Small biotech — big am­bi­tions. Vi­r­a­lyt­ics’ on­colyt­ics ther­a­py makes the show­case round at #AACR17 with pos­i­tive com­bo snap­shot

PHO­TO: © AACR/Todd Buchanan 2017


WASH­ING­TON, DC — When you talk to Vi­r­a­lyt­ics CEO Mal­colm Mc­Coll, he has a habit of re­mind­ing his lis­ten­ers about just how small his pub­lic Aus­tralian biotech is. Small staff, small stud­ies. And there are just so many things a small biotech can do.

Mal­colm Mc­Coll

But he and his com­pa­ny {ASX: VLA} have some very big am­bi­tions in the hot on­colyt­ics field, build­ing their hopes around a ther­a­py called Cox­sack­ievirus A21 (or CVA21 or Ca­vatak). And to­day the pow­ers that be at AACR are steer­ing their lat­est da­ta in­to the show­case morn­ing round.

At the time of the da­ta cut­off for AACR, the com­pa­ny had evalu­able re­spons­es from 22 pa­tients in a small melanoma study com­bin­ing their ther­a­py — us­ing a non­genet­i­cal­ly al­tered cold virus to in­fect can­cer cells, de­signed to de­stroy them and re­cruit T cells for a fol­lowup at­tack — with Yer­voy.

In­ject­ed di­rect­ly in­to le­sions, re­searchers tracked an over­all re­sponse rate of 50%, with 4 pa­tients reg­is­ter­ing a com­plete re­sponse and 7 pa­tients ex­pe­ri­enc­ing a par­tial re­sponse. The me­di­an du­ra­tion of re­sponse was not yet reached, “with a num­ber of re­spons­es greater than six months and sev­er­al still on­go­ing” — a sig­nal of po­ten­tial dura­bil­i­ty.

Among 11 pa­tients who had dis­ease pro­gres­sion fol­low­ing ear­li­er treat­ment with an im­mune check­point in­hibitor, 4 had a re­sponse. The oth­er 7 re­spon­ders had not been treat­ed ear­li­er with a check­point.

“The pre­lim­i­nary over­all re­sponse rate of 50% is very pos­i­tive be­cause pre­vi­ous re­ports in­di­cate an 11% over­all re­sponse rate for ip­il­i­mum­ab alone and an ap­prox­i­mate­ly 28% over­all re­sponse rate for CVA21 alone,” said Bren­dan Cur­ti, the pri­ma­ry in­ves­ti­ga­tor who runs a melanoma clin­ic in Port­land.

The safe­ty pro­file al­so looked good. Of 25 pa­tients who could be eval­u­at­ed for safe­ty, two had grade 3 or high­er ip­il­i­mum­ab-re­lat­ed ad­verse events. No CVA21-re­lat­ed ad­verse events of grade 3 or high­er were re­port­ed.

The next step, says Mc­Coll, will be to add sev­er­al dozen pa­tients to the study who have failed a check­point ther­a­py and then see if they have the kind of re­sults that can be used to mount a reg­is­tra­tion study.

It’s con­ceiv­able, he says, that reg­u­la­tors could sign off on a sin­gle arm study with a cou­ple of hun­dred pa­tients for that piv­otal try, that’s “some­thing a small Aus­tralian com­pa­ny could take on.”

Vi­r­a­lyt­ics has jumped on to a stage that in­cludes a grow­ing num­ber of on­colyt­ics play­ers, like PsiOxus, which re­cent­ly signed a stand­out $936 mil­lion deal to part­ner with Bris­tol-My­ers, which was al­ready help­ing with a com­bo of Op­di­vo plus their on­colyt­ic virus.

All these com­pa­nies are com­ing in be­hind Am­gen’s T-Vec, the pi­o­neer­ing drug in the field which they be­lieve is ripe for sur­pass­ing its re­sults in the clin­ic.

So far, Mc­Coll’s small Aus­tralian com­pa­ny has paid the freight on the work, with Mer­ck pro­vid­ing Keytru­da for com­bi­na­tions in the clin­ic. At some point, if things con­tin­ue to progress, Mc­Coll al­so ex­pects to strike a mon­ey/mar­ket­ing deal.

“I don’t think a small Aus­tralian com­pa­ny will be mar­ket­ing a drug in the US,” he says with a smile. Af­ter to­day, he might find a few new op­por­tu­ni­ties on that score.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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