Foundation Medicine Gains on Approval of Diagnostic Test

Foundation Medicine’s stock rose today following an announcement that its FoundationOne CDx test was approved by the Centers for Medicare and Medicaid Services (CMS) as a new Advanced Diagnostic Laboratory Test (ADLT).

CMS classified FoundationOne CDx as an ADLT under the Protecting Access to Medicare Act (PAMA), a move that Foundation Medicine had said it was expecting.

For a nine-month period starting July 1, CMS will reimburse FoundationOne CDx at a rate of $3,500 per test, in accordance with PAMA and as described by Healthcare Common Procedure Coding System billing code 0037U. During that period, the company will collect and assess private payer rates under PAMA.

Late last year, the US Food and Drug Administration approved FoundationOne CDx for all solid tumors, and as a companion diagnostic for five tumor types. CMS concurrently issued a preliminary national coverage determination for the test, which was later finalized.

https://bit.ly/2KJuGBg

Sesen Bio reports ‘positive’ data from ongoing Phase 3 trial

Sesen Bio announced positive, three-month data from its ongoing Phase 3 VISTA Trial of Vicinium for the treatment of patients with high-grade non-muscle invasive bladder cancer who have been previously treated with bacillus Calmette-Guerin. The efficacy data being reported are based on three-month follow-ups from 111 patients with high-grade NMIBC that is either carcinoma in situ, which is cancer found on the inner lining of the bladder that has not spread into muscle or other tissue, with or without papillary disease, or from patients with papillary disease without CIS, which is cancer that has grown from the bladder lining out into the bladder, but has not spread into muscle or other tissue. “High-grade NMIBC is a devastating cancer that typically occurs later in life and for which treatment options are limited. Over the last 30 years, our industry has seen little innovation for the treatment of this prevalent cancer. The current standard-of-care, BCG, works in many patients, but many will also eventually relapse. For those patients who relapse or who don’t respond at all, the standard alternative is radical cystectomy. In a cystectomy, the bladder is removed along with surrounding lymph nodes and other organs that contain cancer. I am very encouraged by both the safety and these three-month efficacy data with Vicinium, and I look forward to continuing to work with the Sesen Bio team to help bring forward this potential treatment as a safe and effective option for my patients,” said Rian Dickstein, M.D. F.A.C.S., chief of urology, University of Maryland Baltimore Washington Medical Center; medical director of GU oncology, Tate Cancer Center at The University of Maryland Baltimore Washington Medical Center; clinical assistant professor, Department of Surgery, University of Maryland School of Medicine; director, bladder cancer program, Chesapeake Urology; and an investigator in the VISTA Trial.

https://bit.ly/2s1wP3I

Evolus gets CRL tied to its Botox biosimilar manufacturing design

In January, biotech Evolus warned investors that approval of its biosimilar candidate of Allergan’s signature drug Biotox might be delayed. It said FDA had raised questions about the contract manufacturer that will produce the drug. It turns out Evolus was only half right.

The Irvine, California-based biotech last week said the FDA had “fully validated” the Daewoong Pharmaceutical manufacturing facility in South Korea which was built for production of DWP-450. While the plant got an OK, Evolus’ manufacturing processes did not.

The agency issued a complete response letter to the company for its  botulinum neurotoxin candidate, citing concerns about the so-called Chemistry, Manufacturing, and Controls (“CMC”) processes Evolus had established for manufacturing it.

“Overall, we view these updates as positive, which together give us the line of sight necessary to build our commercial infrastructure,” Evolus CEO David Moatazedi said in statement.

He said the company will respond to the FDA’s questions within 90 days.

While it is bad news for Evolus, it is a positive development for Allergan which is facing a slew of biosimilars anxious to cut into Botox’s revenues, which were $2.3 billion in the U.S. last year and $3.2 billion worldwide.

Analysts have said that market share for Allergan’s top-selling Botox should be safe even after new competitors show up but a recent poll of 100 high-volume U.S. aesthetic physicians, suggested otherwise. The poll by analysts at Bernstein found that new entrants from Evolus, Revance  and Hugel could grab up to 34% of the market. Bernstein analyst Ronny Gal told clients a few months ago that it predicted Revance would capture the lion’s share at 18%, with the other two companies splitting a 13% share.

Since then, Mylan announced it had teamed up with Revance on a cheaper copy of Allergan’s top-selling drug.

Last week’s announcement also was good news for South Korea-based Daewoong, which built the new plant specifically to make the Allergan product. That effort seemed to be challenged in January when the FDA issued a Form 483 with 10 observations following the initial preinspection of the plant. But Evolus reported last week the the FDA has now issued a Establishment Inspection Report, or EIR, for Daewoong’s manufacturing facility that confirmed it had passed its most recent preapproval inspection.

https://bit.ly/2khkgxv

Biz development chief Golumbeski is the latest top exec to exit Celgene

Over the past decade, George Golumbeski rose to become an executive vice president at Celgene, frequently lauded for his high-profile role in leading one of the most aggressive BD teams in biotech.

But Golumbeski has quietly slipped out the back door at Celgene at a time CEO Mark Alles has been executing a series of management changes after a string of pratfalls has been raising questions about the company’s competence on executing on a longterm strategy.

According to a note on Golumbeski’s resume posted at MorphoSys AG, which just appointed him as a new supervisory board member, the BD exec officially left Celgene April 16, transitioning to a new role as an independent consultant after gathering a slate of new roles for his post-Celgene life. He’s also a scientific adviser at newly launched KSQ, the startup run by ex-Genzyme chief David Meeker, and is listed as a venture partner at ARCH.

The transition last month has been in the works for close to two and a half years, Golumbeski tells me today. That’s when he sat down with Bob Hugin and Alles and some others to say that after some 20 years on the deal side of the business, with more than 9 years at Celgene, he was ready to do something new and different.

“I communicated I was very happy to stay on,” he adds, “but intended to leave in Q1 or Q2 2017.” For a variety of reasons, his stay lasted until April. And he emphasizes his departure was completely amicable all around.

Now Golumbeski has a variety of new gigs, mostly centered on up and coming biotech companies, where he plans to play a role in recruiting top execs, raising money and focusing on some game-changing medicines. And this way he also gets to spend more time with the family.

“I’m in a good place,” he adds. “I want to help to the extent I can these young companies.”

Working under ex-Celgene CEO Bob Hugin and in close partnership with ex-R&D chief Tom Daniel, Golumbeski was the chief architect of a strategy to snag a multitude of drug rights in an effort to expand beyond their blockbuster Revlimid. Celgene bought into Juno several years ago, then followed up with a $9 billion buyout. Overall, the company liked doing deals that left the partners in charge of the research, as they did successfully with Agios and others.

“We do a better job listening to what a partner has to say,” Golumbeski told me several years ago, when he was in the midst of a deal frenzy worth billions in upfronts and milestones.

Several of his high-profile pacts, though, have been unraveling or hit with severe setbacks. Right on the heels of the implosion of its $710 million cash roll of the dice on the inflammatory bowel disease drug mongersen (GED-301), the FDA hit the company with a refuse-to-file for its would-be MS blockbuster ozanimod.

In response, Alles has engineered some big changes at Celgene. Just days before Golumbeski’s official departure, COO Scott Smith headed out the exits, leaving Alles in charge of everything from clinical development to regulatory, the all-important hematology and oncology franchise as well as inflammation and immunology.

https://bit.ly/2wZxOaj

UroGen Pharma announces ‘positive’ interim results for Phase 3 trial

UroGen Pharma announced new findings from an interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101, an investigational mitomycin formulation for the non-surgical treatment of low-grade upper tract urothelial cancer. Results were presented by Seth Paul Lerner, M.D., FACS, Principal Investigator of the OLYMPUS trial and Professor of Urology at Baylor College of Medicine in Houston in an oral presentation during the plenary session at the 113th American Urological Association’s Annual Meeting in San Francisco. The interim analysis from this international, multi-center trial showed a complete response rate of 59% in 34 patients who were evaluated for primary disease evaluation. PDE is conducted four to six weeks after completion of UGN-101 treatment, which was administered once weekly for six weeks. Results showed that 20 of the interim analysis intent to treat population of 34 patients achieved a CR, defined as a negative ureteroscopic evaluation and a negative wash cytology. In addition, five of 34 patients achieved a partial response. Approximately 39 percent of tumors treated were categorized as unresectable by surgery at baseline. The CRs to date have been durable. Of the 20 patients who achieved a CR, 13 patients have reached three-month follow-up and all remain in CR. Four of these 13 patients have reached six-month follow-up and one of the 13 patients has reached nine-month follow-up. All remain in CR. UGN-101 appeared to be well-tolerated with most treatment-emergent adverse events characterized as mild or moderate and transient. These included urinary tract infection, flank pain, ureteral narrowing and hydronephrosis and time-limited creatinine elevation. The OLYMPUS trial continues to enroll patients, and top-line results are expected in 2H18.

https://bit.ly/2rZwRsX

Takeda to sell majority stake in China JV for $280M

Takeda Pharmaceutical announced it has entered into an agreement to sell its majority shareholding of 51.34% in Guangdong Techpool Bio-Pharma Co. The shareholding is to be acquired in an all-cash, combined Stock Purchase Agreement by Takeda’s joint venture partner in Techpool: Shanghai Pharmaceutical Holding Co. Ltd. and a fund managed by SFund International Investment Fund Management Limited, a wholly-owned subsidiary of Guangzhou Industrial Investment Fund Management Co.The agreement remains subject to approval from the State Administration for Market Regulation in the People’s Republic of China. The Base Payment for the purchase price of Takeda’s shareholding is $280M. Upon completion of the transaction, the Fund will purchase approximately 49% of the Takeda shareholding, and Shanghai Pharma will acquire approximately 51% of it.

https://bit.ly/2IYrQeu

AstraZeneca potassium drug finally approved, threatening Vifor

The decision by U.S. regulators to finally approve AstraZeneca’s much-delayed excess potassium drug Lokelma gives the group another new medicine launch, boosting its portfolio as it strives to offset declining sales of older products.

The U.S. Food and Drug Administration (FDA) had turned down the drug, formerly known as ZS-9, two times previously, casting doubt over AstraZeneca’s decision to buy its original developer ZS Pharma for $2.7 billion (2 billion pounds) in 2015.

Analysts, reacting to the approval announced late on Friday, said on Monday that the label for Lokelma was modestly better than for Vifor Pharma’s rival therapy Veltassa, which also treats excess potassium levels or hyperkalemia.

In particular, AstraZeneca’s drug has a faster onset of action, a better drug-drug interaction profile and can be stored indefinitely at room temperature, Deutsche analysts said. However, both drugs are still deemed unsuitable for acute life-threatening hyperkalemia episodes.

“Whilst we must concede the label is not a best-case, we nonetheless see it as an improvement on the only incumbent option, Vifor’s Veltassa,” Barclays analysts said in a note. “Our thesis remains that Lokelma will be the dominant player in the eventual $3 billion hyperkalemia market.”

AstraZeneca is banking on a range of new drugs to return the company to sales growth in 2018. Last week it reported first-quarter results that showed the impact of generic competition to older medicines, but promising sales of newer ones.

https://bit.ly/2x2v2kC