biohavenBy Mitchell Young

NEW HAVEN: Connecticut’s newest bio-tech star just ran full force into a brick wall.

Biohaven, [NYSE: BHVN] went public in May at $17 raising $168 million in its IPO. Three months later the stock hit $37, and a $1 billion dollar plus valuation in part on expectations of positive results from an Phase 3clinical trial and its strategy of licensing existing drugs in need of further clinical trials and development

Biohaven unique business plan has it licensing rights to drugs from a variety of sources, including Yale, Rutgers, Bristol Myers, AstraZeneca, Mass General Hosptial, creating hopefully a quick route to a diverse pipeline in an industry where a single drug can take a decade or more to develop.

Unfortunately the first study our of the gate, a Phase II/III study of trigriluzole, which was given Fast Track status from the FDA showed that that it didn’t work as well as the placebo in treating spinocerebellar ataxia, a rare genetically generated disease that effects the brains ability to properly control body movements.

The company’s stock initially dropped 28% on the news, but recovered somewhat and still remains at double it’s IPO price.

In September the company announced that it was going to test the drug for Alzheiemers with thes Alzheimer's Disease Cooperative Study [ADCS, an Alzheimer's disease (AD) clinical trials research consortium that receives major support from the U.S. National Institute on Aging (NIA), a part of the US National Institutes of Health.

Biohaven which recently announced in late September it was establishing its headquarters at the former Liberty Bank building on Church Street. Along with the negative results the company said that it has more than $200 million in cash in its coffers, to fund its immediate operations and trials.

Vlad Coric
Biohaven CEO:" Disapointed in the results."

Biohaven CEO Vlad Coric commented on the results saying, “we are obviously disappointed that today’s topline clinical results do not support continued development of trigriluzole as a symptomatic agent for patients with SCA, a devastating neurologic disorder for which novel treatments are urgently needed. This was the largest SCA clinical trial performed to date and important knowledge has been generated — we plan to share our data with the ataxia clinical leaders and the National Ataxia Foundation to help refine clinical trials in this therapeutic area.”

215churchstreet2By Mitchell Young

NEW HAVEN: A newly public Bio-tech holding company Biohaven [ NYSE: BHVN] has chosen a high profile former bank building in the city’s Central Business District for its new headquarters. The moves comes as the company is reporting amazing results in the stock market amid positive announcements in its drug development efforts.

Biohaven is organized as a biotech holding and development company, and has raised funds to invest in New Haven biotech Kleo Pharmaceuticals. The company also is organized to conduct clinical trials and develop drugs it has purchased the rights for from a variety of organizations and companies including; Bristol-Myers Squibb Company, AstraZeneca AB, Yale University, Catalent, RutgersALS Biopharma LLC and Massachusetts General Hospital.

Biohaven went public this past May raising $168 million at approximately $17 per share and the stock has taken a rocket ride to $37 a share in just three months. Its current market value of $1.3 billion, makes it the second most valuable biotech in the state.

The 11,500-square foot free standing building is at 215 Church Street, next to the Quinnipiac Club was previously headquarters for the Bank of New Haven, the Bank of Southern Connecticut and most recently a branch of Liberty Bank.

The building is owned by Netz USA LLC a U.S. subsidiary of Netz Group Ltd., a publicly traded Israeli  company. Nezt, has more than $100 million of real estate investments in the US Capitalize 360 LLC run by Frank Micali is its contracted asset management firm, that negotiated the lease. .

Micali is a long-term Connecticut realtor with deep roots in New Haven’s CBD, including a long-term stint at the C.A. White company and in the commercial leasing of 900 Chapel Street.

Micali said “this is a strategic site for Biohaven’s headquarters which will allow it to take full advantage of the area’s talent and intellectual resources including Yale University, its labs and research, Yale New Haven  and the many biotech firms established in New Haven.”

Biohaven’s stock has grown steadily from its IPO, but took off on the release of information on August 14, about its drug development activities

The company said that it’s clinical trials for a treatment of migraine headaches was moving into a Phase 3 clinical trial, another drug was given Fast Track status by the FDA and was expecting some Phase 2/3 results before the end of the year. A third drug for BHV-5000 to treat Rett syndrome received Orphan Drug Designation from the FDA as well.

melinta therapeutics largeNEW HAVEN: Melinta Therapeutics is merging with Cempra, Inc. [Nasdaq: CEMP],of Chapel Hill, NC. a clinical-stage pharmaceutical company with a $161 milion market value, also focused on serious bacterial infections. The new company will be named Melinta, and current Melinta shareholders will receive 52% of the stock of the combined companies, the merger release said that a new CEO will be named, but did not indicate who. 

The combined companies will have an expanded drug pipeline and the $175 million in cash on Cempra’s balance sheet and a $90 million debt and equity investment will be available to develop Melinta’s recently FDA approved drug.

bigstock Brain head ache migraine Alz 58031558NEW HAVEN: Biohaven Pharmaceutical Holding Company Ltd. [NYSE: BHVN] has begun enrollment in the second of its two Phase 3 clinical trials to evaluate the safety and efficacy of its orally-dosed Rimegepant (BHV-3000) for the acute treatment of migraine.

Rimegepant was acquired from Bristol-Myers Squibb [NYSE: BMY], under a development agreement. BMY had placed the drug on the back burner after developing it. The purchase of the drug was first revealed when the company announced its IPO in May.

The company came public raising $168 million at $17 per share, the company stock is trading at approximately $25.50 per share.

Vlad Coric, M.D., CEO, said, "the launch of our second Phase 3 trial of rimegepant brings us one step closer to our goal of bringing an oral CGRP antagonist to market for patients suffering from migraine headaches." Adding, "we believe our Phase 2b data with rimegepant shows that this investigational agent has the potential to be a best-in-class migraine treatment option, and its oral route of administration will spare patients the need for intravenous or subcutaneous administration associated with other CGRP antagonists in development."

According to the company American’s aren’t the only ones suffering with migraines in large numbers, citing, The Migraine Research Foundation


alexionn

After Columns and Reports in The Hartford Business JournalConnecticut Post and New Haven Register, all questioning the health and even the viability of New Haven’s flagship company Alexion Pharmaceuticals, amidst management changes and media criticism, this writer took the contrary approach.


Writing in May “I said, "On the other hand, all eighteen investment analysts that research the company, have it as a buy, expecting gains from 30% to 70%. Adding, Make some money, buy their stock, before the second quarter results come in. [ I would if I had the dough]."

The stock has continued to gain and you would be up more than 35% from our report, rising more than $37+ per share from the naysayers' low. 

Why did we get it right? 

We’re not content with consensus thinking backed by uninformed opinion and when it comes to a public company, we’re never going to follow the lead of self-serving short sellers or stock promoters.

Our approach, research and information, beyond just regurgitating press releases.

Our mission is not to steer you, but on the rare occasion we do, it will be backed by a foundation of vetted information, hopefully our colleagues will adopt that approach as well, when reporting on important companies in Connecticut. 

If you think we’re boosters of Alexion, you can re-read our stories on the Search [Alexion] page at Conntact.com.

_______________________________________________________________________

Alexion Shakes Off Management Change and Bad Press With Strong Financial Results

By Mitchell Young

NEW HAVEN: Alexion Pharmaceuticals, Inc. (ALXN) blew out estimates of sales and earnings for the second quarter, and new CEO Ludwig Hantson outlined some important directions on the company’s research and “pipeline” direction.

Total revenues in the quarter were $912 million, a 21% percent increase compared to the same period in 2016. Earnings were $1.56 per share, compared to $1.13 per share in the second quarter of 2016, a 38% increase.

The company’s stock price reached as high as $142 per share, after the earnings report before dropping back into the $130s, still up from a low of $96.75 in November. 2016, when the company announced an internal investigation of how it was booking certain sales, which was followed by the resignation of its CEO and CFO. 

The company’s flagship product Soliris continued to move ahead generating a 16% sales increases in spite of media criticism about it marketing approach. Soliris has also been advancing for regulatory approval of other rare diseases. The company is also in clinical development of a replacement to stave off competitor drugs and generics.

The company also laid out markers for future drug development, announcing it was terminating its partnerships with three bio-techs, Moderna TherapeuticsBlueprint Medicines, both in Cambridge, MA and Arbutus Biopharma of Burnaby, Canada. 

The company said it was dropping certain research approaches and sticking with its “complement franchise.” The body’s complement system is an important part of the innate immune system.

On June 13, Hantson floated the idea at the Goldman Sachs Healthcare Conference, that he was considering writing off the investment of one of the company's key drugs, Kanuma. Hantson referred to the commercial results as near the bottom on biotech launches.

Kanuma is an enzyme replacement therapy for an ultra-rare inherited metabolic disorder, it was approved by the FDA at the end of 2015.

Sales of the drug were under $26 million for the full 2016 year. Alexion paid $8.4 billion in June of 2015 for Cambridge based Synageva BioPharma Corp. [Nasdaq:GEVA].

Hantson apparently has changed his mind, as Kanuma sales did double for the quarter to $15 million and was included in the company’s presentation for an ongoing “strategic” effort, with Alexion trying to open more global markets to boost sales.

Alexion’s other acquired drug Strensiq was classified by the company as a new “growth driver,” with sales increasing 85% to $83 million for the quarter.

Hantson described the announcements saying, "Alexion delivered strong performance in the second quarter of 2017 while also executing on several initiatives to position the company for the future, including strengthening the Soliris patent portfolio, reaching a funding agreement for Strensiq in England, advancing the late-stage pipeline, enhancing compliance and culture and building a strong leadership team." He added, "Our strategy for the next phase of growth will focus on our strengths to deliver sustainable long-term performance and increased value for shareholders. We will achieve this by growing our rare disease business, leveraging our expertise in complement, pursuing disciplined business development to expand the pipeline, and taking steps to optimize our infrastructure and operating model."

More detail on company press release

kleoNEW HAVEN: Kleo Pharmaceuticals has entered into a drug development agreement with PeptiDream Inc an “emerging” public pharmaceutical company traded on the Tokyo exchange.

PeptiDream will use its Peptide Discovery Platform System ("PDPS") technology to identify drug development targets for the treatment of Cancer and Kleo will “engineer molecules using its Antibody Recruiting Molecule ("ARM") and Synthetic Antibody Mimic ("SyAM") platform technologies to develop small molecule immunotherapies.”

Kleo will receive an upfront payment and will have the right to develop and commercialize all compounds resulting from the collaboration. PeptiDream is eligible to receive a tiered share of proceeds from any products that arise from the collaboration based on the  financial investment made into the product by PeptiDream, financial terms were not disclosed.

Douglas Manion, MD, CEO of Kleo, said “we think combining PeptiDream's PDPS technology with Kleo's ARM and SyAM platform represents a truly potent partnership. We are looking forward to advancing this new paradigm of small molecule immunotherapies into the clinic."

Kleo Pharmaceuticals Inc. is a biotechnology company initially formed on the basis of intellectual property from the Spiegel Lab at Yale University and funded by a series A round led by New Haven based Biohaven Pharmaceuticals Holding Ltd [NYSE:BHVN] . The company was founded by Dr. David Spiegel and Roy Prieb and is focused on developing a new class of targeted immunotherapies.

flublokBy Mitchell Young

 MERIDEN: Sanofi, [NYSE: SYN] the $111 billion  Paris, France based pharmaceutical giant announced it will purchase Protein Sciences Corp. [PSC] for up to $750 million including an up-front payment of $650 million and another $100 million if the company reaches [unannounced] benchmarks].

Protein Sciences was recognized by Business New Haven in 2011 as a greater New Haven Healthcare Hero for Innovation.

PSC developed an innovative vaccine development technology and its non-egg based Flu vaccine, Flu-blok is the only non-egg based flu vaccine approved by the US Federal Drug Administration. The business information website Owler.com estimates revenues of $34 million and 120 employees for the private company.

Sanofi does more than $40 billion in annual revenues and is one of a handful of major pharmaceutical companies with a significant vaccines’ business, approximately $5 billion annually.

In a world of pharma giants. marketing Flu-blok has been a challenge for PSC, a relatively tiny bio-tech, but that could change under Sanofi’s control

The company claims it is the world’s leading flu vaccine company with more than $1.5 billion in flu vaccine revenues.

"The acquisition of Protein Sciences will allow us to broaden our flu portfolio with the addition of a non-egg based vaccine," said David Loew, Sanofi executive vice president and head of Sanofi Pasteur, the company's vaccines' arm.

Manon M.J. Cox, President and CEO of PSC, said, “as part of Sanofi Pasteur, we expect our Flublok influenza vaccine to benefit from Sanofi Pasteur’s expertise in the field of influenza vaccines.”

Protein Sciences was founded in 1983 with the hopes of creating a vaccine for HIV/AIDS. The company struggled for more than a decade and short of funds recruited Daniel Adams, [current executive chairman of the board] to be its new CEO in 1995 and attract new capital to the company.

Adams had been a biotech executive at several companies and a co-founder [1976] and CEO of Biogen of Cambridge Mass. [now a $58 billion company].

Adams quickly jettisoned the HIV vaccine effort and turned the company toward what was hoped would be a somewhat easier target, a new non-egg based Flu vaccine.

Egg based vaccines take many months to grow and can take up to a year to produce supply once the appropriate viral target is identified.

And while with PSC technology a vaccine could be developed in days and scaled up in production in weeks, the industry’s respect for the technology and the company and its ability to attract capital was still proving difficult.

Things started to change with the September 11, 2001 terrorist attacks however, when Protein Sciences found itself on the front lines of the war against bioterrorism.

In an interview with Business New Haven, published in October 2001, then-CEO Adams revealed that the federal Centers for Disease Control (CDC) told the company that “we were the only ones who could make a vaccine to protect people against the pandemic flu in time to make a difference.”

At the time Adams suggested to the CDC that the deadliest threat might be not from bio-terrorism, but rather a reprise of the “Spanish” flu pandemic that killed 50 million people worldwide immediately following World War I.

Two years later that fear hit home across the globe, when the World Health Organization identified SARS as a new disease in 2003.

SARS spread rapidly and infected thousands of people around the world, including people in Asia, Australia, Europe, Africa as well as the Western Hemisphere.

By 2005 fear that another flu, Avian Flu virus (H5N1), would find its way to the U.S. and promising results by Protein Sciences in treating chickens against the flu convinced the FDA to put a PSC vaccine on the fast track.

By 2007 the U.S. Department of Health & Human Services committed $8.5 billion to address concerns of a pandemic flu through expanding existing vaccine facilities and funding new technologies. However, much of that money continued to be directed to large pharmaceutical companies and much of that to traditional vaccine methods.

Development costs were sinking the company just as the technology effort was turning the corner, cost for a clinical trial was $20 million.

In 2008, Protein Sciences announced the sale of the company for $78 million as word of a major U.S. government contract surfaced.

The development would be funded and finished by the acquirer, Emergent BioSolutions of Rockville Md. Emergent provided immediate funding to shore up Protein Sciences, but the deal soon fell apart with acrimony and lawsuits a plenty.

In early 2009 H1N1 surfaced as a potential pandemic virus. Like many flu strains, H1N1 started in birds and was transferred to pigs, which is why it was initially called swine flu. With pigs as hosts the flu became more suitable for transmission to humans. Eventually more than 17,000 people worldwide would die, in part due to lack of supply of vaccines.

In 2009 PSC won a contract from the Biomedical Advanced Research and Development Authority of the U.S. Department of Health & Human Services for up to $150 million for the development of its vaccine technology, clinical testing and eventual manufacturing of the FluBlok vaccine.

Development was able to continue and FluBlok received its initial FDA approval in 2013 at the time only for adults 18-49, subsequent approvals included adults 18 and over.