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Saturday, October 6, 2018

Pfizer: NICE approval of MYLOTARG for acute myeloid leukaemia


Pfizer comment on the NICE approval of MYLOTARG (gemtuzumab ozogamicin) for treatment of previously untreated, de novo, CD33-positive acute myeloid leukaemia (AML) in combination with chemotherapy.
MYLOTARG will now be available through the National Health Service (NHS) for eligible patients in England and Wales.
AML is a rare and aggressive blood cancer that affects approximately 2,600 people in the UK each year. If left untreated, the average life expectancy is less than 10 months. The goal of AML treatment is to help patients achieve a prolonged complete remission, however, few new treatment options have become available for AML patients in the last few decades, with chemotherapy remaining the standard-of-care since the 1970s.
The Final Appraisal Determination (FAD), NICE approval of MYLOTARG, recommends use in combination with daunorubicin and cytarabine as an option for untreated CD33-positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia, in people 15 years and over, only if they start induction therapy when either the cytogenetic test confirms that the disease has favourable, intermediate or unknown cytogenetics or when their cytogenetic test results are not yet available, and they start consolidation therapy when their cytogenetic test confirms that the disease has favourable, intermediate or unknown cytogenetics (because the test was unsuccessful).
Craig Eagle, Head of Oncology, Pfizer UK said: We welcome the NICE approval of MYLOTARG for eligible patients living with AML. Pfizer has worked tirelessly with the AML community to ensure patients in the UK have access to appropriate treatment options and this important, potentially life-changing medicine offers renewed hope for some people living with AML in England and Wales.
Haematologist Professor Nigel Russell from the Centre for Clinical Haematology, Nottingham University Hospital Trust said Over the past few decades, there has been little innovation in how we treat AML. Despite slow improvements in outcomes, the prognosis for patients with this type of blood cancer remains poor. Gemtuzumab ozogamicin is a targeted therapy that is given with standard chemotherapy and prolongs the time spent in remission compared to standard treatments. It provides a welcome addition to the treatment options for eligible patients with AML and is an important step towards ensuring that some very sick blood cancer patients can access appropriate treatments to achieve a prolonged, complete remission.
Novartis Kymriah was approved for acute lymphoblastic leukaemia earlier this year while NICE rejected Janssens Imbruvica and NICE have reconsidered their view on Pfizers BESPONSA.
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Carl Zeiss Meditec: FDA Approval for SMILE Astigmatism


ZEISS, a technology enterprise operating in the fields of optics and optoelectronics, issued the following news release:
Today, the ZEISS Medical Technology Segment announced the FDA Premarket Approval (PMA) for ReLEx(R) SMILE(R) PMA expanding myopia treatment to patients with astigmatism. The PMA also provides for a small entry incision to be made, allowing the SMILE(R) procedure to be potentially less disruptive to the corneal surface tissue.
“The expansion of Myopia treatment to patients with Astigmatism will enable current and future SMILE(R) surgeons to expand their patient base, paving the way for a new generation of refractive surgery patients,” said Jim Mazzo, Global President Ophthalmic Devices at Carl Zeiss Meditec.
ZEISS ReLEx SMILE utilizes the high-precision femtosecond laser VisuMax(R) to create a lenticule inside the cornea and access incision in a single treatment step. Its outstanding cutting precision, exceptional speed and gentle treatment make it an ideal platform for advanced corneal surgery such as SMILE(R). Incisions are made through microscopic-photodisruptions of tissue, created by ultrashort pulses.
Many patients are patiently awaiting this approval, reported John F. Doane, M.D., F.A.C.S. of Discover Vision Centers. “We and our patients are excited to say the least. Refractive surgery just keeps getting better with Carl Zeiss Meditec’s FDA approval of SMILE(R) for compound myopic astigmatism,” Doane added.
VisuMax laser is the first femtosecond laser to receive FDA PMA approval for the treatment of a refractive indication in addition to 510k clearances for LASIK flap, keratoplasty, and ICR. With the approval of ReLEx(R) SMILE(R), patients can now benefit from a minimally invasive surgery, performed on one laser versus two.
Refractive surgeons benefit from SMILE(R)’s versatility, offering patients a more comfortable treatment option. With the approved new indication range the surgeon has also the option to benefit from extended technical parameters.
“Thanks to our continued collaboration with partner surgeons worldwide, we are able to pave the way for new developments and technologies in the refractive space,” said Dr. Ludwin Monz, President, and CEO of Carl Zeiss Meditec. “Now with the FDA approval for US SMILE(R) Astigmatism, we can now extend this great treatment option to US Astigmatism patients as well.”
ReLEx(R) SMILE(R) from ZEISS made its U.S. debut in 2016. In the last 10 years, over 1.5 million SMILE(R) treatments have been performed worldwide constituting over 10% of global laser vision correction procedures. To date, there are over 1700 surgeons using SMILE(R) in over 70 countries. The technology behind SMILE was recently featured in the Scientific Background on the Nobel Prize in Physics 2018. Dr. Gerard Mourou and Dr. Donna Strickland were awarded the Nobel Prize for his method to generate high-intensity ultrashort optical pulses. Their invention of so-called chirped pulse amplification is essential to generate the ultrashort laser pulses of the ZEISS VisuMax femtosecond laser system. ReLEx(R) SMILE(R) is a registered trademark of Carl Zeiss Meditec. For more information about ReLEx(R) SMILE(R), visit http://www.zeiss.com.
VisuMax(R) and ReLEx(R) SMILE(R) are registered trademarks of Carl Zeiss Meditec. Not all products, services or offers are approved or offered in every market and approved labeling and instructions may vary from one country to another. For country specific product information, see the appropriate country website. Product specifications are subject to change in design and scope of delivery as a result of ongoing technical development.
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Biogen New SPINRAZA Data Presented at World Muscle Society


  • NURTURE study participants were alive and did not require permanent ventilation, in contrast to natural history of spinal muscular atrophy (SMA)
  • Study participants achieved motor milestones with 100 percent sitting independently and 88 percent able to walk
  • Additional data feature biomarkers as an indicator for clinical development work in SMA
Biogen Inc. (Nasdaq: BIIB) today announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety study of SPINRAZA® (nusinersen) in 25 presymptomatic infants with SMA. The data are being presented today in a late-breaking session at the 23rd Annual Congress of the World Muscle Society (WMS) held in Mendoza, Argentina.
“The NURTURE study results demonstrate that early diagnosis and treatment with SPINRAZA has the potential to dramatically change the course of SMA,” said Wildon Farwell, M.D., senior medical director, clinical development at Biogen. “This is the longest available span of data on infants with SMA who began treatment in a presymptomatic period and indicates that children treated early with SPINRAZA can achieve motor milestones they would likely not attain without treatment.”
The interim analysis evaluated survival and respiratory intervention rates in infants (n=25) who were genetically diagnosed with SMA and began treatment in the presymptomatic stage of the disease. As of May 2018 all patients in the study were alive and none required tracheostomy or permanent ventilation. Additionally, 22 of the 25 participants were able to walk either with assistance or independently according to the motor milestone standard of the World Health Organization and all 25 were able to sit without support.
The motor skills of study participants were also evaluated using the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), an assessment which considers 16 different types of movement to create an overall score between zero and 64. The mean CHOP INTEND scores were 62.6 for study participants with three copies of the SMN2 gene and 61.0 for those with two copies of the gene.
All NURTURE study participants were 14 months or older at the time of the analysis. Participants included infants with two copies of the SMN2 gene (n=15) who are likely to develop a fatal, early-onset form of SMA known as Type 1, and infants with three copies of the SMN2 gene (n=10) who typically develop SMA Type 2 or 3. People living with SMA Types 2 and 3 may never be able to walk or will lose that ability over time. No new safety concerns were identified.
Additional research presented at WMS compared levels of phosphorylated neurofilament heavy chain (pNF-H) in plasma in more than 300 patients from SPINRAZA clinical trials, including those in the NURTURE study, and a control group of people without SMA. The data demonstrated that treatment with SPINRAZA is associated with a rapid decline followed by stabilization of pNF-H in plasma at levels close to those of healthy controls. The results are part of Biogen’s ongoing work to identify and validate biomarkers that could provide insight on the disease progression of SMA.
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Cell therapy, now artisanal and costly, heads for mass production


-Not so long ago, manipulating living cells to serve as therapies was a difficult and mysterious art. Only a few biomedical companies and academic labs could claim proficiency.
But in recent years, there’s been an explosion of knowledge about how cells work, how they can become diseased and how they can be cured. Three groundbreaking cell and gene therapy products have been approved since 2017; two for relapsing blood cancers, and one for a genetic disease that leads to blindness.
This transformation attracted nearly 70 presenting companies to La Jolla for this year’s Cell & Gene Meeting on the Mesa, an annual conference in the field.
These treatments are now costly and performed for a small number of patients. But scaling up this artisanal industry through mass production is expected to reduce costs and bring the benefits to many more people.
To prepare for this wave of cell therapies, the U.S. Food and Drug Administration has been updating its standards for assessing therapies, standards developed for pills and injections of drugs, not of living cells.
“It’s the nature of this whole meeting that the process of characterizing something as squishy and variable as human biology, packaging it and heading toward the clinic, is cutting-edge for the FDA,” said Taylor Crouch, CEO of San Diego’sOrganovo.
Organovo made a name for itself by selling “bioprinted” human liver cells for toxicology screening of potential drugs. The San Diego company is preparing to take its technology to patients with end-stage liver disease, Crouch said.
The company collects cells from donor livers, and prints them with a device that arrays the cells into a three-dimensional form, with various cells that support each other, Crouch said.
Transplanted liver cells could supplement function of a diseased liver until a donor liver can be found or perhaps even help the patient’s own liver recover, Crouch said.
The company plans to file in 2020 to begin clinical testing, Crouch said.
Another presenting company, Athersys, is in late-stage or Phase 3 clinical testing of its cell therapy for strokes caused by blood clots. The product, called MultiStem, consists of cells taken from a donor’s bone marrow, grown in the lab and frozen. When needed, they are thawed and then infused into a patient, said Gil Van Bokkelen, president and CEO of the Cleveland-based company.
MultiStem consists of a class of cells that reproduce prolifically in the lab, so the cells from one donor can yield “millions of clinical doses,” he said. These cells reduce inflammation, promote regeneration, and are tolerated by the patient’s immune system, Van Bokkelen said.
“We can administer them just like Type O blood,” he said.
San Diego’sViaCyte is clinically testing a cell therapy for type 1 diabetes. The San Diegocompany’s product replaces insulin-producing pancreatic “beta” cells, which are destroyed in the disease.
ViaCyte turns embryonic stem cells into precursors of these beta cells. These cells are then encapsulated in a device that is implanted into patients. The company is testing two variants of this approach.
Early human testing of one approach, using cells shielded by a semi-permeable membrane, hit a roadblock when the devices caused growth of fibrous tissue. This blocked diffusion of insulin into the patient. Testing has been suspended while ViaCytereworks the encapsulating material, with help from W.L. Gore & Associates, the makers of Gore-Tex.
That trial is expected to be restarted next year once approval is granted, said Paul Laikind, ViaCyte’s CEO.
Meanwhile, ViaCyte has advanced development of a somewhat different device, which allows direct contact between the cells and patient tissue. This risks an immune reaction, so patients receiving the devices must take immune-suppressing drugs.
So far, side effects have been like those expected from taking immune-suppressing drugs, and appear controllable, Laikind said. But it’s too early to know if the cells can produce therapeutic quantities of insulin.
Patients and others looking for more information on clinical trials from these companies can search for the name of the company on http://www.clinicaltrials.gov.
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VistaGen Gets Fast Track, Other Drugmakers Advance Non-Opioids


Opioids have shown tremendous efficacy in pain management. At the same time, the dangers of the addiction to the medication have become well-known in recent years as an epidemic has swept across the United States.
Last week, a pharmacist in Illinois warned me of the dangers of opioids as he filled a prescription for hydrocodone following a minor surgery. The pharmacist recommended taking acetaminophen for pain instead of the hydrocodone if that was possible – it was.
According to the National Institutes of Health, about 115 Americans die daily from overdosing on opioids, which includes illicit drugs, as well as prescription medications. As more and more patients, prescribers and pharmacists are recognizing the dangers of opioid-based pain medications, multiple companies are developing non-opioid treatments for pain. This week South San Francisco-based VistaGen Therapeutics snagged Fast Track designation from the U.S.Food and Drug Administration (FDA) for its neuropathic-pain candidate AV-101, the second such designation in the past year.
Neuropathic pain affects approximately 33 million people in the United States. It is characterized by a steady burning or sensation that results in abnormal neuronal function.
AV-101 is an investigational, orally bioavailable, small molecule NMDA (N-methyl-D-aspartate) receptor glycine B antagonist without psychological or sedative side effects, according to company data. In December, AV-101 was awarded the Fast Track designation for major depressive disorder. It is currently in Phase II development for that indication.
Shawn Singh, chief executive officer of VistaGen, said AV-101 has the potential to “address the high unmet need for a new non-opioid, non-sedating treatment for neuropathic pain.” He made that assertion based on peer-reviewed data published last year in The Journal of Pain coupled with data from the company’s Phase I clinical trial.
Singh pointed to the Fast Track designation awarded to AV-101 as a sign of the FDA’s commitment to addressing the opioid epidemic sweeping the nation.
“The FDA’s Fast Track designation for development of AV-101 for neuropathic pain, together with the previously granted Fast Track designation for major depressive disorder, will allow our team to work closely with the FDA to bring AV-101 to patients affected by two of our country’s most debilitating and widespread healthcare concerns as soon as possible,” Singh said in a statement.
VistaGen’s Fast Track designation comes about a month after the FDA and Commissioner Scott Gottlieb outlined plans for the regulatory agency to address opioid addiction. Part of that plan includes a new guidance to advance the development of non-addictive treatments for pain, which would include VistaGen’s AV-101, as well as other potential candidates. Earlier this year the FDA launched an “innovation challenge” to spur on the development of medical devices that can combat the crisis and help prevent and treat opioid use disorder – a disorder that is often caused by abuse of opioid-based pain treatments.
Other pharma and biotech companies are also focused on developing treatments for acute pain that do not use opioids, which BioSpace has highlighted in the past. For example, Connecticut-based Biowave’s non-opioid Smarter Pain Blocking Technology has been adopted for use by 32 veteran’s hospitals across the country. Likewise, startup Tremeau Pharmaceuticals is developing TRM-201 (rofecoxib), a highly potent cyclooxygenase-2 (COX-2) selective NSAID (nonsteroidal anti-inflammatory drugs) to treat hemophilic arthropathy (HA), a degenerative joint disease occurring in patients with hemophilia. Rofecoxib is a non-narcotic analgesic, unlike opioids, and has no effect on bleeding time relative to placebo.
Another non-opioid pain treatment in development is an NSAID spray. Pennsylvania-based Virpax Pharmaceuticals licensed MedPharm’s MedSpray ‘Patch-in-a-Can’ Technology as a non-opioid treatment for pain. The Patch-in-a-Can product, called DSF100 (NSAID spray film 1.3%), delivers an NSAID through a metered spray to the skin. The medicine is absorbed to target the pain.
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Friday, October 5, 2018

Spinal implant manufacturer SI-BONE sets terms for $84 million IPO


SI-BONE, which manufactures spinal implants for the alleviation of lower back pain, announced terms for its IPO on Friday.
The Santa Clara, CA-based company plans to raise $84 million by offering 6 million shares at a price range of $13 to $15. Insiders intend to purchase up to $32 million of the IPO (38% of the deal). At the midpoint of the proposed range, SI-BONE would command a fully diluted market value of $335 million.
SI-BONE was founded in 2008 and booked $52 million in revenue for the 12 months ended June 30, 2018. It plans to list on the Nasdaq under the symbol SIBN. Morgan Stanley and BofA Merrill Lynch are the joint bookrunners on the deal. It is expected to price during the week of October 15, 2018.
Relevant Profile: SIBN
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Collins defends Kavanaugh’s healthcare record, renders confirmation likely


The senator’s speech defended the nominee’s track record on the ACA and abortion, rejecting claims that confirming him could endanger legal healthcare-related legal precedent.

With a highly anticipated speech on the Senate floor Friday afternoon, Sen. Susan Collins, R-Maine, announced that she will vote in favor of Judge Brett Kavanaugh’s nomination to the U.S. Supreme Court, virtually guaranteeing that a contentious vetting process will end with Kavanaugh’s confirmation.
Due to a lack of corroborating evidence, the allegations of sexual misconduct brought against Kavanaugh did not overcome the presumption of innocence, Collins said, scolding her fellow senators for allowing the process to devolve into a partisan fight.
Collins defended the nominee’s record on a number of topics, specifically rejecting claims that confirming Kavanaugh could spell trouble for the Obama administration’s signature healthcare law and decades of legal precedent on abortion rights.
“One concern that I frequently heard was that the judge would be likely to eliminate the Affordable Care Act’s vital protections for people with preexisting conditions. I disagree with this contention,” Collins said. “In a dissent in Seven-Sky v. Holder, Judge Kavanaugh rejected a challenge to the ACA on narrow procedural grounds, preserving the law in full. Many experts have said that his dissent informed Justice Roberts’ opinion upholding the ACA at the Supreme Court.”
Roberts’ opinion upheld the ACA as constitutionally authorized by congressional power to tax. In light of the ACA’s individual mandate being zeroed out, a Texas-led coalition of conservative states has claimed that dropping the tax penalty to $0 renders the entire law unconstitutional, so they have asked a federal judge to impose an injunction. The judge, who reportedly seemed sympathetic to the plaintiffs’ argument, could rule on the request any day now, setting off a chain of appeals that could land before the Supreme Court.
Collins argued that Kavanaugh would be disinclined to overturn the ACA in its entirety because his approach to severability is narrow.
“When a part of a statute is challenged on constitutional grounds, he has argued for severing the invalid clause as surgically as possible, while allowing the overall law to remain intact,” Collins said, citing Kavanaugh’s dissent last January in PHH Corp. v. Consumer Financial Protection Bureau.
“Given the current challenges to the ACA, proponents—including myself—of protections for people with preexisting conditions should want a justice who would take just this kind of approach,” she added.

ABORTION RIGHTS

On the campaign trail, then-candidate Donald Trump promised to pick Supreme Court nominees who would overturn Roe v. Wade, the 1973 decision affirming a woman’s constitutional right to an abortion prior to a fetus achieving viability.
Collins said Friday, however, that this pledge has been part of the Republican platform in every presidential campaign since at least 1980 and that Kavanaugh views abiding by legal precedent as constitutionally required, “except in the most extraordinary of circumstances.”
“In short,” Collins said, “his views on honoring precedent would preclude attempts to do by stealth that which one has committed not to do overtly.”
The final vote is expected to take place on Saturday.
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