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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

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The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

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Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

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London Stock Exchange-listed PureTech Health announced Wednesday that it’s looking to extend to Nasdaq. But due to its “strong cash position,” the biotech says it isn’t issuing any new shares in the potential secondary listing.

The company’s shares closed at £256.50 Tuesday on the London Stock Exchange. Its candidate LYT-100 is currently in Phase I development for various indications, including lymphatic flow disorders and fibrotic and inflammatory disorders. PureTech is expecting a Phase Ib readout in lymphatic flow disorders later this year, and is also planning to launch a Phase II study for the drug to treat respiratory conditions experienced after Covid-19.

Rep. Andy Harris (R-MD) (Tasos Katopodis/Pool via AP Images)

The mi­cro-cap that tapped a mask-skep­tic con­gress­man for their Covid DSMB is ap­ply­ing for an EUA. Their ev­i­dence? 21 pa­tients

NeuroRx, the tiny biotech that came under fire last week after Politico reported they selected a congressman and two other acquaintances of the CEO to supervise their Covid-19 drug trial, announced today that they will ask the FDA to authorize their drug based on the results of just 21 patients.

Such an application would test the agency’s standards of evidence for an EUA, which have already come under scrutiny after controversial authorizations for convalescent plasma and hydroxychloroquine. The only other company to discuss their intention to file for an EUA, Eli Lilly, did so after results came back from a randomized control study testing their antibody in over 450 patients.

J&J re­leas­es PhI­II safe­ty blue­print for Covid-19 vac­cine tri­al. How does it stack up to Mod­er­na, Pfiz­er and As­traZeneca?

Along with the initiation of its Phase III Covid-19 vaccine study announced Wednesday morning, Johnson & Johnson also released its trial protocol, giving an inside look at how the company is conducting its late-stage research.

The move comes after the other three companies conducting Phase III’s in the US — Moderna, Pfizer and AstraZeneca — each disclosed their own trial blueprints within the last week. Though the release of such protocols is typically done after trials have been completed, drug developers had come under intense pressure after a brief safety scare in an AstraZeneca trial and amid growing concern of a politically motivated vaccine authorization.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.