Macro­mol­e­cules in a pill? Tor­toise-in­spired cap­sule of­fers hope in an ear­ly test de­liv­er­ing in­sulin in­to stom­ach wall

For many di­a­bet­ics, nee­dles that de­liv­er their in­sulin are as cru­cial as taps that run the wa­ter they drink. But a team led by MIT and sci­en­tists from No­vo Nordisk — the world’s biggest in­sulin mak­er — are look­ing to change that. Re­port­ed in Sci­ence, the team has cre­at­ed a blue­ber­ry-sized oral cap­sule that de­liv­ers an in­sulin shot in­side the stom­ach once swal­lowed in rats and swine, and are look­ing to take the work in­to hu­mans in three years.

The cap­sule is in­spired by a leop­ard tor­toise — which bran­dish­es a shell that al­lows the African rep­tile to right it­self if it rolls on­to its back. The cap­sule em­u­lates that, so that no mat­ter how it lands in the gut, a nee­dle in­side the pill made of com­pressed freeze-dried in­sulin will ori­ent it­self to come in con­tact with the stom­ach lin­ing. Wa­ter in the stom­ach dis­solves a sug­ar disk hold­ing the nee­dle in­to place, and since the stom­ach wall does not con­tain pain re­cep­tors, the sci­en­tists an­tic­i­pate that pa­tients will not be able to feel the prick.

In an­i­mal stud­ies, the cap­sule was shown to de­liv­er enough in­sulin to low­er blood sug­ar to lev­els sim­i­lar to in­jec­tions ad­min­is­tered through skin, and the de­vice can po­ten­tial­ly be adapt­ed to de­liv­er oth­er pro­tein drugs, such as im­muno­sup­pres­sants used to treat rheuma­toid arthri­tis or in­flam­ma­to­ry bow­el dis­ease or even nu­cle­ic acids in­clud­ing DNA and RNA.

Bob Langer

“(This) new type of cap­sule could some­day help di­a­bet­ic pa­tients and per­haps any­one who re­quires ther­a­pies that can now on­ly be giv­en by in­jec­tion or in­fu­sion,” said MIT’s Robert Langer who was one of the se­nior au­thor’s of the study. Langer’s lab at MIT has spawned a string of bio­phar­ma com­pa­nies.

“(If) a per­son were to move around or the stom­ach were to growl, the de­vice would not move from its pre­ferred ori­en­ta­tion,” said the study’s lead au­thor and MIT grad­u­ate stu­dent Alex Abram­son.

Once in­ject­ed in­to the stom­ach wall, the cap­sule re­leas­es its con­tents, and dis­in­te­grates as it pass­es harm­less­ly through the di­ges­tive sys­tem and is even­tu­al­ly elim­i­nat­ed, the re­searchers said re­fer­ring to an­i­mal da­ta in their re­port pub­lished on Thurs­day.

The re­search was fund­ed by No­vo Nordisk $NVO, the NIH and oth­ers. To be sure, hu­man tri­als are still to come and must be cleared be­fore the de­vice can be safe­ly and ef­fec­tive­ly used. But for the Dan­ish drug­mak­er, the suc­cess­ful de­ploy­ment of the prod­uct could rein­vig­o­rate its long-term growth as it slow­ly moves away from its de­pen­dence of tra­di­tion­al in­jectable in­sulin amidst in­ten­si­fy­ing com­pe­ti­tion and pric­ing pres­sure with­in the di­a­betes mar­ket.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.