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Saturday, August 3, 2019

Vertex chief targets a slate of new and ‘larger’ deals ahead

After making it crystal clear that Jeff Leiden plans to keep his hand directly on the wheel of business development following his move from CEO to the executive chairman’s spot, Leiden himself stepped up on Wednesday to personally map out plans for a series of new deals he expects to orchestrate to beef up the pipeline.
During the Q2 call with analysts, Leiden heralded the near-term approval expected for their triple combination for cystic fibrosis. And he made it abundantly clear that a string of new deals regarding gene editing programs for Duchenne muscular dystrophy — buying Exonics and more — are just a prelude to more M&A pacts in the very near future as Reshma Kewalramani preps a move to the CEO suite.
Right at the intro, Leiden noted:
These agreements provide us with development candidates that have shown promising preclinical results and also enable us to integrate cutting-edge scientific technology and expertise in diseases that are highly aligned with our business strategy. We plan to execute more of these types of fields as we further expand our pipeline of transformative medicines over the coming months and years.
In highlighting his continued presence in the day-to-day operations, Leiden spelled out his role in “business development, helping to get deals done and secure our access to external innovation in products.”
In the Q&A, Leiden was explicit that Vertex’s cash flow position gives them plenty of firepower for doing more, and larger deals ahead, after spending $600 million over the past 12 months. What they won’t do, though, is buy up commercial or late-stage programs closing in on an approval.
Leiden:
As we said before, first of all, we are accumulating significant financial firepower capital in our balance sheet, and so you should expect to see us do more deals and potentially larger deals. But the strategy will remain the same as it’s been for the last four years. And as you know, we focus on three areas. Anything in CF that could be complementary or were additive to what we’re doing now is triple. Obviously, we’re not seeing any of those because the triple has set such a high bar, but we continue to look at everything out there.
The second one is technologies — our technology platforms that would allow us to better treat the kinds of diseases which you’ve heard about today either alone or potentially in combination with small molecules. And you’ve seen us do the CRISPR deal, the Moderna deal, the Arbor deal, the X-Chem deal, all of those fall into that category.
And then the third area is looking for assets mostly preclinical and early clinical assets that will complement our pipeline in the diseases we’re interested in. In a way, Exonics was a part of that because DMD and DM1 are two diseases we’re interested in and we continue to look for those assets.
Vertex’s decision to go after deals comes during one of the busiest M&A seasons we’ve seen in years, with a host of major players opening up their checkbooks to buy new drugs for the pipeline. Most, Like Vertex, are focused clearly on early and mid-stage assets, where they hope to find a few relative bargains in the R&D bin. In just the last few days we’ve seen Daniel O’Day at Gilead as well as Albert Bourla at Pfizer highlight their interest in more deals. So he’ll have plenty of company at the bargaining table. All of this will also continue to feed into the venture cycle that’s been seeing more and bigger funds come along to back startups.

Salk scientists create the world’s first human/monkey chimeras in China

Scientists at the world-famous Salk Institute have been at work creating embryos in China that are part human, part monkey in a first step toward determining the feasibility of growing organs for human transplants in primates.
Spain’s El Pais talked with Salk investigator Juan Carlos Izpisúa Belmonte — an internationally acclaimed scientist named one of Time magazine’s 50 most influential people in healthcare for 2018 for his work on chimeras.
Izpisúa had earlier been engaged in creating human/pig chimeras with the same goal but had little success with their model, which developed with an overwhelming imbalance of pig versus human cells. He had more success in creating chimeras using cells of similar species, specifically a mouse/rat approach.
In this latest case, explains El Pais, investigators from Salk teamed with scientists from Murcia Catholic University in Spain. Together they worked to deactivate genes in monkey embryos needed for organ development and then injected human stem cells, looking for them to do their work in developing human organs in the monkey embryo. The project never went past the embryo stage, though, and was performed in China to circumvent any legal restrictions.
Four years ago, the NIH stopped accepting grant applications seeking funds for projects like these — infusing human stem cells into primate embryos. That’s been under review, but so far there’s been no change in the official stance.
Estrella Núñez, the vice-chancellor at UCAM, told the Spanish newspaper that the initial results were “very promising.” But others are simply alarmed.
“What happens if the stem cells escape and form human neurons in the brain of the animal?” asks Ángel Raya, the director of the Barcelona Regenerative Medicine Center. “Would it have consciousness? And what happens if these stem cells turn into sperm cells?”
Núñez, though, says they have a built-in self-destruct mechanism that would prevent that from happening.
Pablo Ross, a veterinary researcher at the University of California, Davis who worked on the human/pig chimeras with Salk investigators, told MIT Technology Review the work faces big challenges when it comes to transplant organs.
“I always made the case that it doesn’t make sense to use a primate for that. Typically they are very small, and they take too long to develop,” he says.

BioCardia prices $10M pubic offering, uplists to NASDAQ

BioCardia, Inc. (NasdaqCM: BCDA, BCDAW), a leader in the development of comprehensive solutions for cardiovascular regenerative therapies, today announced the pricing of its public offering of 1,666,667 units at a price to the public of $6.00 per unit.  Each unit issued in the offering consists of one share of common stock and one warrant to purchase one share of common stock at an exercise price of $6.30 per share.  The common stock and warrants will be immediately separable from the units and will be issued separately. The common stock and warrants are expected to begin trading on the Nasdaq Capital Market on August 2, 2019, under the symbols “BCDA” and “BCDAW,” respectively. BioCardia expects to receive gross proceeds of $10 million, before deducting underwriting discounts and commissions and other estimated offering expenses.
BioCardia has granted the underwriters a 45-day option to purchase up to 250,000 additional shares of common stock, and/or warrants or any combination thereof, to cover over-allotments, if any. The offering is expected to close on August 6, 2019, subject to customary closing conditions.
Maxim Group LLC is acting as sole book-running manager for the offering.  Brookline Capital Markets and Dawson James Securities, Inc. are acting as co-managers for the offering.

Shorter AbbVie Maviret regimen OKd in Europe for hepatitis C with cirrhosis

The European Commission approves an eight-week regimen for AbbVie’s (NYSE:ABBV) MAVIRET (glecaprevir/pibrentasvir) for previously untreated patients with hepatitis C virus (HCV) genotypes 1,2,4,5 or 6 infection with compensated cirrhosis. It was previously approved as a 12-week regimen in these patients.
MAVIRET was first approved in Europe two years ago as an eight-week regimen for treatment-naive HCV patients without cirrhosis.
Related ticker: Enanta Pharmaceuticals (NASDAQ:ENTA)

Friday, August 2, 2019

Pfizer Sickle Cell Phase 3 Misses Primary, Secondary Goals

Pfizer Inc. (NYSE:PFE) announced today that the Phase 3 Rivipansel (GMI-1070): Evaluating Safety, Efficacy and Time to Discharge (RESET) pivotal study did not meet its primary or key secondary efficacy endpoints. The objective of the trial was to evaluate the efficacy and safety of rivipansel in patients aged six and older with sickle cell disease (SCD) who were hospitalized for a vaso-occlusive crisis (VOC) and required treatment with intravenous (IV) opioids. The primary endpoint was time to readiness-for-discharge and the key secondary efficacy endpoints were time-to-discharge, cumulative IV opioid consumption, and time to discontinuation of IV opioids.
“We are disappointed with the results, as we have been working in close partnership with the SCD community to advance rivipansel as a potential treatment option for acute VOC. We plan to share the study data at an upcoming scientific meeting as we want to ensure the learnings from this trial help inform future sickle cell programs that aim to improve care for SCD patients experiencing a VOC,” said Brenda Cooperstone, M.D., Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “We express our sincere gratitude to everyone who made this study possible, including the study investigators, and in particular, the patients and their families.”

J&J and Colgate Score Win At 1st Cosmetic Talc Trial in Kentucky

Hayes closings
CVN screenshot of J&J attorney Morton Dubin delivering his opening statement. Click here to see video from the trial. 
Louisville, KY – A Kentucky state court jury returned a defense verdict in favor of Johnson & Johnson and Colgate-Palmolive on Friday in the first trial in the state over alleged cancer risks associated with the company’s cosmetic talc products. 
The jury deliberated for less than an hour after hearing closing arguments earlier in the day in a trial that began in early July. 
The full trial was webcast and recorded gavel-to-gavel by Courtroom View Network, and is available with a subscription to CVN’s video library, along with numerous other talc and asbestos trials.

The trial was closely watched, both due to being the first cosmetic talc trial in Kentucky and also a case involving the California-based plaintiff firm Kazan McClain Satterley & Greenwood, which had previously notched three major wins in cosmetic talc trials, including one in J&J’s home state of New Jersey that ended in a $117 million verdict. 
Friday’s verdict also makes just the second time a jury weighed in on a trial including both J&J and Colgate. The only other such trial to date, also involving Kazan McClain Satterley & Greenwood, ended earlier this summer in California with a plaintiff’s verdict.
Both J&J and Colgate argued that talc does not cause mesothelioma, the form of cancer contracted by plaintiff Donna Hayes and frequently associated with asbestos exposure. Hayes claimed she developed the fatal illness by inhaling asbestos allegedly present in products like Johnson’s Baby Powder and Cashmere Bouquet for decades. 
J&J and Colgate successfully argued that Hayes’ cancer was the result of exposure to asbestos from various garages where her husband worked over the course of many years as a mechanic. 
Another J&J talc/mesothelioma trial that began at roughly the same time as the Kentucky case remains underway in New Jersey, and additional trials are slated for later this month in California. 
State courts will likely see an uptick in cosmetic talc trials over coming months, after a Delaware federal judge rejected an effort by J&J to consolidate the thousands of cosmetic talc-related claims sprawled across state courts throughout the country into the federal court where their talc supplier Imerys’ bankruptcy proceedings are playing out. 
J&J was represented by Orrick Herrington & Sutcliffe LLP, and Colgate was represented by Gordon & Rees LLP. 

Philippines weighs re-use of controversial dengue vaccine

The Philippines is considering re-introducing a dengue vaccine whose use it halted because of links to the deaths of several children, as authorities battle to contain a dengue outbreak that has killed more than 450 people this year.
Concerns over dengue immunization for nearly 734,000 children aged nine or older sparked two congressional inquiries, a criminal investigation and a sharp fall in the number of parents seeking routine vaccinations for their children.
If the government decided to revive the use of Dengvaxia, developed by French drugmaker Sanofi, it would be administered with “utmost caution”, presidential spokesman Salvador Panelo said.

“If Dengvaxia is proven effective to those who already had dengue in the past, then its application to these individuals will surely cause the decline of the overall number of cases,” he told reporters.
The Philippines stopped using Dengvaxia in late 2017 and ordered Sanofi to stop selling, distributing and marketing it after Sanofi warned the vaccine could worsen the disease in some cases.
In March, the Department of Justice said it had found probable cause to indict Sanofi officials, and former and current Philippine health officials, over 10 deaths it said were linked to the use of Dengvaxia, which Sanofi has repeatedly said is safe and effective.

Panelo said the government would follow a protocol set by the World Health Organization for all individuals to be screened before receiving the vaccine, to determine if they have ever been exposed to the infection.
Any decision to start administering the vaccine again would not affect cases against individuals involved in the controversy, he added.
This year, the Philippines has reported more than 100,000 cases of dengue, a mosquito-borne tropical disease that kills about 20,000 people annually and infects hundreds of millions.