Ever optimistic despite concerns, BrainStorm teams with Catalent to prep for possible approval for ALS drug

Catal­ent has en­tered a part­ner­ship with a cell ther­a­py com­pa­ny that fo­cus­es on neu­rode­gen­er­a­tive dis­eases, tak­ing on a tech trans­fer to man­u­fac­ture treat­ments for ALS and PMS.

Brain­Storm Cell Ther­a­peu­tics and Catal­ent have been part­ners since 2020, when Catal­ent start­ed man­u­fac­tur­ing NurOwn, the amy­otroph­ic lat­er­al scle­ro­sis plat­form com­prised of mes­enchy­mal stem cells (MSCs) and neu­rotroph­ic fac­tors, in an­tic­i­pa­tion of a pos­si­ble reg­u­la­to­ry ap­proval. Catal­ent will use its 32,000-square-foot fa­cil­i­ty in Hous­ton, TX to man­u­fac­ture the cell ther­a­py.

‘Our ex­ten­sive ex­pe­ri­ence in cell ther­a­py de­vel­op­ment and scale-up was key to the com­ple­tion of this tech­nol­o­gy trans­fer to our state-of-the-art cell ther­a­py fa­cil­i­ty in Hous­ton,’ pres­i­dent of cell and gene ther­a­py at Catal­ent Man­ja Boer­man said in a state­ment.

NurOwn us­es MSCs de­rived from bone mar­row, and ex­pand­ed and dif­fer­en­ti­at­ed ex vi­vo. They are de­liv­ered di­rect­ly to the site of the dam­aged cells to slow or sta­bi­lize the pro­gres­sion of dis­ease, in this case ALS. Brain­Storm’s Phase III tri­al saw low­ered lev­els of neu­roin­flam­ma­tion, which led to clin­i­cal ben­e­fits in the small group. But it missed its pri­ma­ry and sec­ondary end­points in the study. The drug’s price was once pro­posed at $300,000, but a year af­ter two failed end­points and a stock that plunged 70%, the team at Brain­storm re­mains op­ti­mistic.

Brain­Storm pre­sent­ed new da­ta from a Phase III tri­al at the ALS One Re­search Sym­po­sium at the end of No­vem­ber, which it said ‘re­vealed the po­ten­tial for a mean­ing­ful treat­ment ef­fect across end­points when fo­cus­ing on a sub­set of par­tic­i­pants with less se­vere dis­ease at base­line’ de­spite not meet­ing its end­points, in­clud­ing da­ta from a sub­group that pre­dicts that group to have long to very-long sur­vival.

ALS has been a tough field for biotechs to nav­i­gate. Bio­gen’s SOD1-ALS drug tofersen was the lat­est flop, as it failed in Phase III tri­als in Oc­to­ber, show­ing very lit­tle dif­fer­ence in re­sponse be­tween par­tic­i­pants giv­en the drug ver­sus the place­bo.

The an­nounce­ment comes just days be­fore the US House of Rep­re­sen­ta­tives vot­ed al­most unan­i­mous­ly (423-3) to pass a bill that gives $500 mil­lion over five years to cer­tain small drug­mak­ers to cov­er the costs of R&D and to ex­pand ac­cess to pa­tients not el­i­gi­ble for clin­i­cal tri­als for po­ten­tial ALS drugs.

The bill could pro­vide a boost to com­pa­nies like Amy­lyx, which will be sub­mit­ting its ALS drug for US ap­proval, the com­pa­ny said. This is af­ter the FDA asked for a Phase III study to con­firm pos­i­tive Phase II re­sults, but then walked back that ask.

Ed­i­tor’s Note: For more news and ex­clu­sive cov­er­age from the man­u­fac­tur­ing beat, sub­scribe to the End­points Man­u­fac­tur­ing week­ly re­port in your read­er pro­file.

The newest building at Fujifilm Irvine Scientific’s Netherlands campus is open for business.

The company, amidst a massive amount of expansion that has placed it in the top-five companies by manufacturing capacity, announced the Tilburg site is fully operational as of Wednesday. It will allow for an increase in efficiency and affordability in shipping, thanks to its central Europe location, as well as put some flexibility into the supply chain.

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Bobby Sheng, Bora Pharmaceuticals CEO

Taipei’s Bora Pharmaceuticals has linked up with Taishin Healthcare to invest about $108 million into CDMOs and CROs in response to a booming business environment for both industries.

The two companies plan to enter the macromolecular, and cell and gene therapy manufacturing spaces, and strengthen competitiveness while bolstering Taiwan’s CDMO market. According to a report from Hartford, CT-based research group Global Information, the global CDMO market was valued at $160.1 billion in 2020 and could grow to as much as $242.64 billion by 2026. There are five CDMOs in the world that have revenue above $500 million, while 75% of the field takes in less than $50 million in revenue, the report says.

Sandoz has issued a recall of one lot of its enoxaparin sodium injection, 40 mg/.4 mL single-dose syringes after exposure to high temperatures may have impacted product effectiveness.

The lot, dubbed SAB06761A, could be a risk for patients with health conditions the product is intended to treat, such as stroke or heart attack. The patients, who are already at risk for blood clots, blocked blood vessels or arteries and swelling close to the lungs, are being treated for the prevention of deep vein thrombosis, a condition that occurs when blood clots form in a deep vein, usually in the legs, after surgeries or as a complication from a heart attack.

Just after Merck announced a deal to partner with a manufacturer to make its experimental Covid-19 pill molnupiravir in the US, the drugmaker has reached a similar partnership to make the pill for distribution throughout the rest of the world as well.

Merck will partner with Thermo Fisher Scientific to manufacture the pill for Canada, the UK, several markets in the EU and Asia, as well as Latin America. The Whitby, Ontario site will be one of three in the world making the pill, which was developed with Ridgeback Biotherapeutics.

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Adar Poonawalla, chief executive officer of Serum Institute of India

Covax, which ships Covid-19 vaccines to low- and middle-income nations, is calling for more Indian-made doses of AstraZeneca’s vaccine, Reuters reported, as a key manufacturer plans to halve the vaccine’s production temporarily until it gets more orders.

The Serum Institute of India said uptake has slowed sharply and told an Indian television station that Covax was taking in fewer doses than offered. CEO Adar Poonawalla said uptake is expected to kick up next quarter, but a spokesperson for Gavi, which is providing low-cost shots worldwide as part of a collaboration with Covax, told Reuters the organization is still receiving ‘robust demand’ for Covishield.

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Please signup to continue — it’s fast and free. This article is sponsored by Catalent and produced by Endpoints Studio. Each year, we aim to highlight 20 extraordinary women who are leaving their mark on drug R&D — and this year’s group was no exception.

Our list, while by no means exhaustive, includes scientists, CEOs, researchers and professors who are supercharging the discovery and development of new therapies worldwide. Our team of writers spent time with each honoree (with a few exceptions), learning their stories and sketching profiles, which you’ll find in our special report.

For the second time, we brought the celebration to a live virtual audience, featuring an award presentation followed by a panel on what it takes to break the glass ceiling in biopharma with Kojin Therapeutics CEO Luba Greenwood, AskBio CEO Sheila Mikhail, and Silverback Therapeutics CEO Laura Shawver. Our panelists had a lively discussion on how the industry’s culture has changed, how to handle sexual harassment, the progress we’ve made and the challenges that still hold women back today.

We applaud each of our honorees for scaling the heights of biopharma R&D. You got to meet most of them via brief recordings we played during our live event. Below, you’ll find bonus videos offering a longer glimpse into those interviews. And if you didn’t get a chance to tune in to our main event live, you can replay the entire show.

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Even though many biopharma leaders have come together in recent years to address its gender gap, the consensus is clear: We still have a long way to go.

Companies this year were 2.5 times more likely than last year to have a diversity and inclusion program in place, according to a recent BIO survey, but women are still largely absent from executive roles. Getting women to enter the industry isn’t the problem — studies show that they represent just under half of all biotech employees around the world. But climbing through the ranks can be challenging, as women still report facing stereotypes, and, unfortunately, harassment.

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Matt Kapusta, uniQure CEO

After a five-month hold put a damper on uniQure’s hemophilia B gene therapy program, things are finally looking up as a positive pivotal readout puts the company and partner CSL Behring on track to file for approval in the first half of next year — and analysts spy blockbuster potential.

The companies’ potential first gene therapy for hemophilia B, known as etranacogene dezaparvovec, met its primary non-inferiority endpoint in annualized bleeding rate (ABR) after 18 months compared to baseline Factor IX prophylactic therapy, uniQure announced on Thursday. Factor IX is a protein that’s naturally produced in the body to help form blood clots and stop bleeding, and common treatments today are designed to replace the protein to achieve adequate clotting.

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