Part­ner­ing talks led to Bio­gen’s $800M Night­star buy­out as play­ers clus­tered around the hot deal ta­ble for gene ther­a­pies

Any­one look­ing for some in­sight in­to just how hot gene ther­a­pies have be­come should turn their at­ten­tion to­day to the be­hind-the-scenes ne­go­ti­a­tions that went in­to Bio­gen’s $800 mil­lion deal to buy Night­star Ther­a­peu­tics $NITE.

David Fel­lows, Night­star CEO

Over a pe­ri­od of 7 months, they held buy­out and part­ner­ing talks with 4 dif­fer­ent com­pa­nies be­fore Bio­gen $BI­IB emerged as the win­ner — or at least the last one at the ta­ble talk­ing M&A. Dur­ing that time, Night­star would paint a va­ri­ety of po­ten­tial fu­tures for it­self, topped by a bull ar­gu­ment that it could nail a $1.1 bil­lion prof­it on $1.2 bil­lion in sales come 2025.

The Night­star ac­qui­si­tion came just weeks af­ter Roche com­plet­ed its deal for Spark Ther­a­peu­tics, the US pi­o­neer in high-priced gene ther­a­pies and an­oth­er prime ex­am­ple that main­stream play­ers are in­creas­ing­ly ready to buy up gene ther­a­py as­sets in the clin­ic.

The prof­it fore­casts help tell the sto­ry.

Even on­ly a mod­er­ate suc­cess in Night­star’s books, ac­cord­ing to the proxy, would be close to $650 mil­lion at the peak, with $608 mil­lion in gross prof­it. And a bear case (which in­cludes mar­ket­ing ap­proval) scraped around the $400 mil­lion lev­el for sales and prof­its.

Even be­fore their IPO in the fall of 2017, a new SEC fil­ing re­veals, Bio­gen and a cou­ple of oth­er play­ers in the in­dus­try had been by to kick the tires and dis­cuss strate­gic part­ner­ing am­bi­tions with the ex­ec­u­tive team at Night­star. Those talks were pri­mar­i­ly about li­cens­ing NSR-REP1. Then in the sum­mer of last year things be­gan to heat up when CEO David Fel­lows field­ed a call from Par­ty A look­ing to do some due dili­gence on NSR-REP1 for choroi­deremia.

It was time to spread the word.

A few weeks lat­er, Night­star CFO Senthil Sun­daram had a sit-down with Bio­gen VP Daniel Karp to dis­cuss their mu­tu­al in­ter­est in gene ther­a­pies. Then David Mott, a gen­er­al part­ner at NEA and mem­ber of the board, was chat­ting about “gen­er­al strate­gic top­ics” when he picked up a sig­nal from a cer­tain Par­ty B that they had an in­ter­est in the port­fo­lio. Two months lat­er, yet an­oth­er group stepped up to dis­cuss oph­thal­mol­o­gy gene ther­a­py.

The first hard of­fer came in De­cem­ber, when Par­ty B an­ted up $20.50 a share — a 70% pre­mi­um over its price at the time. And with­in a few weeks Bio­gen, B and C were all giv­en the key to the elec­tron­ic da­ta room. Bio­gen fol­lowed up with a round of­fer of $19 to $21 a share, which opened the door on a sec­ond da­ta vault, even though they said it was still too low.

By ear­ly Feb­ru­ary, though, A, B and C were all on the out­side look­ing in, talk­ing about a col­lab­o­ra­tion in­stead. Bio­gen stuck with the buy­out talks, and came up with $25 a share.

How about $26.50, Night­star coun­tered. 

Bio­gen, though, would on­ly move to $25.50 on March 2. They closed the deal March 4.

Im­age: AP

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.