Olivier Morand (Azafaros)

Dutch biotech scores €25M in small mol­e­cule bet to tack­le lyso­so­mal stor­age dis­or­ders

While a host of biotech com­pa­nies work on gene and en­zyme re­place­ment ther­a­pies for cer­tain lyso­so­mal stor­age dis­or­ders, a Dutch play­er is aim­ing to slot in its small mol­e­cule ap­proach across a range of these rare in­her­it­ed meta­bol­ic dis­or­ders.

The com­pa­ny — Aza­faros — found­ed in 2018 based on sci­ence de­vel­oped from Lei­den Uni­ver­si­ty and Am­s­ter­dam Uni­ver­si­ty Med­ical Cen­ter has se­cured €25 mil­lion in Se­ries A fi­nanc­ing as it works on shep­herd­ing its lead ex­per­i­men­tal drug in­to the clin­ic.

The drug, dubbed AZ-3102, is de­signed to work via a dual mode of ac­tion — by di­min­ish­ing metabo­lite ac­cu­mu­la­tion and en­hanc­ing lyso­so­mal func­tion.

There has been a lot of at­ten­tion for gene ther­a­py in lyso­so­mal stor­age dis­or­ders — but some ef­forts have shown no ben­e­fit and there are some chal­lenges such as the risk of im­muno­genic­i­ty, not­ed Aza­faros chief Olivi­er Morand in an in­ter­view with End­points News. “But still, it ob­vi­ous­ly has a lot of po­ten­tial.”

Lyso­so­mal stor­age dis­or­ders are in­her­it­ed meta­bol­ic dis­eases char­ac­ter­ized by an ab­nor­mal build-up of var­i­ous tox­ic ma­te­ri­als in the body’s cells as a re­sult of en­zyme de­fi­cien­cies. Lyso­somes are like the stom­ach of the cell, in charge of di­gest­ing com­plex com­po­nents like pro­teins, poly­sac­cha­rides, nu­cle­ic acids, or lipids and break­ing them down in­to sim­pler units. When this process doesn’t oc­cur, a sort of a traf­fic jam en­sues — which can cul­mi­nate in a reser­voir of tox­i­c­i­ty in dif­fer­ent or­gans such as the liv­er, spleen, heart, kid­ney, skin, bones and brain.

Al­to­geth­er, there are rough­ly 50 such dis­or­ders — in­clud­ing Tay-Sachs, Fab­ry, Hunter and San­fil­lipo — each char­ac­ter­ized by dif­fer­ent symp­toms re­sult­ing from the tox­ic ac­cu­mu­la­tion of a cer­tain metabo­lite. For ex­am­ple, tox­ic ac­cu­mu­la­tion in the brain can lead to de­vel­op­men­tal de­lays, seizures, res­pi­ra­to­ry in­fec­tions, loss of vi­sion and hear­ing, and cog­ni­tive func­tion.

“I think the re­cur­rent no­tion is that these dis­eases are very com­plex and very se­vere,” Morand not­ed. “So hav­ing sev­er­al treat­ment modal­i­ties and com­bin­ing them may be the way to go for a so­lu­tion.”

In con­trast to gene or en­zyme re­place­ment ther­a­py de­vel­op­ers, Aza­faros’ small mol­e­cule ef­fort has the po­ten­tial for use across sev­er­al in­di­ca­tions, he added, with­out dis­clos­ing how long it will take AZ-3102 to be ready for clin­i­cal test­ing.

The Se­ries A round was led by For­bion, with par­tic­i­pa­tion from Bio­Med­Part­ners and found­ing in­vestor Bio­Gen­er­a­tion Ven­tures.

Months ago, Mer­ck shelled out up to $576 mil­lion to swal­low Cal­por­ta, which has been work­ing on small mol­e­cule ag­o­nists of TRPML1 — de­signed to re­store cal­ci­um ef­flux and nor­mal­ize lyso­so­mal func­tion — to treat lyso­so­mal stor­age dis­or­ders, as well as neu­rode­gen­er­a­tive dis­eases.

Mean­while, Sang­amo Ther­a­peu­tics has test­ed its gene edit­ing zinc fin­ger tech­nol­o­gy in pa­tients with two lyso­so­mal stor­age dis­eases. Lentivi­ral gene ther­a­py de­vel­op­er Avro­bio al­so is in the ear­ly stages of as­sess­ing the po­ten­tial of its tech­nol­o­gy across dif­fer­ent lyso­so­mal stor­age dis­eases.

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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UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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FDA puts pe­di­atric aGVHD drug on pri­or­i­ty re­view lane — will they go vir­tu­al with the ad­comm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

Covid 19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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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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ITeos nabs $125M as they prep Keytru­da com­bi­na­tion tri­al — if Covid-19 will let them

For iTeos, it turned out, $75 million could only last so long.

Two years after announcing their eye-catching Series B raise, the Belgian biotech is back with an even larger Series B-2: $125 million.

The now $200 million financing illustrates the vast capital available for those with promising new immuno-oncology compounds, particularly those that might be used in combination with existing therapies. In December, iTeos announced a collaboration with Merck to test its lead compound with Keytruda this year. The proceeds will push forward that trial and help fund the ongoing Phase I/II trials for that compound, EOS-850, and a second one, EOS-448.

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Dai­ichi Sankyo sinks $200M in­to new gene ther­a­py tech from Ul­tragenyx

In a leap to the gene therapy space, Daiichi Sankyo has dropped $200 million to access Ultragenyx’s manufacturing technology, providing the rare disease biotech with plenty of cash and a stock boost amid a general cash crunch.

For $125 million in cash and a $75 million equity investment, Daiichi Sankyo has bought a non-exclusive license to the IP around two platforms with which it plans to develop AAV-based gene therapy products. The Japanese pharma is purchasing the stock $RARE at $60 per share, more than a third above its current price of $44.43.

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).