A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For near­ly a decade now, the low-pro­file Cam­bridge biotech Kymab has been bat­tling in US, UK, Japan­ese and Aus­tralian courts with the biotech be­he­moth Re­gen­eron.

Re­gen­eron has turned it­self in­to a $70 bil­lion com­pa­ny off of a plat­form of trans­geni­cal­ly hu­man­ized mice they can use to make an­ti­bod­ies for any­thing from Ebo­la to col­orec­tal can­cer. The tech­nol­o­gy took decades and bil­lions to build, 20 years from the com­pa­ny’s found­ing to the first ap­proved drug. And the com­pa­ny guards and touts it zeal­ous­ly, break­ing their pro­duc­tion process down in­to var­i­ous brand­ed com­po­nents — Ve­locim­mune, Ve­loci­gene, Ve­loci­mouse and four oth­er Ve­locis — and some­times su­ing would-be copy­cats. In 2014, most no­tably, they sued two Pfiz­er-backed en­ti­ties for patent in­fringe­ment.

The year be­fore those suits, though, Re­gen­eron al­leged in the Eu­ro­pean High Court that Kymab had in­fringed on a patent they had filed in 2012 around ways of ge­net­i­cal­ly mod­i­fy­ing a mouse. It was the first of sev­er­al dif­fer­ent patent suits be­tween the com­pa­nies. Sev­en years lat­er, that fight is still on­go­ing — a UK Supreme Court de­ci­sion is pend­ing — but Kymab has just won the lat­est sal­vo. The US Patent Tri­al and Ap­peal Board (PT­AB) re­ject­ed in April Re­gen­eron’s re­quest to in­val­i­date 4 Kymab patents, and to­day they re­ject­ed a fifth one.

The case could have im­pact both broad­ly on how biotechs choose to patent their in­ven­tions and specif­i­cal­ly on Kymab, a com­pa­ny that, af­ter rais­ing over $220 mil­lion, is now in the ear­ly clin­i­cal stage and in­di­cat­ed its in­ter­est last year for a US IPO.

Kymab has its own name for their hu­man­ized mice: Ky­mouse. Spun out of the Well­come Trust’s ge­net­ics-fo­cused Sanger In­sti­tute, the com­pa­ny was found­ed in 2010 and their plat­form works sim­i­lar­ly to Re­gen­eron: in­fect a mouse with a virus, for in­stance, and in­stead of murine an­ti­bod­ies, the mouse will cre­ate hu­man an­ti­bod­ies that in the­o­ry can then be giv­en to a pa­tient. The com­pa­ny un­veiled the plat­form in a 2014 Na­ture Biotech­nol­o­gy pa­per.

By 2015, the two com­pa­nies were in patent dis­putes in four dif­fer­ent coun­tries. At the cen­ter of the case were patents Re­gen­eron had placed on a so­lu­tion to a tricky prob­lem mak­ing trans­genic mice. Rough­ly, the first at­tempts to reap hu­man an­ti­bod­ies from ge­net­i­cal­ly mod­i­fied mice had failed be­cause when you give a mouse a ful­ly hu­man im­mune sys­tem, the mouse be­comes “im­muno­log­i­cal­ly sick,” mean­ing it can’t mount as force­ful an im­mune re­sponse and gen­er­ate as many an­ti­bod­ies as a nor­mal, mousi­fied mouse would.

Re­gen­eron fig­ured out that you pre­vent this im­muno­log­i­cal short­fall by leav­ing cer­tain mouse DNA se­quences in­tact and swap­ping them out of the an­ti­bod­ies be­fore you give them to pa­tients. The tech­ni­cal term for this is a “re­verse chimeric lo­cus.” Kymab al­so us­es re­verse chimeric lo­cus to cre­ate their an­ti­bod­ies.

While al­leg­ing Kymab in­fringed on their patents in Eu­rope, in the US, Re­gen­eron al­leged that Kymab’s an­ti­body patents should be thrown out on the grounds that they were an “ob­vi­ous” ex­ten­sion of Re­gen­eron’s work. In April, the PT­AB up­held a pre­vi­ous rul­ing that 4 of Kymab’s patents were valid. To­day’s rul­ing up­held the va­lid­i­ty of the fifth and fi­nal patent in dis­pute.

With the Japan suit over and an Aus­tralian court re­cent­ly rul­ing in fa­vor of Kymab, the last domi­no is in the UK, where Re­gen­eron ar­gued that Kymab’s mice in­fringed on their patent­ed strat­e­gy. Kymab ar­gued that Re­gen­eron’s patents did not give easy in­struc­tion for an ex­pert to car­ry out the pro­tect­ed sci­ence – as patents must. A 2016 rul­ing sided with Kymab, a 2018 rul­ing with Re­gen­eron, and in Feb­ru­ary, the UK Supreme Court heard the case. Both com­pa­nies now await their ver­dict.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.