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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Ken Greenberg, SonoThera CEO

Gene ther­a­py goes acoustic as ARCH-backed biotech launch­es with ul­tra­sound gene de­liv­ery plat­form

After co-founding two biotechs off virus-based therapies, one for pain and one for cancer, Ken Greenberg decided to go in a different direction for his newest biotech, SonoThera.

Based out of San Francisco, SonoThera announced Monday morning that it raised $60.75 million to develop new gene therapies — but delivered by ultrasound, which Greenberg says can address the major challenges facing more conventional viral gene therapies.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

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