Sanofi is in talks to buy Flexion Therapeutics, a Boston-area biotech with its lead product now awaiting FDA approval, for more than $1 billion in cash, according to a source close to the deal.
Flexion’s board has voted to accept Sanofi’s non-binding offer, with a price in the “mid-30s” per share, the source said. Flexion stock closed Wednesday at $19.68.
Flexion’s knee injection for osteoarthritis, Zilretta, would fit right in with Sanofi’s biosurgery division, which markets its own osteoarthritis injectable, Synvisc. And some of Flexion’s staffers know that product well; marketing VP Mark Fraga and medical affairs VP Scott Kelley, M.D., jumped to Flexion last year from jobs handling Synvisc at Sanofi.
Accepted for review in December, Zilretta could win FDA approval in October.
Sanofi refused to comment on the potential deal. Flexion spokesman Scott Young said he “can’t comment on market speculation or rumors.”
“We’re pursuing an independent strategy to commercialize our lead product Zilretta,” Young said Thursday.
The talks are ongoing and the transaction may not come to fruition, the source said. But if due diligence goes well and a binding offer is accepted, the cash deal could close quickly, giving Sanofi’s Genzyme unit several months to gear up for the Zilretta launch.
Zilretta is a sustained-release corticosteroid first up for approval for use in the knee. Analysts have suggested it could bring in $500 million to $600 million in peak sales, and perhaps hit blockbuster status if it wins indications for use in other joints.
Synvisc works differently; it’s a hyaluronon-based injection designed to supplement the natural fluid in the knee. It brought in €408 million for Sanofi in 2016, with €313 million of that in the U.S.
Sanofi has been deal-shopping as its diabetes franchise slows, eyeing companies with price tags in the bolt-on range—as Flexion would be—as well as bigger targets. It’s had some trouble bringing those bigger deals across the finish line, however; it lost out to Pfizer on the cancer biotech Medivation last summer and to Johnson & Johnson in its attempt to snap up Swiss-based Actelion late last year.
XOMA Corporation (Nasdaq: XOMA), a pioneer in the discovery and development of therapeutic antibodies, announced today that clinical data for its two hypoglycemia drug candidates, X358 and X129, and a third drug candidate for hypercalcemic endocrine and oncology conditions, will be presented at the Endocrine Society’s 99th Annual Meeting (ENDO 2017), taking place from April 1-4, 2017 in Orlando, Florida.
“We look forward to sharing additional efficacy and safety data on X358 and X129 with the clinical endocrinology community at this year’s ENDO meeting. We are also looking forward to discussing the positive progress and development candidate selection for our anti-PTH1R program targeting serious hypercalcemia associated with hyperparathyroidism and certain malignancies,” said Jim Neal, Chief Executive Officer of XOMA. “This continued positive data positions each of these programs well for partnering activities. Once they are successfully partnered, these programs would expand our portfolio of fully funded programs producing future milestones and royalties that contribute to our goal of delivering positive cash flow and profitability.”
The Company will deliver one oral presentation and three poster presentations, they include:
Oral Presentation Abstract title: Single Administration of XOMA 358, an Insulin Receptor Attenuator, Improves Post-Meal and Nighttime Hypoglycemia Profiles in Post Gastric Bypass Hypoglycemia (PGBH) Patients
- Session: OR14: Glucose Metabolism and Post Bariatric Surgery
- Date: Monday, April 3, 2017, 12:30 PM – 12:45 PM
- Location: OCCC – W224C
Poster Presentations Abstract title: Activity of XOMA 358, an Inhibitor of Insulin Action Following Short-Term Administration to Congenital Hyperinsulinism Patients
- Session: MON 001-056 Pediatric Endocrine Case Reports: Diabetes, Thyroid, and Beyond
- Poster number: MON 056
- Date: Monday, April 3, 2017, 1:00 PM – 3:00 PM
- Location: West Hall B (EXPO Hall)
Abstract title: XOMA 129, a Novel Insulin Receptor Negative Modulator, Is Efficacious in Treating Insulin- Induced Hypoglycemia in Minipigs
- Session: SAT 575-585 Cellular Signaling Pathways and Regulation of Glucose Metabolism
- Poster number: SAT 583
- Date: Saturday, April 1, 2017, 1:00 PM – 3:00 PM
- Location: West Hall B (EXPO Hall)
Abstract title: A Novel Anti-PTH1R Receptor Antagonist Monoclonal Antibody Reverses Hypercalcemia Induced By PTH or PTHrP: A Potential Treatment of Primary Hyperparathyroidism and Humoral Hypercalcemia of Malignancy
- Session: SAT 338-359 Innovations in Bone Biology
- Poster number: SAT 339
- Date: Saturday, April 1, 2017, 1:00 PM – 3:00 PM
- Location: West Hall B (EXPO Hall)
More information about the ENDO program can be found at: http://www.endocrine.org/endo-2017/meeting-program
U.S. Secretary of State Rex Tillerson has directed U.S. diplomatic missions to identify “populations warranting increased scrutiny” and toughen screening for visa applicants in those groups, according to diplomatic cables seen by Reuters.
He has also ordered a “mandatory social media check” for all applicants who have ever been present in territory controlled by the Islamic State, in what two former U.S. officials said would be a broad, labor-intensive expansion of such screening. Social media screening is now done fairly rarely by consular officials, one of the former officials said.
Four cables, or memos, issued by Tillerson over the last two weeks provide insight into how the U.S. government is implementing what President Donald Trump has called “extreme vetting” of foreigners entering the United States, a major campaign promise. The cables also demonstrate the administrative and logistical hurdles the White House faces in executing its vision.
The memos, which have not been previously reported, provided instructions for implementing Trump’s March 6 revised executive order temporarily barring visitors from six Muslim-majority countries and all refugees, as well as a simultaneous memorandum mandating enhanced visa screening.
The flurry of cables to U.S. missions abroad issued strict new guidelines for vetting U.S. visa applicants, and then retracted some of them in response to U.S. court rulings that challenged central tenets of Trump’s executive order.
The final cable seen by Reuters, issued on March 17, leaves in place an instruction to consular chiefs in each diplomatic mission, or post, to convene working groups of law enforcement and intelligence officials to “develop a list of criteria identifying sets of post applicant populations warranting increased scrutiny.”
Applicants falling within one of these identified population groups should be considered for higher-level security screening, according to the March 17 cable.
Those population groups would likely vary from country to country, according to sources familiar with the cables, as the March 17 memo does not explicitly provide for coordination between the embassies.
Trump has said enhanced screening of foreigners is necessary to protect the country against terrorist attacks.
Advocates and immigration lawyers said the guidance could lead to visa applicants being profiled on the basis of nationality or religion rather than because they pose an actual threat to the United States.
“Most posts already have populations that they look at for fraud and security issues,” said Jay Gairson, a Seattle-based immigration attorney who has many clients from countries that would be affected by Trump’s travel ban.
“What this language effectively does is give the consular posts permission to step away from the focused factors they have spent years developing and revising, and instead broaden the search to large groups based on gross factors such as nationality and religion.”
Virginia Elliott, a spokeswoman for the State Department’s Bureau of Consular Affairs, said the department was working to implement Trump’s presidential memorandum “in accordance with its terms, in an orderly fashion, and in compliance with any relevant court orders, so as to increase the safety and security of the American people.”
State Department officials declined to comment on the specifics of the cables, saying they were internal communications.
In cables dated March 10 and March 15, Tillerson issued detailed instructions to consular officials for implementing Trump’s travel order, which was due to take effect on March 16.
Following successful legal challenges to an earlier, more sweeping travel ban signed by Trump in January, the White House issued a narrower version of the ban earlier this month.
On the same day Tillerson sent out his memo about implementing the new executive order on March 15, a federal court in Hawaii enjoined key parts of the order. That forced him to send another cable on March 16, rescinding much of his earlier guidance.
On March 17, Tillerson issued a fourth cable that set out a new list of instructions for consular officials. At the same time, it withdrew more sections of the March 15 guidance, because they had been issued without approval from the White House Office of Management and Budget (OMB), which is responsible for reviewing all agency rules.
A White House spokesman referred questions about the cables to the State Department and OMB.
Reuters could not determine to what extent the cables departed from guidance given to consular officers under previous administrations, since this type of guidance is not made public.
Some of the language in the cables, including the line that “all visa decisions are national security decisions,” is similar to statements made by U.S. officials in the past.
Some consular officials suggested some of the March 17 guidance – aside from identifying particular populations and doing more social media checks – differed little from current practice, since vetting of visa applicants is already rigorous.
PHONE NUMBERS, EMAIL ADDRESSES
Among the instructions rescinded by Tillerson were a set of specific questions for applicants from Iran, Libya, Somalia, Sudan, Syria and Yemen, the countries targeted by Trump’s March 6 executive order, as well as members of populations identified as security risks.
The questions asked where applicants had lived, traveled and worked over the previous 15 years. Applicants would also have been required to provide prior passport numbers and all phone numbers, email addresses and social media handles used in the previous five years.
The March 16 and 17 cables from Tillerson instructed consular officers not to ask those questions, due to court action and pending approval by the OMB.
Both Republicans and Democrats in Congress have called for wider social media screening for those seeking to enter the United States, saying that such checks could help to spot possible links to terrorist activity.
Some former officials and immigration attorneys cautioned that delving deeper into applicants’ social media use could significantly lengthen processing time of visas.
“There’s so much social media out there,” said Anne Richard, a former U.S. assistant secretary of state in the Obama administration. “It’s not something you can do on a timely basis.”
Both the March 15 and March 17 cables seem to anticipate delays as a result of their implementation. They urged embassies to restrict the number of visa interviews handled per day, acknowledging this “may cause interview appointment backlogs to rise.”
Neurotrope CEO Dr. Susanne Wilke and President Dr. Daniel Alkon presented at Oppenheimer’s 27th Annual Healthcare Conference Tuesday.
- Dr. Wilke ran through a lot of material that Alzheimer’s stakeholders, from patients to equities investors, would have heard about the company before.
- Neurotrope’s unique leading drug candidate, Bryostatin, has a mechanism of action focused on regeneration of brain synopses, which it expects to halt or even reverse the progression of AD even in advanced patients. Bryostatin activates an enzyme called protein kinase C (PKC) epsilon, deficits of which preclinical studies and autopsies have implicated as a cause of Alzheimer’s.
- The prevailing “amyloid hypothesis” of AD treatment is flawed, in their view, as its focus on the removal of amyloid plaque and tau tangles in the brain has failed in numerous clinical trials.
- Bryostatin has been shown in some compassionate-use cases in severely demented patients to return motor skills, increase engagement, return language and vocalizations, among other benefits.
Fortunately for followers of the company yearning for fresh info, the presentation concluded with a Q&A session, highlights of which included:
- Dr. Wilke cited encouraging “anecdotal evidence” that patients involved in the Phase 2 trial wanted to continue the trial.
- Dr. Alkon said the company, in addition to plans for a Phase 3 trial, intends to do an open-label extension for all patients in Phase 2 “to get the best drug dose.”
- Neurotrope continues to expect to uplist to the Nasdaq in March, with top-line data from its Phase 2b trial of Bryostatin expected in April of this year.
Coming To The Stage
The company’s ANAVEX 2-73 has shown 57-week safety and tolerability, and Anavex is both currently developing it in a Phase 2a study, and preparing it for a Phase 2/3, placebo-controlled trial in AD.
ANAVEX 2-73, like Neurotrope’s Bryostatin, seeks to halt or reverse the course of AD. It hopes to do so by activating the sigma-1 receptor (a kind of intracellular protein capable of modulating a variety of cellular processes relevant to neurodegeneration) to restore homeostasis by targeting protein misfolding, oxidative stress, and other factors.
Neurotrope Has An Admirer
Benzinga recently had a discussion with Dr. Maria Maccecchini, CEO of QR Pharma, another biopharma company hoping to develop a novel therapy for AD. QR Pharma is currently evaluating its Posiphen, an inhibitor of several disease-causing proteins (not just amyloid and tau), in a Phase 2A proof-of-concept study. Data is expected later this year.
Of Neurotrope, Maccecchini said, “The reason I kind of like Neurotrope is that I actually believe that attacking PCK (protein kinase C) has a chance. But that’s a totally different approach, which means it’s complimentary.”
Dr. Maccecchini expressed doubts about the amyloid hypothesis, saying, “I think it is important to understand that AD is not Abeta and it is not tau and that a broader approach may be the way to go.”
Origent Data Sciences, Inc. (Origent) and Cytokinetics, Inc. (Nasdaq: CYTK) today announced the advancement of their research collaboration to prospectively validate Origent’s computer model to predict the course of ALS (amyotrophic lateral sclerosis) disease progression using data from VITALITY-ALS, Cytokinetics’ ongoing Phase 3 clinical trial of tirasemtiv. Funded by a grant from The ALS Association to Origent, this joint research program is designed to enable the first prospective validation of the predictive model in a clinical trial. Previously, the Origent models predicting both function and survival of ALS patients have been validated using the placebo arms of retrospective clinical trial datasets.
Because ALS disease progression is heterogeneous among patients, predicting an individual patient’s course is difficult. This heterogeneity creates challenges for the design and conduct of clinical trials as inclusion of patients who progress at variable and unpredictable rates requires larger, longer and more expensive trials in order to observe a potential treatment effect of a therapeutic intervention. Origent’s statistical models are designed to identify the patients whose symptoms are likely to progress quickly or slowly, potentially providing a methodology to address the complexity created by disease heterogeneity. Results from the first part of the research collaboration were presented at the 27th International Symposium on ALS/MND and showed that the Gradient Boosting Machine (GBM) algorithm was the optimal model to predict slow vital capacity (SVC) at times subsequent to baseline and that forced vital capacity (FVC) records could be used to predict SVC scores of ALS patients using this machine learning technique.
“Following the successful completion of the retrospective validation of our predictive models using baseline characteristics data from BENEFIT-ALS, the Phase 2 b trial of tirasemtiv, we look forward to taking this research collaboration to the next level and thank Cytokinetics for providing us access to the first real-time dataset for the prospective analysis of patients with ALS, ” said Dave Ennist, Chief Science Officer, Origent Data Sciences.
“The predictive power of the Origent computer model is encouraging, particularly the ability to predict slow vital capacity subsequent to baseline, as demonstrated in the first phase of the research,” said Jinsy Andrews, M.D., Director of Neuromuscular Clinical Trials at Columbia University and Medical Monitor for VITALITY-ALS. “We are pleased to continue this groundbreaking collaboration which we hope may pave the way towards increased efficiencies in the conduct of clinical trials in patients with ALS.”
About the Research Collaboration
Origent will seek to prospectively validate existing predictive models (including the ALSFRS-R, respiratory, gross, fine, and bulbar sub-scores, SVC and survival models) using baseline characteristics data from VITALITY-ALS, the ongoing Phase 3 clinical trial to assess the effects of tirasemtiv versus placebo on SVC and other measures of skeletal muscle strength in patients with ALS. Tirasemtiv is a fast skeletal troponin activator (FSTA) being developed by Cytokinetics for the potential treatment of patients with ALS. Using existing models in Origent’s library, predictions will be made for each patient using only screening and baseline information from the placebo arm of the trial. Screening and baseline data of placebo patients will be provided following database lock and predictions will be made in the absence of access to the subsequent outcomes of the patients from the placebo arm of VITALITY-ALS. After the predictions are complete, clinical outcomes data from patients in the placebo arm of VITALITY-ALS will be made accessible to Origent for comparing actual placebo outcomes data to the previously escrowed predictions.
The co-founder of a Massachusetts pharmacy was acquitted Wednesday of causing the deaths of 25 people but convicted of other racketeering charges in a nationwide meningitis outbreak in 2012.
Barry Cadden was charged with 25 counts of second-degree murder, conspiracy and other charges under the federal racketeering law. Overall, the outbreak killed 64 people and sickened 700 others in 20 states.
After five days of deliberations, the jury found that he wasn’t responsible for the deaths. But they also found him guilty of racketeering, conspiracy and mail fraud charges.
Cadden, 50, will be released on bail while awaiting sentencing, which is scheduled for June 21.
In September 2012, the Centers for Disease Control and Prevention began investigating an outbreak of fungal meningitis and other infections in patients who had received steroid injections made by the New England Compounding Center in Framingham. The outbreak affected people in 20 states. Indiana, Michigan and Tennessee were hardest hit.
During the two-month trial, prosecutors said Cadden ran the company in an “extraordinarily dangerous” way by skirting industry regulations on sterility and cleanliness in an effort to push production and make more money.
“It was preventable, but it happened because this man, Barry Cadden, decided to put profits before patients,” Assistant U.S. Attorney Amanda Strachan told the jury during closing arguments.
Cadden’s lawyers said he was not responsible for the deaths. They said Glenn Chin, a supervisory pharmacist, ran the so-called clean rooms where drugs were made. Chin has pleaded not guilty to similar charges.
“As horrible as each of these stories is, there is nothing that shows that Mr. Cadden did something that the government can link to the death of that person,” attorney Bruce Singal told the jury.
Prosecutors said NECC also used expired ingredients and falsified logs to make it look like the clean rooms had been disinfected when they had not.
After the outbreak, regulators found multiple potential sources of contamination, including standing water, mold and bacteria in the air and on workers’ gloved fingertips. NECC filed for bankruptcy after it was hit with hundreds of lawsuits from victims or their estates. NECC and several related companies reached a $200 million civil settlement with victims and their families.
In 2013, Congress increased federal oversight of compounding pharmacies, which mix personalized medications for patients and supply them directly to hospitals and doctors.
An influential nonprofit is opening its checkbook to help to develop middle school math and science teachers in Philadelphia.
Lou Bellardine, the Philadelphia School District’s Chief Talent Officer, knows how difficult it is to find qualified math and science teachers for fourth through eighth grade. Enter the Philadelphia School Partnership, which is giving $1.2 million to Drexel University to launch a middle school teacher training program.
“It’s one of our hardest-to-fill positions,” said Bellardine.
PSP Executive Director Mark Gleason says district, charter and catholic schools share the need.
“They’re telling us ‘our teacher pipeline is not strong enough, and in particular in the middle grades it’s especially a problem,’” Gleason said.
The grant will go a long way to put together programs focused on teaching future educators how to teach in inner-city schools. Finding qualified teachers to fill nearly 1,000 open teaching positions yearly can be a challenge.
“Pennsylvania recently switched to where you need a specific certification for grades four through eight and not that many teachers have that certification, and so many schools are struggling to find qualified middle school teachers in those three areas — math, science and education,” Gleason said.
The Dragons Teach Middle Years program aims to produce 40 graduates a year by 2022. Twenty-year-old Aja Sor hopes to be among them.
“There’s kids from the neighborhood and one might be more well-off and one might not be able to eat dinner that night. So I feel as though me being from Philly I’ve prepared myself because I’ve been that kid,” Sor said.
Gleason says the Drexel students will spend 30 weeks as student teachers, more than twice the norm.