Af­ter win­ning an ear­ly deal on XO1, the vir­tu­al biotech gang gets back to­geth­er for Su­perX

A lit­tle less than two years af­ter J&J swooped in to snag the pre­clin­i­cal an­tithrom­bin drug at XO1 in the UK, the vir­tu­al team of en­tre­pre­neurs who cre­at­ed that as­set-cen­tered ven­ture is back to­geth­er again. This time they’re op­er­at­ing as Su­perX, and they’re work­ing in a very fa­mil­iar re­search space.

With a very fa­mil­iar bud­get.

Like XO1, Su­perX is get­ting a liftoff with an $11 mil­lion round — this time drawn from Medicxi and part­ners at John­son & John­son In­no­va­tion. Uni­ver­si­ty of Cam­bridge Pro­fes­sor Jim Hunt­ing­ton is the CSO. Trevor Baglin is the CMO. X01 co-founder and Medicxi part­ner David Grainger is on the team, along with Kevin John­son, al­so at Medicxi, tak­ing the chair­man’s role. XO1 vets Bob Schroff will be op­er­a­tions chief with Jo Davies once again han­dling the in­tel­lec­tu­al prop­er­ty.

The busi­ness mod­el here is some­thing that Medicxi is quite com­fort­able with. It’s a tight bud­get with no room for a ded­i­cat­ed staff. You can ex­pect a CRO to do much of the heavy pre­clin­i­cal lift­ing as they get the man­u­fac­tur­ing squared away.

Medicxi isn’t known for splash­ing large sums on ear­ly-stage drugs. But they have a suc­cess­ful track record in set­ting up ear­ly deals af­ter demon­strat­ing some val­ue ahead of the clin­ic, stay­ing “laser fo­cused,” in Grainger’s words, on a sin­gle project. And while there are no op­tions or strings at­tached on J&J’s in­volve­ment, the same phar­ma gi­ant that is tak­ing XO1’s drug ahead will have a front row seat to see how this new fol­low-up ther­a­py per­forms.

“The whole an­ti­co­ag­u­lant space is sim­ply enor­mous,” Grainger tells me, “with so many dif­fer­ent trig­gers for throm­bo­sis. We still be­lieve the XO1 drug is a mag­nif­i­cent drug,” but there’s still plen­ty of types of throm­bo­sis to tack­le.

Be­sides, he added, the team all agreed that “wouldn’t it be great to do it again?”

Like X01, says Baglin, the com­pa­ny’s sole pro­gram emerged out of ob­ser­va­tions around the case stud­ies of pa­tients that should bleed, but didn’t, build­ing on the same ex­pe­ri­ence that in­spired the pre­de­ces­sor pro­gram but com­ing at it from a dif­fer­ent an­gle. In the XO1 case, a pa­tient that should have been suf­fer­ing from he­mo­phil­ia was able to stop bleed­ing nat­u­ral­ly, and they took the an­ti­body that did that and syn­the­sized it.

The part­ners are en­thu­si­as­tic about this new ven­ture, but they’re al­so play­ing their cards close to the vest. The an­ti­bod­ies they’re work­ing with have prop­er­ties for an­ti­co­ag­u­la­tion, look­ing to block throm­bo­sis that caus­es heart at­tacks and strokes. The goal is to de­vel­op an ide­al an­ti­co­ag­u­lant for chron­ic use, with­out the threat of bleed­ing posed by the cur­rent gen­er­a­tion of ther­a­pies.

But don’t ask about the tar­get yet.

Jim Hunt­ing­ton

“The (new) tar­get we haven’t dis­closed yet,” says Hunt­ing­ton, “so it’s im­pos­si­ble to say why we’ve de­cid­ed on this tar­get. But it’s an in­cred­i­bly cool sto­ry.”

The team is look­ing at a cou­ple of years work be­fore this next drug will be ready for the clin­ic. And then they can see whether they mount the first hu­man study them­selves, or a part­ner comes along to snag it for them­selves.

This new com­pa­ny comes just days af­ter Medicxi joined a syn­di­cate in­volv­ing Touch­stone and Cam­bridge En­ter­prise to back an­oth­er spin­out from the labs of Hunt­ing­ton and Baglin. Apcin­teX Lim­it­ed picked up a £14 mil­lion Se­ries A fund­ing round to back a new he­mo­phil­ia drug.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — and they're prep­ping a pitch to the FDA

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company says that a new analysis of an old dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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Vas Narasimhan. Getty Images

Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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Take­da tees up $420M deal for celi­ac an­ti­dote, con­tin­u­ing R&D re­fo­cus

Sometime in the 1st century AD, a patient presented to Arataeus looking like a varicose ghost. He was “emaciated and atrophied, pale, feeble and incapable of performing any of his accustomed works,” the Greek physician wrote, with hollow temples and huge veins running all over his body.

A dysfunctional digestive system, Arataeus concluded – an imbalance he attributed to a “heat” deficiency in a system he and other Greeks regarded as functioning similarly to an oven – and coined a term: coeliac disease, after the Greek word for abdomen.

UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

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Photo credit: Jacquelyn Martin

Where are the in­ter­change­able biosim­i­lars?

In June 2017, Leah Christl, former biosimilar lead at FDA, told a conference in Chicago that interchangeable biosimilars were likely coming to the US market within two years.

And although no interchangeable biosimilar has been approved by FDA yet, and Christl has since moved on to Amgen, progress on interchangeable biosimilars has been slow in the intervening years.

Most recently, Boehringer Ingelheim announced that it has completed, as of last April, a switching study necessary for launching an interchangeable biosimilar for Humira (adalimumab), although the company did not offer any further details on the timing of its submission to FDA or whether there will be an advisory committee to review the data. Boehringer already has an adalimumab biosimilar approved by FDA, which it will launch in the US on 1 July 2023.

FDA re­buffs lit­tle As­ser­tio Ther­a­peu­tic­s' long-act­ing ACTH for­mu­la­tion, shares sink

Tiny Assertio Therapeutics’ shares plunged pre-market on Tuesday, after the FDA has spurned its man-made version of the hormone ACTH, which was being reviewed as a diagnostic for patients presumed to have adrenocortical insufficiency.

The Lake Forest, Illinois-based drugmaker said its development partner West Therapeutic Development had received a complete response letter from the US regulator, which indicated that that certain “pharmacodynamic parameters were not adequately achieved” for the product.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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That $335M JV Bay­er set up on CRISPR/Cas9? They’re let­ting the biotech part­ner car­ry on

Bayer committed $300 million to set up a joint venture on CRISPR/Cas9 tech with CRISPR Therapeutics $CRSP. But they’re handing off control now to the smaller biotech while retaining a couple of opt-ins for programs nearing an IND.

Bayer $BAY made much of the fact that they were going all-in on gene editing when they did their deal 3 years ago with CRISPR Therapeutics, which pitched $35 million in on their end. This was the cornerstone of their plan to set up new JVs that could make some serious leap forwards in hot new R&D spaces. Now CRISPR will have full management control of Casebia as they pursue programs in hemophilia, ophthalmology and autoimmune diseases.
Samarth Kulkarni, the CEO at CRISPR, made it sound like a natural progression.