Geoff MacKay, Avrobio

Avro­bio is us­ing 'pod­s' to cre­ate gene ther­a­pies, and it be­lieves the ap­proach could change the game al­to­geth­er

Ge­off MacK­ay

Where oth­er com­pa­nies have gone big, Avro­bio is go­ing small. Its man­u­fac­tur­ing op­er­a­tion is no dif­fer­ent.

In­stead of try­ing to keep up with the val­i­da­tion of new fa­cil­i­ties — and spend­ing mon­ey and pre­cious time in the process — the com­pa­ny has opt­ed for a dif­fer­ent ap­proach: small, au­to­mat­ed pods that can be in­stalled in rent­ed clean rooms any­where. Though pro­duc­tion out­put isn’t as high as oth­er com­pa­nies, Avro­bio avoids brick-and-mor­tar and is able to stay flex­i­ble and nim­ble to re­spond to fluc­tu­a­tions.

“We be­lieve it’s the on­ly way to glob­al­ize,” said Ge­off MacK­ay, Avro’s founder and CEO. “We had no ap­petite to build big fa­cil­i­ties every­where in the world.”

The com­pa­ny based in Cam­bridge, MA is fo­cused on sin­gle-dose per­son­al­ized gene ther­a­py to cure Fab­ry dis­ease, Gauch­er dis­ease type 1 and cysti­nosis through the plat­form its dubbed pla­to. Un­like oth­er gene ther­a­pies that are de­liv­ered straight in­to pa­tients, Avro­Bio’s lentivi­ral-based ther­a­pies are de­liv­ered ex vi­vo in­to pa­tients’ stem cells be­fore pro­lif­er­a­tion and then in­fu­sion back in­to pa­tients.

The pods — typ­i­cal­ly used in hos­pi­tals for bone mar­row trans­plants — come from Mil­tenyi Biotec, and MacK­ay and his team re­pur­pose the tech for use in mod­i­fy­ing the pa­tients’ stem cells. The pods are housed in CMO clean rooms all over the world, from Hous­ton to Mel­bourne, San Jose and Maas­tricht, but the biotech even­tu­al­ly plans to scale up ca­pac­i­ty for its own in-house ver­sion of the tech­nol­o­gy.

MacK­ay be­lieves a lo­cal­ized man­u­fac­tur­ing chain is cru­cial be­cause it al­lows for each pa­tients’ stem cells to be stored in rel­a­tive prox­im­i­ty to the pa­tient.  The mod­el al­so al­lows a greater de­gree of flex­i­bil­i­ty — each dis­crete pod can be used for a wide va­ri­ety of ther­a­pies — and easy scal­a­bil­i­ty.

“The beau­ty here is that you val­i­date the pod, but reg­u­la­tors are ag­nos­tic about where you house the pod, be­cause the en­vi­ron­ment doesn’t change,” MacK­ay said. “It’s the ex­act same steps, the ex­act same en­vi­ron­ment, so you kind of just side­step all of the com­plex­i­ties of get­ting new man­u­fac­tur­ing fa­cil­i­ties on­line.”

It’s al­so a boon for Avro­Bio, which at the clin­i­cal stage is ap­proach­ing the point at which most gene ther­a­py com­pa­nies start think­ing of in­vest­ing heavy cap­i­tal in their own plants. With­out the need for a huge foot­print, that could Avro­Bio cap­i­tal flex­i­ble as it moves in­to the next stage of tri­als.

“When you’re a biotech com­pa­ny in ear­ly de­vel­op­ment, the cost of cap­i­tal is ob­vi­ous­ly pre­cious, and if you don’t have to spend hun­dreds of mil­lions of dol­lars with a VC type cost of cap­i­tal, that’s great … Nor­mal­ly, you don’t have that type of lux­u­ry,” MacK­ay said.

Avro­bio cur­rent­ly has 3 prod­ucts in clin­i­cal tri­als. Its gene ther­a­py for Fab­ry dis­ease is cur­rent­ly be­ing stud­ied in two clin­i­cal tri­als: 1 Phase I tri­al in Cana­da, and a Phase II tri­al in Aus­tralia, the US and Cana­da. It al­so has a ther­a­py for Gauch­er dis­ease type 1 that is in Phase I in Cana­da and Aus­tralia, and a cysti­nosis study in a Phase I/II tri­al at the Uni­ver­si­ty of Cal­i­for­nia, San Diego.

To­ward the end of 2018, the gene ther­a­py com­pa­ny saw its stock plum­met, drop­ping down 52% af­ter its da­ta sur­round­ing Fab­ry dis­ease treat­ment showed that vec­tor copy num­bers dropped in pe­riph­er­al blood in all of its pa­tients be­tween 1 and 18 months.

Re­cent­ly, the com­pa­ny an­nounced new da­ta that showed at least in a few pa­tients, the ther­a­py saw 100% re­duc­tion in kid­ney sub­strate, the end­point the FDA us­es for ap­proval. There has been strong safe­ty and ef­fi­ca­cy da­ta in all 14 pa­tients in the tri­al, and so far, a year from dos­ing, 10 pa­tients in clin­i­cal tri­als show no wan­ing ef­fi­ca­cy.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

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FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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Growth hor­mone from No­vo Nordisk is in short­age over man­u­fac­tur­ing de­lays

Novo Nordisk’s growth hormone Norditropin is in shortage because of manufacturing delays, according to an FDA site that tracks drug shortages as well as the American Society of Health-System Pharmacists’ shortages list.

The FDA has shortages of the drug listed for its 5, 10, 15 and 30 mg doses, while the pharmacists’ group, also known as ASHP, reported shortages of the same doses, except for the 15 mg version.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

PhRMA calls for more di­verse in­fra­struc­ture up­grades to US emer­gency tri­als frame­work

The White House’s Office of Science and Technology Policy (OSTP) last year sought to find ways to better coordinate large-scale clinical trials in the US — as the UK lead by example during the pandemic — especially for these emergency clinical trials.

The lobbying group PhRMA Tuesday called for more clinical trial diversity in underserved areas, including by making participation less of a burden, and expanding eligibility criteria when appropriate.