Nab­bing late-stage drug, PTC ac­quires gene ther­a­py start­up Ag­ilis for $200M in cash/stock

Af­ter its bumpy road with a thrice-failed Duchenne drug, the team at PTC Ther­a­peu­tics has been busy putting new irons in its fire. In its lat­est ef­fort, the New Jer­sey com­pa­ny an­nounced Thurs­day that it’s ac­quir­ing a gene ther­a­py start­up with a few promis­ing drugs in its pipeline.

In­vestors, al­ready heart­ened by PTC’s new­ly-re­leased da­ta on a dif­fer­ent drug, are glow­ing. The com­pa­ny’s stock $PTCT has climbed 10% in af­ter-hours trad­ing.

PTC is pay­ing $50 mil­lion in cash and about $150 mil­lion in stock to buy up Ag­ilis Bio­ther­a­peu­tics, a Cam­bridge, MA-based com­pa­ny that has a lead gene ther­a­py can­di­date in late-stage tri­als. The drug is be­ing de­vel­oped to treat a rare CNS dis­or­der called Aro­mat­ic L-Amino Acidd De­car­boxy­lase, or AD­DC. It’s al­ready been test­ed in two prospec­tive clin­i­cal stud­ies, which showed it boost­ed dopamine pro­duc­tion — a key goal for treat­ing AD­DC.

Stu­art Peltz

“Da­ta from tri­als that be­gan in 2010 have demon­strat­ed long-term ev­i­dence of durable clin­i­cal ben­e­fit,” the com­pa­ny stat­ed. “Based on mul­ti-year da­ta from ini­tial clin­i­cal stud­ies with the AADC gene ther­a­py treat­ment can­di­date, PTC plans to sub­mit a Bi­o­log­ics Li­cense Ap­pli­ca­tion (BLA) to the FDA in 2019.”

Ag­ilis al­so has oth­er gene ther­a­py can­di­dates, in­clud­ing a Friedre­ich atax­ia pro­gram near­ing IND, and an An­gel­man syn­drome pro­gram.

“The ad­di­tion of the gene ther­a­py plat­form trans­forms PTC and aligns with our vi­sion of be­ing a leader in the treat­ment of rare dis­or­ders,” said Stu­art Peltz, CEO of PTC, in a state­ment. “We look for­ward to ad­vanc­ing the Friedre­ich atax­ia and An­gel­man syn­drome pro­grams in­to the clin­ic in the next two years.”

The ac­qui­si­tion is good news for PTC,  a com­pa­ny that’s been plagued with bad news for years due to the fail­ure (af­ter fail­ure af­ter fail­ure) of its Duchenne mus­cu­lar dy­s­tro­phy drug ataluren. That drug, ap­proved in Eu­rope, has so far failed to please reg­u­la­tors in the US. Al­though PTC still isn’t giv­ing up on DMD, they’re cer­tain­ly shoring up risk by putting more prod­ucts in the pipeline.

And this isn’t the first time a Duchenne com­pa­ny veered in­to gene ther­a­py land. Sarep­ta, the mak­er of the con­tro­ver­sial Ex­ondys 51 treat­ment for DMD, has made gene ther­a­py a huge area of new fo­cus for the com­pa­ny. I met with Sarep­ta’s CEO Doug In­gram at BIO this year, where he up­dat­ed me on the com­pa­ny’s wide­spread ef­forts in the space. Sarep­ta now has 8 new gene ther­a­py pro­grams, he says, and just re­cruit­ed Louise Rodi­no-Kla­pac, a for­mer Na­tion­wide Chil­dren’s Hos­pi­tal ex­ec, to lead its new gene ther­a­py unit.

“There is no doubt that gene ther­a­py — if it is suc­cess­ful — is a mas­sive, mas­sive op­por­tu­ni­ty,” In­gram said at the time. “I mean bil­lions of dol­lars. The fi­nan­cial rea­son for that is that gene ther­a­py is ag­nos­tic to mu­ta­tion; it spans all mu­ta­tions. In that sense, there’s a lot of big fi­nan­cial op­por­tu­ni­ty for gene ther­a­py.”

PTC has clear­ly board­ed that train. The Ag­ilis ac­qui­si­tion deal in­cludes up to $535 mil­lion in suc­cess-based mile­stones in con­nec­tion with reg­u­la­to­ry ap­provals on the three most ad­vanced pro­grams and re­ceipt of a pri­or­i­ty re­view vouch­er, as well as tiered com­mer­cial mile­stones of $150 mil­lion, and 2-6% of an­nu­al net sales for Friedre­ich atax­ia and An­gel­man syn­drome, the com­pa­ny said in a state­ment.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

Por­tion of Neil Wood­ford’s re­main­ing in­vest­ments, in­clud­ing Nanopore, sold off for $284 mil­lion

It’s been precisely one year and one day since Neil Woodford froze his once-vaunted fund, and while a global pandemic has recently shielded him from the torrent of headlines, the fallout continues.

Today, the California-based patent licensing firm Acacia Research acquired the fund’s shares for 19 healthcare and biotech companies for $284 million.  Those companies include shares for public and private companies and count some of Woodford’s most prominent bio-bets, such as Theravance Biopharma, Oxford Nanopore and Mereo Biopharma, according to Sky News, which first reported the sale. It won’t include shares for BenevelontAI, the machine learning biotech once valued at $2 billion.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,200+ biopharma pros reading Endpoints daily — and it's free.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,200+ biopharma pros reading Endpoints daily — and it's free.

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.