Spark's CSO is wrap­ping up an­i­mal stud­ies on a gene ther­a­py for Pompe dis­ease as new work in the field sees ex­plo­sive growth

When Fed­eri­co Min­gozzi joined Spark Ther­a­peu­tics $ONCE last fall as their new chief sci­en­tif­ic of­fi­cer, he brought along sev­er­al years worth of pre­clin­i­cal work on a new AAV gene ther­a­py for Pompe dis­ease. Now, he’s fin­ish­ing that lyso­so­mal stor­age dis­cov­ery-stage ef­fort with a flour­ish, un­veil­ing late pre­clin­i­cal re­sults for their drug SPK-3006, which they’re now steer­ing to­ward the clin­ic in hopes of prov­ing that they can com­plete­ly sur­pass the pricey en­zyme re­place­ment ther­a­py — and would-be suc­ces­sors in the clin­ic — used to keep pa­tients alive.

It is, with­out a doubt, still ear­ly in the process. But Min­gozzi tells me he’s keen to move on to near-term hu­man stud­ies in search of the first round of proof-of-con­cept da­ta. And you can look for the ri­val­ry among com­peti­tors to heat up fast, as an­oth­er play­er in the field is al­so an­nounc­ing an al­liance with one of the aca­d­e­m­ic pi­o­neers in the field to fo­cus on a new gene ther­a­py for Pompe dis­ease and sev­er­al oth­er rare ge­net­ic ail­ments.

“I’ve been work­ing on that a good 4 years or so,” says the CSO, who had la­bored at IN­SERM and the non­prof­it R&D group Genethon, which out-li­censed the tech to Spark. Over the past year they’ve been re­fin­ing the gene ther­a­py, us­ing a be­nign virus that can de­liv­ery cod­ing for the miss­ing en­zyme, and look­ing at its abil­i­ty to in­crease the GAA pro­tein in plas­ma in mon­keys as they looked to cre­ate a clin­i­cal-stage pro­gram for the com­pa­ny.

Work­ing prop­er­ly, says Min­gozzi, they can turn the liv­er in­to a fac­to­ry for pro­duc­ing the en­zyme, a po­ten­tial once-and-done treat­ment.

In their mouse study re­viewed at the In­ter­na­tion­al Con­gress of the World Mus­cle So­ci­ety in Ar­genti­na, Min­gozzi out­lined the gene ther­a­py’s im­pact on liv­er ex­pres­sion of the GAA en­zyme, the safe­ty pro­file, an abil­i­ty to break down glyco­gen and an im­prove­ment in strength.

The next steps are clear. Spark’s team has to com­plete a GLP tox study, then file the IND and work up a Phase I/II study that can get start­ed next year. From there they are bound for a safe­ty re­view, bio­mark­er ex­am­i­na­tions and will ul­ti­mate­ly, if it all works out, be­gin to clar­i­fy for reg­u­la­tors and pay­ers how durable it is.

In the mean­time, Am­i­cus $FOLD — which has its own Pompe drug — an­nounced plans this morn­ing to al­ly with Penn’s James Wil­son on de­vel­op­ing an AAV gene ther­a­py for Pompe dis­ease, Fab­ry, CD­KL5 and an­oth­er unan­nounced tar­get. Wil­son has been work­ing on AAV tech for decades, and Am­i­cus will add its ex­per­tise on pro­tein en­gi­neer­ing to the cause.

Spark has been the pi­o­neer for gene ther­a­pies in the US, rolling out the first ap­proved pro­gram here for $850,000. The com­pa­ny’s more ad­vanced work in­cludes pro­grams for he­mo­phil­ia, as a grow­ing num­ber of com­pa­nies ad­vance scores of new pro­grams to­ward the clin­ic.

Over the week­end can­cer drug in­vestor Brad Lon­car not­ed that the FDA ex­pects more than 150 gene ther­a­py INDs this year.

The gene ther­a­py race is on.


Im­age: Fed­eri­co Min­gozzi. Uni­ver­si­tat Autòno­ma de Barcelona

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.

RA Cap­i­tal, Hill­house join $310M rush to back Ever­est's climb to com­mer­cial heights in Chi­na

Money has never been an issue for Everest Medicines. With an essentially open tab from their founders at C-Bridge Capital, the biotech has gone two and a half years racking up drug after drug, bringing in top exec after top exec, and issuing clinical update after update.

But now other investors want in — and they’re betting big.

Everest is closing its Series C at $310 million. The first $50 million comes from the Jiashan National Economic and Technological Development Zone; the remaining C-2 tranche was led by Janchor Partners, with RA Capital Management and Hillhouse Capital as co-leaders. Decheng Capital, GT Fund, Janus Henderson Investors, Rock Springs Capital, Octagon Investments all joined.

David Meline (file photo)

Mod­er­na’s new CFO took a cut in salary to jump to the mR­NA rev­o­lu­tion­ary. But then there’s the rest of the com­pen­sa­tion pack­age

David Meline took a little off the top of his salary when he jumped from the CFO post at giant Amgen to become the numbers czar at the upstart vaccines revolutionary Moderna. But the SEC filing that goes with a major hire also illustrates how it puts him in line for a fortune — provided the biotech player makes good as a promising game changer.

To be sure, there’s nothing wrong with the base salary: $600,000. Or the up-to 50% annual cash bonus — an industry standard — that comes with it. True, the 62-year-old earned $999,000 at Amgen in 2019, but it’s the stock options that really count in the current market bliss for all things biopharma. And there Meline did well.

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