In­smed fol­lows a new path­way at the FDA for an­tibac­te­ri­als, win­ning quick OK for Arikayce

The FDA grant­ed ac­cel­er­at­ed ap­proval for In­smed’s drug Arikayce for the treat­ment of a lung dis­ease caused by the bac­te­ria group My­cobac­teri­um avi­um com­plex, or MAC, in cer­tain pa­tients who have not re­spond­ed to con­ven­tion­al treat­ment. And reg­u­la­tors used the mo­ment to flag a new path­way opened at the FDA that is in­tend­ed to speed up the ar­rival of a new gen­er­a­tion of bad­ly need­ed an­tibac­te­ri­als.

Arikayce is the first drug to be ap­proved un­der the new Lim­it­ed Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs, or LPAD, which al­lows for more stream­lined clin­i­cal tri­als in­volv­ing small­er pa­tient pop­u­la­tions.

Scott Got­tlieb

“As bac­te­ria con­tin­ue to grow im­per­vi­ous to cur­rent­ly avail­able an­tibi­otics, we need to en­cour­age the de­vel­op­ment of drugs that can treat re­sis­tant in­fec­tions. That means uti­liz­ing nov­el tools in­tend­ed to stream­line de­vel­op­ment and en­cour­age in­vest­ment in­to these im­por­tant en­deav­ors,” said FDA com­mis­sion­er Scott Got­tlieb in a state­ment is­sued Fri­day.

“This ap­proval is the first time a drug is be­ing ap­proved un­der the Lim­it­ed Pop­u­la­tion Path­way for An­tibac­te­r­i­al and An­ti­fun­gal Drugs, and it marks an im­por­tant pol­i­cy mile­stone. This path­way, ad­vanced by Con­gress, aims to spur de­vel­op­ment of drugs tar­get­ing in­fec­tions that lack ef­fec­tive ther­a­pies. We’re see­ing a lot of ear­ly in­ter­est among spon­sors in us­ing this new path­way, and it’s our hope that it’ll spur more de­vel­op­ment and ap­proval of an­tibac­te­r­i­al drugs for treat­ing se­ri­ous or life-threat­en­ing in­fec­tions in lim­it­ed pop­u­la­tions of pa­tients with un­met med­ical needs,” Got­tlieb added.

Arikayce was ap­proved un­der the agency’s ac­cel­er­at­ed ap­proval path­way and has re­ceived or­phan drug sta­tus. The agency said ap­proval was grant­ed based on clin­i­cal da­ta that showed 29% of pa­tients on a mul­ti-drug an­tibac­te­r­i­al reg­i­men who took Arikayce for six months showed no growth of the bac­te­ria in their spu­tum for at least three con­sec­u­tive months com­pared with on­ly 9% of pa­tients who did not add Arikayce to their reg­i­mens.

Pos­i­tive da­ta from that on­go­ing Phase III study sent shares soar­ing in ear­ly Sep­tem­ber on hopes that it would lead to a swift FDA ap­proval.

As a con­di­tion of Arikayce’s ac­cel­er­at­ed ap­proval, In­smed will be re­quired to run an ad­di­tion­al clin­i­cal tri­al to re­ceive full ap­proval. The com­pa­ny said Fri­day that it is col­lab­o­rat­ing with the FDA on the de­sign of the tri­al, which will like­ly be a ran­dom­ized, dou­ble-blind, place­bo-con­trolled study eval­u­at­ing the ben­e­fit of Arikayce in pa­tients with Non­tu­ber­cu­lous My­cobac­te­r­i­al lung dis­ease caused by MAC.

Arikayce, al­so known as amikacin li­po­some in­hala­tion sus­pen­sion, is de­liv­ered through a neb­u­liz­er. The prod­uct will car­ry a black box warn­ing ad­vis­ing it may trig­ger bron­chospasms, hy­per­sen­si­tiv­i­ty pneu­moni­tis, the ex­ac­er­ba­tion of lung dis­ease, and spit­ting up of blood. Oth­er side ef­fects in­clude dif­fi­cul­ty speak­ing, cough, dam­aged hear­ing, up­per air­way ir­ri­ta­tion, mus­cu­loskele­tal pain, fa­tigue, di­ar­rhea and nau­sea, ac­cord­ing to the FDA.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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.

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.

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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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.

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.

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.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

David Chang steps up to CEO spot at WuX­i's cell and gene ther­a­py CD­MO; David Meline is the new CFO at Mod­er­na

David Meline didn’t stay retired for long. As reported yesterday, the former Amgen exec will replace Lorence Kim as CFO of Moderna as the Big Pharma has sprinted to the front of the Covid-19 vaccine pack, getting selected as one of the finalists for Operation Warp Speed. Meline, who gets started on Monday, announced his retirement as Amgen’s CFO in October 2019, remaining in the role until the end of the year. But he’s back in the saddle at a crucial time as Moderna prepares for Phase III studies of their SARS-CoV-2 vaccine. Meline previously held leadership positions at 3M and General Motors.