Pen­ny stock biotech Syn­thet­ic Bi­o­log­ics sees its 'break­through' sta­tus pulled as FDA pon­ders safe­ty is­sues

A lit­tle un­der a year ago the pen­ny stock of Syn­thet­ic Bi­o­log­ics $SYN gy­rat­ed high­er on the news that its drug SYN-004 (rib­axa­m­ase) — de­signed to pro­tect the gut mi­cro­bio­me from the hav­oc caused by some an­tibi­otics and guard against Clostrid­i­um dif­fi­cile in­fec­tion — had won the FDA’s not-so-rare break­through ther­a­py des­ig­na­tion.

Steven Shall­cross, Syn­thet­ic in­ter­im CEO

To­day, though, af­ter the biotech re­viewed plans for an up­com­ing Phase III pro­gram, they fol­lowed up with the rare news that the FDA is drop­ping the BTD af­ter reg­u­la­tors con­clud­ed that they couldn’t fig­ure out why the drug arm ex­pe­ri­enced a high­er rate of deaths in their Phase IIb tri­al.

From their state­ment:

Fol­low­ing FDA’s re­view of the ad­di­tion­al da­ta, it was de­ter­mined that the re­quire­ments for Break­through Ther­a­py Des­ig­na­tion were no longer met due to the nu­mer­i­cal im­bal­ance in fa­tal ad­verse events ob­served in the study which could not be ful­ly eval­u­at­ed due to the lim­it­ed safe­ty data­base, and the study’s method of sta­tis­ti­cal treat­ment of pa­tients who did not com­plete the study for any rea­son. 

The biotech’s stock slid 12% in pre-mar­ket trad­ing — falling to 30 cents a share.

You don’t of­ten hear about BTD’s be­ing with­drawn, but it hap­pens more of­ten than you might think. The Twit­ter buzz that met the news this morn­ing in­clud­ed a link to the FDA’s page on BTDs that showed 4 break­through ti­tles were re­scind­ed so far in fis­cal year 2018, which got start­ed Oc­to­ber 1.

The agency set up the BTD pro­gram in or­der to make a com­mit­ment to biotechs to help ac­cel­er­ate the re­view of drugs that war­rant­ed spe­cial at­ten­tion. And it’s been gain­ing steam un­der Com­mis­sion­er Scott Got­tlieb, who would like to see more reg­u­la­tors em­brace it the same way the on­col­o­gy di­vi­sion has.

The FDA did ex­press a will­ing­ness to keep help­ing, says Syn­thet­ic Bi­o­log­ics. And the biotech has worked out a plan with reg­u­la­tors on the Phase III de­sign that will keep a care­ful fo­cus on safe­ty, so they can keep a rein on risk.

They al­so agreed that the pri­ma­ry end­point should be “the re­duc­tion of the in­ci­dence of Clostrid­i­um dif­fi­cile in­fec­tion (CDI) in the rib­axa­m­ase treat­ment group rel­a­tive to place­bo.”

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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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.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

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.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

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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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.