of healthcare, especially in US and the continuing debate around ‘the affordable healthcare act’ and its cost are all contributing to a healthy dialogue. Ultimately it’s making the pharma and biotech’s be more conscious of the issues and what’s going to need to happen to address them.

Q: Why do existing profits makes the pharma system work efficiently? How does providing incentives to keep innovating, making enough profit to make it work, and encourage reinvestment in new ideas and products help?

Corin: Companies need to be profitable to continue to deliver product. But, more so in the pharma industry, we are working new drugs and treatments that have a low probability of making it from concept to revenue. So significant amounts of money need to be invested in innovation and Research & Development that doesn’t always deliver a return on that investment. So, if pharma companies are not making a significant amount of money they simply can’t invest capital into those areas and we’ll have less new drug discovery and testing. That’s not the idea. We need to be continually reinvesting in pipelines of drugs and new potential therapeutics and making old ones better and more affordable.

Q: How we can strike the right balance between price and innovation?

Corin: I am not sure if there’s a silver bullet or a simple answer to that. I believe that as technology improves and diagnoses are made earlier, and regulatory hurdles are lowered, more competition will help make that balance more meaningful.

Q: What can CEO do to raise good will and trust among investors?

Corin: I think just by working hard at delivering good product at reasonable prices and providing a decent return. It sounds simple and the kind of principle any business should run on.

Q: Why are capital markets key to fueling innovation and keeping prices manageable?

Corin: So much innovation and new product development is coming out of the junior biotech sector and academia. This is where the capital markets are funding this development and the future pipelines of big pharma. Most new drugs these days are not organically developed at the big name pharma companies, they are coming from smaller biotech start-ups and that requires the capital markets to be actively funding them.

Q: What sectors are going to experience new innovations, new medicines and drive patient care costs down.

Corin: I think there are number of companies looking to repurpose older drugs and generic drugs. Due to the fact that these may have lower development costs, they should provide better access and better price points. I think the new frontier in oncology is immuno-oncology, checkpoint inhibitors and the newer CAR-T type approaches. There have been some very exciting developments in those areas that give hope that there are some major breakthroughs on the horizon. While the results are encouraging, the fact that they are new and costly to produce and administer, I don’t see the price coming down in the near term, but expect that the economic climate and burdened healthcare system, will encourage reasonable price policies.

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Called Off: Pharmaceutical Giants Pfizer and Allergan to abandon $212 billion merger

US pharmaceutical giant Pfizer has announced it is abandoning plans for a US $160 billion ($212 billion) merger with Botox maker Allergan, citing new US rules cracking down on tie ups aimed at saving on taxes.

The deal with the Irish-based firm would have created the world’s largest pharmaceutical company.

Pfizer said in a statement that the two companies “terminated by mutual agreement” plans to merge.

“Pfizer approached this transaction from a position of strength and viewed the potential combination as an accelerator of existing strategies,” company chairman and CEO Ian Read said.

Pfizer also agreed to pay Allergan US$150 million to reimburse its expenses linked to the planned merger.

The decision to call off the merger came after the US Treasury Department announced new rules to discourage mergers between US and foreign businesses designed to sharply lower the US company’s tax bill.

The New York-based pharmaceutical giant said it remains on track to report its 2016 first quarter earnings on May 3.

For all of 2015, Pfizer reported earnings of US$7.7 billion, down 15.2 per cent from 2014. Revenues dipped 1.5 per cent to US$48.9 billion.

Allergan CEO Brent Saunders said in a separate statement that while he is “disappointed” that the merger will not proceed, his company is nevertheless “poised to deliver strong, sustainable growth built on a set of powerful attributes”.

Source AFP