Rare dis­ease play­er rakes in a meaty $81M in Spain's biggest ever pri­vate biotech fundraise

Fund­ing rounds in the Unit­ed States for the life sci­ences in­dus­try of­ten dwarf fi­nanc­ing in Eu­rope. Looks like rare dis­ease biotech San­i­fit is an anom­aly. On Wednes­day, the Span­ish drug de­vel­op­er un­veiled a near­ly $81 mil­lion boun­ty — Spain’s largest-ever pri­vate biotech fundraise — ahead of a piv­otal study for its lead ex­per­i­men­tal drug.

San­i­fit, which was spun out of the Uni­ver­si­ty of the Balearic Is­lands in 2007, is work­ing on a treat­ment for cal­ci­phy­lax­is, a rare dis­ease char­ac­ter­ized by cal­ci­um ac­cu­mu­la­tion in small blood ves­sels of the fat and skin tis­sues — which lead to blood clots, skin ul­cers and po­ten­tial­ly life-threat­en­ing in­fec­tions.

The dis­ease, es­ti­mat­ed to af­fect about 30,000 glob­al­ly, typ­i­cal­ly af­flicts those with chron­ic kid­ney dis­ease. Al­though treat­ments such as painkillers are used to man­age symp­toms, there are no drugs ap­proved to treat the eti­ol­o­gy of the dis­ease — which is what San­i­fit’s SNF472 is de­signed to do.

The small mol­e­cule, de­rived from nat­ur­al sources, is en­gi­neered to have an affin­i­ty for hy­drox­ya­p­atite (HAP) crys­tals — the key com­po­nent in cal­ci­fi­ca­tion de­posits. SNF472 binds to the growth sites of HAP, there­by thwart­ing their for­ma­tion.

Joan Perel­ló Bioib

Last year, SNF472 was shown to help cal­ci­phy­lax­is pa­tients un­der­go­ing dial­y­sis in a Phase II study — a piv­otal study is slat­ed to kick off in the fourth quar­ter. Mean­while, a sep­a­rate mid-stage study test­ing the drug’s ef­fect in slow­ing ar­te­r­i­al cal­ci­fi­ca­tion, a ma­jor risk fac­tor for car­dio­vas­cu­lar dis­ease in pa­tients on dial­y­sis, is ex­pect­ed to read out by the end of the year.

“When we have ad­di­tion­al da­ta avail­able, we will eval­u­ate our op­tions…be it pri­vate fi­nanc­ing or an IPO,”  San­i­fit co-founder and chief Joan Perel­ló told End­points News.

For now, the com­pa­ny has 20 em­ploy­ees — it is head­quar­tered in Pal­ma, Spain, where the in­tel­lec­tu­al prop­er­ty is housed and pre­clin­i­cal re­search and man­u­fac­tur­ing take place. It al­so has op­er­a­tions in San Diego, where a bulk of the clin­i­cal de­vel­op­ment is con­duct­ed.

So far, San­i­fit has raised rough­ly $130 mil­lion, in­clud­ing the Se­ries D round an­nounced on Wednes­day. This round en­com­passed €17 mil­lion in con­vert­ible bonds as well as €55.2 mil­lion in fi­nanc­ing, led by Spain’s Caixa Cap­i­tal Risc, with par­tic­i­pa­tion from Colum­bus Ven­ture Part­ners and Al­ta Life Sci­ences, in ad­di­tion to an in­ter­na­tion­al con­sor­tium of ex­ist­ing share­hold­ers in­clud­ing Lund­beck­fonden Ven­tures, Ys­ios Cap­i­tal, For­bion Cap­i­tal Part­ners, Gilde Health­care, An­dera Part­ners, HealthE­quity and sev­er­al pri­vate in­di­vid­u­als.

“The land­scape of life sci­ences in Spain is grow­ing sig­nif­i­cant­ly. When we start­ed the com­pa­ny…there were no…ven­ture cap­i­tal­ists, al­most no biotech com­pa­nies,” Perel­ló not­ed.

As the Span­ish gov­ern­ment of­fers low-in­ter­est cred­it and in­cen­tives dur­ing the start­up and growth phas­es of life sci­ence com­pa­nies, ac­tiv­i­ty in the South­west­ern Eu­ro­pean coun­try has picked up. Last year, Take­da swal­lowed TiGenix, a Eu­ro­pean cell ther­a­py com­pa­ny head­quar­tered in Bel­gium with op­er­a­tions in Madrid, Spain.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Stephen Hoge, Moderna president (Moderna)

On morn­ing of FDA Covid-19 ad­comm, Mod­er­na com­pletes PhI­II en­roll­ment, putting them neck-and-neck with Pfiz­er

Weeks away from a potential EUA application, Moderna announced they have completed enrollment in their 30,000-person Phase III Covid-19 vaccine trial, with over a third of volunteers non-white and a quarter over the age of 65.

The announcement caps what has been the most closely-watched recruitment race in the history of drug development, as Pfizer and Moderna rushed to get enough volunteers to prove whether or not experimental vaccines could actually protect people from contracting Covid-19. Pfizer reached that mark on Sept. 15. Moderna said around the same time that they would slow down enrollment to ensure they enrolled enough participants from minority and at-risk groups.

Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

With lumasir­an on the FDA's doorstep, Al­ny­lam reads out new PhI­II da­ta in PH1

Just over a month away from its December PDUFA date, Alnylam flaunted new data from two Phase III studies to back lumasiran in primary hyperoxaluria type 1 (PH1), a rare liver condition.

The Cambridge, MA-based biotech snagged a priority review for the candidate back in June, and got positive feedback from the EMA’s Committee for Medicinal Products for Human Use just last week. Lumasiran uses RNA interference (RNAi) to silence the gene for glycolate oxidase, an enzyme used in the production of oxalate.

News brief­ing: UK biotech 4D phar­ma heads for Nas­daq via SPAC; Dr. Red­dy's shuts down man­u­fac­tur­ing af­ter cy­ber­at­tack

Another pharma company is intending to use a SPAC to join the Nasdaq.

4D pharma, a UK-based biotech, is reverse-merging with a blank check company in a deal worth up to $37.6 million. The move will give 4D pharma a new Nasdaq ticker, which will be $LBPS, using the American Depositary Share program. 4D will continue to trade on the London stock exchange under its previous ticker.

As a result of the move, 4D pharma will gain $14.6 million in cash held by the blank check company, dubbed Longevity $LOAC. The merger is expected to be completed in early 2021, after which shares will be immediately tradeable on Nasdaq.

CEO Marc Gleeson (Azura)

Azu­ra Oph­thalmics gets a $20M boost for its R&D work on eye dis­eases

Three years after closing a $16 million Series B, the same group of investors are back to give Azura Ophthalmics a $20 million boost.

That brings the Tel Aviv-Yafo, Israel-based biotech’s total fundraise to $38 million, and should pave the way for a registration study of its lead candidate in Meibomian gland dysfunction (MGD) and related eye diseases, CEO Marc Gleeson told Endpoints News.

The topical candidate, dubbed AZR-MD-001, is designed to address abnormal hyperkeratinization, or the build-up and shedding of proteins at the opening of or within the Meibomian gland. When Meibomian glands become dysfunctional, rapid evaporation of the tear film can occur, leading to dry eye disease.